PTC Therapeutics (PTCT) 10-Q25 Apr 242024 Q1 Quarterly reportFinancial data

Company Profile

Cover

Cover - shares	3 Months Ended
Cover - shares	Mar. 31, 2024	Apr. 23, 2024
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Mar. 31, 2024
Document Transition Report	false
Entity File Number	001-35969
Entity Registrant Name	PTC Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	04-3416587
Entity Address, Address Line One	500 Warren Corporate Center Drive
Entity Address, City or Town	Warren
Entity Address, State or Province	NJ
Entity Address, Postal Zip Code	07059
City Area Code	908
Local Phone Number	222-7000
Title of 12(b) Security	Common Stock, $0.001 par value per share
Trading Symbol	PTCT
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		76,696,785
Entity Central Index Key	0001070081
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q1
Amendment Flag	false

Consolidated Balance Sheets

Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 548,355	$ 594,001
Marketable securities	336,458	282,738
Trade and royalty receivables, net	197,660	160,822
Inventory, net	30,058	30,577
Prepaid expenses and other current assets	56,207	150,491
Total current assets	1,168,738	1,218,629
Fixed assets, net	92,779	87,089
Intangible assets, net	330,040	379,497
Goodwill	82,341	82,341
Operating lease ROU assets	91,186	91,896
Deposits and other assets	24,545	36,246
Total assets	1,789,629	1,895,698
Current liabilities:
Accounts payable and accrued expenses	330,924	391,983
Deferred revenue		801
Operating lease liabilities- current	14,572	13,002
Finance lease liabilities- current	1,948	3,000
Liability for sale of future royalties - current	227,102	194,314
Total current liabilities	574,546	603,100
Long-term debt	284,512	284,213
Contingent consideration payable	36,200	36,300
Deferred tax liability	55,904	55,905
Operating lease liabilities- noncurrent	104,837	97,627
Finance lease liabilities- noncurrent	15,574	17,184
Liability for sale of future royalties- noncurrent	1,611,831	1,619,783
Other long-term liabilities	141	141
Total liabilities	2,683,545	2,714,253
Stockholders' deficit:
Common stock, $0.001 par value. Authorized 250,000,000 shares; issued and outstanding 76,653,960 shares at March 31, 2024. Authorized 250,000,000 shares; issued and outstanding 75,708,889 shares at December 31, 2023	76	75
Additional paid-in capital	2,486,722	2,466,233
Accumulated other comprehensive loss	(5,560)	(1,285)
Accumulated deficit	(3,375,154)	(3,283,578)
Total stockholders' deficit	(893,916)	(818,555)
Total liabilities and stockholders' deficit	$ 1,789,629	$ 1,895,698

Consolidated Balance Sheets (Pa

Consolidated Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2024	Dec. 31, 2023
Statement of Financial Position [Abstract]
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, authorized shares (in shares)	250,000,000	250,000,000
Common stock, issued shares (in shares)	76,653,960	75,708,889
Common stock, outstanding shares (in shares)	76,653,960	75,708,889

Consolidated Statements of Oper

Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Revenues:
Revenue	$ 210,118	$ 220,382
Operating expenses:
Cost of product sales, excluding amortization of acquired intangible assets	14,740	14,144
Amortization of intangible assets	51,530	39,415
Research and development	116,129	195,124
Selling, general and administrative	73,272	86,914
Change in the fair value of contingent consideration	(100)	2,400
Total operating expenses	255,571	337,997
Loss from operations	(45,453)	(117,615)
Interest expense, net	(40,834)	(27,331)
Other income, net	1,591	9,956
Loss before income tax expense	(84,696)	(134,990)
Income tax expense	(6,880)	(3,969)
Net loss attributable to common stockholders	$ (91,576)	$ (138,959)
Weighted-average shares outstanding:
Basic	76,496,127	73,729,284
Diluted	76,496,127	73,729,284
Net loss per share-basic and diluted (in dollars per share)
Basic	$ (1.20)	$ (1.88)
Diluted	$ (1.20)	$ (1.88)
Net product revenue
Revenues:
Revenue	$ 177,604	$ 187,557
Collaboration revenue
Revenues:
Revenue		6
Royalty revenue
Revenues:
Revenue	31,154	30,831
Manufacturing revenue
Revenues:
Revenue	$ 1,360	$ 1,988

Consolidated Statements of Comp

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Statement of Comprehensive Income [Abstract]
Net Income (Loss)	$ (91,576)	$ (138,959)
Other comprehensive (loss) income:
Unrealized (loss) gain on marketable securities, net of tax	(444)	54
Foreign currency translation loss, net of tax	(3,831)	(6,437)
Comprehensive loss	$ (95,851)	$ (145,342)

Consolidated Statements of Stoc

Consolidated Statements of Stockholders' Deficit - USD ($) $ in Thousands	Common stock	Additional paid-in capital	Accumulated other comprehensive (loss) income	Accumulated deficit	Total
Balance (in shares) at Dec. 31, 2022	73,104,692
Balance at the beginning of the period at Dec. 31, 2022	$ 72	$ 2,305,020	$ 4,796	$ (2,656,974)	$ (347,086)
Increase (Decrease) in Stockholders' Equity
Exercise of options (in shares)	211,561
Exercise of options		5,655			5,655
Restricted stock vesting and issuance (in shares)	695,781
Restricted stock vesting and issuance, net	$ 1				1
Share-based compensation expense		28,815			28,815
Receivable from investor		396			396
Net Income (Loss)				(138,959)	(138,959)
Comprehensive (loss) income			(6,383)		(6,383)
Balance (in shares) at Mar. 31, 2023	74,012,034
Balance at the end of the period at Mar. 31, 2023	$ 73	2,339,886	(1,587)	(2,795,933)	(457,561)
Balance (in shares) at Dec. 31, 2023	75,708,889
Balance at the beginning of the period at Dec. 31, 2023	$ 75	2,466,233	(1,285)	(3,283,578)	(818,555)
Increase (Decrease) in Stockholders' Equity
Exercise of options (in shares)	109,892
Exercise of options		2,030			2,030
Restricted stock vesting and issuance (in shares)	835,179
Restricted stock vesting and issuance, net	$ 1				1
Share-based compensation expense		18,378			18,378
Receivable from investor		81			81
Net Income (Loss)				(91,576)	(91,576)
Comprehensive (loss) income			(4,275)		(4,275)
Balance (in shares) at Mar. 31, 2024	76,653,960
Balance at the end of the period at Mar. 31, 2024	$ 76	$ 2,486,722	$ (5,560)	$ (3,375,154)	$ (893,916)

Consolidated Statements of Cash

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Cash flows from operating activities
Net Income (Loss)	$ (91,576)	$ (138,959)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	55,395	42,825
Non-cash operating lease expense	1,792	2,942
Non-cash royalty revenue related to sale of future royalties	(25,228)	(13,237)
Non-cash interest expense on liability related to sale of future royalties	50,064	18,902
Change in valuation of contingent consideration	(100)	2,400
Unrealized (gain) loss on ClearPoint Equity Investments	(9)	39
Unrealized gain on ClearPoint convertible debt security	(166)	(59)
Unrealized gain on marketable securities - equity investments	(710)	(2,166)
Disposal of asset	544
Deferred income taxes	(1)	(4)
Amortization of discounts on investments, net	(3,697)	(85)
Amortization of debt issuance costs	296	438
Share-based compensation expense	18,378	28,815
Unrealized foreign currency transaction losses (gains), net	269	(10,057)
Changes in operating assets and liabilities:
Inventory, net	120	(4,612)
Prepaid expenses and other current assets	93,904	57,611
Trade and royalty receivables, net	(39,500)	(43,426)
Deposits and other assets	11,192	743
Accounts payable and accrued expenses	(5,389)	33,524
Other liabilities	5,984	(3,988)
Deferred revenue	(801)	(1,137)
Net cash provided by (used in) operating activities	70,761	(29,491)
Cash flows from investing activities
Purchases of fixed assets	(9,588)	(10,270)
Purchases of marketable securities - available for sale	(104,373)
Purchases of marketable securities - equity investments	(9,065)
Sale and redemption of marketable securities- available for sale	61,650	12,500
Sale and redemption of marketable securities - equity investments	1,207	2,196
Acquisition of product rights and licenses	(54,763)	(33,397)
Net cash used in investing activities	(114,932)	(28,971)
Cash flows from financing activities
Proceeds from exercise of options	2,030	5,655
Debt issuance costs related to secured term loan		(182)
Payment of finance lease principal	(1,490)	(1,379)
Net cash provided by financing activities	540	4,094
Effect of exchange rate changes on cash	(2,030)	(7,963)
Net decrease in cash and cash equivalents	(45,661)	(62,331)
Cash and cash equivalents, and restricted cash beginning of period	610,284	295,925
Cash and cash equivalents, and restricted cash end of period	564,623	233,594
Supplemental disclosure of cash information
Cash paid for interest	3,666	12,956
Cash paid for income taxes	2,042	2,215
Supplemental disclosure of non-cash investing and financing activity
Unrealized (loss) gain on marketable securities, net of tax	(444)	54
Right-of-use assets obtained in exchange for operating lease obligations	1,723
Acquisition of product rights and licenses	2,296	19,406
Milestone payable	$ 2,500	32,500
Debt issuance costs related to senior secured term loan		45
Capital expenditures unpaid at the end of period		$ 28

The Company

The Company	3 Months Ended
The Company	Mar. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
The Company	1. The Company PTC Therapeutics, Inc. (the “Company” or “PTC”) is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes that this allows it to maximize value for all of its stakeholders. PTC has a diversified therapeutic portfolio pipeline that includes several commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology and metabolism. The Company has two products, Translarna™ (ataluren) and Emflaza® (deflazacort), for the treatment of Duchenne muscular dystrophy (“DMD”), a rare, life threatening disorder. Translarna has marketing authorization in the European Economic Area (the “EEA”) for the treatment of nonsense mutation Duchenne muscular dystrophy (“nmDMD”) in ambulatory patients aged 2 years and older. Translarna also has marketing authorization in Russia for the treatment of nmDMD in patients aged two years and older and in Brazil for the treatment of nmDMD in ambulatory patients two years and older and for continued treatment of patients that become non-ambulatory, as well as in various other countries. Emflaza is approved in the United States for the treatment of DMD in patients two years and older. The Company’s marketing authorization for Translarna in the EEA is subject to annual review and renewal by the European Commission (“EC”) following reassessment by the European Medicines Agency (“EMA”) of the benefit-risk balance of the authorization, which the Company refers to as the annual EMA reassessment. In September 2022, the Company submitted a Type II variation to the EMA to support conversion of the conditional marketing authorization for Translarna to a standard marketing authorization, which included a report on the placebo-controlled trial of Study 041 and data from the open-label extension. In February 2023, the Company also submitted an annual marketing authorization renewal request to the EMA. In September 2023, the Committee for Medicinal Products for Human Use (“CHMP”), gave a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna for the treatment of nmDMD and a negative opinion on the renewal of the existing conditional marketing authorization of Translarna for the treatment of nmDMD. On January 25, 2024, the CHMP issued a negative opinion for the renewal of the conditional marketing authorization following a re-examination procedure. In accordance with EMA regulations, the EC has approximately 67 days to adopt the opinion. At this time, the EC has not yet adopted the negative opinion. If the EC adopts the negative opinion, Translarna would no longer have marketing authorization in the member states of the EEA. Translarna is an investigational new drug in the United States. Following the Company’s announcement of top-line results from the placebo-controlled trial of Study 041 in June 2022, the Company submitted a meeting request to the U.S. Food and Drug Administration (“FDA”) to gain clarity on the regulatory pathway for a potential re-submission of a New Drug Application (“NDA”) for Translarna. The FDA provided initial written feedback that Study 041 does not provide substantial evidence of effectiveness to support an NDA re-submission. The Company held a Type C meeting with the FDA in the fourth quarter of 2023 to discuss the totality of Translarna data. Based on feedback from the FDA, the Company plans to re-submit the NDA based on results from Study 041 and from the Company’s international drug registry study for nmDMD patients receiving Translarna by mid-year 2024. The Company has developed Upstaza (eladocagene exuparvovec), a gene therapy used for the treatment of Aromatic L-Amino Acid Decarboxylase (“AADC”) deficiency (“AADC deficiency”), a rare central nervous system (“CNS”) disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene. In July 2022, the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. In November 2022, the Medicines and Healthcare Products Regulatory Agency approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the United Kingdom. In March 2024, the Company submitted a biologics license application (“BLA”) for Upstaza for the treatment of AADC deficiency in the United States. The Company holds the rights for the commercialization of Tegsedi ® ® The Company also has a spinal muscular atrophy (“SMA”) collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc. (referred to collectively as “Roche”) and the Spinal Muscular Atrophy Foundation (“SMA Foundation”). The SMA program has one approved product, Evrysdi® (risdiplam), which was approved by the FDA in August 2020 for the treatment of SMA in adults and children two months and older and by the EC in March 2021 for the treatment of 5q SMA in patients two months and older with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. Evrysdi has also received marketing authorization for the treatment of SMA in over 100 countries. In May 2022, the FDA approved a label expansion for Evrysdi to include infants under two months old with SMA. In August 2023, the EC approved an extension of the Evrysdi marketing authorization to include infants under two months old in the EU. One of the Company’s most advanced clinical stage molecules is sepiapterin. Sepiapterin is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in metabolism and synthesis of numerous metabolic products. In May 2023, the Company announced that the primary endpoint was achieved in its registration-directed Phase 3 trial for sepiapterin for phenylketonuria (“PKU”). The primary endpoint of the study was the achievement of statistically-significant reduction in blood Phe level. In March 2024, the Company submitted a marketing authorization application (“MAA”) to the EMA for sepiapterin for the treatment of PKU in the EEA. Additionally, the Company participated in a pre-NDA meeting with the FDA in the third quarter of 2023. At that meeting, the FDA stated that the sepiapterin clinical safety and efficacy data supported NDA submission for the treatment of pediatric and adult PKU patients. However, the FDA has requested that PTC completes a 26-week nonclinical mouse study to assess sepiapterin carcinogenicity potential prior to NDA submission. Based on the timing to complete this study, PTC expects to submit an NDA to the FDA for sepiapterin for the treatment of PKU no later than the third quarter of 2024 and PTC intends to discuss with the FDA the potential for an earlier submission if PTC is permitted to submit the 26-week mouse study report during the NDA review. PTC also expects to make regulatory submissions for sepiapterin for the treatment of PKU in Japan and Brazil in 2024. In addition to the Company’s SMA program, the Company’s splicing platform also includes PTC518, which is being developed for the treatment of Huntington’s disease (“HD”). The Company initiated a Phase 2 study of PTC518 for the treatment of HD in the first quarter of 2022, which consists of an initial 12-week placebo-controlled phase focused on safety, pharmacology and pharmacodynamic effects followed by a nine-month placebo-controlled phase focused on PTC518 biomarker effect. In June 2023, the Company announced interim data from the 12-week placebo-controlled phase. The Phase 2 study is actively ongoing outside the United States, while it has been paused within the United States as the FDA requested additional data to allow the Phase 2 study to proceed. The Company expects results from the 12-month interim data from the Phase 2 study of PTC518 for the treatment of HD in the second quarter of 2024. The Company expects to submit a safety data update to the FDA in the second quarter of 2024 to support lifting of the partial clinical hold on the program. The Company’s ferroptosis and inflammation platform consists of small molecule compounds that target oxidoreductase enzymes that regulate oxidative stress and inflammatory pathways central to the pathology of a number of CNS diseases. The two most advanced molecules in the Company’s ferroptosis and inflammation platform are vatiquinone and utreloxastat. The Company announced topline results from a registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich ataxia, called MOVE-FA, in May 2023. While the study did not meet its primary endpoint, vatiquinone treatment did demonstrate significant benefit on key disease subscales, including the upright stability subscale, as well as on other disease relevant endpoints. In the first quarter of 2024, the Company met with the FDA, who expressed willingness to review an NDA for vatiquinone for the treatment of Friedreich ataxia based on the MOVE-FA trial as well as data from the ongoing open label extension study following the MOVE-FA trial, potentially allowing for the submission of an NDA in late 2024. In the first quarter of 2024, the Company also received scientific advice from the EMA on the MOVE-FA trial results, in which the EMA stated that the MOVE-FA data would likely not be sufficient for conditional authorization. The Company initiated a Phase 2 registration directed trial of utreloxastat for amyotrophic lateral sclerosis, or ALS, in the first quarter of 2022. The Company expects topline results from this trial in the fourth quarter of 2024. In addition, the Company has a pipeline of product candidates and discovery programs that are in early clinical, pre-clinical and research and development stages focused on the development of new treatments for multiple therapeutic areas for rare diseases. As of March 31, 2024, the Company had an accumulated deficit of approximately $3,375.2 million. The Company has financed its operations to date primarily through the private offerings of convertible senior notes (see Note 9), public and “at the market offerings” of common stock, proceeds from royalty purchase agreements (see Note 2), net proceeds from the Company’s’ borrowings under its credit agreement with Blackstone (see Note 9), private placements of its convertible preferred stock and common stock, collaborations, bank and institutional lender debt, other convertible debt, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease area addressed by the Company’s product candidates. The Company has also relied on revenue generated from net sales of Translarna for the treatment of nmDMD in territories outside of the United States since 2014, Emflaza for the treatment of DMD in the United States since 2017, and Upstaza for the treatment of AADC deficiency in the EEA since May 2022. The Company has also relied on revenue associated with milestone and royalty payments from Roche pursuant to the License and Collaboration Agreement (the “SMA License Agreement”) dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation, under its SMA program. The Company expects that cash flows from the sales of its products, milestone and royalty payments from Roche, together with the Company’s cash, cash equivalents and marketable securities, will be sufficient to fund its operations for at least the next twelve months.

Summary of significant accounti

Summary of significant accounting policies	3 Months Ended
Summary of significant accounting policies	Mar. 31, 2024
Accounting Policies [Abstract]
Summary of significant accounting policies	2. Summary of significant accounting policies The Company’s complete listing of significant accounting policies is set forth in Note 2 of the notes to the Company’s audited financial statements as of December 31, 2023 included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC") on February 29, 2024 (the "2023 Form 10-K"). Selected significant accounting policies are discussed in further detail below. Basis of presentation The accompanying financial information as of March 31, 2024 and for the three months ended March 31, 2024 and 2023 has been prepared by the Company, without audit, pursuant to the rules and regulations of the SEC. Certain information and footnote disclosures normally included in financial statements prepared in accordance with generally accepted accounting principles in the United States ("GAAP") have been condensed or omitted pursuant to such rules and regulations. These interim financial statements should be read in conjunction with the Company’s audited financial statements as of December 31, 2023 and notes thereto included in the 2023 Form 10-K. In the opinion of management, the unaudited financial information as of March 31, 2024 and for the three months ended March 31, 2024 and 2023 reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of financial position, results of operations, stockholders’ (deficit) equity, and cash flows. The results of operations for the three months ended March 31, 2024 are not necessarily indicative of the results to be expected for the year ended December 31, 2024 or for any other interim period or for any other future year. Use of estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of net product sales, royalty revenue, certain accruals related to the Company’s research and development expenses, valuation procedures for liability for sale of future royalties, fair value of the contingent consideration, and the provision for or benefit from income taxes. Actual results could differ from those estimates. Changes in estimates are reflected in reported results in the period in which they become known. Restricted cash Restricted cash included in deposits and other assets on the consolidated balance sheet contains an unconditional, irrevocable and transferable letter of credit that was entered into during the twelve-month period ended December 31, 2019 in connection with obligations under a facility lease for the Company’s leased biologics manufacturing facility in Hopewell Township, New Jersey. The amount of the letter of credit is $7.5 million, is to be maintained for a term of not less than five years and has the potential to be reduced to $3.8 million if after five years the Company is not in default of its lease. Restricted cash also contains an unconditional, irrevocable and transferable letter of credit that was entered into during June 2022 in connection with obligations for the Company’s new facility lease in Warren, New Jersey. The amount of the letter of credit is $8.1 million and has the potential to be reduced to $4.1 million if after five years the Company is not in default of its lease. Both amounts are classified within deposits and other assets on the consolidated balance sheet due to the long-term nature of the respective letters of credit. Restricted cash also includes a bank guarantee of $0.6 million denominated in a foreign currency. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same amounts shown in the statement of cash flows: End of Beginning of period- period- March 31, December 31, 2024 2023 Cash and cash equivalents $ 548,355 $ 594,001 Restricted cash included in deposits and other assets 16,268 16,283 Total Cash, cash equivalents and restricted cash per statement of cash flows $ 564,623 $ 610,284 Marketable securities The Company’s marketable securities consists of both debt securities and equity investments. The Company considers its investments in debt securities with original maturities of greater than 90 days to be available for sale securities. Securities under this classification are recorded at fair value and unrealized gains and losses within accumulated other comprehensive income. The estimated fair value of the available for sale securities is determined based on quoted market prices or rates for similar instruments. In addition, the cost of debt securities in this category is adjusted for amortization of premium and accretion of discount to maturity. For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. If the criteria are not met, the Company evaluates whether the decline in fair value has resulted from a credit loss or other factors. In making this assessment, management considers, among other factors, the extent to which fair value is less than amortized cost, any changes to the rating of the security by a rating agency, and adverse conditions specifically related to the security. If this assessment indicates that a credit loss exists, the present value of cash flows expected to be collected from the security are compared to the amortized cost basis of the security. If the present value of the cash flows expected to be collected is less than the amortized cost basis, a credit loss exists and an allowance for credit losses is recorded for the credit loss, limited by the amount that the fair value is less than the amortized costs basis. Any impairment that has not been recorded through an allowance for credit losses is recognized in other comprehensive income. For the three months ended March 31, 2024 and 2023, no allowance was recorded for credit losses. Marketable securities that are equity investments are measured at fair value, as it is readily available, and as such are classified as Level 1 assets. Unrealized holding gains and losses for these equity investments are components of other income (expense), net within the consolidated statement of operations. Inventory and cost of product sales Inventory Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis by product. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. Products which may be used in clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. Inventory used for marketing efforts are charged to selling, general and administrative expense. Amounts related to clinical development programs and marketing efforts are immaterial. The following table summarizes the components of the Company’s inventory for the periods indicated: March 31, 2024 December 31, 2023 Raw materials $ 1,121 $ 952 Work in progress 22,178 17,991 Finished goods 6,759 11,634 Total inventory $ 30,058 $ 30,577 The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. For the three months ended March 31, 2024, the Company recorded inventory write-downs of $3.7 million primarily related to adjustments to inventory reserves and product approaching expiration. For the three months ended March 31, 2023, the Company recorded inventory write-downs of $0.1 million primarily related to product approaching expiration. Additionally, though the Company’s product is subject to strict quality control and monitoring which it performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications resulting in a charge to cost of product sales. For the three months ended March 31, 2024 and 2023, these amounts were immaterial. Cost of product sales Cost of product sales consists of the cost of inventory sold, manufacturing and supply chain costs, storage costs, amortization of the acquired intangible asset, royalty payments associated with net product sales, and royalty payments to collaborative partners associated with royalty revenues and collaboration revenue related to milestones. Production costs are expensed as cost of product sales when the related products are sold or royalty revenues and collaboration revenue milestones are earned. Revenue recognition Net product revenue The Company’s net product revenue primarily consists of sales of Translarna in territories outside of the U.S. for the treatment of nmDMD and sales of Emflaza in the U.S. for the treatment of DMD. The Company recognizes revenue when its performance obligations with its customers have been satisfied. The Company’s performance obligations are to provide products based on customer orders from distributors, hospitals, specialty pharmacies or retail pharmacies. The performance obligations are satisfied at a point in time when the Company’s customer obtains control of the product, which is typically upon delivery. The Company invoices its customers after the products have been delivered and invoice payments are generally due within 30 to 90 days of the invoice date. The Company determines the transaction price based on fixed consideration in its contractual agreements. Contract liabilities arise in certain circumstances when consideration is due for goods the Company has yet to provide. As the Company has identified only one distinct performance obligation, the transaction price is allocated entirely to product sales. In determining the transaction price, a significant financing component does not exist since the timing from when the Company delivers product to when the customers pay for the product is typically less than one year. Customers in certain countries pay in advance of product delivery. In those instances, payment and delivery typically occur in the same month. The Company records product sales net of any variable consideration, which includes discounts, allowances, rebates related to Medicaid and other government pricing programs, and distribution fees. The Company uses the expected value or most likely amount method when estimating its variable consideration, unless discount or rebate terms are specified within contracts. The identified variable consideration is recorded as a reduction of revenue at the time revenues from product sales are recognized. These estimates for variable consideration are adjusted to reflect known changes in factors and may impact such estimates in the quarter those changes are known. Revenue recognized does not include amounts of variable consideration that are constrained. For the three months ended March 31, 2024 and 2023, net product sales outside of the United States were $120.1 million and $133.0 million, respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net revenues made up $103.6 million and $115.1 million of the net product sales outside of the United States for the three months ended March 31, 2024 and 2023, respectively. For the three months ended March 31, 2024 and 2023, net product sales in the United States were $57.5 million and $54.6 million, respectively, consisting solely of sales of Emflaza. During the three months ended March 31, 2024, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $57.5 million and $52.6 million of net product sales, respectively. During the three months ended March 31, 2023, three countries, the United States, Russia, and Brazil, accounted for at least 10% of the Company’s net product sales, representing $54.6 million, $44.6 million, and $25.9 million of net product sales, respectively. In relation to customer contracts, the Company incurs costs to fulfill a contract but does not incur costs to obtain a contract. These costs to fulfill a contract do not meet the criteria for capitalization and are expensed as incurred. The Company considers any shipping and handling costs that are incurred after the customer has obtained control of the product as a cost to fulfill a promise. Shipping and handling costs associated with finished goods delivered to customers are recorded as a selling expense. Collaboration and royalty revenue The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license fees; milestone payments; research funding and royalties on future product sales. In addition, the Company generates service revenue through agreements that generally provide for fees for research and development services and may include additional payments upon achievement of specified events. At the inception of a collaboration arrangement, the Company needs to first evaluate if the arrangement meets the criteria in Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 808 “Collaborative Arrangements” to then determine if ASC Topic 606 is applicable by considering whether the collaborator meets the definition of a customer. If the criteria are met, the Company assesses the promises in the arrangement to identify distinct performance obligations. For licenses of intellectual property, the Company assesses, at contract inception, whether the intellectual property is distinct from other performance obligations identified in the arrangement. If the licensing of intellectual property is determined to be distinct, revenue is recognized for nonrefundable, upfront license fees when the license is transferred to the customer and the customer can use and benefit from the license. If the licensing of intellectual property is determined not to be distinct, then the license will be bundled with other promises in the arrangement into one distinct performance obligation. The Company needs to determine if the bundled performance obligation is satisfied over time or at a point in time. If the Company concludes that the nonrefundable, upfront license fees will be recognized over time, the Company will need to assess the appropriate method of measuring proportional performance. For milestone payments, the Company assesses, at contract inception, whether the development or sales-based milestones are considered probable of being achieved. If it is probable that a significant revenue reversal will occur, the Company will not record revenue until the uncertainty has been resolved. Milestone payments that are contingent upon regulatory approval are not considered probable of being achieved until the applicable regulatory approvals or other external conditions are obtained as such conditions are not within the Company’s control. If it is probable that a significant revenue reversal will not occur, the Company will estimate the milestone payments using the most likely amount method. The Company will re-assess the development and sales-based milestones each reporting period to determine the probability of achievement. The Company recognizes royalties from product sales at the later of when the related sales occur or when the performance obligation to which the royalty has been allocated has been satisfied. If it is probable that a significant revenue reversal will not occur, the Company will estimate the royalty payments using the most likely amount method. The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities. For the three months ended March 31, 2024, the Company did not recognize collaboration revenue related to the SMA License Agreement with Roche. For the three months ended March 31, 2023, the amounts recognized for the collaboration revenue related to the SMA License Agreement with Roche were immaterial. For the three months ended March 31, 2024 and 2023, the Company has recognized $31.2 million and $30.8 million of royalty revenue, respectively, related to Evrysdi. Manufacturing Revenue The Company has manufacturing services related to the production of plasmid deoxyribonucleic acid (“DNA”) and adeno-associated virus (“AAV”) vectors for gene therapy applications for external customers. Performance obligations vary but may include manufacturing plasmid DNA and/or AAV vectors, material testing, stability studies, and other services related to material development. The transaction prices for these arrangements are fixed and include amounts stated in the contracts for each promised service. Typically, the performance obligations within a manufacturing contract are highly interdependent, in which case, the Company will combine them into a single performance obligation. The Company has determined that the assets created have no alternative use to the Company, and the Company has an enforceable right to payment for the performance completed to date, therefore revenue related to these services are recognized over time and is measured using an output method based on performance of manufacturing milestones completed to date. Manufacturing service contracts may also include performance obligations related to project management services or obtaining materials from third parties. The Company has determined that these are separate performance obligations for which revenue is recognized at the point in time the services are performed. For performance obligations related to obtaining third party materials, the Company has determined that it is the principal as the Company has control of the materials and has discretion in setting the price. Therefore, the Company recognizes revenue on a gross basis related to obtaining third party materials. Certain arrangements require a portion of the contract consideration to be received in advance at the commencement of the contract, and such advance payment is initially recorded as a contract liability. A contract asset may be recognized in the event the Company’s satisfaction of performance obligations outpaces customer billings. For the three months ended March 31, 2024 and 2023, the Company recognized $1.4 million and $2.0 million of manufacturing revenue, respectively, related to plasmid DNA and AAV vector production for external customers. As of March 31, 2024, the Company has contract assets of $0.8 million and no remaining performance obligations related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. For the period ended December 31, 2023, the Company had contract assets of $0.2 million and remaining performance obligations of $0.8 million related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. Allowance for doubtful accounts The Company maintains an allowance for estimated losses resulting from the inability of its customers to make required payments. The Company estimates uncollectible amounts based upon current customer receivable balances, the age of customer receivable balances, the customer’s financial condition and current economic trends. The Company also assesses whether an allowance for expected credit losses may be required which includes a review of the Company’s receivables portfolio, which are pooled on a customer basis or country basis. In making its assessment of whether an allowance for credit losses is required, the Company considers its historical experience with customers, current balances, levels of delinquency, regulatory and legal environments, and other relevant current and future forecasted economic conditions. For the three months ended March 31, 2024 and 2023, no allowance was recorded for credit losses. The allowance for doubtful accounts was $1.1 million as of March 31, 2024, and $1.2 million as of December 31, 2023. For the three months ended March 31, 2024 and 2023, bad debt expense was immaterial. Liability for sale of future royalties On July 17, 2020, the Company, RPI Intermediate Finance Trust (“RPI”), and, for the limited purposes set forth in the agreement, Royalty Pharma PLC, entered into a royalty purchase agreement (the “Original Royalty Purchase Agreement”). Pursuant to the Original Royalty Purchase Agreement, the Company sold to RPI 42.933% (the “ Original Assigned Royalty Rights”) of the Company’s right to receive sales-based royalty payments (the “Royalty”) on worldwide net sales of Evrysdi and any other product developed pursuant to the License and Collaboration Agreement (the “License Agreement”), dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation under the SMA program. In consideration for the sale of the Original Assigned Royalty Rights, RPI paid the Company $650.0 million in cash consideration. The Company has retained a 57.067% interest in the Royalty and all economic rights to receive the remaining potential regulatory and sales milestone payments under the License Agreement, which remaining milestone payments equal $150.0 million in the aggregate as of March 31, 2024. The Original Royalty Purchase Agreement was set to terminate 60 days following the earlier of the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement and the date on which RPI has received $1.3 billion in respect of the Original Assigned Royalty Rights. Pursuant to the guidance in ASC 470-10-25-2, the Company determined that the $650.0 million cash consideration obtained pursuant to the Original Royalty Purchase Agreement should be classified as debt and recorded it as “liability for sale of future royalties-current” and “liability for sale of future royalties-noncurrent” on the Company’s consolidated balance sheet based on the timing of the expected payments to be made to RPI at the time of the transaction. The liability was subsequently amortized using the effective interest method over the life of the arrangement, in accordance with the respective guidance, utilizing the prospective method to account for subsequent changes in the estimated future payments to be made to RPI. On October 18, 2023, the Company, Royalty Pharma Investments 2019 ICAV (“Royalty Pharma”), and, for the limited purposes set forth in the agreement, Royalty Pharma plc, entered into an Amended and Restated Royalty Purchase Agreement (the “A&R Royalty Purchase Agreement”), which amends and restates in its entirety the Original Royalty Purchase Agreement. Pursuant to the A&R Royalty Purchase Agreement, the Company has sold or agreed to sell to Royalty Pharma certain portions of the Company’s remaining Royalty on worldwide net sales of Evrysdi and any other product (the “Products”) developed pursuant to the SMA License Agreement (all such retained Royalty rights of the Company, the “Retained Royalty Rights,” and all such Royalty rights that are sold to Royalty Pharma pursuant to the A&R Royalty Purchase Agreement, the (“A&R Assigned Royalty Rights”). At closing, Royalty Pharma paid the Company $1.0 billion in cash consideration for 38.0447% of the Company’s Retained Royalty Rights (which is in addition to the 42.9330% assigned to Royalty Pharma in connection with the Original Royalty Purchase Agreement, for a total of 80.9777% of the total Royalty) until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 66.6667% of the total Royalty. In addition, the Company may sell to Royalty Pharma the remainder of the Company’s Retained Royalty Rights in exchange for an aggregate of $500.0 million in additional cash consideration after the closing of the A&R Royalty Purchase Agreement, less royalties received in respect of the Retained Royalty Rights put to Royalty Pharma, which will be payable by Royalty Pharma pursuant to five put options held by the Company that are exercisable at the Company’s option between January 1, 2024 and December 31, 2025. If the Company exercises two or fewer of the put options, Royalty Pharma may exercise a call option during the period from and after January 1, 2026 until and including March 31, 2026 for up to 50% of the remainder of the Company’s Retained Royalty Rights less amounts exercised by the Company via its put options at a purchase price that is proportional to the purchase price of the Company’s unexercised put options. Royalty Pharma’s exercise of the call option would result in Royalty Pharma owning 90.4888% of the total Royalty until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 83.3333% of the total Royalty. The A&R Royalty Purchase Agreement will terminate 60 days following the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement. The change in rights and obligations from the A&R Royalty Purchase Agreement resulted in a change in the terms of the liability for sale of future royalties, which was evaluated by the Company in accordance with ASC 470-50, Debt —Modifications and Extinguishments. The Company determined that the present value of the cash flows under the A&R Royalty Purchase Agreement were substantially different from the present value of the cash flows under the Original Royalty Purchase Agreement. This resulted in the derecognition of the old liability for sale of future royalties and the new liability for sale of future royalties being recorded at fair value, which was determined to be $1,809.9 million as of the date of the A&R Royalty Purchase Agreement. This resulted in an extinguishment loss of $44.9 million, which was recorded within loss on extinguishment of debt, within the Company’s statement of operations for the year ended December 31, 2023. The fair value for the new liability for sale of future royalties on the date of the A&R Royalty Purchase Agreement was based on the Company’s estimates of future royalties expected to be paid to Royalty Pharma over the life of the arrangement, which was determined using forecasts from market data sources, which are considered Level 3 inputs. The liability is being amortized using the effective interest method over the life of the arrangement, in accordance with ASC 470 and ASC 835. The initial annual effective interest rate was determined to be 10.8%. The Company utilizes the prospective method to account for subsequent changes in the estimated future payments to be made to Royalty Pharma and updates the effective interest rate on a quarterly basis. Issuance costs related to the transaction were determined to be immaterial. Refer to Note 9 for further details. Indefinite-lived intangible assets Indefinite-lived intangible assets consist of in process research and development ("IPR&D"). IPR&D acquired directly in a transaction other than a business combination is capitalized if the projects will be further developed or have an alternative future use; otherwise they are expensed. The fair values of IPR&D projects and license agreement assets acquired in business combinations are capitalized. Several methods may be used to determine the estimated fair value of the IPR&D and license agreement asset acquired in a business combination. The Company utilizes the "income method" and uses estimated future net cash flows that are derived from projected sales revenues and estimated costs. These projections are based on factors such as relevant market size, patent protection, and expected pricing and industry trends. The estimated future net cash flows are then discounted to the present value using an appropriate discount rate. These assets are treated as indefinite-lived intangible assets until completion or abandonment of the projects, at which time the assets are amortized over the remaining useful life or written off, as appropriate. Intangible assets with indefinite lives, including IPR&D, are tested for impairment if impairment indicators arise and, at a minimum, annually. However, an entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that an indefinite-lived intangible asset’s fair value is less than its carrying amount. Otherwise, no further impairment testing is required. The indefinite-lived intangible asset impairment test consists of a one-step analysis that compares the fair value of the intangible asset with its carrying amount. If the carrying amount of an intangible asset exceeds its fair value, an impairment loss is recognized in an amount equal to that excess. The Company considers many factors in evaluating whether the value of its intangible assets with indefinite lives may not be recoverable, including, but not limited to, expected growth rates, the cost of equity and debt capital, general economic conditions, the Company’s outlook and market performance of the Company’s industry and recent and forecasted financial performance. Goodwill Goodwill represents the amount of consideration paid in excess of the fair value of net assets acquired as a result of the Company’s business acquisitions accounted for using the acquisition method of accounting. Goodwill is not amortized and is subject to impairment testing at a reporting unit level on an annual basis or when a triggering event occurs that may indicate the carrying value of the goodwill is impaired. The Company reassesses its reporting units as part of its annual segment review. An entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that the fair value of the reporting unit is less than its carrying amount. Income Taxes On December 15, 2022, the EU Member States formally adopted the EU’s Pillar Two Directive, which generally provides for a minimum effective tax rate of 15%, as established by the Organization for Economic Co-operation and Development (“OECD”) Pillar Two Framework that was supported by over 130 countries worldwide. The EU effective dates are January 1, 2024, and January 1, 2025, for different aspects of the directive. A significant number of other countries are also implementing similar legislation. As a result, the tax laws in the U.S. and other countries in which PTC and its affiliates do business could change on a prospective or retroactive basis and any such changes could materially adversely affect the Company’s business. The Company is continuing to evaluate the potential impact on future periods of the Pillar Two Framework, pending legislative adoption by additional individual countries, including those within the European Union. On December 22, 2017, the U.S. government enacted the 2017 Tax Act, which significantly revised U.S. tax law by, among other provisions, lowering the U.S. federal statutory corporate income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Global Intangible Low-Taxed Income (“GILTI”) provisions of the 2017 Tax Act require the Company to include in its U.S. income tax return foreign subsidiary earnings in excess of an allowable return on the foreign subsidiary’s tangible assets. The Company has elected to account for GILTI tax in the period in which it is incurred, and therefore has not provided any deferred tax imp

Leases

Leases	3 Months Ended
Leases	Mar. 31, 2024
Leases
Leases	3. Leases The Company leases office space in South Plainfield, New Jersey for its principal office under two noncancelable operating leases through August 2024, in addition to office and laboratory space in Bridgewater, New Jersey and other locations throughout the United States and office space in various countries for international employees primarily through workspace providers. Effective April 2024, the Company will utilize the Warren Premises, as described below, as its principal office space. The Company also leases approximately 220,500 square feet of office, manufacturing and laboratory space at a facility located in Hopewell Township, New Jersey pursuant to a Lease Agreement (the “Hopewell Lease”) with Hopewell Campus Owner LLC. The rental term of the Hopewell Lease commenced on July 1, 2020 and has an initial term of fifteen years (the “Hopewell Initial Term”), with two consecutive ten year renewal periods, each at the Company’s option. The aggregate rent for the Hopewell Initial Term will be approximately $111.5 million. The rental rate for the renewal periods will be 95% of the Prevailing Market Rate (as defined in the Hopewell Lease) and determined at the time of the exercise of the renewal. The Company is also responsible for maintaining certain insurance and the payment of proportional taxes, utilities and common area operating expenses. The Hopewell Lease contains customary events of default, representations, warranties and covenants. In May 2022, the Company entered into a Lease Agreement (the “Warren Lease”) with Warren CC Acquisitions, LLC (the “Warren Landlord”) relating to the lease of two entire buildings comprised of approximately 360,000 square feet of shell condition, modifiable space (the “Warren Premises”) at a facility located in Warren, New Jersey. The rental term of the Warren Lease commenced on June 1, 2022, with an initial term of seventeen years (the “Warren Initial Term”), followed by three consecutive five-year renewal periods at the Company’s option. The aggregate base rent for the Warren Initial Term will be approximately $163.0 million; provided, however, that if the Company is not subject to an Event of Default (as defined in the Warren Lease), the Company will be entitled to a base rent abatement over the first three years of the Warren Initial Term of approximately $18.6 million, reducing the Company’s total base rent obligation to $144.4 million. The rental rate for the renewal periods will be at the Fair Market Rental Value (as defined in the Warren Lease) and determined at the time of the exercise of the renewal. Beginning in the second lease year, the Company is also responsible for the payment of all taxes and operating expenses for the Warren Premises. As a result, the Company recorded an operating lease ROU asset of $28.9 million and an operating lease ROU liability of $28.9 million as of the commencement date. The Company is entitled to an allowance of approximately $36.2 million to be provided by the Warren Landlord to be used towards such improvements. The Landlord is providing the allowance to cover those assets that are real property improvements, such as structural components, roofs, flooring, etc., whose useful lives are typically longer in nature. The Company evaluated the leasehold improvements under ASC 842 and determined that the Company will be the owner of the improvements, and therefore the $36.2 million allowance and $5.0 million due from the Landlord were treated as lease incentives at the commencement of the lease and included in the calculation of the lease ROU asset and lease ROU liability, effectively reducing both at Commencement Date. In connection with the execution of the Warren Lease, the Company also committed to fund a construction account with $3.6 million to go towards the Company’s improvements of the Warren Premises. Subject to the terms of the Warren Lease, the Company has a right of first offer to purchase the Warren Premises if the Warren Landlord receives a bona fide third party offer to purchase the Warren Premises or the Warren Landlord decides to sell the Warren Premises. The Company also has a finance lease related to its commercial manufacturing agreement with MassBiologics of the University of Massachusetts Medical School (“MassBio”). As of March 31, 2024, the balance of the finance lease liabilities-current and finance lease liabilities-noncurrent are $1.9 million and $15.6 million, respectively, and are directly related to the Company’s MassBio agreement. As of December 31, 2023, the balance of the finance lease liabilities-current and finance lease liabilities-noncurrent were $3.0 million and $17.2 million, respectively. Additionally, the Company recorded finance lease costs of $0.3 million and $0.4 million related to interest on the lease liability during the three months ended March 31, 2024 and 2023, respectively. The Company also leases certain vehicles, lab equipment, and office equipment under operating leases. The Company’s leases have remaining operating lease terms ranging from 0.2 years to 15.2 years and certain of the leases include renewal options to extend the lease for up to 15 years. Rent expense was $6.9 million and $7.1 million for the three months ended March 31, 2024 and 2023, respectively. The components of operating lease expense were as follows: Three Months Ended Three Months Ended March 31, 2024 March 31, 2023 Operating Lease Cost Fixed lease cost $ 5,575 $ 5,473 Variable lease cost 1,069 1,353 Short-term lease cost 214 303 Total operating lease cost $ 6,858 $ 7,129 Total operating lease cost is a component of operating expenses on the consolidated statements of operations. Supplemental lease term and discount rate information related to leases was as follows as March 31, 2024 and December 31, 2023: March 31, 2024 December 31, 2023 Weighted-average remaining lease terms - operating leases (years) 11.66 11.55 Weighted-average discount rate - operating leases 8.75 % 8.69 % Weighted-average remaining lease terms - finance lease (years) 8.76 9.01 Weighted-average discount rate - finance lease 7.80 % 7.80 % Supplemental cash flow information related to leases was as follows as of March 31, 2024 and 2023: Three Months Ended March 31, 2024 2023 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 3,948 $ 3,811 Financing cash flows from finance lease 1,490 1,379 Operating cash flows from finance lease 1,510 1,621 Right-of-use assets obtained in exchange for lease obligations: Operating leases $ 1,723 $ — Future minimum lease payments under non-cancelable leases as of March 31, 2024 were as follows: Operating Leases Finance Lease 2024 (excludes the three months ended March 31, 2024) $ 14,981 $ — 2025 20,995 3,000 2026 20,539 3,000 2027 18,400 3,000 2028 and thereafter 175,853 15,000 Total lease payments 250,768 24,000 Less: Imputed Interest expense 131,359 6,478 Total $ 119,409 $ 17,522

Fair value of financial instrum

Fair value of financial instruments and marketable securities	3 Months Ended
	Mar. 31, 2024
Fair Value Disclosures [Abstract]
Fair value of financial instruments and marketable securities	4. Fair value of financial instruments and marketable securities The Company follows the fair value measurement rules, which provide ● Level 1—Unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the balance sheet date. ● Level 2—Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs include quoted prices for similar assets and liabilities in active markets, quoted prices for identical or similar assets or liabilities in markets that are not active, inputs other than quoted prices that are observable for the asset or liability (i.e., interest rates, yield curves, etc.), and inputs that are derived principally from or corroborated by observable market data by correlation or other means (market corroborated inputs). ● Level 3—Inputs are unobservable and reflect the Company’s assumptions as to what market participants would use in pricing the asset or liability. The Company develops these inputs based on the best information available. Cash equivalents and marketable securities are reflected in the accompanying financial statements at fair value. The carrying amount of receivables and accounts payable and accrued expenses approximates fair value due to the short-term nature of those instruments. The Company owns common stock in ClearPoint Neuro, Inc. (“ClearPoint”) (formerly MRI Interventions, Inc.), a publicly traded medical device company. The ClearPoint equity investments (collectively, the “ClearPoint Equity Investments”) represent financial instruments, and therefore, are recorded at fair value, which is readily determinable. The ClearPoint Equity Investments are components of prepaids and other current assets on the consolidated balance sheet as of March 31, 2024 and December 31, 2023. In January 2020, the Company purchased a $10.0 million convertible note from ClearPoint that the Company can convert into ClearPoint shares at a conversion rate of $6.00 per share at any point throughout the term of the loan, which matures five years from the purchase date. The Company determined that the convertible note represents an available for sale debt security and the Company has elected to record it at fair value under ASC 825. The Company classifies its ClearPoint convertible debt security as a Level 2 asset within the fair value hierarchy, as the value is based on inputs other than quoted prices that are observable. The fair value of the ClearPoint convertible debt security is determined at each reporting period by utilizing a Black-Scholes option pricing model, as well as a present value of expected cash flows from the debt security utilizing the risk free rate and the estimated credit spread as of the valuation date as the discount rate. The convertible debt security is included as a component of prepaids and other current assets on the consolidated balance sheet as of March 31, 2024 and as a component of deposits and other assets as of December 31, 2023. Other than the ClearPoint Equity Investments and the ClearPoint convertible debt security, no other items included in prepaids and other current assets on the consolidated balance sheets are fair valued. The Company has investments in mutual funds, including one that is denominated in a foreign currency. All of these are equity investments and are classified as marketable securities on the Company’s consolidated balance sheets. These equity investments are reported at fair value, as they are readily available, and as such are classified as Level 1 assets. Unrealized holding gains and losses for these equity investments are included as components of other expense, net within the consolidated statement of operations. The table presented below is a summary of changes in the fair value for the Company’s marketable securities – equity investments, ClearPoint Equity Investments, and ClearPoint convertible debt security for the three months ended March 31, 2024 and March 31, 2023: Ending Foreign Ending Balance at Currency Balance at December 31, Unrealized Unrealized Investments Redemptions/ March 31, 2023 Gain Loss Purchased Sale 2024 Marketable securities - equity investments $ 22,634 710 (823) 9,065 (1,207) $ 30,379 ClearPoint Equity Investments 6,074 9 — — — 6,083 ClearPoint convertible debt security 12,553 166 — — — 12,719 Total Fair Value $ 41,261 $ 885 $ (823) $ 9,065 $ (1,207) $ 49,181 Ending Foreign Ending Balance at Currency Balance at December 31, Unrealized Unrealized Investments Redemptions/ March 31, 2022 Gain/(Loss) Gain Purchased Sale 2023 Marketable securities - equity investments $ 108,261 2,166 328 — (2,196) $ 108,559 ClearPoint Equity Investments 10,965 (39) — — — 10,926 ClearPoint convertible debt security 15,231 59 — — — 15,290 Total Fair Value $ 134,457 $ 2,186 $ 328 $ — $ (2,196) $ 134,775 Fair value of marketable securities that are classified as available for sale debt securities is based upon market prices using quoted prices in active markets for identical assets quoted on the last day of the period. In establishing the estimated fair value of the remaining available for sale debt securities, the Company used the fair value as determined by its investment advisors using observable inputs other than quoted prices. The following represents the fair value using the hierarchy described above for the Company’s financial assets and liabilities that are required to be measured at fair value on a recurring basis as of March 31, 2024 and December 31, 2023: March 31, 2024 Quoted prices Significant in active other Significant markets for observable unobservable identical assets inputs inputs Total (level 1) (level 2) (level 3) Marketable securities - available for sale $ 306,079 $ — $ 306,079 $ — Marketable securities - equity investments $ 30,379 $ 30,379 $ — $ — ClearPoint Equity Investments $ 6,083 $ 6,083 $ — $ — ClearPoint convertible debt security $ 12,719 $ — $ 12,719 $ — Contingent consideration payable- development and regulatory milestones $ 26,900 $ — $ — $ 26,900 Contingent consideration payable- net sales milestones $ 9,300 $ — $ — $ 9,300 December 31, 2023 Quoted prices Significant in active other Significant markets for observable unobservable identical assets inputs inputs Total (level 1) (level 2) (level 3) Marketable securities - available for sale $ 260,104 $ — $ 260,104 $ — Marketable securities - equity investments $ 22,634 $ 22,634 $ — $ — ClearPoint Equity Investments $ 6,074 $ 6,074 $ — $ — ClearPoint convertible debt security $ 12,553 $ — $ 12,553 $ — Contingent consideration payable- development and regulatory milestones $ 26,600 $ — $ — $ 26,600 Contingent consideration payable- net sales milestones and royalties $ 9,700 $ — $ — $ 9,700 No transfers of assets between Level 1, Level 2, or Level 3 of the fair value measurement hierarchy occurred during the periods ended March 31, 2024 and December 31, 2023. The following is a summary of marketable securities accounted for as available for sale debt securities at March 31, 2024 and December 31, 2023: March 31, 2024 Amortized Gross Unrealized Cost Gains Losses Fair Value Commercial paper $ 132,080 $ 26 $ (121) $ 131,985 Government Obligations 174,118 41 (65) 174,094 Total $ 306,198 $ 67 $ (186) $ 306,079 December 31, 2023 Amortized Gross Unrealized Cost Gains Losses Fair Value Commercial paper $ 117,044 $ 128 $ (12) $ 117,160 Corporate debt securities 1,650 — (2) 1,648 Government Obligations 141,084 212 — 141,296 Total $ 259,778 $ 340 $ (14) $ 260,104 For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. For the three months ended March 31, 2024 and 2023, no write downs occurred. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis, which may be maturity. The Company also reviews its available for sale debt securities in an unrealized loss position and evaluates whether the decline in fair value has resulted from credit losses or other factors. This review is subjective, as it requires management to evaluate whether an event or change in circumstances has occurred in that period that may be related to credit issues. For the three months ended March 31, 2024 and 2023, no allowance was recorded for credit losses. Unrealized gains and losses are reported as a component of accumulated other comprehensive (loss) income in stockholders’ deficit. For the three months ended March 31, 2024 and 2023, the Company did not have any realized gains or losses from the sale of available for sale debt securities. Realized gains and losses are reported as a component of interest expense, net in the consolidated statement of operations. Reclassified amounts from other comprehensive items were determined using the actual realized gains and losses from the sales of marketable securities. The unrealized losses and fair values of available for sale debt securities that have been in an unrealized loss position for a period of less than and greater than or equal to 12 months as of March 31, 2024 are as follows: March 31, 2024 Securities in an unrealized loss Securities in an unrealized loss position less than 12 months position greater than or equal to 12 months Total Unrealized losses Fair Value Unrealized losses Fair Value Unrealized losses Fair Value Commercial paper $ (121) 73,250 — — (121) 73,250 Government bonds $ (65) 40,302 — — (65) $ 40,302 Total $ (186) $ 113,552 $ — $ — $ (186) $ 113,552 The unrealized losses and fair values of available for sale debt securities that have been in an unrealized loss position for a period of less than and greater than or equal to 12 months as of December 31, 2023 are as follows: December 31, 2023 Securities in an unrealized loss Securities in an unrealized loss position less than 12 months position greater than or equal to 12 months Total Unrealized losses Fair Value Unrealized losses Fair Value Unrealized losses Fair Value Commercial paper $ (12) 44,446 — — (12) 44,446 Corporate debt securities $ — — (2) 1,648 (2) $ 1,648 Total $ (12) $ 44,446 $ (2) $ 1,648 $ (14) $ 46,094 Available for sale debt securities at March 31, 2024 and December 31, 2023 mature as follows: March 31, 2024 Less Than More Than 12 Months 12 Months Commercial paper $ 131,985 $ — Government obligations 174,094 — Total $ 306,079 $ — December 31, 2023 Less Than More Than 12 Months 12 Months Commercial paper $ 117,160 $ — Corporate debt securities 1,648 — Government obligations 141,296 — Total $ 260,104 $ — The Company classifies all of its marketable securities as current as they are all either available for sale debt securities or equity investments and are available for current operations. Convertible senior notes In September 2019, the Company issued $287.5 million of 1.50% convertible senior notes due September 15, 2026 (the “2026 Convertible Notes,”). The fair value of the 2026 Convertible Notes, which differs from their carrying values, is influenced by interest rates, the Company’s stock price and stock price volatility and is determined by prices for the 2026 Convertible Notes observed in market trading which are Level 2 inputs. The estimated fair value of the 2026 Convertible Notes at March 31, 2024 and December 31, 2023 was $267.7 million and $265.3 million, respectively. Level 3 valuation The contingent consideration payable is fair valued each reporting period with the change in fair value recorded as a gain or loss within the change in the fair value of contingent consideration on the consolidated statements of operations. The fair value of the development and regulatory milestones is estimated utilizing a probability adjusted, discounted cash flow approach. The discount rates are estimated utilizing Corporate B rated bonds maturing in the years of expected payments based on the Company’s estimated development timelines for the acquired product candidate. The fair value of the net sales milestones is determined utilizing a valuation framework that estimates net sales volatility to simulate a range of possible payment scenarios. The average of the payments in these scenarios is then discounted to calculate present fair value. As of March 31, 2024, the contingent consideration balance, consisting solely of the Upstaza intangible asset, is $36.2 million. As of March 31, 2024, the weighted average discount rate for the Upstaza development and regulatory milestones was 6.0% and the weighted average probability of success was 90%. As of March 31, 2024, the weighted average discount rate for the Upstaza net sales milestones was 13.5% and the weighted average probability of success for the net sales milestones was 93%. The table presented below is a summary of changes in the fair value of the Company’s Level 3 valuations for the contingent consideration payable for the periods ended March 31, 2024 and March 31, 2023: Level 3 liabilities Contingent consideration payable- Contingent consideration payable- development and regulatory net sales milestones and royalties milestones Beginning balance as of December 31, 2023 $ 26,600 $ 9,700 Additions — — Change in fair value 300 (400) Payments — — Ending balance as of March 31, 2024 $ 26,900 $ 9,300 Level 3 liabilities Contingent consideration payable- Contingent consideration payable- development and regulatory net sales milestones and royalties milestones Beginning balance as of December 31, 2022 $ 82,500 $ 81,500 Additions — — Change in fair value 3,000 (600) Payments — — Ending balance as of March 31, 2023 $ 85,500 $ 80,900 The following significant unobservable inputs were used in the valuation of the contingent consideration payable for the periods ended March 31, 2024 and December 31, 2023: March 31, 2024 Fair Value Valuation Technique Unobservable Input Range Contingent consideration payable- $26,900 Probability-adjusted discounted cash flow Potential development and regulatory milestones $0 - $31 million Contingent considerable payable- net sales $9,300 Option-pricing model with Monte Carlo simulation Potential net sales milestones $0 - $50 million December 31, 2023 Fair Value Valuation Technique Unobservable Input Range Contingent consideration payable- $26,600 Probability-adjusted discounted cash flow Potential development and regulatory milestones $0 - $31 million Contingent considerable payable- net sales $9,700 Option-pricing model with Monte Carlo simulation Potential net sales milestones $0 - $50 million The contingent consideration payables are classified Level 3 liabilities as their valuation requires substantial judgment and estimation of factors that are not currently observable in the market. If different assumptions were used for the various inputs to the valuation approaches, including but not limited to, assumptions involving probability adjusted sales estimates for the gene therapy platform and estimated discount rates, the estimated fair value could be significantly higher or lower than the fair value determined.

Accounts payable and accrued ex

Accounts payable and accrued expenses	3 Months Ended
Accounts payable and accrued expenses	Mar. 31, 2024
Payables and Accruals [Abstract]
Accounts payable and accrued expenses	5. Accounts payable and accrued expenses Accounts payable and accrued expenses at March 31, 2024 and December 31, 2023 consist of the following: March 31, December 31, 2024 2023 Employee compensation, benefits, and related accruals $ 35,060 $ 62,643 Income tax payable 1,833 — Consulting and contracted research 22,745 27,500 Professional fees 2,380 2,246 Sales allowance 67,075 77,176 Sales rebates 132,047 131,334 Royalties 28,952 74,111 Accounts payable 27,459 6,045 Other 13,373 10,928 Total $ 330,924 $ 391,983 As of March 31, 2024 and December 31, 2023, there were $1.7 million and $9.0 million, respectively, of accrued restructuring costs included above within employee compensation, benefits, and related accruals from a reduction in workforce in the year ended December 31, 2023 in connection with the Company’s strategic pipeline prioritization and discontinuation of its preclinical and early research programs in its gene therapy platform.

Capitalization

Capitalization	3 Months Ended
Capitalization	Mar. 31, 2024
Stockholders' Equity Note [Abstract]
Capital structure	6. Capitalization In August 2019, the Company entered into an At the Market Offering Sales Agreement (the “Sales Agreement”) with Cantor Fitzgerald and RBC Capital Markets, LLC (together, the “Sales Agents”), pursuant to which, the Company may offer and sell shares of its common stock, having an aggregate offering price of up to $125.0 million from time to time through the Sales Agents by any method that is deemed to be an “at the market offering” as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended. No shares were sold during the three months ended March 31, 2024 and 2023. The remaining shares of the Company’s common stock available to be issued and sold, under the At the Market Offering, have an aggregate offering price of up to $93.0 million as of March 31, 2024.

Net loss per share

Net loss per share	3 Months Ended
Net loss per share	Mar. 31, 2024
Earnings Per Share [Abstract]
Net loss per share	7. Net loss per share Basic and diluted net loss per share is computed by dividing net loss by the weighted-average number of common shares outstanding. Potentially dilutive securities were excluded from the diluted calculation because their effect would be anti-dilutive. The following tables set forth the computation of basic and diluted net loss per share: Three Months Ended March 31, 2024 2023 Numerator Net loss $ (91,576) $ (138,959) Denominator Denominator for basic and diluted net loss per share 76,496,127 73,729,284 Net loss per share: Basic and diluted $ (1.20) * $ (1.88) * * In the three months ended March 31, 2024 and 2023, the Company experienced a net loss and therefore did not report any dilutive share impact. The following table shows historical dilutive common share equivalents outstanding, which are not included in the above historical calculation, as the effect of their inclusion is anti-dilutive during each period. As of March 31, 2024 2023 Stock Options 9,530,185 12,100,550 Unvested restricted stock awards and units 3,730,166 3,649,742 Total 13,260,351 15,750,292

Stock award plan

Stock award plan	3 Months Ended
Stock award plan	Mar. 31, 2024
Share-based Payment Arrangement [Abstract]
Stock award plan	8. Stock award plan In May 2013, the Company’s Board of Directors and stockholders approved the 2013 Long-Term Incentive Plan, which became effective upon the closing of the Company’s initial public offering. On June 8, 2022 (the “Restatement Effective Date”), the Company’s stockholders approved the Amended and Restated 2013 Long-Term Incentive Plan (the “Amended 2013 LTIP”). The Amended 2013 LTIP provides for the grant of incentive stock options, nonstatutory stock options, restricted stock units and other stock-based awards. The number of shares of common stock reserved for issuance under the Amended 2013 LTIP is the sum of (A) the number of shares of the Company’s common stock (up to 16,724,212 shares) that is equal to the sum of (1) the number of shares issued under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date, (2) the number of shares that remain available for issuance under the 2013 Long-Term Incentive Plan immediately prior to the Restatement Effective Date and (3) the number of shares subject to awards granted under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date that are outstanding as of the Restatement Effective Date, plus (B) from and after the Restatement Effective Date, an additional 8,475,000 shares of Common Stock. As of March 31, 2024, awards for 6,618,698 shares of common stock are available for issuance under the Amended 2013 LTIP. There are no additional shares of common stock available for issuance under the Company’s 1998 Employee, Director and Consultant Stock Option Plan, 2009 Equity and Long Term Incentive Plan or 2013 Stock Incentive Plan. In January 2020, the Company’s Board of Directors approved the 2020 Inducement Stock Incentive Plan. The 2020 Inducement Stock Incentive Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards and other stock-based awards for, initially, up to at the time, an aggregate of 1,000,000 shares of common stock. Any grants made under the 2020 Inducement Stock Incentive Plan must be made pursuant to the Nasdaq Listing Rule 5635(c)(4) inducement grant exception as a material component of the Company’s new hires’ employment compensation. In December 2020, the Company’s Board of Directors approved an additional 1,000,000 shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan. In April 2022, the Company’s Board of Directors approved a reduction in the total number of shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan to 1,300,000 shares. In December 2022, the Company’s Board of Directors approved an additional 1,700,000 shares of common stock that may be issued under the 2020 Inducement Stock Incentive Plan. The Board of Directors has the authority to select the individuals to whom options are granted and determine the terms of each option, including (i) the number of shares of common stock subject to the option; (ii) the date on which the option becomes exercisable; (iii) the option exercise price, which, in the case of incentive stock options, must be at least 100% (110% in the case of incentive stock options granted to a stockholder owning in excess of 10% of the Company’s stock) of the fair market value of the common stock as of the date of grant; and (iv) the duration of the option (which, in the case of incentive stock options, may not exceed ten years). Options typically vest over a four-year period. Inducement stock option awards From January 1, 2024 through March 31, 2024, the Company issued a total of 855,385 stock options to various employees. Of those, 1,840 were inducement grants for non-statutory stock options, all of which were made pursuant to the 2020 Inducement Stock Incentive Plan. Stock option activity Weighted- Weighted- average Aggregate average remaining intrinsic Number of exercise contractual value(in options price term thousands) Outstanding at December 31, 2023 9,600,399 $ 43.59 Granted 855,385 $ 25.69 Exercised (109,892) $ 19.22 Forfeited/Cancelled (815,707) $ 45.32 Outstanding at March 31, 2024 9,530,185 $ 42.12 5.90 years $ 12,025 Vested or Expected to vest at March 31, 2024 2,112,348 $ 38.57 8.67 years $ 2,949 Exercisable at March 31, 2024 7,173,552 $ 43.44 4.98 years $ 8,598 The fair value of grants made in the three months ended March 31, 2024 was contemporaneously estimated on the date of grant using the following assumptions: Three months ended March 31, 2024 Risk-free interest rate 4.23% Expected volatility 53% Expected term 5.5 years The Company assumed no expected dividends for all grants. The weighted average grant date fair value of options granted during the three months ended March 31, 2024 was $13.49 per share. The expected term of options was estimated based on the Company’s historical exercise data and the expected volatility of options was estimated based on the Company’s historical stock volatility. The risk-free rate of the options was based on U.S. Government Securities Treasury Constant Maturities yields at the date of grant for a term similar to the expected term of the option. Restricted Stock Awards and Restricted Stock Units the Company issued a total of 1,785,660 restricted stock units to various employees. Of those, 4,275 were inducement grants for restricted stock units, all of which were made pursuant to the 2020 Inducement Stock Incentive Plan. The following table summarizes information on the Company’s restricted stock awards and units: Restricted Stock Awards and Units Weighted Average Grant Number of Date Shares Fair Value Unvested at December 31, 2023 2,866,270 $ 41.82 Granted 1,785,660 25.69 Vested (835,179) 44.56 Forfeited (86,585) 40.98 Unvested at March 31, 2024 3,730,166 $ 33.51 Performance-based Restricted Stock Units— Employee Stock Purchase Plan The Company recorded share-based compensation expense in the statement of operations related to incentive stock options, nonstatutory stock options, restricted stock awards, restricted stock units and the ESPP as follows: Three Months Ended March 31, 2024 2023 Research and development $ 8,967 $ 15,314 Selling, general and administrative 9,411 13,501 Total $ 18,378 $ 28,815 As of March 31, 2024, there was approximately $156.3 million of total unrecognized compensation cost related to unvested share-based compensation arrangements granted under the Company’s equity award plans. This cost is expected to be recognized as share-based compensation expense over the weighted average remaining service period of approximately 2.4 years.

Debt

Debt	3 Months Ended
Debt	Mar. 31, 2024
Debt Disclosure [Abstract]
Debt	9. Debt Liability for sale of future royalties The following table shows the activity within the “liability for sale of future royalties- current” and “liability for sale of future royalties- noncurrent” accounts for the three months ended March 31, 2024: Three Months Ended March 31, Liability for sale of future royalties- (current and noncurrent) 2024 Beginning balance as of December 31, 2023 $ 1,814,097 Less: Non-cash royalty revenue payable to RPI (25,228) Plus: Non-cash interest expense recognized 50,064 Ending balance $ 1,838,933 Effective interest rate as of March 31, 2024 10.8% Non-cash interest expense is recorded in the statement of operations within “Interest expense, net”. Senior Secured Term Loan On October 27, 2022 (the “Closing Date”), the Company entered into a credit agreement (the “Blackstone Credit Agreement”) for fundings of up to $950.0 million consisting of a committed loan facility of $450.0 million and further contemplating the potential for up to $500.0 million of additional financing, to the extent that the Company requested such additional financing and subject to the Lenders’ agreement to provide such additional financing and to mutual agreement on terms, among the Company, certain subsidiaries of the Company (together with the Company, the “Loan Parties”) and funds and other affiliated entities advised or managed by Blackstone Life Sciences and Blackstone Credit (collectively, “Blackstone”, and such lenders, together with their permitted assignees, the “Lenders” and each a “Lender”) and Wilmington Trust, National Association, as the administrative agent for the Lenders. The Blackstone Credit Agreement provided for a senior secured term loan facility funded on the Closing Date in the aggregate principal amount of $300.0 million (the “Initial Loans”) and a committed delayed draw term loan facility of up to $150.0 million (the “Delayed Draw Loans” and, together with the Initial Loans, the “Loans”) to be funded at the Company’s request within 18 months of the Closing Date subject to specified conditions. In addition, the Blackstone Credit Agreement contemplated the potential for further financings by Blackstone, by providing for incremental discretionary uncommitted further financings of up to $500.0 million. The Company capitalized approximately $11.6 million of debt issuance costs which are presented on the balance sheet as a direct deduction from the debt liability and are being amortized over the term of the senior secured term loan facility using the effective interest rate method. The Loans were to mature on the date that is seven years from the Closing Date. Borrowings under the Blackstone Credit Agreement bore interest at a variable rate equal to, at the Company’s option, either an adjusted Term SOFR rate plus seven and a quarter percent (7.25%) or the Base Rate plus six and a quarter percent (6.25%) , subject to a floor of one percent (1%) and two percent (2%) with respect to Term SOFR rate and Base Rate (each as defined in the Blackstone Credit Agreement), respectively. Payment of the Loans were subject to certain premiums specified in the Blackstone Credit Agreement, in each case, from the date of the applicable Loan funded. On October 19, 2023, the Company terminated the Blackstone Credit Agreement. In connection with the termination of the Credit Agreement, the Company repaid outstanding principal of $300.0 million, accrued interest of $2.1 million, an additional $82.0 million in prepayment premiums, exit fees, and creditor expenses, and $0.2 million in legal fees. The Company recorded a loss on the extinguishment of debt of $92.7 million which was included on the statement of operations for the period ended December 31, 2023. The loss on extinguishment of debt consisted of $82.0 million in prepayment premiums, exit fees, and creditor expenses and debt issuance costs of $10.7 million. All liens and security interests securing the loans made pursuant to the Blackstone Credit Agreement were released upon termination. The Blackstone Credit Agreement consisted of the following: March 31, 2024 December 31, 2023 Principal $ — $ 300,000 Less: Debt issuance costs — — Repayment of senior secured term loan — (300,000) Net carrying amount $ — $ — The following table sets forth total interest expense recognized related to the Blackstone Credit Agreement: Three Months Ended Three Months Ended March 31, March 31, 2024 2023 Contractual interest expense $ — $ 9,178 Amortization of debt issuance costs — 150 Total $ — $ 9,328 Effective interest rate — % 13.2 % 2026 Convertible Notes In September 2019, the Company issued, at par value, $287.5 million aggregate principal amount of 1.50% convertible senior notes due 2026, which included an option to purchase up to an additional $37.5 million in aggregate principal amount of the 2026 Convertible Notes, which was exercised in full by the initial purchasers. The 2026 Convertible Notes bear cash interest at a rate of 1.50% per year, payable semi-annually on March 15 and September 15 of each year, beginning on March 15, 2020. The 2026 Convertible Notes will mature on September 15, 2026, unless earlier repurchased or converted. The net proceeds to the Company from the offering were $279.3 million after deducting the initial purchasers’ discounts and commissions and the offering expenses payable by the Company. The 2026 Convertible Notes are governed by an indenture (the "2026 Convertible Notes Indenture") with U.S Bank National Association as trustee (the "2026 Convertible Notes Trustee"). Holders of the 2026 Convertible Notes may convert their 2026 Convertible Notes at their option at any time prior to the close of business on the business day immediately preceding March 15, 2026 only under the following circumstances: ● during any calendar quarter commencing on or after December 31, 2019 (and only during such calendar quarter), if the last reported sale price of the Company’s common stock for at least 20 trading days (whether or not consecutive) during a period of 30 consecutive trading days ending on the last trading day of the immediately preceding calendar quarter is greater than or equal to 130% of the conversion price on each applicable trading day; ● during the five business day period after any five consecutive trading day period (the “measurement period”) in which the trading price (as defined in the 2026 Convertible Notes Indenture) per $1,000 principal amount of 2026 Convertible Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price of the Company’s common stock and the conversion rate on each such trading day; ● during any period after the Company has issued notice of redemption until the close of business on the scheduled trading day immediately preceding the relevant redemption date; or ● upon the occurrence of specified corporate events. On or after March 15, 2026, until the close of business on the business day immediately preceding the maturity date, holders may convert their 2026 Convertible Notes at any time, regardless of the foregoing circumstances. Upon conversion, the Company will pay or deliver, as the case may be, cash, shares of the Company’s common stock or any combination thereof at the Company’s election. The conversion rate for the 2026 Convertible Notes was initially, and remains, 19.0404 shares of the Company’s common stock per $1,000 principal amount of the 2026 Convertible Notes, which is equivalent to an initial conversion price of approximately $52.52 per share of the Company’s common stock. The conversion rate may be subject to adjustment in some events but will not be adjusted for any accrued and unpaid interest. The Company was not permitted to redeem the 2026 Convertible Notes prior to September 20, 2023. The Company may redeem for cash all or any portion of the 2026 Convertible Notes, at its option, if the last reported sale price of its common stock has been at least 130% of the conversion price then in effect on the last trading day of, and for at least 19 other trading days (whether or not consecutive) during, any 30 consecutive trading day period ending on, and including, the trading day immediately preceding the date on which the Company provides notice of redemption, at a redemption price equal to 100% of the principal amount of the 2026 Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date. No sinking fund is provided for the 2026 Convertible Notes, which means that the Company is not required to redeem or retire the 2026 Convertible Notes periodically. If the Company undergoes a “fundamental change” (as defined in the 2026 Convertible Notes Indenture), subject to certain conditions, holders of the 2026 Convertible Notes may require the Company to repurchase for cash all or part of their 2026 Convertible Notes at a repurchase price equal to 100% of the principal amount of the 2026 Convertible Notes to be repurchased, plus accrued and unpaid interest to, but excluding, the fundamental change repurchase date. The 2026 Convertible Notes represent senior unsecured obligations and will rank senior in right of payment to the Company’s future indebtedness that is expressly subordinated in right of payment to the notes, equal in right of payment to the Company’s existing and future unsecured indebtedness that is not so subordinated, effectively junior in right of payment to any of the Company’s secured indebtedness to the extent of the value of the assets securing such indebtedness, and structurally subordinated to all existing and future indebtedness and other liabilities (including trade payables) incurred by the Company’s subsidiaries. The 2026 Convertible Notes Indenture contains customary events of default with respect to the 2026 Convertible Notes, including that upon certain events of default (including the Company’s failure to make any payment of principal or interest on the 2026 Convertible Notes when due and payable) occurring and continuing, the 2026 Convertible Notes Trustee by notice to the Company, or the holders of at least 25% in principal amount of the outstanding 2026 Convertible Notes by notice to the Company and the Convertible Notes Trustee, may, and the 2026 Convertible Notes Trustee at the request of such holders (subject to the provisions of the 2026 Convertible Notes Indenture) shall, declare 100% of the principal of and accrued and unpaid interest, if any, on all the 2026 Convertible Notes to be due and payable. In case of certain events of bankruptcy, insolvency or reorganization, involving the Company or a significant subsidiary, 100% of the principal of and accrued and unpaid interest on the 2026 Convertible Notes will automatically become due and payable. Upon such a declaration of acceleration, such principal and accrued and unpaid interest, if any, will be due and payable immediately. The 2026 Convertible Notes consist of the following: March 31, 2024 December 31, 2023 Principal $ 287,500 $ 287,500 Less: Debt issuance costs (2,988) (3,287) Net carrying amount $ 284,512 $ 284,213 As of March 31, 2024, the remaining contractual life of the 2026 Convertible Notes is approximately 2.5 years. The following table sets forth total interest expense recognized related to the 2026 Convertible Notes: Three Months Ended March 31, Three Months Ended March 31, 2024 2023 Contractual interest expense $ 1,076 $ 1,069 Amortization of debt issuance costs 296 288 Total $ 1,372 $ 1,357 Effective interest rate 1.9 % 1.9 %

Commitments and contingencies

Commitments and contingencies	3 Months Ended
Commitments and contingencies	Mar. 31, 2024
Commitments and Contingencies Disclosure [Abstract]
Commitments and contingencies	10. Commitments and contingencies Under various agreements, the Company will be required to pay royalties and milestone payments upon the successful development and commercialization of products. The Company has entered into funding agreements with The Wellcome Trust Limited ("Wellcome Trust") for the research and development of small molecule compounds in connection with the Company’s oncology and antibacterial programs. As the Company has discontinued development under its antibacterial program, it no longer expects that milestone and royalty payments from the Company to Wellcome Trust will apply under that agreement, resulting in a change to the total amount of development and regulatory milestone payments the Company may become obligated to pay for this program. Under the oncology program funding agreement, to the extent that the Company develops and commercializes program intellectual property on a for-profit basis itself or in collaboration with a partner (provided the Company retains overall control of worldwide commercialization), the Company may become obligated to pay to Wellcome Trust development and regulatory milestone payments and single-digit royalties on sales of any research program product. The Company’s obligation to pay such royalties would continue on a country-by-country basis until the longer of the expiration of the last patent in the program intellectual property in such country covering the research program product and the expiration of market exclusivity of such product in such country. The Company made the first development milestone payment of $0.8 million to Wellcome Trust under the oncology platform funding agreement during the second quarter of 2016. During the year ended December 31, 2022, the Company incurred $2.5 million of development milestones in connection with the enrollment of patients in the registration-directed Phase 2/3 trial of unesbulin for the treatment of LMS, which is recorded in accounts payable and accrued expenses on the balance sheet and will be payable upon the earlier to occur of the first dose administered to the last patient enrolled in the study or the termination of dosing of all patients in the study. However, as part the Company's continuous platform review, the Company has decided to deprioritize its programs for unesbulin for the treatment of diffuse intrinsic pontine glioma and leiomyosarcoma. Accordingly, the Company no longer expects to pay additional milestones to Wellcome Trust under this agreement. The Company has also entered into a collaboration agreement with the SMA Foundation. The Company was obligated to pay the SMA Foundation single-digit royalties on worldwide net product sales of any collaboration product that is successfully developed and subsequently commercialized or, with respect to collaboration products the Company outlicenses, including Evrysdi, a specified percentage of certain payments the Company receives from its licensee. The Company was not obligated to make such payments unless and until annual sales of a collaboration product exceed a designated threshold. Pursuant to the asset purchase agreement ("Asset Purchase Agreement") between the Company and Marathon Pharmaceuticals, LLC (now known as Complete Pharma Holdings, LLC) (“Marathon”), Marathon is entitled to receive contingent payments from the Company based on annual net sales of Emflaza up to a specified aggregate maximum amount over the expected commercial life of the asset. Pursuant to the Agilis Merger Agreement, Agilis equityholders were previously entitled to receive contingent consideration payments from the Company based on (i) the achievement of certain development milestones up to an aggregate maximum amount of $60.0 million, (ii) the achievement of certain regulatory approval milestones together with a milestone payment following the receipt of a priority review voucher up to an aggregate maximum amount of $535.0 million, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $150.0 million, and (iv) a percentage of annual net sales for Friedreich ataxia and Angelman syndrome during specified terms, ranging from 2%-6%. The Company was required to pay $40.0 million of the development milestone payments upon the passing of the second anniversary of the closing of the Agilis Merger, regardless of whether the applicable milestones have been achieved. On April 29, 2020, the Company, certain of the former equity holders of Agilis (“the Participating Rightholders”), and, for the limited purposes set forth in the agreement, Shareholder Representative Services LLC, entered into a Rights Exchange Agreement (the “Rights Exchange Agreement”). The Rights Exchange Agreement has no effect on the Agilis Merger Agreement other than to provide for the cancellation and forfeiture of the Participating Rightholders’ rights to receive $211.6 million, in the aggregate, of the milestone payments described above. As a result, all other rights and obligations under the Agilis Merger Agreement remain in effect pursuant to their terms, including the Company’s obligation to pay up to an aggregate maximum amount of $20.0 million upon the achievement of certain development milestones (representing the remaining portion of potential development milestone payments for which rights were not canceled and forfeited pursuant to the Rights Exchange Agreement while excluding the remaining $2.4 million milestone payment that was due and paid upon the passing of the second anniversary of the closing of the Agilis Merger), up to an aggregate maximum amount of $361.0 million upon the achievement of certain regulatory milestones (representing the remaining portion of potential regulatory milestone payments for which rights were not canceled and forfeited pursuant to the Rights Exchange Agreement), up to a maximum aggregate amount of $150.0 million upon the achievement of certain net sales milestones and a percentage of annual net sales for Friedreich ataxia and Angelman syndrome during specified terms, ranging from 2% to 6%, pursuant to the terms of the Agilis Merger Agreement. In July 2022, the European Commission approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. As a result of such approval, the Company paid the former equityholders of Agilis $50.0 million in accordance with the terms of the Agilis Merger Agreement in the year ended December 31, 2022. In May 2023, as part of the Company’s strategic portfolio prioritization, the Company decided to discontinue its preclinical and early research programs in its gene therapy platform, which included Friedreich ataxia and Angelman syndrome. As a result, the Company does not expect the milestones related to Friedreich ataxia and Angelman syndrome to be achieved. In addition, the Company does not expect to pay the 2% to 6% royalties on annual net sales related to Friedreich ataxia and Angelman syndrome. As of March 31, 2024, the remaining potential development and regulatory milestones the Company expects to achieve is $31.1 million, and the remaining potential sales milestones the Company expects to achieve is $50.0 million, both of which relate solely to Upstaza. On October 25, 2019, the Company completed the acquisition of substantially all of the assets of BioElectron Technology Corporation (“BioElectron”), a Delaware corporation, including certain compounds that the Company has begun to develop as part of its Bio-e platform, pursuant to an asset purchase agreement by and between the Company and BioElectron, dated October 1, 2019 (the “BioElectron Asset Purchase Agreement”). BioElectron was a private company with a pipeline focused on inflammatory and central nervous system (CNS) disorders. The lead program, vatiquinone, is in late stage development for Friedreich ataxia Subject to the terms and conditions of the BioElectron Asset Purchase Agreement, BioElectron may become entitled to receive contingent milestone payments of up to $200.0 million (in cash or in shares of the Company’s common stock, as determined by the Company) from the Company based on the achievement of certain regulatory and net sales milestones. Subject to the terms and conditions of the BioElectron Asset Purchase Agreement, BioElectron may also become entitled to receive contingent payments based on a percentage of net sales of certain products. Subject to the terms and conditions of the Agreement and Plan of Merger, dated as of May 5, 2020 (the “Censa Merger Agreement”) by and among the Company, Hydro Merger Sub, Inc., the Company’s wholly owned, indirect subsidiary, and, solely in its capacity as the representative, agent and attorney-in-fact of the securityholders of Censa, Shareholder Representative Services LLC (such merger pursuant thereto, the “Censa Merger”), former Censa securityholders may become entitled to receive contingent payments from the Company based on (i) the achievement of certain development and regulatory milestones up to an aggregate maximum amount of $217.5 million for sepiapterin sepiapterin sepiapterin In February 2023, the Company completed enrollment of its Phase 3 placebo-controlled clinical trial for sepiapterin for PKU. In connection with this event and pursuant to the Censa Merger Agreement, the Company paid a $30.0 million development milestone to the former Censa securityholders during the three months ended March 31, 2023. The Company elected to pay this milestone in the form of shares of its common stock, less certain cash payments in accordance with the Censa Merger Agreement. Pursuant to such election, the Company issued 657,462 shares of its common stock and paid $0.4 million to the former Censa securityholders. The Company expects to make payments to the former Censa securityholders of $65.0 million in the aggregate in cash upon the potential achievement in 2024 of regulatory milestones relating to sepiapterin pursuant to the Censa Merger Agreement. The Company also has the Tegsedi-Waylivra Agreement for the commercialization of Tegsedi and Waylivra, and products containing those compounds in countries in Latin America and the Caribbean. Akcea is entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement. The Company has employment agreements with certain employees which require the funding of a specific level of payments, if certain events, such as a change in control or termination without cause, occur. Additionally, the Company has royalty payments associated with Translarna, Emflaza, and Upstaza net product revenue, payable quarterly or annually in accordance with the terms of the related agreements. From time to time in the ordinary course of its business, the Company is subject to claims, legal proceedings and disputes. The Company is not currently aware of any material legal proceedings against it.

Revenue recognition

Revenue recognition	3 Months Ended
Revenue recognition	Mar. 31, 2024
Revenue from Contract with Customer [Abstract]
Revenue recognition	11. Revenue recognition Net product sales The Company views its operations and manages its business in one operating segment. During the three months ended March 31, 2024 and 2023, net product sales outside of the United States were $120.1 million and $133.0 million, respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net revenues made up $103.6 million and $115.1 million of the net product sales outside of the United States for the three months ended March 31, 2024 and 2023, respectively. During the three months ended March 31, 2024 and 2023, net product sales in the United States were $57.5 million and $54.6 million, respectively, consisting solely of sales of Emflaza. During the three months ended March 31, 2024, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $57.5 million and $52.6 million of the net product sales, respectively. During the three months ended March 31, 2023, three countries, the United States, Russia, and Brazil, accounted for at least 10% of the Company’s net product sales, representing $54.6 million, $44.6 million, and $25.9 million of the net product sales, respectively. For the three months ended March 31, 2024 and 2023, two of the Company’s distributors each accounted for over 10% of the Company’s net product sales. As of March 31, 2024 and December 31, 2023, the Company does not have a contract liabilities balance related to net product sales, and has not made significant changes to the judgments made in applying ASC Topic 606. Collaboration and Royalty revenue In November 2011, the Company and the Spinal Muscular Atrophy (“SMA Foundation”) entered into a licensing and collaboration agreement with F. Hoffman-La Roche Ltd and Hoffman- La Roche Inc. (collectively, “Roche”). Under the terms of the SMA License Agreement, Roche acquired an exclusive worldwide license to the Company’s SMA program. Under the agreement, the Company is eligible to receive additional payments from Roche if specified events are achieved with respect to each licensed product, including up to $135.0 million in research and development event milestones, up to $325.0 million in sales milestones upon achievement of specified sales events, and up to double digit royalties on worldwide annual net sales of a commercial product. The SMA program currently has one approved product, Evrysdi, which was approved in August 2020 by the FDA for the treatment of SMA in adults and children two months and older. As of March 31, 2024, the Company does not have any remaining research and development event milestones that can be received. The remaining potential sales milestones that can be received is $150.0 million. For the three months ended March 31, 2024, the Company did not recognize collaboration revenue related to the licensing and collaboration agreement with Roche. For the three months ended March 31, 2023, the amounts recognized for the collaboration revenue related to the licensing and collaboration agreement with Roche were immaterial. In addition to research and development and sales milestones, the Company is eligible to receive up to double-digit royalties on worldwide annual net sales of a commercial product under the SMA License Agreement. For the three months ended March 31, 2024 and 2023, the Company has recognized $31.2 million and $30.8 million of royalty revenue, respectively, related to Evrysdi. Manufacturing Revenue For the three months ended March 31, 2024 and 2023, the Company recognized $1.4 million and $2.0 million of manufacturing revenue, respectively, related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. As of March 31, 2024, the Company does not have a contract liabilities balance related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. As of December 31, 2023, the Company had a contract liabilities balance of $0.8 million related to the production of plasmid DNA and AAV vectors for external customers, which is recorded within deferred revenue on the consolidated balance sheet. For the three-month period ended March 31, 2024, the Company recognized $0.8 million related to the amounts included in the contract liability balance at the beginning of the period. As of March 31, 2024, the Company has contract assets of $0.8 million related to plasmid DNA and AAV production for external customers, which is recorded within prepaid expenses and other current assets on the consolidated balance sheet. As of December 31, 2023, the Company had contract assets of $0.2 million related to plasmid DNA and AAV production for external customers, which is recorded within prepaid expenses and other current assets on the consolidated balance sheet. Remaining performance obligations There are no remaining performance obligations as of March 31, 2024. The Company’s remaining performance obligations of $0.8 million as of December 31, 2023 were fully recognized during the three months ended March 31, 2024.

Intangible assets and goodwill

Intangible assets and goodwill	3 Months Ended
Intangible assets and goodwill	Mar. 31, 2024
Goodwill and Intangible Assets Disclosure [Abstract]
Intangible assets and goodwill	12. Intangible assets and goodwill Definite-lived intangibles Definite-lived intangible assets consisted of the following at March 31, 2024 and December 31, 2023: Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency March 31, intangibles assets, gross 2023 Additions translation 2024 Emflaza $ 527,417 $ — $ — $ 527,417 Waylivra 10,218 1,658 (246) 11,630 Tegsedi 13,322 792 (320) 13,794 Upstaza 89,550 — — 89,550 Total definite-lived intangibles, gross $ 640,507 $ 2,450 $ (566) $ 642,391 Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency March 31, intangibles assets, accumulated amortization 2023 Amortization translation 2024 Emflaza $ (478,618) $ (48,799) $ — $ (527,417) Waylivra (3,965) (366) 106 (4,225) Tegsedi (3,311) (499) 83 (3,727) Upstaza (10,882) (1,866) — (12,748) Total definite-lived intangibles, accumulated amortization $ (496,776) $ (51,530) $ 189 $ (548,117) Total definite-lived intangibles, net $ 94,274 Marathon is entitled to receive contingent payments from the Company based on annual net sales of Emflaza beginning in 2018, up to a specified aggregate maximum amount over the expected commercial life of the asset, which expired February 2024. In accordance with the guidance for an asset acquisition, the Company recorded the milestone payments when they became payable to Marathon and increased the cost basis for the Emflaza rights intangible asset. As of March 31, 2024, the Emflaza rights intangible asset was fully amortized, therefore for the three months ended March 31, 2024, the milestone payment was recorded on the consolidated statement of operations within cost of product sales, excluding amortization of acquired intangible assets. Akcea is also entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement related to sales of Waylivra and Tegsedi. In accordance with the guidance for an asset acquisition, the Company records royalty payments when they become payable to Akcea and increase the cost basis for the Waylivra and Tegsedi intangible assets. For the three months ended March 31, 2024, royalty payments of $0.8 million and $1.7 million were recorded for Tegsedi and Waylivra, respectively. As of March 31, 2024, a royalty payable of $0.8 million and $1.6 million for Tegsedi and Waylivra, respectively, was recorded on the consolidated balance sheet within accounts payable and accrued expenses. For the three months ended March 31, 2024 and 2023, the Company recognized amortization expense of $51.5 million and $39.4 million, respectively, related to the Emflaza rights, Upstaza, Waylivra, and Tegsedi intangible assets. The estimated future amortization of the Upstaza, Waylivra, and Tegsedi intangible assets is expected to be as follows: As of March 31, 2024 2024 $ 8,182 2025 10,898 2026 10,898 2027 10,898 2028 and thereafter 53,398 Total $ 94,274 The weighted average remaining amortization period of the definite-lived intangibles as of March 31, 2024 is 9.4 years. Indefinite-lived intangibles Indefinite-lived intangible assets consisted of the following at March 31, 2024 and December 31, 2023: Ending Balance at Ending Balance at Indefinite-lived December 31, March 31, intangibles assets 2023 Additions Impairment 2024 Upstaza $ 235,766 $ — $ — $ 235,766 Total indefinite-lived intangibles $ 235,766 $ — $ — $ 235,766 Total intangible assets, net $ 330,040 In connection with the acquisition of the Company’s gene therapy platform from Agilis, the Company acquired rights to Upstaza, for the treatment of AADC deficiency. AADC deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase gene. In accordance with the acquisition method of accounting, the Company allocated the acquisition cost for the Agilis Merger to the underlying assets acquired and liabilities assumed, based upon the estimated fair values of those assets and liabilities at the date of acquisition. The Company classified the fair value of the acquired IPR&D as indefinite lived intangible assets until the successful completion or abandonment of the associated research and development efforts. There have been no changes to the indefinite lived intangible assets balance since the year ended December 31, 2023. Accordingly, the indefinite lived intangible asset balance as of March 31, 2024 is $235.8 million. Goodwill As a result of the Agilis Merger on August 23, 2018, the Company recorded $82.3 million of goodwill. As of March 31, 2024, there have been no changes to the balance of goodwill since the date of the Agilis Merger. Accordingly, the goodwill balance as of March 31, 2024 is $82.3 million.

Subsequent events

Subsequent events	3 Months Ended
Subsequent events	Mar. 31, 2024
Subsequent Events [Abstract]
Subsequent events	13. Subsequent events The Company has evaluated all subsequent events and transactions through the filing date. There were no material events that impacted the unaudited consolidated financial statements or disclosures.

Summary of significant accoun_2

Summary of significant accounting policies (Policies)	3 Months Ended
Summary of significant accounting policies (Policies)	Mar. 31, 2024
Accounting Policies [Abstract]
Basis of presentation	Basis of presentation The accompanying financial information as of March 31, 2024 and for the three months ended March 31, 2024 and 2023 has been prepared by the Company, without audit, pursuant to the rules and regulations of the SEC. Certain information and footnote disclosures normally included in financial statements prepared in accordance with generally accepted accounting principles in the United States ("GAAP") have been condensed or omitted pursuant to such rules and regulations. These interim financial statements should be read in conjunction with the Company’s audited financial statements as of December 31, 2023 and notes thereto included in the 2023 Form 10-K. In the opinion of management, the unaudited financial information as of March 31, 2024 and for the three months ended March 31, 2024 and 2023 reflects all adjustments, which are normal recurring adjustments, necessary to present a fair statement of financial position, results of operations, stockholders’ (deficit) equity, and cash flows. The results of operations for the three months ended March 31, 2024 are not necessarily indicative of the results to be expected for the year ended December 31, 2024 or for any other interim period or for any other future year.
Use of estimates	Use of estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of net product sales, royalty revenue, certain accruals related to the Company’s research and development expenses, valuation procedures for liability for sale of future royalties, fair value of the contingent consideration, and the provision for or benefit from income taxes. Actual results could differ from those estimates. Changes in estimates are reflected in reported results in the period in which they become known.
Restricted Cash	Restricted cash Restricted cash included in deposits and other assets on the consolidated balance sheet contains an unconditional, irrevocable and transferable letter of credit that was entered into during the twelve-month period ended December 31, 2019 in connection with obligations under a facility lease for the Company’s leased biologics manufacturing facility in Hopewell Township, New Jersey. The amount of the letter of credit is $7.5 million, is to be maintained for a term of not less than five years and has the potential to be reduced to $3.8 million if after five years the Company is not in default of its lease. Restricted cash also contains an unconditional, irrevocable and transferable letter of credit that was entered into during June 2022 in connection with obligations for the Company’s new facility lease in Warren, New Jersey. The amount of the letter of credit is $8.1 million and has the potential to be reduced to $4.1 million if after five years the Company is not in default of its lease. Both amounts are classified within deposits and other assets on the consolidated balance sheet due to the long-term nature of the respective letters of credit. Restricted cash also includes a bank guarantee of $0.6 million denominated in a foreign currency. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the consolidated balance sheet that sum to the total of the same amounts shown in the statement of cash flows: End of Beginning of period- period- March 31, December 31, 2024 2023 Cash and cash equivalents $ 548,355 $ 594,001 Restricted cash included in deposits and other assets 16,268 16,283 Total Cash, cash equivalents and restricted cash per statement of cash flows $ 564,623 $ 610,284
Marketable securities	Marketable securities The Company’s marketable securities consists of both debt securities and equity investments. The Company considers its investments in debt securities with original maturities of greater than 90 days to be available for sale securities. Securities under this classification are recorded at fair value and unrealized gains and losses within accumulated other comprehensive income. The estimated fair value of the available for sale securities is determined based on quoted market prices or rates for similar instruments. In addition, the cost of debt securities in this category is adjusted for amortization of premium and accretion of discount to maturity. For available for sale debt securities in an unrealized loss position, the Company assesses whether it intends to sell or if it is more likely than not that the Company will be required to sell the security before recovery of its amortized cost basis. If either of the criteria regarding intent or requirement to sell is met, the security’s amortized cost basis is written down to fair value. If the criteria are not met, the Company evaluates whether the decline in fair value has resulted from a credit loss or other factors. In making this assessment, management considers, among other factors, the extent to which fair value is less than amortized cost, any changes to the rating of the security by a rating agency, and adverse conditions specifically related to the security. If this assessment indicates that a credit loss exists, the present value of cash flows expected to be collected from the security are compared to the amortized cost basis of the security. If the present value of the cash flows expected to be collected is less than the amortized cost basis, a credit loss exists and an allowance for credit losses is recorded for the credit loss, limited by the amount that the fair value is less than the amortized costs basis. Any impairment that has not been recorded through an allowance for credit losses is recognized in other comprehensive income. For the three months ended March 31, 2024 and 2023, no allowance was recorded for credit losses. Marketable securities that are equity investments are measured at fair value, as it is readily available, and as such are classified as Level 1 assets. Unrealized holding gains and losses for these equity investments are components of other income (expense), net within the consolidated statement of operations.
Inventory and cost of product sales	Inventory and cost of product sales Inventory Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis by product. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. Products which may be used in clinical development programs are included in inventory and charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. Inventory used for marketing efforts are charged to selling, general and administrative expense. Amounts related to clinical development programs and marketing efforts are immaterial. The following table summarizes the components of the Company’s inventory for the periods indicated: March 31, 2024 December 31, 2023 Raw materials $ 1,121 $ 952 Work in progress 22,178 17,991 Finished goods 6,759 11,634 Total inventory $ 30,058 $ 30,577 The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. For the three months ended March 31, 2024, the Company recorded inventory write-downs of $3.7 million primarily related to adjustments to inventory reserves and product approaching expiration. For the three months ended March 31, 2023, the Company recorded inventory write-downs of $0.1 million primarily related to product approaching expiration. Additionally, though the Company’s product is subject to strict quality control and monitoring which it performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications resulting in a charge to cost of product sales. For the three months ended March 31, 2024 and 2023, these amounts were immaterial. Cost of product sales Cost of product sales consists of the cost of inventory sold, manufacturing and supply chain costs, storage costs, amortization of the acquired intangible asset, royalty payments associated with net product sales, and royalty payments to collaborative partners associated with royalty revenues and collaboration revenue related to milestones. Production costs are expensed as cost of product sales when the related products are sold or royalty revenues and collaboration revenue milestones are earned.
Revenue recognition	Revenue recognition Net product revenue The Company’s net product revenue primarily consists of sales of Translarna in territories outside of the U.S. for the treatment of nmDMD and sales of Emflaza in the U.S. for the treatment of DMD. The Company recognizes revenue when its performance obligations with its customers have been satisfied. The Company’s performance obligations are to provide products based on customer orders from distributors, hospitals, specialty pharmacies or retail pharmacies. The performance obligations are satisfied at a point in time when the Company’s customer obtains control of the product, which is typically upon delivery. The Company invoices its customers after the products have been delivered and invoice payments are generally due within 30 to 90 days of the invoice date. The Company determines the transaction price based on fixed consideration in its contractual agreements. Contract liabilities arise in certain circumstances when consideration is due for goods the Company has yet to provide. As the Company has identified only one distinct performance obligation, the transaction price is allocated entirely to product sales. In determining the transaction price, a significant financing component does not exist since the timing from when the Company delivers product to when the customers pay for the product is typically less than one year. Customers in certain countries pay in advance of product delivery. In those instances, payment and delivery typically occur in the same month. The Company records product sales net of any variable consideration, which includes discounts, allowances, rebates related to Medicaid and other government pricing programs, and distribution fees. The Company uses the expected value or most likely amount method when estimating its variable consideration, unless discount or rebate terms are specified within contracts. The identified variable consideration is recorded as a reduction of revenue at the time revenues from product sales are recognized. These estimates for variable consideration are adjusted to reflect known changes in factors and may impact such estimates in the quarter those changes are known. Revenue recognized does not include amounts of variable consideration that are constrained. For the three months ended March 31, 2024 and 2023, net product sales outside of the United States were $120.1 million and $133.0 million, respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net revenues made up $103.6 million and $115.1 million of the net product sales outside of the United States for the three months ended March 31, 2024 and 2023, respectively. For the three months ended March 31, 2024 and 2023, net product sales in the United States were $57.5 million and $54.6 million, respectively, consisting solely of sales of Emflaza. During the three months ended March 31, 2024, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $57.5 million and $52.6 million of net product sales, respectively. During the three months ended March 31, 2023, three countries, the United States, Russia, and Brazil, accounted for at least 10% of the Company’s net product sales, representing $54.6 million, $44.6 million, and $25.9 million of net product sales, respectively. In relation to customer contracts, the Company incurs costs to fulfill a contract but does not incur costs to obtain a contract. These costs to fulfill a contract do not meet the criteria for capitalization and are expensed as incurred. The Company considers any shipping and handling costs that are incurred after the customer has obtained control of the product as a cost to fulfill a promise. Shipping and handling costs associated with finished goods delivered to customers are recorded as a selling expense. Collaboration and royalty revenue The terms of these agreements typically include payments to the Company of one or more of the following: nonrefundable, upfront license fees; milestone payments; research funding and royalties on future product sales. In addition, the Company generates service revenue through agreements that generally provide for fees for research and development services and may include additional payments upon achievement of specified events. At the inception of a collaboration arrangement, the Company needs to first evaluate if the arrangement meets the criteria in Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) Topic 808 “Collaborative Arrangements” to then determine if ASC Topic 606 is applicable by considering whether the collaborator meets the definition of a customer. If the criteria are met, the Company assesses the promises in the arrangement to identify distinct performance obligations. For licenses of intellectual property, the Company assesses, at contract inception, whether the intellectual property is distinct from other performance obligations identified in the arrangement. If the licensing of intellectual property is determined to be distinct, revenue is recognized for nonrefundable, upfront license fees when the license is transferred to the customer and the customer can use and benefit from the license. If the licensing of intellectual property is determined not to be distinct, then the license will be bundled with other promises in the arrangement into one distinct performance obligation. The Company needs to determine if the bundled performance obligation is satisfied over time or at a point in time. If the Company concludes that the nonrefundable, upfront license fees will be recognized over time, the Company will need to assess the appropriate method of measuring proportional performance. For milestone payments, the Company assesses, at contract inception, whether the development or sales-based milestones are considered probable of being achieved. If it is probable that a significant revenue reversal will occur, the Company will not record revenue until the uncertainty has been resolved. Milestone payments that are contingent upon regulatory approval are not considered probable of being achieved until the applicable regulatory approvals or other external conditions are obtained as such conditions are not within the Company’s control. If it is probable that a significant revenue reversal will not occur, the Company will estimate the milestone payments using the most likely amount method. The Company will re-assess the development and sales-based milestones each reporting period to determine the probability of achievement. The Company recognizes royalties from product sales at the later of when the related sales occur or when the performance obligation to which the royalty has been allocated has been satisfied. If it is probable that a significant revenue reversal will not occur, the Company will estimate the royalty payments using the most likely amount method. The Company recognizes revenue for reimbursements of research and development costs under collaboration agreements as the services are performed. The Company records these reimbursements as revenue and not as a reduction of research and development expenses as the Company has the risks and rewards as the principal in the research and development activities. For the three months ended March 31, 2024, the Company did not recognize collaboration revenue related to the SMA License Agreement with Roche. For the three months ended March 31, 2023, the amounts recognized for the collaboration revenue related to the SMA License Agreement with Roche were immaterial. For the three months ended March 31, 2024 and 2023, the Company has recognized $31.2 million and $30.8 million of royalty revenue, respectively, related to Evrysdi. Manufacturing Revenue The Company has manufacturing services related to the production of plasmid deoxyribonucleic acid (“DNA”) and adeno-associated virus (“AAV”) vectors for gene therapy applications for external customers. Performance obligations vary but may include manufacturing plasmid DNA and/or AAV vectors, material testing, stability studies, and other services related to material development. The transaction prices for these arrangements are fixed and include amounts stated in the contracts for each promised service. Typically, the performance obligations within a manufacturing contract are highly interdependent, in which case, the Company will combine them into a single performance obligation. The Company has determined that the assets created have no alternative use to the Company, and the Company has an enforceable right to payment for the performance completed to date, therefore revenue related to these services are recognized over time and is measured using an output method based on performance of manufacturing milestones completed to date. Manufacturing service contracts may also include performance obligations related to project management services or obtaining materials from third parties. The Company has determined that these are separate performance obligations for which revenue is recognized at the point in time the services are performed. For performance obligations related to obtaining third party materials, the Company has determined that it is the principal as the Company has control of the materials and has discretion in setting the price. Therefore, the Company recognizes revenue on a gross basis related to obtaining third party materials. Certain arrangements require a portion of the contract consideration to be received in advance at the commencement of the contract, and such advance payment is initially recorded as a contract liability. A contract asset may be recognized in the event the Company’s satisfaction of performance obligations outpaces customer billings. For the three months ended March 31, 2024 and 2023, the Company recognized $1.4 million and $2.0 million of manufacturing revenue, respectively, related to plasmid DNA and AAV vector production for external customers. As of March 31, 2024, the Company has contract assets of $0.8 million and no remaining performance obligations related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. For the period ended December 31, 2023, the Company had contract assets of $0.2 million and remaining performance obligations of $0.8 million related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers.
Allowance for doubtful accounts	Allowance for doubtful accounts The Company maintains an allowance for estimated losses resulting from the inability of its customers to make required payments. The Company estimates uncollectible amounts based upon current customer receivable balances, the age of customer receivable balances, the customer’s financial condition and current economic trends. The Company also assesses whether an allowance for expected credit losses may be required which includes a review of the Company’s receivables portfolio, which are pooled on a customer basis or country basis. In making its assessment of whether an allowance for credit losses is required, the Company considers its historical experience with customers, current balances, levels of delinquency, regulatory and legal environments, and other relevant current and future forecasted economic conditions. For the three months ended March 31, 2024 and 2023, no allowance was recorded for credit losses. The allowance for doubtful accounts was $1.1 million as of March 31, 2024, and $1.2 million as of December 31, 2023. For the three months ended March 31, 2024 and 2023, bad debt expense was immaterial.
Liability for sale of future royalties	Liability for sale of future royalties On July 17, 2020, the Company, RPI Intermediate Finance Trust (“RPI”), and, for the limited purposes set forth in the agreement, Royalty Pharma PLC, entered into a royalty purchase agreement (the “Original Royalty Purchase Agreement”). Pursuant to the Original Royalty Purchase Agreement, the Company sold to RPI 42.933% (the “ Original Assigned Royalty Rights”) of the Company’s right to receive sales-based royalty payments (the “Royalty”) on worldwide net sales of Evrysdi and any other product developed pursuant to the License and Collaboration Agreement (the “License Agreement”), dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation under the SMA program. In consideration for the sale of the Original Assigned Royalty Rights, RPI paid the Company $650.0 million in cash consideration. The Company has retained a 57.067% interest in the Royalty and all economic rights to receive the remaining potential regulatory and sales milestone payments under the License Agreement, which remaining milestone payments equal $150.0 million in the aggregate as of March 31, 2024. The Original Royalty Purchase Agreement was set to terminate 60 days following the earlier of the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement and the date on which RPI has received $1.3 billion in respect of the Original Assigned Royalty Rights. Pursuant to the guidance in ASC 470-10-25-2, the Company determined that the $650.0 million cash consideration obtained pursuant to the Original Royalty Purchase Agreement should be classified as debt and recorded it as “liability for sale of future royalties-current” and “liability for sale of future royalties-noncurrent” on the Company’s consolidated balance sheet based on the timing of the expected payments to be made to RPI at the time of the transaction. The liability was subsequently amortized using the effective interest method over the life of the arrangement, in accordance with the respective guidance, utilizing the prospective method to account for subsequent changes in the estimated future payments to be made to RPI. On October 18, 2023, the Company, Royalty Pharma Investments 2019 ICAV (“Royalty Pharma”), and, for the limited purposes set forth in the agreement, Royalty Pharma plc, entered into an Amended and Restated Royalty Purchase Agreement (the “A&R Royalty Purchase Agreement”), which amends and restates in its entirety the Original Royalty Purchase Agreement. Pursuant to the A&R Royalty Purchase Agreement, the Company has sold or agreed to sell to Royalty Pharma certain portions of the Company’s remaining Royalty on worldwide net sales of Evrysdi and any other product (the “Products”) developed pursuant to the SMA License Agreement (all such retained Royalty rights of the Company, the “Retained Royalty Rights,” and all such Royalty rights that are sold to Royalty Pharma pursuant to the A&R Royalty Purchase Agreement, the (“A&R Assigned Royalty Rights”). At closing, Royalty Pharma paid the Company $1.0 billion in cash consideration for 38.0447% of the Company’s Retained Royalty Rights (which is in addition to the 42.9330% assigned to Royalty Pharma in connection with the Original Royalty Purchase Agreement, for a total of 80.9777% of the total Royalty) until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 66.6667% of the total Royalty. In addition, the Company may sell to Royalty Pharma the remainder of the Company’s Retained Royalty Rights in exchange for an aggregate of $500.0 million in additional cash consideration after the closing of the A&R Royalty Purchase Agreement, less royalties received in respect of the Retained Royalty Rights put to Royalty Pharma, which will be payable by Royalty Pharma pursuant to five put options held by the Company that are exercisable at the Company’s option between January 1, 2024 and December 31, 2025. If the Company exercises two or fewer of the put options, Royalty Pharma may exercise a call option during the period from and after January 1, 2026 until and including March 31, 2026 for up to 50% of the remainder of the Company’s Retained Royalty Rights less amounts exercised by the Company via its put options at a purchase price that is proportional to the purchase price of the Company’s unexercised put options. Royalty Pharma’s exercise of the call option would result in Royalty Pharma owning 90.4888% of the total Royalty until such time as Royalty Pharma has received payments in respect of the Original Assigned Royalty Rights equal to $1.3 billion in the aggregate, and thereafter 83.3333% of the total Royalty. The A&R Royalty Purchase Agreement will terminate 60 days following the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement. The change in rights and obligations from the A&R Royalty Purchase Agreement resulted in a change in the terms of the liability for sale of future royalties, which was evaluated by the Company in accordance with ASC 470-50, Debt —Modifications and Extinguishments. The Company determined that the present value of the cash flows under the A&R Royalty Purchase Agreement were substantially different from the present value of the cash flows under the Original Royalty Purchase Agreement. This resulted in the derecognition of the old liability for sale of future royalties and the new liability for sale of future royalties being recorded at fair value, which was determined to be $1,809.9 million as of the date of the A&R Royalty Purchase Agreement. This resulted in an extinguishment loss of $44.9 million, which was recorded within loss on extinguishment of debt, within the Company’s statement of operations for the year ended December 31, 2023. The fair value for the new liability for sale of future royalties on the date of the A&R Royalty Purchase Agreement was based on the Company’s estimates of future royalties expected to be paid to Royalty Pharma over the life of the arrangement, which was determined using forecasts from market data sources, which are considered Level 3 inputs. The liability is being amortized using the effective interest method over the life of the arrangement, in accordance with ASC 470 and ASC 835. The initial annual effective interest rate was determined to be 10.8%. The Company utilizes the prospective method to account for subsequent changes in the estimated future payments to be made to Royalty Pharma and updates the effective interest rate on a quarterly basis. Issuance costs related to the transaction were determined to be immaterial. Refer to Note 9 for further details.
Indefinite-lived intangible assets	Indefinite-lived intangible assets Indefinite-lived intangible assets consist of in process research and development ("IPR&D"). IPR&D acquired directly in a transaction other than a business combination is capitalized if the projects will be further developed or have an alternative future use; otherwise they are expensed. The fair values of IPR&D projects and license agreement assets acquired in business combinations are capitalized. Several methods may be used to determine the estimated fair value of the IPR&D and license agreement asset acquired in a business combination. The Company utilizes the "income method" and uses estimated future net cash flows that are derived from projected sales revenues and estimated costs. These projections are based on factors such as relevant market size, patent protection, and expected pricing and industry trends. The estimated future net cash flows are then discounted to the present value using an appropriate discount rate. These assets are treated as indefinite-lived intangible assets until completion or abandonment of the projects, at which time the assets are amortized over the remaining useful life or written off, as appropriate. Intangible assets with indefinite lives, including IPR&D, are tested for impairment if impairment indicators arise and, at a minimum, annually. However, an entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that an indefinite-lived intangible asset’s fair value is less than its carrying amount. Otherwise, no further impairment testing is required. The indefinite-lived intangible asset impairment test consists of a one-step analysis that compares the fair value of the intangible asset with its carrying amount. If the carrying amount of an intangible asset exceeds its fair value, an impairment loss is recognized in an amount equal to that excess. The Company considers many factors in evaluating whether the value of its intangible assets with indefinite lives may not be recoverable, including, but not limited to, expected growth rates, the cost of equity and debt capital, general economic conditions, the Company’s outlook and market performance of the Company’s industry and recent and forecasted financial performance.
Goodwill	Goodwill Goodwill represents the amount of consideration paid in excess of the fair value of net assets acquired as a result of the Company’s business acquisitions accounted for using the acquisition method of accounting. Goodwill is not amortized and is subject to impairment testing at a reporting unit level on an annual basis or when a triggering event occurs that may indicate the carrying value of the goodwill is impaired. The Company reassesses its reporting units as part of its annual segment review. An entity is permitted to first assess qualitative factors to determine if a quantitative impairment test is necessary. Further testing is only required if the entity determines, based on the qualitative assessment, that it is more likely than not that the fair value of the reporting unit is less than its carrying amount.
Income Taxes	Income Taxes On December 15, 2022, the EU Member States formally adopted the EU’s Pillar Two Directive, which generally provides for a minimum effective tax rate of 15%, as established by the Organization for Economic Co-operation and Development (“OECD”) Pillar Two Framework that was supported by over 130 countries worldwide. The EU effective dates are January 1, 2024, and January 1, 2025, for different aspects of the directive. A significant number of other countries are also implementing similar legislation. As a result, the tax laws in the U.S. and other countries in which PTC and its affiliates do business could change on a prospective or retroactive basis and any such changes could materially adversely affect the Company’s business. The Company is continuing to evaluate the potential impact on future periods of the Pillar Two Framework, pending legislative adoption by additional individual countries, including those within the European Union. On December 22, 2017, the U.S. government enacted the 2017 Tax Act, which significantly revised U.S. tax law by, among other provisions, lowering the U.S. federal statutory corporate income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Global Intangible Low-Taxed Income (“GILTI”) provisions of the 2017 Tax Act require the Company to include in its U.S. income tax return foreign subsidiary earnings in excess of an allowable return on the foreign subsidiary’s tangible assets. The Company has elected to account for GILTI tax in the period in which it is incurred, and therefore has not provided any deferred tax impacts of GILTI in its consolidated financial statements for the period ended March 31, 2024. Starting in 2022, TCJA amendments to IRC Section 174 no longer permits an immediate deduction for research and development expenditures in the tax year that such costs are incurred. Instead, these IRC Section 174 development costs must now be capitalized and amortized over either a five- or 15-year period, depending on the location of the activities performed. The new amortization period begins with the midpoint of any taxable year that IRC Section 174 costs are first incurred, regardless of whether the expenditures were made prior to or after July 1, and runs until the midpoint of year five for activities conducted in the United States or year 15 in the case of development conducted on foreign soil. This tax law change is anticipated to result in an increased current taxable income of the Company by $42.6 million for the year ending December 31, 2024. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases and net operating loss and credit carryforwards. Deferred tax assets and liabilities are measured at rates expected to apply to taxable income in the years in which those temporary differences and carryforwards are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in the statement of operations in the period that includes the enactment date. A valuation allowance is recorded when it is not more likely than not that all or a portion of the net deferred tax assets will be realized. On August 23, 2018, the Company completed its acquisition of Agilis Biotherapeutics, Inc. (“Agilis”), pursuant to an Agreement and Plan of Merger, dated as of July 19, 2018 (the “Agilis Merger Agreement”), by and among the Company, Agility Merger Sub, Inc., a Delaware corporation and the Company’s wholly owned, indirect subsidiary, Agilis and, solely in its capacity as the representative, agent and attorney-in-fact of the equityholders of Agilis, Shareholder Representative Services LLC, (the “Agilis Merger”). The Company recorded a deferred tax liability in conjunction with the Agilis Merger of $122.0 million in 2018, related to the tax basis difference in the IPRD indefinite-lived intangibles acquired. The Company’s policy is to record a deferred tax liability related to acquired IPR&D which may eventually be realized either upon amortization of the asset when the research is completed, and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful. In July 2022, the Company received EMEA approval for a portion of the IPR&D assets, and thus, began the amortization of the intangible. In May 2023, the Company announced the discontinuation of its preclinical and early research programs in gene therapy as part of a strategic portfolio prioritization. In conjunction with the announcement, the Company recorded an impairment to its indefinite-lived intangible for IP research and development relating to the Friedreich ataxia and Angelman syndrome gene therapy assets. As a result of the impairment, the Company recorded a deferred tax benefit of $46.9 million during the 2023 tax year.
Leases	Leases The Company determines if an arrangement is a lease at inception. This determination generally depends on whether the arrangement conveys to the Company the right to control the use of an explicitly or implicitly identified fixed asset for a period of time in exchange for consideration. Control of an underlying asset is conveyed to the Company if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. The Company has lease agreements which include lease and non-lease components, which the Company accounts for as a single lease component for all leases. Operating and finance leases are classified as right of use ("ROU") assets, short term lease liabilities, and long term lease liabilities. Operating and finance lease ROU assets and lease liabilities are recognized at the commencement date based on the present value of lease payments over the lease term. ROU assets are amortized and lease liabilities accrete to yield straight-line expense over the term of the lease. Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments associated with the Company’s leases are recognized when the event, activity, or circumstance in the lease agreement on which those payments are assessed occurs. Variable lease payments are presented in the Company’s consolidated statements of operations in the same line item as expense arising from fixed lease payments for operating leases. Leases with an initial term of 12 months or less are not recorded on the consolidated balance sheet and the Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories. A lessee is required to discount its unpaid lease payments using the interest rate implicit in the lease or, if that rate cannot be readily determined, its incremental borrowing rate. As most of the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate based on the information available at the commencement date in determining the present value of lease payments. The Company gives consideration to its recent debt issuances as well as publicly available data for instruments with similar characteristics when calculating its incremental borrowing rates. The lease term for all of the Company’s leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leasehold improvements are capitalized and depreciated over the lesser of useful life or lease term. See Note 3 Leases for additional information.
Recently issued accounting standards	Recently issued accounting standards In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280) – Improvements to Reportable Segment Disclosures. This ASU requires that a public entity provide additional segment disclosures on an interim and annual basis. The amendments in this ASU should be applied retrospectively to all prior periods presented in the financial statements, unless impracticable. Upon transition, the segment expense categories and amounts disclosed in the prior periods should be based on the significant segment expense categories identified and disclosed in the period of adoption. The ASU is effective for fiscal years beginning after December 15, 2023 and interim periods within fiscal years beginning after December 15, 2024. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes. In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures. ASU 2023-09 enhances the transparency about income tax information through improvements to income tax disclosures primarily related to the rate reconciliation and income taxes paid information. The guidance is effective for public business entities for annual periods beginning after December 15, 2024. For entities other than public business entities, the amendments are effective for annual periods beginning after December 15, 2025. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Company’s consolidated financial statements and accompanying footnotes.

Summary of significant accoun_3

Summary of significant accounting policies (Tables)	3 Months Ended
Summary of significant accounting policies (Tables)	Mar. 31, 2024
Accounting Policies [Abstract]
Reconciliation of cash	End of Beginning of period- period- March 31, December 31, 2024 2023 Cash and cash equivalents $ 548,355 $ 594,001 Restricted cash included in deposits and other assets 16,268 16,283 Total Cash, cash equivalents and restricted cash per statement of cash flows $ 564,623 $ 610,284
Schedule of Inventory	March 31, 2024 December 31, 2023 Raw materials $ 1,121 $ 952 Work in progress 22,178 17,991 Finished goods 6,759 11,634 Total inventory $ 30,058 $ 30,577

Leases - (Tables)

Leases - (Tables)	3 Months Ended
Leases - (Tables)	Mar. 31, 2024
Leases
Schedule of lease costs	Three Months Ended Three Months Ended March 31, 2024 March 31, 2023 Operating Lease Cost Fixed lease cost $ 5,575 $ 5,473 Variable lease cost 1,069 1,353 Short-term lease cost 214 303 Total operating lease cost $ 6,858 $ 7,129 March 31, 2024 December 31, 2023 Weighted-average remaining lease terms - operating leases (years) 11.66 11.55 Weighted-average discount rate - operating leases 8.75 % 8.69 % Weighted-average remaining lease terms - finance lease (years) 8.76 9.01 Weighted-average discount rate - finance lease 7.80 % 7.80 %
Schedule of supplemental cash flow information related to leases	Three Months Ended March 31, 2024 2023 Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 3,948 $ 3,811 Financing cash flows from finance lease 1,490 1,379 Operating cash flows from finance lease 1,510 1,621 Right-of-use assets obtained in exchange for lease obligations: Operating leases $ 1,723 $ —
Schedule of future lease payments - Operating leases	Operating Leases Finance Lease 2024 (excludes the three months ended March 31, 2024) $ 14,981 $ — 2025 20,995 3,000 2026 20,539 3,000 2027 18,400 3,000 2028 and thereafter 175,853 15,000 Total lease payments 250,768 24,000 Less: Imputed Interest expense 131,359 6,478 Total $ 119,409 $ 17,522
Schedule of future lease payments - Finance leases	Operating Leases Finance Lease 2024 (excludes the three months ended March 31, 2024) $ 14,981 $ — 2025 20,995 3,000 2026 20,539 3,000 2027 18,400 3,000 2028 and thereafter 175,853 15,000 Total lease payments 250,768 24,000 Less: Imputed Interest expense 131,359 6,478 Total $ 119,409 $ 17,522

Fair value of financial instr_2

Fair value of financial instruments and marketable securities - (Tables)	3 Months Ended
	Mar. 31, 2024
Fair Value Disclosures [Abstract]
Schedule of marketable securities	Ending Foreign Ending Balance at Currency Balance at December 31, Unrealized Unrealized Investments Redemptions/ March 31, 2023 Gain Loss Purchased Sale 2024 Marketable securities - equity investments $ 22,634 710 (823) 9,065 (1,207) $ 30,379 ClearPoint Equity Investments 6,074 9 — — — 6,083 ClearPoint convertible debt security 12,553 166 — — — 12,719 Total Fair Value $ 41,261 $ 885 $ (823) $ 9,065 $ (1,207) $ 49,181 Ending Foreign Ending Balance at Currency Balance at December 31, Unrealized Unrealized Investments Redemptions/ March 31, 2022 Gain/(Loss) Gain Purchased Sale 2023 Marketable securities - equity investments $ 108,261 2,166 328 — (2,196) $ 108,559 ClearPoint Equity Investments 10,965 (39) — — — 10,926 ClearPoint convertible debt security 15,231 59 — — — 15,290 Total Fair Value $ 134,457 $ 2,186 $ 328 $ — $ (2,196) $ 134,775
Schedule of financial assets and liabilities that are required to be measured at fair value on a recurring basis	March 31, 2024 Quoted prices Significant in active other Significant markets for observable unobservable identical assets inputs inputs Total (level 1) (level 2) (level 3) Marketable securities - available for sale $ 306,079 $ — $ 306,079 $ — Marketable securities - equity investments $ 30,379 $ 30,379 $ — $ — ClearPoint Equity Investments $ 6,083 $ 6,083 $ — $ — ClearPoint convertible debt security $ 12,719 $ — $ 12,719 $ — Contingent consideration payable- development and regulatory milestones $ 26,900 $ — $ — $ 26,900 Contingent consideration payable- net sales milestones $ 9,300 $ — $ — $ 9,300 December 31, 2023 Quoted prices Significant in active other Significant markets for observable unobservable identical assets inputs inputs Total (level 1) (level 2) (level 3) Marketable securities - available for sale $ 260,104 $ — $ 260,104 $ — Marketable securities - equity investments $ 22,634 $ 22,634 $ — $ — ClearPoint Equity Investments $ 6,074 $ 6,074 $ — $ — ClearPoint convertible debt security $ 12,553 $ — $ 12,553 $ — Contingent consideration payable- development and regulatory milestones $ 26,600 $ — $ — $ 26,600 Contingent consideration payable- net sales milestones and royalties $ 9,700 $ — $ — $ 9,700
Summary of marketable securities accounted for as available-for-sale debt securities	March 31, 2024 Amortized Gross Unrealized Cost Gains Losses Fair Value Commercial paper $ 132,080 $ 26 $ (121) $ 131,985 Government Obligations 174,118 41 (65) 174,094 Total $ 306,198 $ 67 $ (186) $ 306,079 December 31, 2023 Amortized Gross Unrealized Cost Gains Losses Fair Value Commercial paper $ 117,044 $ 128 $ (12) $ 117,160 Corporate debt securities 1,650 — (2) 1,648 Government Obligations 141,084 212 — 141,296 Total $ 259,778 $ 340 $ (14) $ 260,104
Summary of unrealized losses and fair values of available-for-sale debt securities in a continuous unrealized loss position	March 31, 2024 Securities in an unrealized loss Securities in an unrealized loss position less than 12 months position greater than or equal to 12 months Total Unrealized losses Fair Value Unrealized losses Fair Value Unrealized losses Fair Value Commercial paper $ (121) 73,250 — — (121) 73,250 Government bonds $ (65) 40,302 — — (65) $ 40,302 Total $ (186) $ 113,552 $ — $ — $ (186) $ 113,552 December 31, 2023 Securities in an unrealized loss Securities in an unrealized loss position less than 12 months position greater than or equal to 12 months Total Unrealized losses Fair Value Unrealized losses Fair Value Unrealized losses Fair Value Commercial paper $ (12) 44,446 — — (12) 44,446 Corporate debt securities $ — — (2) 1,648 (2) $ 1,648 Total $ (12) $ 44,446 $ (2) $ 1,648 $ (14) $ 46,094
Schedule of available-for-sale debt securities	March 31, 2024 Less Than More Than 12 Months 12 Months Commercial paper $ 131,985 $ — Government obligations 174,094 — Total $ 306,079 $ — December 31, 2023 Less Than More Than 12 Months 12 Months Commercial paper $ 117,160 $ — Corporate debt securities 1,648 — Government obligations 141,296 — Total $ 260,104 $ —
Summary of changes in the fair value of the Company's Level 3 valuation for contingent consideration payable	Level 3 liabilities Contingent consideration payable- Contingent consideration payable- development and regulatory net sales milestones and royalties milestones Beginning balance as of December 31, 2023 $ 26,600 $ 9,700 Additions — — Change in fair value 300 (400) Payments — — Ending balance as of March 31, 2024 $ 26,900 $ 9,300 Level 3 liabilities Contingent consideration payable- Contingent consideration payable- development and regulatory net sales milestones and royalties milestones Beginning balance as of December 31, 2022 $ 82,500 $ 81,500 Additions — — Change in fair value 3,000 (600) Payments — — Ending balance as of March 31, 2023 $ 85,500 $ 80,900
Fair value measurement inputs and valuation techniques	March 31, 2024 Fair Value Valuation Technique Unobservable Input Range Contingent consideration payable- $26,900 Probability-adjusted discounted cash flow Potential development and regulatory milestones $0 - $31 million Contingent considerable payable- net sales $9,300 Option-pricing model with Monte Carlo simulation Potential net sales milestones $0 - $50 million December 31, 2023 Fair Value Valuation Technique Unobservable Input Range Contingent consideration payable- $26,600 Probability-adjusted discounted cash flow Potential development and regulatory milestones $0 - $31 million Contingent considerable payable- net sales $9,700 Option-pricing model with Monte Carlo simulation Potential net sales milestones $0 - $50 million

Accounts payable and accrued _2

Accounts payable and accrued expenses - (Tables)	3 Months Ended
Accounts payable and accrued expenses - (Tables)	Mar. 31, 2024
Payables and Accruals [Abstract]
Schedule of components of accounts payable and accrued expenses	March 31, December 31, 2024 2023 Employee compensation, benefits, and related accruals $ 35,060 $ 62,643 Income tax payable 1,833 — Consulting and contracted research 22,745 27,500 Professional fees 2,380 2,246 Sales allowance 67,075 77,176 Sales rebates 132,047 131,334 Royalties 28,952 74,111 Accounts payable 27,459 6,045 Other 13,373 10,928 Total $ 330,924 $ 391,983

Net loss per share - (Tables)

Net loss per share - (Tables)	3 Months Ended
Net loss per share - (Tables)	Mar. 31, 2024
Earnings Per Share [Abstract]
Schedule of computation of basic and diluted net loss available to common stockholders	Three Months Ended March 31, 2024 2023 Numerator Net loss $ (91,576) $ (138,959) Denominator Denominator for basic and diluted net loss per share 76,496,127 73,729,284 Net loss per share: Basic and diluted $ (1.20) * $ (1.88) * * In the three months ended March 31, 2024 and 2023, the Company experienced a net loss and therefore did not report any dilutive share impact.
Schedule of historical dilutive common share equivalents outstanding	As of March 31, 2024 2023 Stock Options 9,530,185 12,100,550 Unvested restricted stock awards and units 3,730,166 3,649,742 Total 13,260,351 15,750,292

Stock award plan - (Tables)

Stock award plan - (Tables)	3 Months Ended
Stock award plan - (Tables)	Mar. 31, 2024
Share-based Payment Arrangement [Abstract]
Summary of stock option activity	Weighted- Weighted- average Aggregate average remaining intrinsic Number of exercise contractual value(in options price term thousands) Outstanding at December 31, 2023 9,600,399 $ 43.59 Granted 855,385 $ 25.69 Exercised (109,892) $ 19.22 Forfeited/Cancelled (815,707) $ 45.32 Outstanding at March 31, 2024 9,530,185 $ 42.12 5.90 years $ 12,025 Vested or Expected to vest at March 31, 2024 2,112,348 $ 38.57 8.67 years $ 2,949 Exercisable at March 31, 2024 7,173,552 $ 43.44 4.98 years $ 8,598
Schedule of assumptions used to estimate fair values of grants made on the date of grant	Three months ended March 31, 2024 Risk-free interest rate 4.23% Expected volatility 53% Expected term 5.5 years
Summary of information on the Company's restricted stock	Restricted Stock Awards and Units Weighted Average Grant Number of Date Shares Fair Value Unvested at December 31, 2023 2,866,270 $ 41.82 Granted 1,785,660 25.69 Vested (835,179) 44.56 Forfeited (86,585) 40.98 Unvested at March 31, 2024 3,730,166 $ 33.51
Schedule of share-based compensation expense recorded in the statement of operations	Three Months Ended March 31, 2024 2023 Research and development $ 8,967 $ 15,314 Selling, general and administrative 9,411 13,501 Total $ 18,378 $ 28,815

Debt - (Tables)

Debt - (Tables)	3 Months Ended
Debt - (Tables)	Mar. 31, 2024
Debt Instrument, Redemption [Line Items]
Summary of liability for sale of future royalties	Three Months Ended March 31, Liability for sale of future royalties- (current and noncurrent) 2024 Beginning balance as of December 31, 2023 $ 1,814,097 Less: Non-cash royalty revenue payable to RPI (25,228) Plus: Non-cash interest expense recognized 50,064 Ending balance $ 1,838,933 Effective interest rate as of March 31, 2024 10.8%
1.50% Convertible senior notes due 2026
Debt Instrument, Redemption [Line Items]
Summary of convertible notes	March 31, 2024 December 31, 2023 Principal $ 287,500 $ 287,500 Less: Debt issuance costs (2,988) (3,287) Net carrying amount $ 284,512 $ 284,213
Summary of interest expense recognized related to the Convertible Notes	Three Months Ended March 31, Three Months Ended March 31, 2024 2023 Contractual interest expense $ 1,076 $ 1,069 Amortization of debt issuance costs 296 288 Total $ 1,372 $ 1,357 Effective interest rate 1.9 % 1.9 %
Blackstone Credit Agreement
Debt Instrument, Redemption [Line Items]
Schedule of credit agreement	March 31, 2024 December 31, 2023 Principal $ — $ 300,000 Less: Debt issuance costs — — Repayment of senior secured term loan — (300,000) Net carrying amount $ — $ —
Summary of interest expense recognized related to debt	Three Months Ended Three Months Ended March 31, March 31, 2024 2023 Contractual interest expense $ — $ 9,178 Amortization of debt issuance costs — 150 Total $ — $ 9,328 Effective interest rate — % 13.2 %

Intangible assets and goodwill

Intangible assets and goodwill - (Tables)	3 Months Ended
Intangible assets and goodwill - (Tables)	Mar. 31, 2024
Goodwill and Intangible Assets Disclosure [Abstract]
Schedule of definite lived intangible assets	Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency March 31, intangibles assets, gross 2023 Additions translation 2024 Emflaza $ 527,417 $ — $ — $ 527,417 Waylivra 10,218 1,658 (246) 11,630 Tegsedi 13,322 792 (320) 13,794 Upstaza 89,550 — — 89,550 Total definite-lived intangibles, gross $ 640,507 $ 2,450 $ (566) $ 642,391 Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency March 31, intangibles assets, accumulated amortization 2023 Amortization translation 2024 Emflaza $ (478,618) $ (48,799) $ — $ (527,417) Waylivra (3,965) (366) 106 (4,225) Tegsedi (3,311) (499) 83 (3,727) Upstaza (10,882) (1,866) — (12,748) Total definite-lived intangibles, accumulated amortization $ (496,776) $ (51,530) $ 189 $ (548,117) Total definite-lived intangibles, net $ 94,274
Schedule of estimated future amortization of intangible assets	As of March 31, 2024 2024 $ 8,182 2025 10,898 2026 10,898 2027 10,898 2028 and thereafter 53,398 Total $ 94,274
Schedule of indefinite lived intangible assets	Ending Balance at Ending Balance at Indefinite-lived December 31, March 31, intangibles assets 2023 Additions Impairment 2024 Upstaza $ 235,766 $ — $ — $ 235,766 Total indefinite-lived intangibles $ 235,766 $ — $ — $ 235,766 Total intangible assets, net $ 330,040

The Company (Details)

The Company (Details) $ in Thousands	3 Months Ended
The Company (Details) $ in Thousands	Mar. 31, 2024 USD ($) product	Dec. 31, 2023 USD ($)
Long-term debt
Number of products \| product	2
Accumulated deficit \| $	$ (3,375,154)	$ (3,283,578)

Summary of significant accoun_4

Summary of significant accounting policies - Narrative (Details) $ in Thousands		1 Months Ended	3 Months Ended		12 Months Ended
	Dec. 22, 2017	Jun. 30, 2022 USD ($)	Mar. 31, 2024 USD ($) country segment	Mar. 31, 2023 USD ($) segment country	Dec. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2019 USD ($)	Dec. 31, 2018 USD ($)
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Number of operating segments \| segment			1	1
Allowance for credit loss			$ 0	$ 0
Increase in allowance for credit losses			0	0
Allowance for doubtful accounts receivable			1,100			$ 1,200
Revenue			210,118	220,382
Income taxes
Federal income tax statutory rate	21%
Deferred tax benefit			1	$ 4		$ 46,900
Forecast
Income taxes
Increase in current taxable income resulting from the elimination of current deduction of qualifying R&D costs under Section 174.					$ 42,600
Agilis
Income taxes
Deferred tax liability								$ 122,000
Deposits and other assets
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Bank guarantee denominated in foreign currency			$ 600
Hopewell Campus
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Period after which letter of credit may be reduced if lease is not in default							5 years
Hopewell Campus \| Minimum
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Term for letter of credit (in years)							5 years
Hopewell Campus \| Deposits and other assets
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Letters of credit							$ 7,500
Hopewell Campus \| Fifth Anniversary
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Letters of credit							$ 3,800
Warren Premises
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Period after which letter of credit may be reduced if lease is not in default		5 years
Warren Premises \| Deposits and other assets
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Letters of credit		$ 8,100
Warren Premises \| Fifth Anniversary
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Letters of credit		$ 4,100
Net product revenue \| Geographic concentration risk
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Number of countries exceeding 10% of net product sales \| country			2	3
Percentage of net product sales threshold			10%	10%
Net product revenue \| Geographic concentration risk \| Non-US
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue			$ 120,100	$ 133,000
Net product revenue \| Geographic concentration risk \| Russia
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue			52,600	44,600
Net product revenue \| Geographic concentration risk \| Brazil
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue				25,900
Net product revenue \| Geographic concentration risk \| United States
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue			$ 57,500	$ 54,600
Net product revenue \| Customer concentration risk
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Percentage of net product sales threshold			10%	10%
Net product revenue
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue			$ 177,604	$ 187,557
Emflaza \| Net product revenue \| Geographic concentration risk \| United States
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue			57,500	54,600
Translarna \| Net product revenue \| Geographic concentration risk \| Non-US
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Revenue			$ 103,600	$ 115,100

Summary of significant accoun_5

Summary of significant accounting policies - Reconciliation of cash (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023	Mar. 31, 2023	Dec. 31, 2022
Accounting Policies [Abstract]
Cash and cash equivalents	$ 548,355	$ 594,001
Restricted cash included in deposits and other assets	16,268	16,283
Total Cash, cash equivalents and restricted cash per statement of cash flows	$ 564,623	$ 610,284	$ 233,594	$ 295,925

Summary of significant accoun_6

Summary of significant accounting policies - Inventory (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Accounting Policies [Abstract]
Raw materials	$ 1,121		$ 952
Work in progress	22,178		17,991
Finished goods	6,759		11,634
Total inventory	30,058		$ 30,577
Inventory write-down	$ 3,700	$ 100

Summary of significant accoun_7

Summary of significant accounting policies - Collaboration and Royalty Revenue (Details) - USD ($)	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Collaboration and royalty revenue
Revenue	$ 210,118,000	$ 220,382,000
Collaboration revenue
Collaboration and royalty revenue
Revenue		6,000
Royalty revenue
Collaboration and royalty revenue
Revenue	31,154,000	30,831,000
SMA License Agreement \| Collaboration revenue
Collaboration and royalty revenue
Revenue	0	0
SMA License Agreement \| Royalty revenue
Collaboration and royalty revenue
Revenue	$ 31,200,000	$ 30,800,000

Summary of significant accoun_8

Summary of significant accounting policies - Manufacturing Revenue (Details) - USD ($)	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Revenue recognition
Revenue	$ 210,118,000	$ 220,382,000
Remaining performance obligations	0		$ 800,000
Manufacturing revenue
Revenue recognition
Revenue	1,360,000	$ 1,988,000
Contract with customer, asset	800,000		200,000
Remaining performance obligations	$ 0		$ 800,000

Summary of significant accoun_9

Summary of significant accounting policies - Liability for Sale of Future Royalties (Details) $ in Millions	Oct. 18, 2023 USD ($) Option	Jul. 17, 2020 USD ($)	Mar. 31, 2024 USD ($)
Assigned royalty payments
Assigned royalty payment, retained percentage		57.067%
Remaining potential milestones that can be achieved			$ 150
Effective interest rate of the liability component			10.80%
Aggregate Royalty Purchase Agreements \| Royalty Pharma
Assigned royalty payments
Total percentage of royalty rights assigned	80.9777%
Original Royalty Purchase Agreement
Assigned royalty payments
Assigned royalty payment, percentage		42.933%
Cash consideration received from Royalty Purchase Agreement		$ 650
Royalty purchase agreement termination period once there are no further royalty payment obligations		60 days
Royalty purchase agreement, payment maximum		$ 1,300
Original Royalty Purchase Agreement \| Royalty Pharma
Assigned royalty payments
Royalty purchase agreement, payment maximum	$ 1,300
A&R Royalty Purchase Agreement
Assigned royalty payments
Cash consideration received from Royalty Purchase Agreement	$ 1,000
Royalty purchase agreement termination period once there are no further royalty payment obligations	60 days
Effective interest rate of the liability component	10.80%
Retained Royalty Rights percentage sold	38.0447%
Percentage of total royalty payable after threshold royalties received	66.6667%
Additional cash consideration	$ 500
Number of put options held by the company	5
Maximum number of put options which can be exercised before Royalty Pharma can exercise a call option \| Option	2
Maximum percentage of remaining retained royalty rights that are available upon exercise of call option	50%
Fair value of liability for sale of future royalties.	$ 1,809.9
Loss on extinguishment of debt	$ 44.9
A&R Royalty Purchase Agreement \| Royalty Pharma
Assigned royalty payments
Percentage of maximum royalty payable until assigned royalty rights	90.4888%
Percentage of maximum royalty payable thereafter assigned royalty rights	83.3333%

Leases - Narrative (Details)

Leases - Narrative (Details) $ in Thousands		1 Months Ended	3 Months Ended
Leases - Narrative (Details) $ in Thousands	Jul. 01, 2020 USD ($) ft² item	May 31, 2022 USD ($) ft² period building	Mar. 31, 2024 USD ($) lease	Mar. 31, 2023 USD ($)	Dec. 31, 2023 USD ($)
Leases
Number of operating leases \| lease			2
Operating lease, expense			$ 6,900	$ 7,100
Finance lease liabilities- current			1,948		$ 3,000
Finance lease liabilities- noncurrent			15,574		17,184
Aggregate rent, Initial term			250,768
Operating lease ROU assets			91,186		91,896
Operating Lease, Liability			119,409
MassBio
Leases
Finance lease cost			300	$ 400
Finance lease liabilities- current			1,900		3,000
Finance lease liabilities- noncurrent			$ 15,600		$ 17,200
Minimum
Leases
Operating lease, term of contract			2 months 12 days
Maximum
Leases
Operating lease, term of contract			15 years 2 months 12 days
Renewal term			15 years
Hopewell Campus
Leases
Operating lease, term of contract	15 years
Area of real estate property \| ft²	220,500
Renewal term	10 years
Number of renewable terms \| item	2
Lessee, Operating Lease, Existence of Option to Extend [true false]	true
Aggregate rent, Initial term	$ 111,500
Percent of market rate	95%
Warren Premises
Leases
Operating lease, term of contract		17 years
Number of buildings leased \| building		2
Area of real estate property \| ft²		360,000
Renewal term		5 years
Number of renewable terms \| period		3
Lessee, Operating Lease, Existence of Option to Extend [true false]		true
Aggregate rent, Initial term		$ 163,000
Operating lease ROU assets		28,900
Operating Lease, Liability		$ 28,900
Base rent abatement period		3 years
Base rent abatement		$ 18,600
Aggregate rent net of base rate abatement, initial term		144,400
Allowance for lease improvements		36,200
Commitment to fund construction account		3,600
Payment due from lessor at issuance of temporary certificate of occupancy		$ 5,000

Leases - Lease costs (Details)

Leases - Lease costs (Details) - USD ($) $ in Thousands	3 Months Ended
Leases - Lease costs (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Mar. 31, 2023
Leases
Fixed lease cost	$ 5,575	$ 5,473
Variable lease cost	1,069	1,353
Short-term lease cost	214	303
Total operating lease cost	$ 6,858	$ 7,129

Leases - Supplemental lease ter

Leases - Supplemental lease terms (Details)	Mar. 31, 2024	Dec. 31, 2023
Leases
Weighted-average remaining lease terms - operating leases (years)	11 years 7 months 28 days	11 years 6 months 18 days
Weighted-average discount rate - operating leases	8.75%	8.69%
Weighted-average remaining lease terms - finance lease (years)	8 years 9 months 3 days	9 years 3 days
Weighted-average discount rate - finance lease	7.80%	7.80%

Leases - Cash flow (Details)

Leases - Cash flow (Details) - USD ($) $ in Thousands	3 Months Ended
Leases - Cash flow (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Mar. 31, 2023
Leases
Operating cash flows from operating leases	$ 3,948	$ 3,811
Financing cash flows from finance lease	1,490	1,379
Operating cash flows from finance leases	1,510	$ 1,621
Right-of-use assets obtained in exchange for operating lease obligations	$ 1,723

Leases - Lease payments (Detail

Leases - Lease payments (Details) $ in Thousands	Mar. 31, 2024 USD ($)
Operating leases
2024 (excludes the three months ended March 31, 2024)	$ 14,981
2025	20,995
2026	20,539
2027	18,400
2028 and thereafter	175,853
Total lease payments	250,768
Less: Imputed Interest expense	131,359
Total	119,409
Finance leases
2025	3,000
2026	3,000
2027	3,000
2028 and thereafter	15,000
Total lease payments	24,000
Less: Imputed Interest expense	6,478
Total	$ 17,522

Fair value of financial instr_3

Fair value of financial instruments and marketable securities - Marketable Securities (Details) $ / shares in Units, $ in Thousands	1 Months Ended	3 Months Ended
	Jan. 31, 2020 USD ($) $ / shares	Mar. 31, 2024 USD ($) fund	Mar. 31, 2023 USD ($)
Changes in fair value
Unrealized Gain/(Loss)		$ 710	$ 2,166
Investments Purchased		9,065
Redemptions/Sale		(1,207)	(2,196)
Unrealized Gain/(Loss)		9	(39)
Unrealized Gain/(Loss)		166	59
Beginning Balance - Total		41,261	134,457
Unrealized Gain/(Loss) - Total		885	2,186
Foreign Currency Unrealized Gain/(Loss) - Total		(823)	328
Investments Purchased - Total		9,065
Redemptions/Sale - Total		(1,207)	(2,196)
Ending Balance - Total		49,181	134,775
ClearPoint Convertible note
Purchase of convertible note		104,373
Marketable securities - equity investments
Changes in fair value
Beginning Balance		22,634	108,261
Unrealized Gain/(Loss)		710	2,166
Foreign Currency Unrealized Gain/(Loss)		(823)	328
Investments Purchased		9,065
Redemptions/Sale		(1,207)	(2,196)
Ending Balance		30,379	108,559
ClearPoint Equity Investment
Changes in fair value
Beginning Balance		6,074	10,965
Unrealized Gain/(Loss)		9	(39)
Ending Balance		6,083	10,926
ClearPoint convertible debt
Changes in fair value
Beginning Balance		12,553	15,231
Unrealized Gain/(Loss)		166	59
Ending Balance		$ 12,719	$ 15,290
ClearPoint Convertible note
Purchase of convertible note	$ 10,000
Conversion price - convertible note \| $ / shares	$ 6
Term of convertible note	5 years
Mutual Funds
Equity Investments
Number of mutual funds denominated in a foreign currency \| fund		1

Fair value of financial instr_4

Fair value of financial instruments and marketable securities - Hierarchy (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	$ 306,079	$ 260,104
Contingent consideration payable	36,200	36,300
Transfers of assets measured between Level 1, Level 2, and Level 3	0	0
Recurring basis
Financial assets and liabilities measured at fair value on recurring basis
ClearPoint Equity Investments	6,083	6,074
ClearPoint convertible debt security	12,719	12,553
Recurring basis \| Quoted prices in active markets for identical assets (Level 1)
Financial assets and liabilities measured at fair value on recurring basis
ClearPoint Equity Investments	6,083	6,074
Recurring basis \| Significant other observable inputs (Level 2)
Financial assets and liabilities measured at fair value on recurring basis
ClearPoint convertible debt security	12,719	12,553
Recurring basis \| Development and Regulatory Milestones
Financial assets and liabilities measured at fair value on recurring basis
Contingent consideration payable	26,900	26,600
Recurring basis \| Development and Regulatory Milestones \| Significant unobservable inputs (Level 3)
Financial assets and liabilities measured at fair value on recurring basis
Contingent consideration payable	26,900	26,600
Recurring basis \| Net Sales Milestones and Royalties
Financial assets and liabilities measured at fair value on recurring basis
Contingent consideration payable	9,300	9,700
Recurring basis \| Net Sales Milestones and Royalties \| Significant unobservable inputs (Level 3)
Financial assets and liabilities measured at fair value on recurring basis
Contingent consideration payable	9,300	9,700
Recurring basis \| Marketable securities
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	306,079	260,104
Marketable securities - equity investments	30,379	22,634
Recurring basis \| Marketable securities \| Quoted prices in active markets for identical assets (Level 1)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - equity investments	30,379	22,634
Recurring basis \| Marketable securities \| Significant other observable inputs (Level 2)
Financial assets and liabilities measured at fair value on recurring basis
Marketable securities - available for sale	$ 306,079	$ 260,104

Fair value of financial instr_5

Fair value of financial instruments and marketable securities - Marketable Securities, Unrealized Gains (Losses) (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	$ 306,198		$ 259,778
Gross Unrealized, Gain	67		340
Gross Unrealized, Loss	(186)		(14)
Fair Value	306,079		260,104
Write downs of available for sale debt securities	0	$ 0
Increase in allowance for credit losses	0	0
Realized gain (loss) from sale of marketable securities	0	$ 0
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	132,080		117,044
Gross Unrealized, Gain	26		128
Gross Unrealized, Loss	(121)		(12)
Fair Value	131,985		117,160
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Amortized Cost			1,650
Gross Unrealized, Loss			(2)
Fair Value			1,648
Government obligations
Debt Securities, Available-for-sale [Line Items]
Amortized Cost	174,118		141,084
Gross Unrealized, Gain	41		212
Gross Unrealized, Loss	(65)
Fair Value	$ 174,094		$ 141,296

Fair value of financial instr_6

Fair value of financial instruments and marketable securities - Available-for-sale securities (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Securities in an unrealized loss position less than 12 months - Unrealized losses	$ (186)	$ (12)
Securities in an unrealized loss position less than 12 months - Fair Value	113,552	44,446
Securities in an unrealized loss position greater than 12 months - Unrealized losses		(2)
Securities in an unrealized loss position greater than 12 months - Fair Value		1,648
Total - Unrealized losses	(186)	(14)
Total - Fair Value	113,552	46,094
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Securities in an unrealized loss position less than 12 months - Unrealized losses	(121)	(12)
Securities in an unrealized loss position less than 12 months - Fair Value	73,250	44,446
Total - Unrealized losses	(121)	(12)
Total - Fair Value	73,250	44,446
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Securities in an unrealized loss position greater than 12 months - Unrealized losses		(2)
Securities in an unrealized loss position greater than 12 months - Fair Value		1,648
Total - Unrealized losses		(2)
Total - Fair Value		$ 1,648
Government obligations
Debt Securities, Available-for-sale [Line Items]
Securities in an unrealized loss position less than 12 months - Unrealized losses	(65)
Securities in an unrealized loss position less than 12 months - Fair Value	40,302
Total - Unrealized losses	(65)
Total - Fair Value	$ 40,302

Fair value of financial instr_7

Fair value of financial instruments and marketable securities - Marketable Securities, Balance Sheet Disclosures (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Marketable securities, Less Than 12 Months	$ 306,079	$ 260,104
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Marketable securities, Less Than 12 Months	131,985	117,160
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Marketable securities, Less Than 12 Months		1,648
Government obligations
Debt Securities, Available-for-sale [Line Items]
Marketable securities, Less Than 12 Months	$ 174,094	$ 141,296

Fair value of financial instr_8

Fair value of financial instruments and investments - Convertible senior notes (Details) - 1.50% Convertible senior notes due 2026 - USD ($)	Mar. 31, 2024	Dec. 31, 2023	Sep. 30, 2019
Long-term debt
Principal	$ 287,500,000	$ 287,500,000
Convertible debt
Long-term debt
Principal			$ 287,500,000
Interest rate ( as a percent )	1.50%		1.50%
Convertible debt \| Significant other observable inputs (level 2)
Long-term debt
Fair value of convertible notes	$ 267,700,000	$ 265,300,000

Fair value of financial instr_9

Fair value of financial instruments and investments - Summary of changes in the fair value of the Company's Level 3 valuation (Details) - Significant unobservable inputs (Level 3) - Agilis - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Upstaza
Changes in the fair value of warrant liability and SARs liability
Ending Balance	$ 36,200
Development and Regulatory Milestones \| Commitments
Changes in the fair value of warrant liability and SARs liability
Beginning Balance	26,600	$ 82,500
Additions	0	0
Change in fair value	300	3,000
Rights Exchange settlement	0	0
Ending Balance	26,900	85,500
Net Sales Milestones and Royalties \| Commitments
Changes in the fair value of warrant liability and SARs liability
Beginning Balance	9,700	81,500
Additions	0	0
Change in fair value	(400)	(600)
Rights Exchange settlement	0	0
Ending Balance	$ 9,300	$ 80,900

Fair value of financial inst_10

Fair value of financial instruments and investments - Fair Value Liabilities Measured (Details) - Agilis $ in Thousands	Mar. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	Mar. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)
Significant unobservable inputs (Level 3) \| Upstaza
Fair Value Valuation Inputs
Fair value	$ 36,200
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Development and Regulatory Milestone \| Minimum
Fair Value Valuation Inputs
Potential milestones	0	$ 0
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Development and Regulatory Milestone \| Maximum
Fair Value Valuation Inputs
Potential milestones	$ 31,000	$ 31,000
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Probability of Success \| Minimum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.85	0.85
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Probability of Success \| Maximum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.92	0.92
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Measurement Input, Discount Rate \| Minimum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.059	0.058
Development and Regulatory Milestones \| Commitments \| Valuation Technique, Discounted Cash Flow \| Measurement Input, Discount Rate \| Maximum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.062	0.061
Development and Regulatory Milestones \| Upstaza \| Commitments \| Valuation Technique, Discounted Cash Flow \| Probability of Success \| Weighted Average
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.90
Development and Regulatory Milestones \| Upstaza \| Commitments \| Valuation Technique, Discounted Cash Flow \| Measurement Input, Discount Rate \| Weighted Average
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.060
Development and Regulatory Milestones \| Significant unobservable inputs (Level 3) \| Commitments
Fair Value Valuation Inputs
Fair value	$ 26,900	$ 26,600	$ 85,500	$ 82,500
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Sales Milestones \| Minimum
Fair Value Valuation Inputs
Potential milestones	0	0
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Sales Milestones \| Maximum
Fair Value Valuation Inputs
Potential milestones	$ 50,000	$ 50,000
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Probability of Success \| Minimum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.85	0.85
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Probability of Success \| Maximum
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	1	1
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Percentage of Sales for Royalties
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0	0
Net Sales Milestones and Royalties \| Commitments \| Valuation Technique, Option Pricing Model \| Measurement Input, Discount Rate
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.135	0.11
Net Sales Milestones and Royalties \| Upstaza \| Commitments \| Valuation Technique, Option Pricing Model \| Probability of Success \| Weighted Average
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.93
Net Sales Milestones and Royalties \| Upstaza \| Commitments \| Valuation Technique, Option Pricing Model \| Measurement Input, Discount Rate \| Weighted Average
Fair Value Valuation Inputs
Rights Exchange Settlement measurement	0.135
Net Sales Milestones and Royalties \| Significant unobservable inputs (Level 3) \| Commitments
Fair Value Valuation Inputs
Fair value	$ 9,300	$ 9,700	$ 80,900	$ 81,500

Accounts payable and accrued _3

Accounts payable and accrued expenses - Components (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Payables and Accruals [Abstract]
Employee compensation, benefits, and related accruals	$ 35,060	$ 62,643
Income tax payable	1,833
Consulting and contracted research	22,745	27,500
Professional fees	2,380	2,246
Sales allowance	67,075	77,176
Sales rebates	132,047	131,334
Royalties	28,952	74,111
Accounts payable	27,459	6,045
Other	13,373	10,928
Accounts payable and accrued expenses	$ 330,924	$ 391,983

Accounts payable and accrued _4

Accounts payable and accrued expenses - Restructuring (Details) - USD ($) $ in Millions	Mar. 31, 2024	Dec. 31, 2023
Restructuring costs
Accrued restructuring costs	$ 1.7	$ 9

Capitalization - Narrative (Det

Capitalization - Narrative (Details) - USD ($) $ in Millions	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023	Aug. 31, 2019
Common stock
Common stock, authorized shares (in shares)	250,000,000		250,000,000
Sales Agreement
Common stock
Number of shares issued in transaction (in shares)	0	0
Sales Agreement \| Maximum
Common stock
Value of shares available to be issued and sold in transaction				$ 125
Aggregate value of remaining shares to be issued and sold	$ 93

Net loss per share - Numerator

Net loss per share - Numerator and Denominator (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Numerator
Net loss - Basic	$ (91,576)	$ (138,959)
Denominator
Basic	76,496,127	73,729,284
Diluted	76,496,127	73,729,284
Net loss per share - Basic	$ (1.20)	$ (1.88)
Net loss per share - Diluted	$ (1.20)	$ (1.88)

Net loss per share - Antidiluti

Net loss per share - Antidilutive (Details) - shares	3 Months Ended
Net loss per share - Antidilutive (Details) - shares	Mar. 31, 2024	Mar. 31, 2023
Net loss per share
Total shares excluded from calculation (in shares)	13,260,351	15,750,292
Employee Stock Option
Net loss per share
Total shares excluded from calculation (in shares)	9,530,185	12,100,550
Restricted stock awards and units
Net loss per share
Total shares excluded from calculation (in shares)	3,730,166	3,649,742

Stock award plan - Narrative (D

Stock award plan - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands		1 Months Ended			3 Months Ended
	Jun. 08, 2022	Dec. 31, 2022	Dec. 31, 2020	Jun. 30, 2016	Mar. 31, 2024	Mar. 31, 2023	Apr. 30, 2022	Jun. 30, 2021	May 31, 2021	Jan. 31, 2020
Stock option plan
Share-based compensation expense					$ 18,378	$ 28,815
Unrecognized compensation cost					$ 156,300
Weighted average remaining service period for recognition of unrecognized compensation cost					2 years 4 months 24 days
Stock option
Stock option plan
Granted (in shares)					855,385
Options forfeited (in shares)					815,707
Expected dividend yield (as a percent)					0%
Weighted average grant date fair value (in dollars per share)					$ 13.49
Restricted stock units
Stock option plan
Granted (in shares)					1,785,660
Employee Stock Purchase Plan
Stock option plan
Number of shares authorized (in shares)								2,000,000	1,000,000
Award requisite service period				6 months
Purchase price of common stock, percent				85%
Employee stock purchase plan, voting percentage limit				5%
Share-based compensation expense					$ 600
1998 Employee, Director, and Consultant Stock Option Plan, 2009 Equity and Long-term Incentive Plan, and 2013 Stock Incentive Plan \| Common stock
Stock option plan
Number of shares available for issuance					0
Amended 2013 LTIP
Stock option plan
Number of shares available for issuance					6,618,698
Number of additional shares authorized (in shares)	8,475,000
Amended 2013 LTIP \| Maximum
Stock option plan
Number of shares authorized (in shares)	16,724,212
2020 Inducement Stock Incentive Plan \| Common stock
Stock option plan
Number of shares available for issuance					1,989,827
Number of additional shares authorized (in shares)		1,700,000	1,000,000
2020 Inducement Stock Incentive Plan \| Common stock \| Maximum
Stock option plan
Number of shares authorized (in shares)							1,300,000			1,000,000
2020 Inducement Stock Incentive Plan \| Stock option
Stock option plan
Granted (in shares)					855,385
Stockholder's specified ownership percentage					10%
Inducement grants for non-statutory stock options (in shares)					1,840
Vesting period					4 years
2020 Inducement Stock Incentive Plan \| Stock option \| Minimum
Stock option plan
Stock options granted, exercise price as percentage of the fair market value of common stock at grant date					100%
2020 Inducement Stock Incentive Plan \| Stock option \| Maximum
Stock option plan
Stock options granted to stockholder with specified ownership percentage, exercise price as percentage of the fair market value of common stock at grant date					110%
Expiration period					10 years
2020 Inducement Stock Incentive Plan \| Restricted stock units
Stock option plan
Granted (in shares)					4,275

Stock award plan - Stock Option

Stock award plan - Stock Option Activity (Details) - Stock option $ / shares in Units, $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($) $ / shares shares
Number of options
Outstanding at the beginning of the period (in shares) \| shares	9,600,399
Granted (in shares) \| shares	855,385
Exercised (in shares) \| shares	(109,892)
Forfeited/Cancelled (in shares) \| shares	(815,707)
Outstanding at the end of the period (in shares) \| shares	9,530,185
Vested or Expected to vest at the end of the period (in shares) \| shares	2,112,348
Exercisable at the end of the period (in shares) \| shares	7,173,552
Weighted- average exercise price
Outstanding at the beginning of the period (in dollars per share) \| $ / shares	$ 43.59
Granted (in dollars per share) \| $ / shares	25.69
Exercised (in dollars per share) \| $ / shares	19.22
Forfeited/Cancelled (in dollars per share) \| $ / shares	45.32
Outstanding at the end of the period (in dollars per share) \| $ / shares	42.12
Vested or Expected to vest at the end of the period (in dollars per share) \| $ / shares	38.57
Exercisable at the end of the period (in dollars per share) \| $ / shares	$ 43.44
Weighted- average remaining contractual term
Outstanding at the end of the period	5 years 10 months 24 days
Vested or Expected to vest at the end of the period	8 years 8 months 1 day
Exercisable at the end of the period	4 years 11 months 23 days
Aggregate intrinsic value (in thousands)
Outstanding at the end of the period (in dollars) \| $	$ 12,025
Vested or Expected to vest at the end of the period (in dollars) \| $	2,949
Exercisable at the end of the period (in dollars) \| $	$ 8,598

Stock award plan - Assumptions

Stock award plan - Assumptions Used (Details) - Stock option	3 Months Ended
	Mar. 31, 2024
Valuation assumptions
Risk-free interest rate (as a percent)	4.23%
Expected volatility (as a percent)	53%
Expected term (in years)	5 years 6 months

Stock award plan - Restricted S

Stock award plan - Restricted Stock (Details) - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended	3 Months Ended
	Dec. 31, 2023	Mar. 31, 2024	Mar. 31, 2023
Number of Shares
Share-based compensation expense		$ 18,378	$ 28,815
Restricted stock awards and units
Number of Shares
Balance at the beginning of the period (in shares)		2,866,270
Granted (in shares)		1,785,660
Vested (in shares)		(835,179)
Forfeited (in shares)		(86,585)
Balance at the end of the period (in shares)	2,866,270	3,730,166
Weighted Average Grant Date Fair Value
Balance at the beginning of the period (in dollars per share)		$ 41.82
Granted (in dollars per share)		25.69
Vested (in dollars per share)		44.56
Forfeited (in dollars per share)		40.98
Balance at the end of the period (in dollars per share)	$ 41.82	$ 33.51
Performance-based restricted stock units (PSUs)
Number of Shares
Granted (in shares)	150,000
Share-based compensation expense	$ 0
Performance period	2 years

Stock award plan - Share-based

Stock award plan - Share-based compensation expense (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Stock option plan
Share-based compensation expense	$ 18,378	$ 28,815
Unrecognized compensation cost	$ 156,300
Weighted average remaining service period for recognition of unrecognized compensation cost	2 years 4 months 24 days
Research and development
Stock option plan
Share-based compensation expense	$ 8,967	15,314
Selling, general and administrative
Stock option plan
Share-based compensation expense	$ 9,411	$ 13,501

Debt - Liability for Sale of Fu

Debt - Liability for Sale of Future Royalties (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($)
Debt Disclosure [Abstract]
Beginning balance	$ 1,814,097
Less: Non-cash royalty revenue payable to RPI	(25,228)
Plus: non-cash interest expense recognized	50,064
Ending balance	$ 1,838,933
Effective interest rate of the liability component	10.80%

Debt - Senior Secured Term Loan

Debt - Senior Secured Term Loan (Details) - USD ($) $ in Thousands			3 Months Ended		12 Months Ended
	Oct. 19, 2023	Oct. 27, 2022	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Interest expense
Amortization of debt issuance costs			$ 296	$ 438
Effective interest rate			10.80%
Blackstone Credit Agreement
Debt Facility
Maximum borrowing capacity		$ 950,000
Debt issuance costs capitalized		$ 11,600
Principal					$ 300,000
Repayment of senior secured term loan					(300,000)
Interest expense
Contractual interest expense				9,178
Amortization of debt issuance costs				150
Total				$ 9,328
Effective interest rate				13.20%
Blackstone Credit Agreement \| Adjusted Term SOFR
Debt Facility
Basis spread on variable rate		7.25%
Floor interest rate		1%
Blackstone Credit Agreement \| Base Rate
Debt Facility
Basis spread on variable rate		6.25%
Floor interest rate		2%
Committed Loan Facility
Debt Facility
Maximum borrowing capacity		$ 450,000
Fund draw term		18 months
Loan term		7 years
Senior Secured Term Loan
Debt Facility
Principal		$ 300,000
Repayment of senior secured term loan	$ (300,000)
Payment of accrued interest	2,100
Prepayment premiums, expenses and other exit fees	82,000				82,000
Legal fees	$ 200
Loss on extinguishment of debt					92,700
Debt issuance costs					$ 10,700
Committed Delayed Draw Term Loan Facility
Debt Facility
Maximum borrowing capacity		150,000
Uncommitted Incremental Facility
Debt Facility
Maximum borrowing capacity		$ 500,000

Debt - Narrative (Details)

Debt - Narrative (Details)	1 Months Ended	3 Months Ended
Debt - Narrative (Details)	Sep. 30, 2019 USD ($) D $ / shares	Mar. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)
Long-term debt
Long-term debt		$ 284,512,000	$ 284,213,000
Additional paid-in capital		2,486,722,000	2,466,233,000
1.50% Convertible senior notes due 2026
Long-term debt
Net carrying amount		284,512,000	284,213,000
Debt principal amount		$ 287,500,000	$ 287,500,000
Convertible debt \| 1.50% Convertible senior notes due 2026
Long-term debt
Debt principal amount	$ 287,500,000
Debt instrument additional amount available for repurchase	$ 37,500,000
Interest rate ( as a percent )	1.50%	1.50%
Net proceeds from issuance of convertible notes	$ 279,300,000
Trading days, number \| D	20
Consecutive trading days, period \| D	30
Stock price trigger	130%
Business days, period	5 days
Consecutive trading-day period	5 days
Maximum product of the closing sale price of shares of the Company's common stock and the applicable conversion rate for such trading day	98%
Conversion ratio	19.0404
Conversion price per share (in dollars per share) \| $ / shares	$ 52.52
Redemption price as percent of principal amount	100%
Convertible instruments principal and unpaid interest payable upon events of default	100%
Minimum percentage of principal held by convertible debt instrument holders required to issue notice for declaration of principal and unpaid interest payable upon events of default	25%
Term of the convertible notes		2 years 6 months
Convertible debt \| 1.50% Convertible senior notes due 2026 \| Redemption on or after September 20, 2023
Long-term debt
Trading days, number \| D	19
Consecutive trading days, period \| D	30
Stock price trigger	130%
Redemption price as percent of principal amount	100%
Sinking fund	$ 0

Debt - Convertible Notes (Detai

Debt - Convertible Notes (Details) - 1.50% Convertible senior notes due 2026 - USD ($)	Mar. 31, 2024	Dec. 31, 2023	Sep. 30, 2019
Long-term debt
Principal	$ 287,500,000	$ 287,500,000
Less: Debt issuance costs	(2,988,000)	(3,287,000)
Net carrying amount	$ 284,512,000	$ 284,213,000
Convertible debt
Long-term debt
Principal			$ 287,500,000

Debt - Convertible Notes Intere

Debt - Convertible Notes Interest Expense (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Long-term debt
Amortization of debt issuance costs	$ 296	$ 438
Effective interest rate of the liability component	10.80%
1.50% Convertible senior notes due 2026
Long-term debt
Contractual interest expense	$ 1,076	1,069
Amortization of debt issuance costs	296	288
Total	$ 1,372	$ 1,357
Effective interest rate of the liability component	1.90%	1.90%

Commitments and Contingencies -

Commitments and Contingencies - Narrative (Details) - USD ($) $ in Thousands		3 Months Ended			12 Months Ended
	Apr. 29, 2020	Mar. 31, 2024	Mar. 31, 2023	Jun. 30, 2016	Dec. 31, 2023	Dec. 31, 2022	May 29, 2020
Other Commitments
Milestone payable		$ 2,500	$ 32,500
Accrued royalties		28,952			$ 74,111
Wellcome Trust
Other Commitments
Development milestone payment obligations						$ 2,500
Milestone obligation payments made				$ 800
Censa
Other Commitments
Asset acquisition, development and regulatory milestones							$ 109,000
Censa \| Maximum
Other Commitments
Asset acquisition, milestone, amount							217,500
Asset acquisition, net sales milestone							$ 160,000
Former Censa Securityholders
Other Commitments
Potential developmental and regulatory milestones		65,000
Development milestone obligation payments made			$ 30,000
Development milestone obligation cash payments made		$ 400
Shares issued for milestone payment		657,462
SMA License Agreement \| SMA Foundation
Other Commitments
Royalty expense since inception					52,500
Royalty payments					35,300
Accrued royalties		$ 17,200
SMA License Agreement \| SMA Foundation \| Maximum
Other Commitments
Potential royalty payments due on net product sales					$ 52,500
Agilis
Other Commitments
Development milestone payments which the entity is obligated to pay		40,000
Potential developmental and regulatory milestones		31,100
Potential sales milestones		50,000
Milestone obligation payments made						$ 50,000
Development milestone obligation payments made		$ 2,400
Agilis \| Minimum
Other Commitments
Product sales (as a percent)		2%
Agilis \| Maximum
Other Commitments
Development milestone payments which the entity is obligated to pay		$ 60,000
Priority review voucher amount		535,000
Potential sales milestones		$ 150,000
Product sales (as a percent)		6%
Development milestone payment obligations, net of cancellation and forfeiture		$ 20,000
Contingent liability, milestone, potential achievements, priority review voucher amount, net of cancellation and forfeiture		361,000
Agilis \| Rights Exchange Agreement
Other Commitments
Contingent liability cancellation and forfeiture of potential milestone payments	$ 174,000
Development milestone payment obligations, cancellation and forfeiture	37,600
Contingent liability, cancellation and forfeiture	$ 211,600
BioElectron \| Maximum
Other Commitments
Development milestone payments which the entity is obligated to pay		$ 200,000

Revenue recognition - Net Produ

Revenue recognition - Net Product Sales (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($) Distributor country segment	Mar. 31, 2023 USD ($) segment country Distributor	Dec. 31, 2023 USD ($)
Revenue recognition
Number of operating segments \| segment	1	1
Revenue	$ 210,118	$ 220,382
Net product revenue \| Geographic concentration risk
Revenue recognition
Number of countries exceeding 10% of net product sales \| country	2	3
Percentage of net product sales threshold	10%	10%
Net product revenue \| Geographic concentration risk \| United States
Revenue recognition
Revenue	$ 57,500	$ 54,600
Net product revenue \| Geographic concentration risk \| Non-US
Revenue recognition
Revenue	120,100	133,000
Net product revenue \| Geographic concentration risk \| Russia
Revenue recognition
Revenue	$ 52,600	44,600
Net product revenue \| Geographic concentration risk \| Brazil
Revenue recognition
Revenue		$ 25,900
Net product revenue \| Customer concentration risk
Revenue recognition
Number of distributors \| Distributor	2	2
Percentage of net product sales threshold	10%	10%
Net product revenue
Revenue recognition
Revenue	$ 177,604	$ 187,557
Contract with customer, liability	0		$ 0
Emflaza \| Net product revenue \| Geographic concentration risk \| United States
Revenue recognition
Revenue	57,500	54,600
Translarna \| Net product revenue \| Geographic concentration risk \| Non-US
Revenue recognition
Revenue	$ 103,600	$ 115,100

Revenue recognition - Collabora

Revenue recognition - Collaboration and Royalty Revenue (Details)	3 Months Ended
	Mar. 31, 2024 USD ($) product	Mar. 31, 2023 USD ($)	Nov. 30, 2011 USD ($)
Revenue recognition
Number of approved products \| product	2
Remaining potential milestones that can be achieved	$ 150,000,000
Revenue	210,118,000	$ 220,382,000
Collaboration revenue
Revenue recognition
Revenue		6,000
Royalty revenue
Revenue recognition
Revenue	$ 31,154,000	30,831,000
SMA License Agreement
Revenue recognition
Number of approved products \| product	1
SMA License Agreement \| Collaboration revenue
Revenue recognition
Revenue	$ 0	0
SMA License Agreement \| Royalty revenue
Revenue recognition
Revenue	31,200,000	$ 30,800,000
Research And Development Event Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved	0
Sales Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved	$ 150,000,000
Maximum \| Research And Development Event Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved			$ 135,000,000
Maximum \| Sales Milestones \| SMA License Agreement
Revenue recognition
Remaining potential milestones that can be achieved			$ 325,000,000

Revenue recognition - Manufactu

Revenue recognition - Manufacturing Revenue (Details) - USD ($)	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Revenue recognition
Revenue	$ 210,118,000	$ 220,382,000
Manufacturing revenue
Revenue recognition
Revenue	1,360,000	$ 1,988,000
Contract with customer, asset	800,000		$ 200,000
Contract liability revenues recognized in period	800,000
Manufacturing revenue \| Deferred revenue
Revenue recognition
Contract with customer, liability	0		800,000
Manufacturing revenue \| Prepaids and other current assets
Revenue recognition
Contract with customer, asset	$ 800,000		$ 200,000

Revenue recognition - Performan

Revenue recognition - Performance Obligations (Details) - USD ($)	Mar. 31, 2024	Dec. 31, 2023
Revenue from Contract with Customer [Abstract]
Remaining performance obligations	$ 0	$ 800,000

Intangible assets and goodwil_2

Intangible assets and goodwill - Definite-lived Intangibles (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Definite-lived intangible assets
Milestone Payable	$ 2,500	$ 32,500
Accrued royalties	28,952		$ 74,111
Definite-lived intangibles
Amortization	(51,530)	(39,415)
Finite-Lived Intangible Assets, Net	$ 94,274
Weighted Average
Definite-lived intangible assets
Remaining amortization period	9 years 4 months 24 days
Akcea \| Waylivra
Definite-lived intangible assets
Royalty payments	$ 1,700
Akcea \| Waylivra \| Accounts payable and accrued expenses
Definite-lived intangible assets
Accrued royalties	1,600
Akcea \| Tegsedi
Definite-lived intangible assets
Royalty payments	800
Akcea \| Tegsedi \| Accounts payable and accrued expenses
Definite-lived intangible assets
Accrued royalties	800
Definite-lived intangible assets
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	640,507
Additions	2,450
Foreign Currency Translation	(566)
Ending balance - Definite-lived assets, Gross	642,391
Beginning balance - Accumulated amortization	(496,776)
Amortization	(51,530)	$ (39,400)
Foreign Currency Translation - Accumulated amortization	189
Ending balance - Accumulated amortization	(548,117)
Finite-Lived Intangible Assets, Net	94,274
Definite-lived intangible assets \| Emflaza
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	527,417
Ending balance - Definite-lived assets, Gross	527,417
Beginning balance - Accumulated amortization	(478,618)
Amortization	(48,799)
Ending balance - Accumulated amortization	(527,417)
Definite-lived intangible assets \| Waylivra
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	10,218
Additions	1,658
Foreign Currency Translation	(246)
Ending balance - Definite-lived assets, Gross	11,630
Beginning balance - Accumulated amortization	(3,965)
Amortization	(366)
Foreign Currency Translation - Accumulated amortization	106
Ending balance - Accumulated amortization	(4,225)
Definite-lived intangible assets \| Tegsedi
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	13,322
Additions	792
Foreign Currency Translation	(320)
Ending balance - Definite-lived assets, Gross	13,794
Beginning balance - Accumulated amortization	(3,311)
Amortization	(499)
Foreign Currency Translation - Accumulated amortization	83
Ending balance - Accumulated amortization	(3,727)
Definite-lived intangible assets \| Upstaza
Definite-lived intangibles
Beginning balance - Definite-lived assets, Gross	89,550
Ending balance - Definite-lived assets, Gross	89,550
Beginning balance - Accumulated amortization	(10,882)
Amortization	(1,866)
Ending balance - Accumulated amortization	$ (12,748)

Intangible assets and goodwil_3

Intangible assets and goodwill - Future Amortization (Details) $ in Thousands	Mar. 31, 2024 USD ($)
Goodwill and Intangible Assets Disclosure [Abstract]
2024	$ 8,182
2025	10,898
2026	10,898
2027	10,898
2028 and thereafter	53,398
Total	$ 94,274

Intangible assets and goodwil_4

Intangible assets and goodwill - Indefinite-lived Intangibles (Details) - USD ($)	3 Months Ended
	Mar. 31, 2024	Dec. 31, 2023
Indefinite-lived intangibles
Ending Balance	$ 330,040,000
Intangible assets, net	330,040,000	$ 379,497,000
Change in indefinite-lived intangibles	0
Indefinite-lived intangible assets
Indefinite-lived intangibles
Beginning Balance	235,766,000
Ending Balance	235,766,000
Indefinite-lived intangible assets \| Upstaza
Indefinite-lived intangibles
Beginning Balance	235,766,000
Ending Balance	$ 235,766,000

Intangible assets and goodwil_5

Intangible assets and goodwill - Goodwill (Details) - USD ($) $ in Thousands	67 Months Ended
	Mar. 31, 2024	Dec. 31, 2023	Aug. 23, 2018
Goodwill
Goodwill	$ 82,341	$ 82,341
Changes in goodwill	$ 0
Agilis
Goodwill
Goodwill			$ 82,300

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Mar. 31, 2024	Mar. 31, 2023
Pay vs Performance Disclosure
Net Income (Loss)	$ (91,576)	$ (138,959)

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Mar. 31, 2024
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	Name Action Taken Type of Trading Nature of Trading Duration of Trading Aggregate Number Matthew Klein Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2028 Indeterminable (1) Pierre Gravier Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2028 Indeterminable (1) Pierre Gravier Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2026 Indeterminable (1) Neil Almstead Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2028 Indeterminable (1) Neil Almstead Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2026 Indeterminable (1) Mark Boulding Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2028 Indeterminable (1) Mark Boulding Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2026 Indeterminable (1) Lee Golden Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2028 Indeterminable (1) Lee Golden Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2026 Indeterminable (1) Eric Pauwels Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2028 Indeterminable (1) Eric Pauwels Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2026 Indeterminable (1) Christine Utter Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2028 Indeterminable (1) Christine Utter Adoption Rule 10b5-1 trading arrangement for sell-to-cover transactions for RSUs granted on February 15, 2024 Sale Until final settlement of RSUs on or around February 15, 2026 Indeterminable (1) (1) The number of shares subject to this RSU grant that will be sold to satisfy applicable tax withholding obligations upon vesting is unknown as the number will vary based on the extent to which vesting conditions are satisfied and the market price of the Company’s common stock at the time of settlement. This trading arrangement provides for the automatic sale of shares that would otherwise be issuable on each settlement date of the RSU in an amount sufficient to satisfy the applicable withholding obligation, with the proceeds of the sale delivered to the Company in satisfaction of the applicable withholding obligation.
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false
Restricted Stock Unit Trading Arrangement Expiring 2026 \| Pierre Gravier
Trading Arrangements, by Individual
Name	Pierre Gravier
Title	Chief Financial Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2026
Restricted Stock Unit Trading Arrangement Expiring 2026 \| Neil Almstead
Trading Arrangements, by Individual
Name	Neil Almstead
Title	Chief Technical Operations Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2026
Restricted Stock Unit Trading Arrangement Expiring 2026 \| Mark Boulding
Trading Arrangements, by Individual
Name	Mark Boulding
Title	EVP & Chief Legal Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2026
Restricted Stock Unit Trading Arrangement Expiring 2026 \| Lee Golden
Trading Arrangements, by Individual
Name	Lee Golden
Title	EVP & Chief Medical Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2026
Restricted Stock Unit Trading Arrangement Expiring 2026 \| Eric Pauwels
Trading Arrangements, by Individual
Name	Eric Pauwels
Title	Chief Business Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2026
Restricted Stock Unit Trading Arrangement Expiring 2026 \| Christine Utter
Trading Arrangements, by Individual
Name	Christine Utter
Title	SVP,Chief Accounting Officer and Head of People Services
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2026
Restricted Stock Unit Trading Arrangement Expiring 2028 \| Matthew Klein
Trading Arrangements, by Individual
Name	Matthew Klein
Title	Chief Executive Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2028
Restricted Stock Unit Trading Arrangement Expiring 2028 \| Pierre Gravier
Trading Arrangements, by Individual
Name	Pierre Gravier
Title	Chief Financial Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2028
Restricted Stock Unit Trading Arrangement Expiring 2028 \| Neil Almstead
Trading Arrangements, by Individual
Name	Neil Almstead
Title	Chief Technical Operations Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2028
Restricted Stock Unit Trading Arrangement Expiring 2028 \| Mark Boulding
Trading Arrangements, by Individual
Name	Mark Boulding
Title	EVP & Chief Legal Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2028
Restricted Stock Unit Trading Arrangement Expiring 2028 \| Lee Golden
Trading Arrangements, by Individual
Name	Lee Golden
Title	EVP & Chief Medical Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2028
Restricted Stock Unit Trading Arrangement Expiring 2028 \| Eric Pauwels
Trading Arrangements, by Individual
Name	Eric Pauwels
Title	Chief Business Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2028
Restricted Stock Unit Trading Arrangement Expiring 2028 \| Christine Utter
Trading Arrangements, by Individual
Name	Christine Utter
Title	SVP,Chief Accounting Officer and Head of People Services
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	February 15, 2024
Expiration Date	February 15, 2028