CRISPR Therapeutics (CRSP) 10-Q8 May 232023 Q1 Quarterly reportFinancial data

Company Profile

Document and Entity Information

Document and Entity Information - shares	3 Months Ended
Document and Entity Information - shares	Mar. 31, 2023	May 05, 2023
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Mar. 31, 2023
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q1
Title of 12(b) Security	Common Shares, nominal value CHF 0.03
Trading Symbol	CRSP
Security Exchange Name	NASDAQ
Entity Registrant Name	CRISPR THERAPEUTICS AG
Entity Central Index Key	0001674416
Entity Tax Identification Number	00-0000000
Current Fiscal Year End Date	--12-31
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Common Stock, Shares Outstanding		78,935,098
Entity Current Reporting Status	Yes
Entity Shell Company	false
Entity File Number	001-37923
Entity Address, Address Line One	Baarerstrasse 14
Entity Address, City or Town	Zug
Entity Address, Country	CH
Entity Address, Postal Zip Code	6300
City Area Code	41 (0)41
Local Phone Number	561 32 77
Entity Interactive Data Current	Yes
Entity Incorporation, State or Country Code	V8
Document Quarterly Report	true
Document Transition Report	false

Condensed Consolidated Balance

Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022	Sep. 30, 2022	Dec. 31, 2021
Current assets:
Cash and cash equivalents	$ 344,407	$ 211,885
Marketable securities	1,538,763	1,603,433
Prepaid expenses and other current assets	24,796	37,708
Total current assets	1,907,966	1,853,026
Property and equipment, net	162,198	163,634
Marketable securities, non-current	6,320	53,130
Intangible assets, net	57	71
Restricted cash	11,717	11,635
Operating lease assets	153,346	156,921
Other non-current assets	2,760	4,640
Total assets	2,244,364	2,243,057
Current liabilities:
Accounts payable	45,153	27,428
Accrued expenses	85,631	77,682
Accrued tax liabilities	273	135
Operating lease liabilities	15,717	15,842
Other current liabilities	20	20
Total current liabilities	146,794	121,107
Deferred revenue, non-current	12,323	12,323
Operating lease liabilities, net of current portion	224,552	228,179
Other non-current liabilities	5,799	5,969
Total liabilities	389,468	367,578
Commitments and contingencies, see Note 7
Shareholders’ equity:
Common shares, CHF 0.03 par value, 150,347,467 shares authorized at March 31, 2023 and at December 31, 2022, 79,044,050 and 78,692,766 shares issued at March 31, 2023 and December 31, 2022, respectively,78,863,734 and 78,512,450 shares outstanding at March 31, 2023 and December 31, 2022, respectively	2,452	2,441
Treasury shares, at cost, 180,316 shares at March 31, 2023 and at December 31, 2022	(63)	(63)
Additional paid-in capital	2,761,050	2,734,838
Accumulated deficit	(899,155)	(846,090)
Accumulated other comprehensive loss	(9,388)	(15,647)
Total shareholders' equity	1,854,896	1,875,479		$ 2,399,460
Total liabilities and shareholders’ equity	$ 2,244,364	$ 2,243,057

Condensed Consolidated Balanc_2

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Common stock, par value	$ 0.03	$ 0.03
Common stock, shares authorized	150,347,467	150,347,467
Common stock, shares issued	79,044,050	78,692,766
Common stock, shares outstanding	78,863,734	78,512,450
Treasury stock, shares	180,316	180,316

Condensed Consolidated Statemen

Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Revenue:
Total revenue	$ 100,000	$ 940
Operating expenses:
Research and development	99,935	118,245
General and administrative	22,360	28,021
Collaboration expense, net	42,192	30,646
Total operating expenses	164,487	176,912
Loss from operations	(64,487)	(175,972)
Other income:
Other income, net	12,742	363
Total other income, net	12,742	363
Net loss before income taxes	(51,745)	(175,609)
Provision for income taxes	(1,320)	(3,608)
Net loss	(53,065)	(179,217)
Foreign currency translation adjustment	32	(27)
Unrealized gain (loss) on marketable securities	6,227	(11,799)
Comprehensive loss	$ (46,806)	$ (191,043)
Net loss per common share - basic	$ (0.67)	$ (2.32)
Basic weighted-average common shares outstanding	78,676,986	77,098,319
Net loss per common share - diluted	$ (0.67)	$ (2.32)
Diluted weighted-average common shares outstanding	78,676,986	77,098,319
Collaboration Revenue [Member]
Revenue:
Total revenue	$ 100,000	$ 178
Grant Revenue [Member]
Revenue:
Total revenue	$ 0	$ 762

Condensed Consolidated Statem_2

Condensed Consolidated Statements of Shareholders' Equity - USD ($) $ in Thousands	Total	Common Shares [Member]	Treasury Shares [Member]	Additional Paid-in Capital [Member]	Accumulated Deficit [Member]	Accumulated Other Comprehensive Income (Loss) [Member]
Beginning balance at Dec. 31, 2021	$ 2,399,460	$ 2,391	$ (63)	$ 2,598,114	$ (195,915)	$ (5,067)
Beginning balance (in shares) at Dec. 31, 2021		76,990,066	180,316
Vesting of restricted shares	4	$ 4
Vesting of restricted shares (in shares)		123,564
Exercise of vested options, net of issuance costs, value	10,010	$ 12		9,998
Exercise of vested options, net of issuance costs (in shares)		261,280
Purchase of common stock under ESPP	740			740
Purchase of common stock under ESPP (in shares)		11,495
Stock-based compensation expense	25,745			25,745
Other comprehensive income (loss)	(11,826)					(11,826)
Net loss	(179,217)				(179,217)
Ending balance at Mar. 31, 2022	2,244,916	$ 2,407	$ (63)	2,634,597	(375,132)	(16,893)
Ending balance (in shares) at Mar. 31, 2022		77,386,405	180,316
Beginning balance at Dec. 31, 2022	1,875,479	$ 2,441	$ (63)	2,734,838	(846,090)	(15,647)
Beginning balance (in shares) at Dec. 31, 2022		78,512,450	180,316
Vesting of restricted shares	5	$ 5
Vesting of restricted shares (in shares)		172,995
Exercise of vested options, net of issuance costs, value	$ 4,683	$ 6		4,677
Exercise of vested options, net of issuance costs (in shares)	159,184	159,184
Purchase of common stock under ESPP	$ 660			660
Purchase of common stock under ESPP (in shares)		19,105
Stock-based compensation expense	20,875			20,875
Other comprehensive income (loss)	6,259					6,259
Net loss	(53,065)				(53,065)
Ending balance at Mar. 31, 2023	$ 1,854,896	$ 2,452	$ (63)	$ 2,761,050	$ (899,155)	$ (9,388)
Ending balance (in shares) at Mar. 31, 2023		78,863,734	180,316

Condensed Consolidated Statem_3

Condensed Consolidated Statements of Shareholders' Equity (Parenthetical) - USD ($) $ in Millions	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Stock Options [Member]
Issuance costs	$ 0.2	$ 0.2

Condensed Consolidated Statem_4

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended	9 Months Ended
	Mar. 31, 2023	Sep. 30, 2022
Operating activities:
Net loss	$ (53,065)	$ (179,217)
Reconciliation of net loss to net cash used in operating activities:
Depreciation and amortization	5,051	6,039
Equity-based compensation	20,875	25,745
Other non-cash items, net	(969)	6,099
Changes in:
Accounts receivable	0	162
Prepaid expenses and other assets	12,040	4,611
Accounts payable and accrued expenses	25,214	(1,616)
Deferred revenue	0	(762)
Operating lease assets and liabilities	(177)	3,854
Other liabilities, net	(170)	(154)
Net cash provided by (used in) operating activities	8,799	(135,239)
Investing activities:
Purchase of property, plant and equipment	(3,059)	(15,350)
Purchases of marketable securities	(265,627)	(323,140)
Maturities of marketable securities	386,515	223,975
Net cash provided by (used in) investing activities	117,829	(114,515)
Financing activities:
Proceeds from exercise of options and ESPP contributions, net of issuance costs	5,404	10,649
Net cash provided by financing activities	5,404	10,649
Effect of exchange rate changes on cash	32	(27)
Increase (decrease) in cash	132,064	(239,132)
Cash, cash equivalents and restricted cash, beginning of period	224,060	939,944
Cash, cash equivalents and restricted cash, end of period	356,124
Supplemental disclosure of non-cash investing and financing activities
Property and equipment purchases in accounts payable and accrued expenses	2,663	5,888
Equity issuance costs in accounts payable and accrued expenses	155	217
Leasehold improvements paid directly by landlord	$ 0	$ 13,015

Condensed Consolidated Statem_5

Condensed Consolidated Statements of Cash Flows (Parenthetical) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022	Mar. 31, 2022	Dec. 31, 2021
Cash and cash equivalents	$ 344,407	$ 211,885	$ 683,906
Prepaid expenses and other current assets	0
Restricted cash	11,717	11,635	12,123
Cash, cash equivalents and restricted cash at end of period	$ 356,124	$ 224,060	700,812	$ 939,944
Prepaid Expenses and Other Current Assets [Member]
Prepaid expenses and other current assets			$ 4,783

Basis of Presentation and Signi

Basis of Presentation and Significant Accounting Policies	3 Months Ended
Basis of Presentation and Significant Accounting Policies	Mar. 31, 2023
Accounting Policies [Abstract]
Basis of Presentation and Significant Accounting Policies	1. Basis of Presentation and Significant Accounting Policies Basis of Presentation The accompanying condensed consolidated financial statements are unaudited and have been prepared by the Company in accordance with accounting principles generally accepted in the United States of America, or U.S. GAAP. The accompanying condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The Company views its operations and manages its business in one operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. Certain information and footnote disclosures normally included in the Company’s annual financial statements have been condensed or omitted. These interim financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the financial position and results of operations for the three-month interim periods ended March 31, 2023 and 2022. Beginning in 2022, "collaboration expense, net" in the condensed consolidated statements of operations and comprehensive loss present collaboration costs related to the exagamglogene autotemcel, or exa-cel (formerly known as CTX001), program under the Company's collaboration with Vertex Pharmaceuticals Incorporated and certain of its subsidiaries, or Vertex, accounted for under ASC 808, Collaborative Agreements , or ASC 808. Refer to Note 6 to these condensed consolidated financial statements for further discussion on the Company's agreements with Vertex, including the exa-cel program. The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year. These interim financial statements should be read in conjunction with the audited financial statements for the year ended December 31, 2022, which are contained in the 2022 Annual Report on Form 10-K filed with the Securities and Exchange Commission, or the SEC, on February 21, 2023 . Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, revenue recognition, equity-based compensation expense and reported amounts of expenses during the period. Significant estimates in these consolidated financial statements have been made in connection with revenue recognition and equity-based compensation expense. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Changes in estimates are reflected in reported results in the period in which they become known. Significant Accounting Policies The significant accounting policies used in preparation of these condensed consolidated financial statements for the three months ended March 31, 2023 are consistent with those discussed in Note 2 to the consolidated financial statements in the Company’s 2022 Annual Report on Form 10-K filed with the SEC on February 21, 2023. New Accounting Pronouncements – Recently Adopted From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board, or FASB, or other standard setting bodies that the Company adopts as of the specified effective date. The Company does not believe that the adoption of recently issued standards have or may have a material impact on its consolidated financial statements and disclosures.

Marketable Securities

Marketable Securities	3 Months Ended
Marketable Securities	Mar. 31, 2023
Investments, Debt and Equity Securities [Abstract]
Marketable Securities	2. Marketable Securities The following table summarizes cash equivalents and marketable securities held at March 31, 2023 and December 31, 2022 (in thousands), which are recorded at fair value. The table below excludes $ 263.6 million and $ 159.3 million of cash at March 31, 2023 and December 31, 2022, respectively. Amortized Gross Gross Fair Value March 31, 2023 Cash equivalents: Money market funds $ 19,387 $ — $ — $ 19,387 Corporate debt securities 2,358 — — 2,358 Commercial paper 13,527 — ( 1 ) 13,526 U.S. Treasury securities 45,557 9 — 45,566 Total cash equivalents 80,829 9 ( 1 ) 80,837 Marketable securities: U.S. Treasury securities 2,991 — ( 7 ) 2,984 Corporate debt securities 1,034,507 1,143 ( 9,773 ) 1,025,877 Certificates of deposit 81,948 — — 81,948 Government-sponsored enterprise securities 145,034 81 ( 477 ) 144,638 Commercial paper 289,987 6 ( 357 ) 289,636 Total marketable securities 1,554,467 1,230 ( 10,614 ) 1,545,083 Total cash equivalents and marketable securities $ 1,635,296 $ 1,239 $ ( 10,615 ) $ 1,625,920 December 31, 2022 Cash equivalents: Money market funds $ 17,766 $ — $ — $ 17,766 Corporate debt securities 2,151 — ( 2 ) 2,149 Commercial paper 32,675 — — 32,675 Total cash equivalents 52,592 — ( 2 ) 52,590 Marketable securities: Corporate debt securities 1,236,770 615 ( 15,006 ) 1,222,379 Certificates of deposit 92,417 — — 92,417 Government-sponsored enterprise securities 79,746 11 ( 712 ) 79,045 Commercial paper 263,231 — ( 509 ) 262,722 Total marketable securities 1,672,164 626 ( 16,227 ) 1,656,563 Total cash equivalents and marketable securities $ 1,724,756 $ 626 $ ( 16,229 ) $ 1,709,153 As of March 31, 2023 and December 31, 2022 , marketable securities were in a net unrealized loss position of $ 9.4 million and $ 15.6 million, respectively. The Company has recorded a net unrealized gain of $ 6.2 million and a net unrealized loss of $ 11.8 million during the three months ended March 31, 2023 and 2022, respectively, related to its debt securities, which is included in comprehensive loss on the condensed consolidated statements of operations and comprehensive loss. As of March 31, 2023 and December 31, 2022, the aggregate fair value of marketable securities that were in an unrealized loss position for less than twelve months was $ 630.5 million and $ 628.4 million, respectively. As of March 31, 2023 and December 31, 2022, the aggregate fair value of marketable securities that were in an unrealized loss position for more than twelve months was $ 468.0 million and $ 619.2 million, respectively. Of this amount, securities totaling $ 6.3 million and $ 53.1 million as of March 31, 2023 and December 31, 2022, respectively, will mature beyond one year. The Company determined that there is no material credit risk associated with the above investments as of March 31, 2023. The Company has the intent and ability to hold such securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable securities for the three months ended March 31, 2023 and 2022 . No available-for-sale debt securities held as of March 31, 2023 had remaining maturities greater than thirty months.

Fair Value Measurements

Fair Value Measurements	3 Months Ended
Fair Value Measurements	Mar. 31, 2023
Fair Value Disclosures [Abstract]
Fair Value Measurements	3. Fair Value Measurements The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the fair value hierarchy classification of such fair values as of March 31, 2023 and December 31, 2022 (in thousands): Fair Value Measurements at March 31, 2023 Total Level 1 Level 2 Level 3 Cash and cash equivalents: Cash $ 263,570 $ 263,570 $ — $ — Money market funds 19,387 19,387 — — Corporate debt securities 2,358 — 2,358 — Commercial paper 13,526 — 13,526 — U.S. Treasury securities 45,566 — 45,566 — Marketable securities: U.S. Treasury securities 2,984 — 2,984 — Corporate debt securities 1,025,877 — 1,025,877 — Certificates of deposit 81,948 — 81,948 — Government-sponsored enterprise securities 144,638 — 144,638 — Commercial paper 289,636 — 289,636 — Total $ 1,889,490 $ 282,957 $ 1,606,533 $ — Fair Value Measurements at December 31, 2022 Total Level 1 Level 2 Level 3 Cash and cash equivalents: Cash $ 159,295 $ 159,295 $ — $ — Money market funds 17,766 17,766 — — Corporate debt securities 2,149 — 2,149 — Commercial paper 32,675 — 32,675 — Marketable securities: Corporate debt securities 1,222,379 — 1,222,379 — Certificates of deposit 92,417 — 92,417 — Government-sponsored enterprise securities 79,045 — 79,045 — Commercial paper 262,722 — 262,722 — Other non-current assets 2,212 — — 2,212 Total $ 1,870,660 $ 177,061 $ 1,691,387 $ 2,212 Marketable securities classified as Level 2 within the valuation hierarchy generally consist of U.S. treasury securities and government agency securities, certificates of deposit, corporate bonds and commercial paper. The Company estimates the fair value of these marketable securities by taking into consideration valuations obtained from third-party pricing sources.

Property and Equipment, net

Property and Equipment, net	3 Months Ended
Property and Equipment, net	Mar. 31, 2023
Property, Plant and Equipment [Abstract]
Property and Equipment, net	4. Property and Equipment, net Property and equipment, net, consists of the following (in thousands): As of March 31, December 31, 2023 2022 Computer equipment $ 3,618 $ 3,618 Furniture, fixtures and other 8,109 8,109 Laboratory equipment 40,176 37,897 Leasehold improvements 143,060 141,680 Construction work in process 6,104 6,162 Total property and equipment, gross 201,067 197,466 Accumulated depreciation ( 38,869 ) ( 33,832 ) Total property and equipment, net $ 162,198 $ 163,634 Depreciation expense for the three months ended March 31, 2023 and 2022 was $ 5.0 million and $ 6.0 million, respectively.

Accrued Expenses

Accrued Expenses	3 Months Ended
Accrued Expenses	Mar. 31, 2023
Payables and Accruals [Abstract]
Accrued Expenses	5. Accrued Expenses Accrued expenses consist of the following (in thousands): As of March 31, December 31, 2023 2022 Payroll and employee-related costs $ 8,351 $ 19,241 Research costs 22,354 35,010 Collaboration costs 44,824 11,177 Licensing fees 1,068 983 Professional fees 4,041 4,927 Intellectual property costs 2,985 3,936 Accrued property and equipment 949 1,244 Other 1,059 1,164 Total $ 85,631 $ 77,682

Commitments and Contingencies

Commitments and Contingencies	3 Months Ended
Commitments and Contingencies	Mar. 31, 2023
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	. Commitments and Contingencies Leases Refer to Note 7 to the consolidated financial statements in the Company’s 2022 Annual Report on Form 10-K filed with the SEC on February 21, 2023 for discussion of the Company’s lease arrangements. Litigation In the ordinary course of business, the Company is from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, the Company’s intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office involving the Company’s intellectual property estate including certain in-licensed intellectual property. The outcome of any of the foregoing, regardless of the merits, is inherently uncertain. In addition, litigation and related matters are costly and may divert the attention of Company’s management and other resources that would otherwise be engaged in other activities. If the Company is unable to prevail in any such proceedings, the Company’s business, results of operations, liquidity and financial condition could be adversely affected. Letters of Credit As of March 31, 2023, the Company had restricted cash of $ 11.7 million, representing letters of credit securing the Company’s obligations under certain leased facilities. The letters of credit are secured by cash held in a restricted depository account and included in “Restricted cash” on the Company's condensed consolidated balance sheets as of March 31, 2023. Research, Manufacturing, License and Intellectual Property Agreements The Company has engaged several research institutions and companies to identify new delivery strategies and applications of the Company’s gene editing technology. The Company is also a party to a number of license agreements which require significant upfront payments and may be required to make future royalty payments and potential milestone payments from time to time. In addition, the Company is also a party to intellectual property agreements, which require maintenance and milestone payments from time to time. Further, the Company is a party to a number of manufacturing agreements that require upfront payments for the future performance of services. In association with these agreements, on a product-by-product basis, the counterparties are eligible to receive up to low eight-digit potential payments upon specified research, development and regulatory milestones. In addition, on a product-by-product basis, the counterparties are eligible to receive potential commercial milestone payments based on specified annual sales thresholds. The potential payments are low-single digit percentages of the specified annual sales thresholds. The counterparties are also eligible to receive low single-digit royalties on future net sales. Under certain circumstances and if certain contingent future events occur, Vertex is eligible to receive up to $ 395.0 million in potential specified research, development, regulatory and commercial milestones and tiered single-digit percentage royalties on future net sales related to a specified target under an amendment to the 2015 Collaboration Agreement (as such term is defined in Note 6 above). In addition, Vertex has the option to conduct research at its own cost in certain defined areas that, if beneficial to the exa-cel program and ultimately achieves regulatory approval, could result in the Company owing Vertex certain milestone payments aggregating to high eight digits, subject to certain limitations on the profitability of the exa-cel program. Under the A&R Vertex JDCA, the Company deferred $ 36.1 million of its share of costs incurred under the arrangement for the year ended December 31, 2022, as spending on the exa-cel program exceeded a specified amount. Any deferred amounts are only payable to Vertex as an offset against future profitability of the exa-cel program and the amounts payable are capped at a specified maximum amount per year. These deferred costs on the exa-cel program will be accrued for when it is probable that a liability has been incurred and the amount can be reasonably estimated. As of March 31, 2023 , no contingent payments have been accrued. Refer to Note 6 for further discussion of the Company’s arrangements with Vertex.

Significant Contracts

Significant Contracts	3 Months Ended
Significant Contracts	Mar. 31, 2023
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Significant Contracts	. Significant Contracts Agreements with Vertex 2015 collaboration In 2015, the Company entered into a strategic collaboration, option and license agreement, or the 2015 Collaboration Agreement, with Vertex. The 2015 Collaboration Agreement is focused on the use of the Company’s CRISPR/Cas9 gene editing technology to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. The Company and Vertex amended the 2015 Collaboration Agreement in 2017 and 2019 with Amendment No. 1 and Amendment No. 2, respectively, namely to clarify Vertex’s option rights under the 2015 Collaboration Agreement and to modify certain definitions and provisions of the 2015 Collaboration Agreement to make them consistent with the JDA (as defined below) and the 2019 Collaboration Agreement (as defined below). In 2017, Vertex exercised an option granted to it under the 2015 Collaboration Agreement to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, and in 2019, Vertex exercised the remaining options granted to it under the 2015 Collaboration Agreement to exclusively license certain collaboration targets developed under the 2015 Collaboration Agreement. Exa-cel collaboration In 2017, following Vertex's exercise of its option to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, the Company and Vertex entered into a joint development and commercialization agreement, or the JDA, and agreed for potential hemoglobinopathy treatments, including exa-cel, the Company and Vertex would share equally all research and development costs and worldwide revenues. I n 2021, the Company and Vertex amended and restated the JDA, or the A&R Vertex JDCA, pursuant to which the parties agreed to, among other things, (a) adjust the governance structure for the collaboration and adjust the responsibilities of each party thereunder, whereby Vertex leads and has all decision making (i.e., control) in relation to the exa-cel program prospectively; (b) adjust the allocation of net profits and net losses between the parties with respect to exa-cel only, which will be allocated 40 % to the Company and 60 % to Vertex, prospectively; and (c) exclusively license (subject to the Company’s reserved rights to conduct certain activities) certain intellectual property rights to Vertex relating to the specified product candidates and products (including exa-cel) that may be researched, developed, manufactured and commercialized on a worldwide basis under the A&R Vertex JDCA. Additionally, the A&R Vertex JDCA allows the Company to defer a portion of its share of costs under the arrangement if spending on the exa-cel program exceeds specified amounts. Any deferred amounts are only payable to Vertex as an offset against future profitability of the exa-cel program and the amounts payable are capped at a specified maximum amount per year. DMD and DM1 exclusive license In 2019, the Company and Vertex entered into a series of agreements, including a strategic collaboration and license agreement, or the 2019 Collaboration Agreement, for the development and commercialization of products for the treatment of Duchenne muscular dystrophy, or DMD, and Myotonic Dystrophy Type 1, or DM1. For the DMD and DM1 programs, Vertex is responsible for all research, development, manufacturing and commercialization activities and all related costs. Upon IND filing, the Company has the option to forego the DM1 milestones and royalties, and instead, co-develop and co-commercialize all DM1 products globally in exchange for payment of 50 % of research and development costs incurred by Vertex from the effective date of the agreement through IND filing. Collaboration in the field of diabetes In 2021, CRISPR and ViaCyte, Inc., or ViaCyte, entered into a joint development and commercialization agreement, or the ViaCyte JDCA, to jointly develop and commercialize product candidates and shared products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. In the third quarter of 2022, Vertex acquired ViaCyte, and ViaCyte became a wholly-owned subsidiary of Vertex. In March 2023, (1) the Company and ViaCyte entered into an amendment to the ViaCyte JDCA, or the ViaCyte JDCA Amendment, and adjusted certain rights and obligations of the Company and ViaCyte under the ViaCyte JDCA, and (2) the Company and Vertex entered into a non-exclusive license agreement, or the Non-Ex License Agreement, pursuant to which the Company agreed to license to Vertex, on a non-exclusive basis, certain of its gene editing intellectual property to exploit certain products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. In connection with entering into these agreements, the Company received a $ 100.0 million up front payment from Vertex. Under the Non-Ex License Agreement, the Company is eligible to receive milestone payments from Vertex of up to $ 230.0 million in the aggregate, depending on the achievement of pre-determined research, development and commercial milestones for certain products utilizing the licensed intellectual property. Additionally, the Company is eligible to receive tiered royalties on the sales of certain products in the low to mid-single digits. Accounting Analysis The 2015 Collaboration Agreement, Amendment No. 1, Amendment No. 2, A&R Vertex JDCA, and 2019 Collaboration Agreement are collectively the “Vertex Agreements” for purposes of this Note 6. The Non-Ex License Agreement and ViaCyte JDCA Amendment are collectively the “March 2023 Agreements.” The Vertex Agreements and the March 2023 Agreements include components of a customer-vendor relationship as defined under ASC 606, Revenue from Contracts with Customers , or ASC 606, collaborative arrangements as defined under ASC 808, Collaborative Agreements , or ASC 808, and research and development costs as defined under ASC 730, Research and Development , or ASC 730. Specifically, with regards to the March 2023 Agreements, the Company concluded that the non-exclusive license is a performance obligation under ASC 606 and the ongoing research and development services under the ViaCyte JDCA Amendment are a unit of account under ASC 808. Accounting Analysis Under ASC 606 March 2023 Agreements Identification of the contract The March 2023 Agreements were negotiated as a package with a single commercial objective and, as such, the March 2023 Agreements were combined for accounting purposes and treated as a single arrangement. The Company determined for accounting purposes that the combined contract terminates the original ViaCyte JDCA and created a new contract. Identification of performance obligations The Company concluded the transfer of the non-exclusive license, including certain modified rights and obligations provided as part of the ViaCyte JDCA Amendment to support the delivery of the license, was both capable of being distinct and distinct within the context of the contract. Determination of transaction price The transaction price was comprised of the upfront payment of $ 100.0 million. The Company determined that all other possible variable consideration resulting from milestones and royalties discussed below was fully constrained at the time of the transaction. The Company will reevaluate the transaction price in each reporting period. Allocation of transaction price to performance obligations The Company identified one performance obligation for the March 2023 Agreements and, as a result, no allocation of the transaction price was required. Recognition of revenue The Company determined the non-exclusive license, including certain modified rights and obligations provided as part of the ViaCyte JDCA Amendment to support the delivery of the license, represented functional intellectual property, as the intellectual property provides Vertex with the ability to perform a function or task in the form of research and development in the field of diabetes. The Company recognized revenue for the non-exclusive license at the onset of the arrangement, as this was the point in time in which the non-exclusive license was delivered. Revenue recognized under the March 2023 Agreements was $ 100.0 million for the three months ended March 31, 2023. Milestones under the Non-Ex License Agreement The Company is eligible to receive milestone payments from Vertex of up to $ 230.0 million in the aggregate under the Non-Ex License Agreement, depending on the achievement of pre-determined research, development and commercial milestones for certain products utilizing the licensed intellectual property. Additionally, the Company is eligible to receive tiered royalties on the sales of certain products in the low to mid-single digits. Each of the milestones under the Non-Ex License Agreement are fully constrained as of March 31, 2023. There is uncertainty as to whether the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs. Vertex Agreements Deferred revenue As of March 31, 2023 and December 31, 2022, there was no current deferred revenue related to the Vertex Agreements. As of March 31, 2023, there was $ 12.3 million of non-current deferred revenue related to the Vertex Agreements, which is unchanged from December 31, 2022. The transaction price allocated to the remaining performance obligations was $ 12.3 million. Milestones The Company has evaluated the milestones that may be received in connection with the Vertex Agreements. Under the 2015 Collaboration Agreement and subsequent amendments, the Company is eligible to receive up to $ 410.0 million in additional development, regulatory and commercial milestones and royalties on net product sales for each of the three collaboration targets that Vertex licensed in 2019. Each milestone is payable only once per collaboration target, regardless of the number of products directed to such collaboration target that achieve the relevant milestone event. The Company is eligible to receive potential future payments of up to $ 775.0 million under the 2019 Collaboration Agreement based upon the successful achievement of specified development, regulatory and commercial milestones for the DMD and DM1 programs. The Company is also eligible to receive tiered royalties on future net sales on any products that may result from this collaboration; however, the Company has the option to forego the DM1 milestones and royalties to co-develop and co-commercialize all DM1 products globally. The Company is eligible to receive potential future payments of up to $ 200.0 million under the A&R Vertex JDCA upon receipt by Vertex of the first marketing approval of the initial product candidate from the FDA or the European Commission. In addition, the Company has the option to conduct research at their own cost in certain defined areas that, if beneficial to the exa-cel program and exa-cel ultimately achieves regulatory approval in such areas, then the Company could be entitled to certain milestone payments aggregating to high eight digits from Vertex. Each of the remaining milestones described above are fully constrained as of March 31, 2023. There is uncertainty that the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs. Accounting Analysis under ASC 808 Vertex Agreements In connection with the Vertex Agreements, the Company identified the following collaborative elements, which are accounted for under ASC 808: (i) development and commercialization services for shared products, including any transition services related to exa-cel under the A&R Vertex JDCA; (ii) R&D Services for follow-on products; and (iii) committee participation. The related impact of the cost sharing is included within collaboration expense, net, in the condensed consolidated statements of operations and comprehensive loss. During the three months ended March 31, 2023 and 2022 , the Company recognized $ 42.2 million and $ 30.6 million of collaboration expense, net, related to the exa-cel program, respectively. Collaboration expense, net, was net of $ 2.8 million and $ 7.4 million of reimbursements from Vertex related to the exa-cel program, respectively.

Share Capital

Share Capital	3 Months Ended
Share Capital	Mar. 31, 2023
Equity [Abstract]
Share Capital	8. Share Capital The Company had 150,347,467 authorized common shares as of March 31, 2023 , with a par value of CHF 0.03 per share. Share Capital consisted of the following: As of Type of Share Capital Conditional Capital March 31, 2023 December 31, 2022 Common shares Registered share capital 83,538,347 82,028,328 Common shares Authorized share capital 39,316,975 39,316,975 Common shares Conditional share capital - Bonds or similar debt instruments 8,202,832 8,202,832 Common shares Conditional share capital - Employee benefit plans 19,289,313 20,799,332 Total 150,347,467 150,347,467 Common Share Issuances At-the-Market Offering In August 2019, the Company entered into an Open Market Sale Agreement SM with Jefferies LLC, or Jefferies, under which the Company was able to offer and sell, from time to time at its sole discretion through Jefferies, as its sales agent, its common shares, or the August 2019 Sales Agreement. In January 2021, in connection with the August 2019 Sales Agreement, the Company filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $ 600.0 million. In July 2021, the Company filed a new prospectus supplement with the SEC, which replaced the previous prospectus supplement filed in January 2021, to offer and sell, from time to time, the common shares remaining under the original prospectus supplement having aggregate gross proceeds of up to $ 419.8 million, or, together with the January 2021 prospectus supplement, the 2021 ATM. As of March 31, 2023 , the Company has issued and sold an aggregate of 1.1 million common shares under the 2021 ATM at an average price of $ 168.79 per share for aggregate proceeds of $ 178.8 million, which were net of equity issuance costs of $ 2.4 million.

Stock-based Compensation

Stock-based Compensation	3 Months Ended
Stock-based Compensation	Mar. 31, 2023
Share-Based Payment Arrangement [Abstract]
Stock-based Compensation	9. Stock-based Compensation During the three months ended March 31, 2023 and 2022, the Company recognized the following stock-based compensation expense (in thousands): Three Months Ended March 31, 2023 2022 Research and development $ 11,676 $ 14,589 General and administrative 9,199 11,156 Total $ 20,875 $ 25,745 Stock option activity The following table summarizes stock option activity for the three months ended March 31, 2023: Shares Weighted- Outstanding at December 31, 2022 7,230,233 $ 60.22 Granted 1,236,882 44.05 Exercised ( 159,184 ) 30.37 Cancelled or forfeited ( 390,947 ) 86.12 Outstanding at March 31, 2023 7,916,984 $ 57.01 Exercisable at March 31, 2023 4,853,602 $ 52.83 Vested and expected to vest at March 31, 2023 7,916,984 $ 57.01 As of March 31, 2023, total unrecognized compensation expense related to stock options was $ 117.4 million, which the Company expects to recognize over a remaining weighted-average period of 2.6 years. Restricted stock activity The following table summarizes restricted stock activity for the three months ended March 31, 2023: Shares Weighted- Unvested balance at December 31, 2022 1,325,185 $ 80.13 Granted 643,724 43.76 Vested ( 172,995 ) 74.21 Cancelled or forfeited ( 125,084 ) 86.11 Unvested balance at March 31, 2023 1,670,830 $ 66.29 As of March 31, 2023, total unrecognized compensation expense related to unvested restricted common shares was $ 82.0 million, which the Company expects to recognize over a remaining weighted-average vesting period of 2.8 years.

Net Loss Per Share Attributable

Net Loss Per Share Attributable to Common Shareholders	3 Months Ended
Net Loss Per Share Attributable to Common Shareholders	Mar. 31, 2023
Earnings Per Share [Abstract]
Net Loss Per Share Attributable to Common Shareholders	10. Net Loss Per Share Attributable to Common Shareholders Basic net loss per share is calculated by dividing net loss attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net loss per share is calculated by dividing the net loss attributable to common shareholders by the weighted-average number of common share equivalents outstanding for the period, including any dilutive effect from outstanding stock options and warrants using the treasury stock method. The Company’s net loss is net loss attributable to common shareholders for all periods presented. The following common stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect (in thousands): Three Months Ended March 31, 2023 2022 Outstanding options 7,916,984 8,220,458 Unvested restricted common shares 1,670,830 1,147,364 ESPP 18,110 10,969 Total 9,605,924 9,378,791

Income Taxes

Income Taxes	3 Months Ended
Income Taxes	Mar. 31, 2023
Income Tax Disclosure [Abstract]
Income Taxes	11. Income Taxes During the three months ended March 31, 2023 and 2022 , the Company recorded an income tax provision of $ 1.3 million and $ 3.6 million, respectively, representing an effective tax rate of ( 2.6 %) and ( 2.1 %), respectively. The income tax provision for the three months ended March 31, 2023 and 2022 is primarily attributable to the Company's U.S. subsidiaries. The change in the rate for the three months ended March 31, 2023 is primarily attributable to reduced forecasted capitalized R&D expense addback offset by an increase in forecasted interest income in the United States. The difference in the statutory tax rate a nd effective tax rate is primarily a result of the jurisdictional mix of earnings, research credits generated, and the valuation allowance recorded against certain deferred tax assets. The Company maintains a valuation allowance against certain deferred tax assets that are not more-likely-than-not realizable.

Basis of Presentation and Sig_2

Basis of Presentation and Significant Accounting Policies (Policies)	3 Months Ended
	Mar. 31, 2023
Accounting Policies [Abstract]
Basis of Presentation	Basis of Presentation The accompanying condensed consolidated financial statements are unaudited and have been prepared by the Company in accordance with accounting principles generally accepted in the United States of America, or U.S. GAAP. The accompanying condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The Company views its operations and manages its business in one operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. Certain information and footnote disclosures normally included in the Company’s annual financial statements have been condensed or omitted. These interim financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the financial position and results of operations for the three-month interim periods ended March 31, 2023 and 2022. Beginning in 2022, "collaboration expense, net" in the condensed consolidated statements of operations and comprehensive loss present collaboration costs related to the exagamglogene autotemcel, or exa-cel (formerly known as CTX001), program under the Company's collaboration with Vertex Pharmaceuticals Incorporated and certain of its subsidiaries, or Vertex, accounted for under ASC 808, Collaborative Agreements , or ASC 808. Refer to Note 6 to these condensed consolidated financial statements for further discussion on the Company's agreements with Vertex, including the exa-cel program. The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year. These interim financial statements should be read in conjunction with the audited financial statements for the year ended December 31, 2022, which are contained in the 2022 Annual Report on Form 10-K filed with the Securities and Exchange Commission, or the SEC, on February 21, 2023 .
Use of Estimates	Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, revenue recognition, equity-based compensation expense and reported amounts of expenses during the period. Significant estimates in these consolidated financial statements have been made in connection with revenue recognition and equity-based compensation expense. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Changes in estimates are reflected in reported results in the period in which they become known.
New Accounting Pronouncements - Recently Adopted	New Accounting Pronouncements – Recently Adopted From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board, or FASB, or other standard setting bodies that the Company adopts as of the specified effective date. The Company does not believe that the adoption of recently issued standards have or may have a material impact on its consolidated financial statements and disclosures.

Marketable Securities (Tables)

Marketable Securities (Tables)	3 Months Ended
Marketable Securities (Tables)	Mar. 31, 2023
Investments, Debt and Equity Securities [Abstract]
Summary of Cash Equivalents and Marketable Securities Recorded at Fair Value	The following table summarizes cash equivalents and marketable securities held at March 31, 2023 and December 31, 2022 (in thousands), which are recorded at fair value. The table below excludes $ 263.6 million and $ 159.3 million of cash at March 31, 2023 and December 31, 2022, respectively. Amortized Gross Gross Fair Value March 31, 2023 Cash equivalents: Money market funds $ 19,387 $ — $ — $ 19,387 Corporate debt securities 2,358 — — 2,358 Commercial paper 13,527 — ( 1 ) 13,526 U.S. Treasury securities 45,557 9 — 45,566 Total cash equivalents 80,829 9 ( 1 ) 80,837 Marketable securities: U.S. Treasury securities 2,991 — ( 7 ) 2,984 Corporate debt securities 1,034,507 1,143 ( 9,773 ) 1,025,877 Certificates of deposit 81,948 — — 81,948 Government-sponsored enterprise securities 145,034 81 ( 477 ) 144,638 Commercial paper 289,987 6 ( 357 ) 289,636 Total marketable securities 1,554,467 1,230 ( 10,614 ) 1,545,083 Total cash equivalents and marketable securities $ 1,635,296 $ 1,239 $ ( 10,615 ) $ 1,625,920 December 31, 2022 Cash equivalents: Money market funds $ 17,766 $ — $ — $ 17,766 Corporate debt securities 2,151 — ( 2 ) 2,149 Commercial paper 32,675 — — 32,675 Total cash equivalents 52,592 — ( 2 ) 52,590 Marketable securities: Corporate debt securities 1,236,770 615 ( 15,006 ) 1,222,379 Certificates of deposit 92,417 — — 92,417 Government-sponsored enterprise securities 79,746 11 ( 712 ) 79,045 Commercial paper 263,231 — ( 509 ) 262,722 Total marketable securities 1,672,164 626 ( 16,227 ) 1,656,563 Total cash equivalents and marketable securities $ 1,724,756 $ 626 $ ( 16,229 ) $ 1,709,153

Fair Value Measurements (Tables

Fair Value Measurements (Tables)	3 Months Ended
Fair Value Measurements (Tables)	Mar. 31, 2023
Fair Value Disclosures [Abstract]
Schedule of Financial Assets Measured at Fair Value on Recurring Basis	The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the fair value hierarchy classification of such fair values as of March 31, 2023 and December 31, 2022 (in thousands): Fair Value Measurements at March 31, 2023 Total Level 1 Level 2 Level 3 Cash and cash equivalents: Cash $ 263,570 $ 263,570 $ — $ — Money market funds 19,387 19,387 — — Corporate debt securities 2,358 — 2,358 — Commercial paper 13,526 — 13,526 — U.S. Treasury securities 45,566 — 45,566 — Marketable securities: U.S. Treasury securities 2,984 — 2,984 — Corporate debt securities 1,025,877 — 1,025,877 — Certificates of deposit 81,948 — 81,948 — Government-sponsored enterprise securities 144,638 — 144,638 — Commercial paper 289,636 — 289,636 — Total $ 1,889,490 $ 282,957 $ 1,606,533 $ — Fair Value Measurements at December 31, 2022 Total Level 1 Level 2 Level 3 Cash and cash equivalents: Cash $ 159,295 $ 159,295 $ — $ — Money market funds 17,766 17,766 — — Corporate debt securities 2,149 — 2,149 — Commercial paper 32,675 — 32,675 — Marketable securities: Corporate debt securities 1,222,379 — 1,222,379 — Certificates of deposit 92,417 — 92,417 — Government-sponsored enterprise securities 79,045 — 79,045 — Commercial paper 262,722 — 262,722 — Other non-current assets 2,212 — — 2,212 Total $ 1,870,660 $ 177,061 $ 1,691,387 $ 2,212

Property and Equipment, net (Ta

Property and Equipment, net (Tables)	3 Months Ended
Property and Equipment, net (Tables)	Mar. 31, 2023
Property, Plant and Equipment [Abstract]
Summary of Property and Equipment, Net	Property and equipment, net, consists of the following (in thousands): As of March 31, December 31, 2023 2022 Computer equipment $ 3,618 $ 3,618 Furniture, fixtures and other 8,109 8,109 Laboratory equipment 40,176 37,897 Leasehold improvements 143,060 141,680 Construction work in process 6,104 6,162 Total property and equipment, gross 201,067 197,466 Accumulated depreciation ( 38,869 ) ( 33,832 ) Total property and equipment, net $ 162,198 $ 163,634

Accrued Expenses (Tables)

Accrued Expenses (Tables)	3 Months Ended
Accrued Expenses (Tables)	Mar. 31, 2023
Payables and Accruals [Abstract]
Schedule of Accrued Expenses	Accrued expenses consist of the following (in thousands): As of March 31, December 31, 2023 2022 Payroll and employee-related costs $ 8,351 $ 19,241 Research costs 22,354 35,010 Collaboration costs 44,824 11,177 Licensing fees 1,068 983 Professional fees 4,041 4,927 Intellectual property costs 2,985 3,936 Accrued property and equipment 949 1,244 Other 1,059 1,164 Total $ 85,631 $ 77,682

Share Capital (Tables)

Share Capital (Tables)	3 Months Ended
Share Capital (Tables)	Mar. 31, 2023
Equity [Abstract]
Schedule of Share Capital	The Company had 150,347,467 authorized common shares as of March 31, 2023 , with a par value of CHF 0.03 per share. Share Capital consisted of the following: As of Type of Share Capital Conditional Capital March 31, 2023 December 31, 2022 Common shares Registered share capital 83,538,347 82,028,328 Common shares Authorized share capital 39,316,975 39,316,975 Common shares Conditional share capital - Bonds or similar debt instruments 8,202,832 8,202,832 Common shares Conditional share capital - Employee benefit plans 19,289,313 20,799,332 Total 150,347,467 150,347,467

Stock-based Compensation (Table

Stock-based Compensation (Tables)	3 Months Ended
Stock-based Compensation (Tables)	Mar. 31, 2023
Share-Based Payment Arrangement [Abstract]
Schedule of Stock-Based Compensation Expense	During the three months ended March 31, 2023 and 2022, the Company recognized the following stock-based compensation expense (in thousands): Three Months Ended March 31, 2023 2022 Research and development $ 11,676 $ 14,589 General and administrative 9,199 11,156 Total $ 20,875 $ 25,745
Summary of Stock Option Activity	The following table summarizes stock option activity for the three months ended March 31, 2023: Shares Weighted- Outstanding at December 31, 2022 7,230,233 $ 60.22 Granted 1,236,882 44.05 Exercised ( 159,184 ) 30.37 Cancelled or forfeited ( 390,947 ) 86.12 Outstanding at March 31, 2023 7,916,984 $ 57.01 Exercisable at March 31, 2023 4,853,602 $ 52.83 Vested and expected to vest at March 31, 2023 7,916,984 $ 57.01
Summary of Restricted Stock Activity	The following table summarizes restricted stock activity for the three months ended March 31, 2023: Shares Weighted- Unvested balance at December 31, 2022 1,325,185 $ 80.13 Granted 643,724 43.76 Vested ( 172,995 ) 74.21 Cancelled or forfeited ( 125,084 ) 86.11 Unvested balance at March 31, 2023 1,670,830 $ 66.29

Net Loss Per Share Attributab_2

Net Loss Per Share Attributable to Common Shareholders (Tables)	3 Months Ended
	Mar. 31, 2023
Earnings Per Share [Abstract]
Schedule of Antidilutive Securities not Include in Computation of Net (Loss) Income per Share	The following common stock equivalents were excluded from the calculation of diluted net loss per share for the periods indicated because including them would have had an anti-dilutive effect (in thousands): Three Months Ended March 31, 2023 2022 Outstanding options 7,916,984 8,220,458 Unvested restricted common shares 1,670,830 1,147,364 ESPP 18,110 10,969 Total 9,605,924 9,378,791

Basis of Presentation and Sig_3

Basis of Presentation and Significant Accounting Policies - Additional Information (Detail)	3 Months Ended
	Mar. 31, 2023 Segment
Accounting Policies [Abstract]
Number of operating segments	1

Marketable Securities - Additio

Marketable Securities - Additional Information (Detail) - USD ($)	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022	Dec. 31, 2022
Marketable Securities [Line Items]
Marketable securities	$ 263,600,000		$ 159,300,000
Marketable securities unrealized loss, Less than twelve months	630,500,000		628,400,000
Marketable securities unrealized loss, more than twelve months	468,000,000		619,200,000
Marketable securities totaling	6,320,000		53,130,000
Marketable securities unrealized loss	9,400,000		$ 15,600,000
Available-for-sale debt securities remaining maturities greater than thirty months	0
Other Comprehensive Income (Loss) [Member]
Marketable Securities [Line Items]
Unrealized Gain	6,200,000	$ 11,800,000
Unrealized loss, net	$ 6,200,000	$ 11,800,000

Marketable Securities - Summary

Marketable Securities - Summary of Cash Equivalents and Marketable Securities Recorded at Fair Value (Detail) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Marketable Securities [Line Items]
Cash equivalents, amortized cost	$ 80,829	$ 52,592
Cash equivalents, gross unrealized gains	9	0
Cash equivalents, gross unrealized losses	(1)	(2)
Cash equivalents, fair value	80,837	52,590
Marketable securities, amortized cost	1,554,467	1,672,164
Marketable securities, gross unrealized gains	1,230	626
Marketable securities, gross unrealized losses	(10,614)	(16,227)
Marketable securities, fair value	1,545,083	1,656,563
Cash equivalents and marketable securities, amortized cost	1,635,296	1,724,756
Cash equivalents and marketable securities, gross unrealized gains	1,239	626
Cash equivalents and marketable securities, gross unrealized losses	(10,615)	(16,229)
Cash equivalents and marketable securities, fair value	1,625,920	1,709,153
Money Market Funds [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	19,387	17,766
Cash equivalents, gross unrealized gains	0	0
Cash equivalents, gross unrealized losses	0	0
Cash equivalents, fair value	19,387	17,766
Certificates of Deposit [Member]
Marketable Securities [Line Items]
Marketable securities, amortized cost	81,948	92,417
Marketable securities, gross unrealized gains	0	0
Marketable securities, gross unrealized losses	0	0
Marketable securities, fair value	81,948	92,417
Commercial Paper [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	13,527	32,675
Cash equivalents, gross unrealized gains	0	0
Cash equivalents, gross unrealized losses	1	0
Cash equivalents, fair value	13,526	32,675
Marketable securities, amortized cost	289,987	263,231
Marketable securities, gross unrealized gains	6	0
Marketable securities, gross unrealized losses	(357)	(509)
Marketable securities, fair value	289,636	262,722
US Treasury Securities [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	45,557
Cash equivalents, gross unrealized gains	9
Cash equivalents, gross unrealized losses	0
Cash equivalents, fair value	45,566
Marketable securities, amortized cost	2,991
Marketable securities, gross unrealized gains	0
Marketable securities, gross unrealized losses	(7)
Marketable securities, fair value	2,984
Corporate Debt Securities [Member]
Marketable Securities [Line Items]
Cash equivalents, amortized cost	2,358	2,151
Cash equivalents, gross unrealized gains	0	0
Cash equivalents, gross unrealized losses	0	2
Cash equivalents, fair value	2,358	2,149
Marketable securities, amortized cost	1,034,507	1,236,770
Marketable securities, gross unrealized gains	1,143	615
Marketable securities, gross unrealized losses	(9,773)	(15,006)
Marketable securities, fair value	1,025,877	1,222,379
Government-sponsored Enterprise Securities [Member]
Marketable Securities [Line Items]
Marketable securities, amortized cost	145,034	79,746
Marketable securities, gross unrealized gains	81	11
Marketable securities, gross unrealized losses	(477)	(712)
Marketable securities, fair value	$ 144,638	$ 79,045

Fair Value Measurements - Sched

Fair Value Measurements - Schedule of Financial Assets Measured at Fair Value on Recurring Basis (Detail) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Marketable securities, fair value	$ 1,545,083	$ 1,656,563
Recurring Basis [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Other non-current assets	2,212
Total	1,889,490	1,870,660
Recurring Basis [Member] \| Cash [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	263,570	159,295
Recurring Basis [Member] \| Money Market Funds [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	19,387	17,766
Recurring Basis [Member] \| Corporate Debt Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	2,358	2,149
Marketable securities, fair value	1,025,877	1,222,379
Recurring Basis [Member] \| Certificates of Deposit [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Marketable securities, fair value	81,948	92,417
Recurring Basis [Member] \| Commercial Paper [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	13,526	32,675
Marketable securities, fair value	289,636	262,722
Recurring Basis [Member] \| US Treasury Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	45,566
Marketable securities, fair value	2,984
Recurring Basis [Member] \| Government-sponsored Enterprise Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Marketable securities, fair value	144,638	79,045
Recurring Basis [Member] \| Level 1 [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total	282,957	177,061
Recurring Basis [Member] \| Level 1 [Member] \| Cash [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	263,570	159,295
Recurring Basis [Member] \| Level 1 [Member] \| Money Market Funds [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	19,387	17,766
Recurring Basis [Member] \| Level 2 [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Total	1,606,533	1,691,387
Recurring Basis [Member] \| Level 2 [Member] \| Corporate Debt Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	2,358	2,149
Marketable securities, fair value	1,025,877	1,222,379
Recurring Basis [Member] \| Level 2 [Member] \| Certificates of Deposit [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Marketable securities, fair value	81,948	92,417
Recurring Basis [Member] \| Level 2 [Member] \| Commercial Paper [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	13,526	32,675
Marketable securities, fair value	289,636	262,722
Recurring Basis [Member] \| Level 2 [Member] \| US Treasury Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Cash and cash equivalents	45,566
Marketable securities, fair value	2,984
Recurring Basis [Member] \| Level 2 [Member] \| Government-sponsored Enterprise Securities [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Marketable securities, fair value	144,638	79,045
Recurring Basis [Member] \| Level 3 [Member]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Other non-current assets	$ 2,212
Total		$ 2,212

Property and Equipment, net - S

Property and Equipment, net - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	$ 201,067	$ 197,466
Accumulated depreciation	(38,869)	(33,832)
Total property and equipment, net	162,198	163,634
Computer Equipment [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	3,618	3,618
Furniture, Fixtures and Other [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	8,109	8,109
Laboratory Equipment [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	40,176	37,897
Leasehold Improvements [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	143,060	141,680
Construction Work in Process [Member]
Property, Plant and Equipment [Line Items]
Total property and equipment, gross	$ 6,104	$ 6,162

Property and Equipment, net - A

Property and Equipment, net - Additional Information (Detail) - USD ($) $ in Millions	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Property, Plant and Equipment [Abstract]
Depreciation expense	$ 5	$ 6

Accrued Expenses - Schedule of

Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands	Mar. 31, 2023	Dec. 31, 2022
Accrued Liabilities, Current [Abstract]
Payroll and employee-related costs	$ 8,351	$ 19,241
Research costs	22,354	35,010
Collaboration costs	44,824	11,177
Licensing fees	1,068	983
Professional fees	4,041	4,927
Intellectual property costs	2,985	3,936
Accrued property and equipment	949	1,244
Other	1,059	1,164
Total	$ 85,631	$ 77,682

Commitments and Contingencies -

Commitments and Contingencies - Additional Information (Detail) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Mar. 31, 2023	Mar. 31, 2022
Contingencies And Commitments [Line Items]
Letters of credit secured by cash held in restricted depository account		$ 0
Deferred Finance Costs, Share Lending Arrangement, Issuance Costs	$ 36,100
Prepaid expenses and other current assets		0
Restricted cash		0
Prepaid Expenses and Other Current Assets [Member]
Contingencies And Commitments [Line Items]
Letters of credit secured by cash held in restricted depository account			$ 4,783
Prepaid expenses and other current assets			4,783
Restricted cash			$ 4,783
Letter of Credit [Member]
Contingencies And Commitments [Line Items]
Letters of credit secured by cash held in restricted depository account		11,700
Prepaid expenses and other current assets		11,700
Restricted cash		11,700
2015 Collaboration Agreement [Member] \| Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member]
Contingencies And Commitments [Line Items]
Milestone payment receivable		$ 395,000

Significant Contracts - Additio

Significant Contracts - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended		12 Months Ended
	Mar. 31, 2023	Mar. 31, 2022	Dec. 31, 2021	Dec. 31, 2019	Dec. 31, 2022
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Non-current deferred revenue	$ 12,323				$ 12,323
Research and development expense	99,935	$ 118,245
General and Administrative Expense	22,360	28,021
Collaboration Expense	42,192	30,646
A&R JDCA [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Percentage of net profits and net losses			40%
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Collaboration Expense	42,200	30,600
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| Collaboration Target Options [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Milestone payment receivable	410,000
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| 2019 Collaboration Agreement [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Percentage of exchange payment of research and development costs				50%
Maximum potential future payments	775,000
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| 2015 Collaboration Agreement [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Milestone payment receivable	395,000
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| A&R JDCA [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Up-front payment received	100,000
Percentage of net profits and net losses			60%
Revenue	100,000
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] \| Non Ex License Agreement [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Up-front payment received			$ 100,000
Milestone payment receivable	230,000		$ 230,000
Vertex Pharmaceuticals Inc [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Non-current deferred revenue	12,300
Transaction price allocated to remaining performance obligations	12,300
Current deferred revenue	0				$ 0
Reimbursements from research and license agreements	2,800	$ 7,400
Vertex Pharmaceuticals Inc [Member] \| A&R JDCA [Member]
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Maximum potential future payments	$ 200,000

Share Capital - Additional Info

Share Capital - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions	1 Months Ended		3 Months Ended
	Jul. 31, 2021	Jan. 31, 2021	Mar. 31, 2023	Dec. 31, 2022
Class of Stock [Line Items]
Common Stock, Shares Authorized			150,347,467	150,347,467
Common shares, par value			$ 0.03	$ 0.03
Sales Agreement With Jefferies LLC [Member] \| 2021 ATM [Member]
Class of Stock [Line Items]
Average issue price of common shares			$ 168.79
Proceeds from issuance of common shares			$ 178.8
Equity issuance costs			$ 2.4
Shares, Issued			1,100,000
Sales Agreement With Jefferies LLC [Member] \| Maximum [Member] \| 2021 ATM [Member]
Class of Stock [Line Items]
Aggregate estimated gross proceeds	$ 419.8	$ 600

Share Capital - Schedule of Sha

Share Capital - Schedule of Share Capital (Detail) - shares	Mar. 31, 2023	Dec. 31, 2022
Class of Stock [Line Items]
Authorized share capital	150,347,467	150,347,467
Common Shares [Member]
Class of Stock [Line Items]
Registered share capital	83,538,347	82,028,328
Authorized share capital	39,316,975	39,316,975
Common Shares [Member] \| Employee Benefit Plans [Member]
Class of Stock [Line Items]
Conditional share capital	19,289,313	20,799,332
Common Shares [Member] \| Bonds or Similar Debt Instruments [Member]
Class of Stock [Line Items]
Conditional share capital	8,202,832	8,202,832

Stock-based Compensation - Sche

Stock-based Compensation - Schedule of Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Stock-based compensation expense	$ 20,875	$ 25,745
Research and Development [Member]
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Stock-based compensation expense	11,676	14,589
General and Administrative [Member]
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Stock-based compensation expense	$ 9,199	$ 11,156

Stock-based Compensation - Summ

Stock-based Compensation - Summary of Stock Option Activity (Detail)	3 Months Ended
	Mar. 31, 2023 $ / shares shares
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]
Shares, Outstanding, Beginning Balance \| shares	7,230,233
Shares, Granted \| shares	1,236,882
Shares, Exercised \| shares	(159,184)
Shares, Cancelled or forfeited \| shares	(390,947)
Shares, Outstanding, Ending Balance \| shares	7,916,984
Shares, Exercisable \| shares	4,853,602
Shares, Vested or expected to vest \| shares	7,916,984
Weighted-Average Exercise Price, Outstanding, Beginning Balance \| $ / shares	$ 60.22
Weighted-Average Exercise Price, Granted \| $ / shares	44.05
Weighted-Average Exercise Price, Exercised \| $ / shares	30.37
Weighted-Average Exercise Price, Cancelled or forfeited \| $ / shares	86.12
Weighted-Average Exercise Price, Outstanding, Ending Balance \| $ / shares	57.01
Weighted-Average Exercise Price, Exercisable \| $ / shares	52.83
Weighted-Average Exercise Price, Vested or expected to vest \| $ / shares	$ 57.01

Stock-based Compensation - Addi

Stock-based Compensation - Additional Information (Detail) $ in Millions	3 Months Ended
	Mar. 31, 2023 USD ($)
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total unrecognized compensation expense related to stock options	$ 117.4
Stock Options [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation expense, remaining weighted-average period for recognition	2 years 7 months 6 days
Restricted Common Shares [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation expense, remaining weighted-average period for recognition	2 years 9 months 18 days
Total unrecognized compensation expense	$ 82

Stock-based Compensation - Su_2

Stock-based Compensation - Summary of Restricted Stock Activity (Detail) - Restricted Stock [Member]	3 Months Ended
	Mar. 31, 2023 $ / shares shares
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unvested restricted common shares, Number of Shares, Beginning Balance \| shares	1,325,185
Unvested restricted common shares, Number of Shares, Granted \| shares	643,724
Unvested restricted common shares, Number of Shares, Vested \| shares	(172,995)
Unvested restricted common shares, Number of Shares, Cancelled or forfeited \| shares	(125,084)
Unvested restricted common shares, Number of Shares, Ending Balance \| shares	1,670,830
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Beginning Balance \| $ / shares	$ 80.13
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Granted \| $ / shares	43.76
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Vested \| $ / shares	74.21
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Cancelled or forfeited \| $ / shares	86.11
Unvested restricted common shares, Weighted-Average Grant Date Fair Value, Ending Balance \| $ / shares	$ 66.29

Net Loss Per Share Attributab_3

Net Loss Per Share Attributable to Common Shareholders - Schedule of Antidilutive Securities not Include in Computation of Net (Loss) Income per Share (Detail) - shares	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities not include in computation of net income (loss) per share	9,605,924	9,378,791
Outstanding Options [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities not include in computation of net income (loss) per share	7,916,984	8,220,458
Unvested Restricted Common Shares [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities not include in computation of net income (loss) per share	1,670,830	1,147,364
ESPP [Member]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Anti-dilutive securities not include in computation of net income (loss) per share	18,110	10,969

Income Taxes - Additional Infor

Income Taxes - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2023	Mar. 31, 2022
Income Tax Disclosure [Abstract]
Income tax provision (benefit)	$ 1,320	$ 3,608
Effective income tax rate	(2.60%)	(2.10%)