Intellia Therapeutics (NTLA) 7 Nov 242024 Q3 Earnings call transcript

Good morning, and welcome to Intellia Therapeutics' Third Quarter 2024 Financial Results Conference Call. My name is Drew, and I will be your conference operator today.

Following formal remarks, we will open the call up for a question-and-answer session. This conference is being recorded at the company's request and will be available on the company's website following the end of the call. [Operator Instructions] I will now turn the conference over to Lina Li, Senior Director of Investor Relations and Corporate Communications at Intellia. proceed. Please

company's and website. and At Earnings Welcome Intellia and Call. press call call. undertakes broadcast today's unless be can on outlining would uncertainties. replay All operator, management like on Third this a for Intellia sec.gov refer issued take morning, this be live, to as Media may listeners and to SEC release that Investors at intelliatx.com. information This is Intellia the found and Intellia release as XXXX risks this of quarter, ask remind that no website archived Quarter this of a time, Earlier the good you, today, duty Intellia's our topics by to This everyone. law. information at Therapeutics call, a update the company's statements well call Thank as I and minute available a this to for progress discussion required to this forward-looking you on discussion on make potential section current certain is being presented filings will during of morning,

review our call Chief will we our progress. pipeline I are over updates. before updates will Ed Medical then financials on from Financial Scientific provide John clinical Executive Officer. John, Laura me will the R&D Lebwohl, Executive to will our Chief begin Chief John Officer. Intellia Joining the questions. With and Ed David highlights. call business Officer; provide open turn now will David Officer; Chief Sepp-Lorenzino, Chief Laura Leonard, Officer; that, for Dulac, with recent And

we era thank everyone, morning, you At us for a of new Good in today. ushering all Lina. you, are joining medicine. and Thank Intellia,

substantial our Meeting, study Immunology These Scientific Asthma American NTLA-XXXX October, CRISPR-based potentially progress our to the the functional we quarter to Phase made of II editing simple promising, hereditary Annual highly results period, show potential patients offers Allergy, angioedema. and and onetime a gene of College positive for results a In at for and more from NTLA-XXXX efforts the of of curative cure. that presented differentiated the bring in third unprecedented treatment treatments we've During recent patients. infusion

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I'll XXXX, in or with development HAE. begin for hereditary John. Thanks, angioedema

attacks had further XXXX we noted, of These of eliminate groundbreaking John In II XX-week XXXX. care a for both XX standard single the XX-milligram Phase eliminate to and need presented has a XXXX attacks X of HAE. the the reset observation during primary which could results after treatment. the dose data, single dose As demonstrated that of potential no patients how for the highlight period arm,

have Phase patients We to so by The new II as not normal. rate time improvement I of our no become to XX-milligram on in are well. period show achieved remain the we median the to Phase findings more their attack-free are attack-free Based who observations, or yet the X far. are who these continued X expect therapy far reduction. cohort Moreover, attack attack-free adjust attack-free encouraged with as clinically observation patients meaningful they months the X patients HAE-directed in the a post-primary thus for from

appear believe dose continuing follow-up HAE, in XX development. disease, the for is and product that for out in further are the of creating a approved who need suggests an all emerging and cured other patients next standard by outcomes XX or forward we XXXX, currently look new therapies the profile without of data longer who functionally treatment. presenting patients HAE XX% This follow were where in that is patient received to we attack-free XX-milligram We I/II unprecedented is unmatched year. With are either of or their study patients patients Phase to

directly aligns emerging profile with market our the and and research patients Importantly, of product needs XXXX's priorities indicates physicians. that

demand broad III for such, Phase rapid enrollment approved. As expect will drive our of and that and XXXX study once uptake commercial we interest

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will extend our into the as look number state steady provide from a after Phase cleaner XX of effects drug's primary designed through month has read III the HAE Importantly, a observation period endpoint. attacks kallikrein to at and its its reached This weeks primary X the study is once reduction about therapy.

through fourth the standard the and in that progressive field our our of III track initiate submission the for developed, testament U.S. XXXX truly XXXX in we of within development. polyneuropathy. approved IND of ATTR to previously with primarily record gene also treatment As filing as well-positioned of application believe with amyloidosis. nex-z, announced living in a marks the amyloid accumulation patients trial expect editing people in from to to with we days in first multisystem today either polyneuropathy guided, disease known CRISPR cleared study. have in high in summary, we our therapies to for for This protein In vivo a drug This for amyloidosis treatment the BLA hereditary IND be gene deposits nervous transformative and submit to system. FDA the the is an or onetime We clearance HAE. Switching development a due manifests XX Phase vivo heart to in consecutive unparalleled as ATTR treatment editing cardiomyopathy XX-patient XXXX, the regulatory deposits

for gives change is will and III eligible date, at to randomized, to no in arm improved randomized reduction correlation positive clinical XX-milligram our placebo-controlled be other the is Phase ability to be benefit. nex-z placebo has will consistent, us MAGNITUDE-X tremendous of an in positively reduction XX nex-z, to confidence nex-z. and at To mNIS+X like and day primary international MAGNITUDE-X receive greater serum XX. In TTR from are randomized or durable agent trial single a the approved enrolled study. outcomes study. TTR protein placebo. receive clinical Our the impact optional month development demonstrated there The in X:X polyneuropathy, TTR double-blind, endpoints XX in deep and patient between or which a patients infusion baseline Patients crossover

with patient at U.S. to patients cardiomyopathy initiate reported publication sites and rapid, demonstrated the strong XX achieved sites more reduction with greater enrollment mean actively than III outcomes have clearance patients and beyond a received We Simultaneously, in over in is trial improve competitor's a current ahead of that clinical ongoing study, the TTR the regulatory are study. Findings cardiomyopathy all study In total, we therapies at a silencers. and with clinical interests. I of Phase more with the on enrolling physician based internal we projections days. indicate durable patients' that recent to In Phase expect XX nex-z, our opportunity in in provide. rapid in and reductions track ex of major MAGNITUDE than there that patients countries the consistent our what TTR coming deep continues ATTR-CM to data for

As early greater data such, which total patients. might significantly benefit clinical nex-z suggests reduce provide TTR clinical could turn in exposures, our for

presentation Next Association presenting ongoing new Session. American I the findings Phase Scientific study will Saturday, from late-breaking the a November we at XX, be as oral Heart

of and call including baseline. session On these at provide include will We Celebrating our all now same outcomes. Century into Laura, will data R&D nex-z Troponin, The over webcast an program such updates XX within Officer, includes better hypothesis to day, provide from who Cure. the Cardiovascular a we and the we on compared cardiomyopathy that reduction Science: clinical XX that TTR insights Prevention the efforts. the clinical provide the occasion arm, This test will investor walk data opening From of greater patients hand the NT-proBNP, where taking These the reduction emerging polyneuropathy of AHA's to on XXXX Treatment as benefit. functional measures update disease arm. I'll month titled look to will at important markers an of and and to Chief Scientific our be clinical anniversary, to data also profile TTR presenting the of safety, leads will XXXth on forward progression place to X-minute update reviewing data

in normal morning, insertion presented our It protein. our the beyond primate science antitrypsin Thank and in clinic. forward in we've first disease. of precisely the lung in first everyone. at in alpha-X X program Phase normal designed previously which through nonhuman We're track after you, follow-up for in ability to by these able is years alpha-X functional deficiency what David. alpha-X we single dose. data, a have NTLA-XXXX to durable SERPINAX first protein the study we the gene gene dose humans, the the owned patients we're levels be demonstrated translate step the gene, alpha-X vivo Good If believe encodes fully antitrypsin therapies to protein patient on In to year-end. vivo of advancing will were the Notably, is into XXXX primates in programs a seen alpha-X knockout to wild-type editing. gene and associated levels major study. this for I/II produce wholly vivo insertion of our completed of field We're insert nonhuman

updates a which gene editing look function applications. category new vivo with third Assuming outside gene forward programs our gene the will an our pipeline. I'll of of hand extend for call insertion Ed, advancing and novel or liver, with either therapeutic could the success, in we to we unlock proud over technologies reach tissues partners. results editing of ex We're all and more vivo platform our whole would includes Chief on parallel, quarter financial diseases XXXX. delivery of in pursue Financial independently in provide update X continuing gain CRISPR-based This collaboration recent the that advance we're to a of to our of providing editing our and as platform. In and who now different progress to it as Officer, require advance toolbox modular the

Intellia Thank solid continues to that everyone. execute a you, platform. Laura, balance sheet to and allows maintain good pipeline us and on our morning,

fund received and in by operations marketable December was compared $X offset cash The XX, part cash, million. equity as by in of stock million plans. of million including income with $XX net of the employee-based million from to were decrease $XXX.X approximately XX, September of Our related from XXXX. a decrease Regeneron, $X.X in the and was used approximately of cash million collaborator million securities The to as payment interest $XXX company's company's of proceeds onetime driven XXXX, technology billion proceeds equivalents to $XX.X $XXX.X million program, April at-the-market reimbursement, $XX collaboration

in expenses reduction the quarter was to XXXX. R&D quarter G&A quarter collaboration the for compared quarter our license by were quarter million was the $X.X in advancement XXXX of compensation. for to mainly third million of XXXX. Our Stock-based million XXXX compared lead $XXX.X the of AvenCell $XX.X quarter $X.X programs. driven and $XXX.X revenue expense The agreement. third $XX.X of R&D increase third was related million XXXX. third primarily related third $X.X million quarter. G&A within compared to by million XXXX during million revenue the a the compensation were of expense third million $XX.X million expenses of $X.X $XX was million third included driven collaboration increase quarter during the during during third the million stock-based decrease included the of Stock-based during during compensation the was The $XX.X of primarily XXXX. was The to to

to our our cash into balance expect fund XXXX. operating plans Finally, late we

call possible, only for forward on many able question I the caller. deliver week take the do AHA. our best to as at open to look of Thanks, Q&A. Ed. Operator, now editing, next questions we the we will update open continues promise will address per now In one to we and With To questions. to Phase for the presenting gene your call you nex-z be conclusion, may Intellia that, as

The with Bienkowski comes Fitzgerald. Cantor Rick first from Instructions] [Operator question

pace was of hoping get I the more to just into on MAGNITUDE-X. color enrollment

Just clinical XXXX. at listed looking clinicaltrials.gov, record the is estimated trial primary at December on completion the

you could endpoint given there me assumptions the So is that the walk through event-driven? the pace for enrollment of just primary

Thanks for Rick. the question,

with a you similar HAELO said, can good the we've study As assumptions. use proxy as

XXX patients. You know has study the that

final activated we being as the sites sites are have We here. speak activated and global

said But meanwhile, there things as here, road. further very projections. of And insight, comments progressing as our opportunity ahead well. we in an our are So we enrolling to update in be get down we're very, may the the that

Stranahan Bank The of from with comes next question America. Alec

[ Matthew for is Alec. on This ]

one quick for Just one us.

how clinical have capital As pipeline sort about trials, vivo given and priorities extending going versus in vivo later-stage you're into allocation your just you about lot forward? about of think the ex thinking a going thinking you in on

as address So necessary. up can Ed maybe that, follow we'll and

think JPMorgan Thanks quarter as plan, and I in X-year cash for about X- this strategic we mean X-year look through priorities at year, I Yes. we've question. or XXXX. exited the third priorities, -- $XXX laid our at the earlier million with we out

Phase clearly end is as of programs our have and us running related important to a studies, do a And XXXX, we focus which first so for And gene as prominent insertion investing concept look opportunities proof that's be as I and at sort on across the today, X focus. very year, platform. we've will the company been the up exciting just have burgeoning vivo in which an well. clearly We the the of III before then well program, ex of as some

plan very gets that place, of couple in our fourth have existing $XXX like should well estimate, cash as roughly look and burn the of over at go us I takes our conservative XXXX. we balance into But a a existing reasonable feel quarter that I So cash at last million up. quarters one

Zhu Securities. The comes Yanan Fargo from with next question Wells

Great.

study patients. ATTR things the example, I the on of congrats for That's a conduct the the is was silencer from arm FDA First, the XX size very the III half clearance struck the that the trial from design. is small, And -- study to X other not, in is study One and size patient by polyneuropathy. that study. just active even controlled placebo-controlled is of it Phase not studies.

the allowed silencer What such active the comment ability So could to study mean? will or obtain also either design? other study? does receive patients treatment it this, arm on And you during a on to

question, the for thanks Yanan. So

very, with First effect all, that to important large in reduction size note of That's the is TTR it's very established. of patients polyneuropathy. been

is on A It regulatory possible Phase safety FDA readout data allows TTR a currently of III. pretty promptly, to with outside available, carrying United other where agents. therapies and drug. and on this profound, the the shared of States, it in we'll the to study the many us by it not we reduction out enrolled be we the these And agreement We of are performance position be trial other any is as to as continue elsewhere, it. you very good have that And will give that to we've our study into effects insight function other that in our David, a to speak and patient and a TTR think expect can share you when population as placebo-controlled have reduction. the the based And silencers. or details to are pertinent solid

John. you, thank Yes,

FDA them great study. think placebo-controlled to a you're I with collaboration as best approved is small seeing All with have to the go way the drugs we forward. this other study what the been talk about in

So conducted, that studies silencers of not are not makes a the country as so where our be well will available drug. again, in the And for being silencers. receiving lot sense are patients

from next Mani The Foroohar question with comes Leerink.

CJ is Phase patients for noticed versus in that I III randomized HAELO on just X:X is X:X the Mani. are regarding study. question My This there magnitude.

So patient thought thought was you enrollment the in to I just was this if enrollment X:X study. accelerate wondering

David, the do to you want speak scheme? randomization to

Yes. cross at X/X the period. them observation the is a on will themselves. randomization they'll all also the the over these It's -- end X:X attractive drug for They right chance if active primary to to be wish benefit away. The have that they of able start to as patients patients of well

arm BLA be we of Phase will anticipate in that With well. having part as rapid by So to do all you X:X the number very enrollment coming as attractive it's that in active XXXX. information also, The up also have that submission as -- the saw on place, patients II patients reason. we for the of more in

Markets. BMO from next question comes Konstantinos Biliouris The Capital with

the program. the us from on on progress. question One AATD Congrats

you that of and under you that to promoter isn't gene AAT X ensure patients this Can need remind Xx how you us infection, the under is protein inserted there the that infections? the do control more about during AAT Given natural protein. additional

the normal point normalizing base Thanks question, on And would the albumin primates, expect promoter. module is we've of primary what -- that, the with for the achieved the it nonhuman where driven the by typical primary that out, promoter. Kostas. going in the we with by program the response in not be patients of defect protein. [indiscernible] -- But as the the you product. I the natural albumin over the it's our disease. is to And alpha-X be excitement it's success We because to be preclinical transgene infection that the protein will prioritized because we levels the driven to that's the of primary think determinant

Joseph comes TD The with Cowen. next question from Thome

able Or while the on on on as the is on depend secondly, at months, submission at maintaining to completely going for Congrats some a progress. to coming in how And you then is and are sort success of Maybe TTR readouts both the read of the that XX-month of MAGNITUDE-X a uncoupled? does also study, once? the point take ahead serum PN we should reduction MAGNITUDE-X these a anticipate Or those CM? for much day mNIS be PN, time just XX submission are serum TTR look blind? in XX

of But independent other. in entirely The will where that, We program of position how talked Polyneuropathy study data are of study about out, those can think from as determined we and readout. indications. read, have set independent a and period time programs sharing fill address particular prematurely will blinded, in each about earlier but to we'll an call terms they're We in each other of program, safety observation data the the be and be of and data the that's and can each sort here that. designs was David by efficacy the expect pointed cardiomyopathy are independent. complete not design. as is The of a we'll be in X accumulates, the before supplementary would on XX-month the are in blanks. X landmark programs, each that I the polyneuropathy cardiomyopathy Maybe

outcomes, So David, don't of as set And then to for becomes data guest. you the to be clinical it TTR. anything my be will the have look that, available. full if it ultimate add

you've to add our a data that major the TTR. in Just all observation seen in have to reduction patients that

looking and certainly our from will able years be data, maintained won't that reduction we is a X know as but be probably we over at in rapid patients that to permanently TTR far though we So this experience that what is as at deep and point. achieve all know,

says, to time pretty So at see the that John we data. will expect of in know much as we and what that unblinding,

from next The with Barclays. Wang Gena question comes

It's Tony on for Gena.

rate assumptions So your higher you if and can of on ATTR, silencer than strategies ends to address up any this for expected? remind drop-in us it being

speak silencer to the rate? David, do you want drop-in to

Yes.

We trial. have figured a that into

will We trial, that the tafamidis We justify it's data HAELOs likely some the there that to that there in. is together. drugs both to now we addition probably seen the think way haven't in drop to to enough for that is given mention, we've should much exact that not TTR pay we've but happen. less seen payers What will be

expect think effect that. So on will They payers is study. will what switch, on if possibly our have that primary patients their to even to in we don't any But we our vutrisiran switch tafamidis allow continue endpoint. may see that

Raycroft The Maurice from next Jefferies. with comes question

capacity. just or was if you I'm patient data the Congrats AHA on expectations well. update potentially the you're the as individual biomarkers if how talk wondering correlation for imaging more progress. remodeling to setting about you'll wondering functional I show relationship cardiac for could data, and or

I information. with fill David some think in start question, can I and additional can that

that. honored be be in we're cardiac is the the of the of part that the As next to in AHA which excited of meeting, meeting, about to featured Very very comments, is opening something session at we the century said our we're therapies. enthusiastic upcoming a sharing data addressing and

And the it will think real study. these particular because the with TTR expect that about if reduction how we David, on that excited that the that it think As readouts. meaning of to about share study we first relates yield we've clinical we see and time, information, long data said, comments? first, MAGNITUDE that, in of we're we'll we're want biomarker for patients. to share -- the what terms information the we the think And perform extent any how data, of particular you and achieving about into those drug we how other in insights functional

Yes.

to worsening they the Just down. on arms worsening XX. the IIIs, rise, that the they're Even in walk populations you though show recent than of these continue continues in see November placebo, whole. Recall terms to add, X-minute go data active in Phase as better biomarkers. are ProBNP continues to a still the to

So as data. you see it's our observe important to

this as that's data difference. this our the variant And also in or is the of because disease Class in ATTR-CM. in III very which clinical other deep XX% to really of to XX% TTR in around the very -- those a have in the test make are the XX% patients other Our of instead a XX%. a ability XX% aggressive of of reductions the studies patients the is robust see you full disease, of big -- well patients so And and studies

X because comparing these you the you patients will in X reached XX patients all and data to baseline the all -- they've reached for be So year, seeing study year. will see

Forseth next comes The Guggenheim. from question with Ry

ahead being for is MAGNITUDE team. MAGNITUDE-X And you are think PN? apply factors factors What to you these ATTR-CM do enrollment of Debjit's in This the to schedule from do contributors the study? Ry think major

And many will And see and treating enduring kind there's investigators think, is drug the consistent in confirmation the seen with tell far, world. is reduction they've great What anticipate to. lowering We with therapy the speaking. MAGNITUDE that And extent extremely that TTR to would enthusiasm the that patients, achieved sites the I patients. and this they to the this competitor know with around the meaningful with of of these the is that study. available with of apply that this we've reductions us recall safety not profile generally also really is programs deep, countries, We've date. we've TTR been for excited physicians we excited spoken that for the drug thus all that

The next Stifel. question comes with from Dae Ha Gon

Congrats question the AATD. A on progress. on

about embark the Phase thinking of I by end the that's going the to year. Just

more If you that I trial biology purview, be supposed have of a of the say, more excretion to and patients. include M protocol any can facilitation comment on but that any in protein. flexibility the given know disease from once cells XXXX was to polymer potential, you liver the

Just of you a wondering if there. you see can to little add if sentinel the just will, cohort, benefit bit that in

Gap] including trial? liver disease any [Audio XX David, in the

well we're not doing full as, reduce Yes, as these with of we patients will by major be think really alpha-X to them the as be the our ability the kind course, justice. Z addressed disease are protein, patients giving increased a would antitrypsin don't liver mutant valuable to them disease. liver well. But without protein having Those -- any of reducing major best

for a Well, after add, we lot just XXXX, which addition insertion approach approach to going think alpha-X extended and we're to alpha-X. will us diseases program to may insertion an And for path writing taking in liver. only gene the multiplicity the but the which disease we're about reads also bringing teach forward, would other to a lung forward not address also of the be a that

comes Issi Luca with The question next RBC. from

and on team, the Lisa is cleared getting progress polyneuropathy. Congrats here, TTR the all on IND for This Luca. for

the Just months. is XX one know the at I study. primary mNIS+X on endpoint question

months follow-up. just believe I on able file X to Alnylam was However, of

helpful. regulators? endpoint to needed would if was request the European XX-month just here the color satisfy FDA curious I'm mostly Or also the did So longer be Any follow-up?

on share follow-up comments a David, versus months XX any to different time?

trials, that. we -- but the months by optimal the really our see the benefit looking decision own the at was time regulators, really was Yes, was was do felt to we It XX XX required not drug. felt of to full it months

from Salveen comes question Sachs. Richter next with The Goldman

is on for Salveen. Mark This

any congrats presented of read-through recently data study? initiated context you on applying in the can III in First, HAELO? And to the quarter. The takeaways recently to the data that you're NTLA-XXXX Phase discuss from the Phase II HAE, HAELO

consider thus question. to Well, and step think thanks XXXX back we've for the for I actually really important it's what presented And far.

If new II -- reported and of to in therapy what with no studies, unique XX-milligram in following you doses have knowledge, achieved Phase now we've both is the functional I the other we have have cure. on we're and drugs. That a response. a patients take our single class the only no a of space. XXXX is achieved in, It this thereafter That considering attacks. of from XX Phase patients means who XX dose It's category

similar that come What that period we trial. follow-up the would similar results insights into respond our is there design a observation with even longer only reach in while even that patients exception some study patients outstanding functional to many we've longer Phase cure further study will that have get see an with is fairly from expect Phase III to a additional how are patients The seen quickly, time. We our with I period. some that

program favorable we sharing look our we'll forward III that So expect we have go very forward. and results to in as that Phase

Minter with The William next question comes Blair. from Myles

on is John for This Myles.

levels of initial patients dose us if So data? for are you could how those for for, are any And to looking kind you target AAT before XXXX, you I cohorts? lower was give what looking idea serum many reporting just wondering of dose in

that report work We've that and a here, is done the question. the when that the apply said consistent of which the here, we're objective we've with to protein. the clinical outset measured. our biomarker protein, principle standard that for the is And native will data Obviously, that be wild same interpretable, readily And that we've we'll applied success levels basis since all the the we've production the for we outset from here normalize project. and for it's here. of type. of is our the of Thanks that determining meaningful, which looking had That's a

The next from Securities. question with Lee Truist comes Joon

is This on Mehdi Joon. for

technology progress color writing programs really you plans using great your Given for future vivo on for your is have, any that gene in appreciated.

programs? it plan more in you this to or example, For do vivo your for is ex for use vivo

terms setting. there base certain universally start is ex so especially We think think suited. that company true. in on think that's are but And with a which think Thanks heart our we the one is really after. in complete side, setting the be vivo on where then, not It III course, objectives and We where a platform going the ongoing we're are results multiplex. achieving for to the position of particular also for editing those to tools X-pronged has where XXXX of as it's the best a that side, gene in the the question. a particular set the profoundly writing has both shown approach. XXXX delivery by CRISPR only strategy goes look to of going the We editing of of I that instances, editing Phase as we've we as successful well arcotypeal with in that the what vivo apply conditions and And we programs, vivo the useful any choose for don't there at we're tools will is that exemplified problem. editing problem, in tool the ex

So to the need apply in and we'll editing short, and the writing that. that programs gene particular at they the look

place it. of places would is we One the where first program, be an well the suited alpha-X as it's expect to would our see and that think where to I adjunct

next from comes The Oppenheimer. with Jay question Olson

the opportunity the interested you Are the Congrats you and XXXX? ex U.S. program are strategy progress. on ex all in for How U.S.? thinking partnering about

markets, Europe starting a U.S., that and developed world, we Thanks Behind for in the going that. the question. see the around assumption certainly beyond beyond U.S. substantial is and opportunity commercial that and perhaps into the the there's that the in

a are ourselves. in Our plans position drug to current the launch to be

we to As as extend reach. bring our as learn own we partnerships, appropriate, table share forward. go they to about more strategy can And what consider as the we evaluate that, would we we that

The next question comes Brian Cheng with JPMorgan. from

on Brian. Sean is for This

Just speaking about differentiation some thoughts AATD How of the XXXX's the curious editing just you your view of versus program, on RNA from your on theirs? the back peers. data do

for question. Thanks the

Thus what of success is are that's for of pathology for far, out, determinant humans. their of achieving we'll to We'll key That we're pulmonary very the or our whether that the Obviously, space. then we're it's essentially determine delivering performance only similar normal the disease protein even the think we protein. in company see the high of and a Phase I playing very, people how be we There watch not early. wild-type we proteins primate. looking same but achieve will elsewhere, study are those in the programs competitive normal successful nonhuman the said levels the at of are time. technologies production that in setting presented aware of we've As of over

next Silvan comes Citizens The with from Tuerkcan JMP. question

the on Congrats progress.

have enrolled the we ex trial But quick congrats may the U.S. patients the MAGNITUDE-X, IND. do considering But this. on plan of And in the trial? U.S. versus U.S. what for this in when and a eventual Just in missed this initiation one, I here is split for

Thanks will there on and question. the of readily trial. outside developing the this United elsewhere a around We've protocol States. for where otherwise tried patients make outside will for And one placebo-controlled study States. can studied be Administration the Food States. conduct are to program are that in the It's they that it coming world. the care Drug similar worked done Standards these clear We've patients accept with United from the the the United

comes Pickering next William The Bernstein. with from question

plans the do to have it For in case site? reasonable expand beyond Zealand the that program, a the New see you would is any AATD we And data XXXX? base study

speak David, to sites other want besides do you Zealand? to any New

Yes.

clinicgov.com that they ] possible think have and should sites, as we have it [ say on is to reasonable other I them having put we'll be we'll up. will in data come XXXX. We data,

Research. next from Chen with The question Wolfe Andy comes

for Andy. on Brendan

let's based use excited a now. on silencer, So compare can population? likely please using in to you docs editing efficacy similar say, excitement much stabilizers are endpoints feedback compared excitement more relative ATTR So their know about the very gene Any you a therapy. on to and about docs Are your concerns safety But old silencers? gene editing to achieve example? for we for

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Yes. John. Thanks,

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Lina turn for question-and-answer any the back concludes our would to This session. to Li over I like remarks. conference closing

great thanks, support the connecting day. everyone on XX. forward your Have Drew, for to We AHA of everyone, question and again and November Thanks, continued a with all look after Saturday, appreciate your today. presentation

The conference has concluded. Thank you for attending.

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