Intellia Therapeutics (NTLA) 9 May 242024 Q1 Earnings call transcript

Good morning, and welcome to Intellia Therapeutics First 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 Ian Karp, Senior Vice President of Investor Relations and Corporate Communications at Intellia. proceed. Please

company's and Earnings Welcome Intellia discussion Call. press outlining operator, First Intellia today's being be on broadcast issued morning, as archived a will & release live, the at release intelliatx.com. on XXXX can quarter Quarter Intellia's be Earlier website. the section and good of website This the you, topics This to call. Investors on everyone. Therapeutics a well Thank as this for found progress this Media call company's is morning, replay

and I'd call undertakes on to information risks to statements refer time, sec.gov this Intellia and uncertainties. to during this management like listeners All is law. call, as Intellia and for unless this you that our a At to no today, SEC duty information of take update minute this forward-looking ask presented remind of by filings a may available current discussion certain required make that potential at

Intellia Chief Medical Goddard, Officer; Joining me Chief Chief David Glenn Sepp-Lorenzino, and are Officer; Executive from Officer; Scientific Officer. Laura Lebwohl, Financial Chief Leonard, John

updates our John Laura update up provide review Glenn pipeline on clinical your recent with progress. overview the financials review questions. and before R&D for highlights. we an David call our our will an will business begin will will of open

Officer. With that, I'll now turn to John, the Chief Executive call our over

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an clinical hand to David? David our Chief programs. who update now will the over our call Officer, provide I'll Lebwohl, Medical on

and XXXX, in as in or This protein vivo progressive to disease John manifests amyloidosis. in through deposits I'll treatment deposits polyneuropathy either the CRISPR ATTR nervous primarily cardiomyopathy for welcome, due system. heart the Thanks, accumulation our with multisystem of amyloid everyone. candidate of begin the the

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highly deeper dosed with from improvements silencing diagnosed data of neuropathy. PN ATTR on progresses measures typically than and earlier standard demonstrate in are disease chronically Importantly, TTR that often are patients ATTR-CM. adulthood, TTR of rapidly more correlated therapies Public their reductions

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such Additionally, the well we summary, dramatically as time, X-minute reset and potentially plan disease data landscape. mNIS+X. In as the TTR NT-proBNP, may beyond first to continue include levels, walk as ATTR we and to treatment reverse believe test for the halt XXXX

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developments with our modular turn insertion now exciting me platform. Let to gene

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development pipeline as is rapidly leader medicine. is Our positioned a this in new accelerating, era and clinical Intellia well of of the in vivo

our the Officer, Laura, updates over hand now efforts what's call to R&D and who next. coming on provide I'll will our Scientific Chief

Intellia, therapeutic Thank we're technologies you, At for everyone. and applications. vivo advancing ex and David. delivery vivo Good novel gene in morning, editing

development. mentioned, is strategy type on the safety emphasis and As core our John each to at performance of

take by Our success don't my to that we in become and a chance, the of just common and a making have editing to But for lead it's experience misconception industry have word leveraging and platform able world-class our date it. entire adaptations delivery is to technologies, tools been commoditized. By the dramatic gene forward. not improvements team

give [indiscernible] therapy. our you Let examples. Intellia FDA the world in some me by now INDs is approved for investigational real

the Groundbreaking NTLA-XXXX in Medicine. been sets NTLA-XXXX Journal of have both and data clinical published for New

success Intellia gained help already which us Phase a regulatory Regeneron, genetic III the foster of clinical in conducting the have company, study become We to of clearance with the that medicine. has as important X Together we're trials work different advocacy reference CRISPR-based be world's relationships countries. the multiple has largest for And global with that gene-editing expanding in technology. building experts the cases our we're medical well liver, initiatives as our leading on the organizations. with now originating can targeted [indiscernible]

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[indiscernible] pipeline both We're autoimmune for in collaboration treatment and of diseases. owned programs, also the and partners with of advancing immuno-oncology wholly the

is of partners our already allogeneic our which platform, of fact, X in clinic. are the one In leveraging

is uniquely solutions, [indiscernible] avoid anti-cell and T-cell Our and both control. disease to [indiscernible] results and including allogeneic processes rejection mediator differentiated positioned

on combined platform tumors. function, our is our this year. target [indiscernible] allogeneic forward Further, you broadly our more with offering to on enhance updating a look to edits solid R&D new across progress with platforms We to approach engineered

provide the an our to our hand Glenn, first XXXX. over who financial for update call quarter will Chief Financial now results I'll on as Officer,

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

million of marketable XX, driven securities $XXX.X million as million of to of interest from approximately of in March proceeds by Our cash, $X the XXXX, million. by cash compared XXXX. decrease in to as equivalents and The fund $XXX.X at-the-market offset $X cash X. program, approximately of million collaborator X $XX.X proceeds part was billion December operations was XX, stock and used reimbursements of plans. income,$ decrease million company's equity net were employee-based $XX The from

of was [indiscernible] first during mainly Our during by million driven first million million million noncash quarter a quarter related was the $XX.X $XX revenue recognition XXXX adjustment The to compared revenue $XX.X the collaboration $XX.X the increase to XXXX. of collaboration.

in our expenses advancement of million the XXXX. of was million $XX.X during compared $XXX.X compensation the XXXX million quarter quarter of R&D expenses for the by the to during $XX.X Stock-based were first mainly driven included lead R&D programs. XXXX. first million quarter $XX.X the increase was of first

Stock-based during XXXX. $X.X XXXX. of $XX.X million first XXXX quarter million during the expenses the primarily for included of compensation. increase $XX.X related million in quarter first quarter the was compared $XX to was expenses first to compensation million G&A were The stock-based G&A of

late our laid gives we Finally, cash this until X-year expect our execute power of XXXX. the priorities strategic out on us year. to at balance financial sheet plans Notably, our strong the beginning operating balance the to fund

execute trials to pivotal our to and deploy next vivo in modalities. editing to therapies; First, vivo two delivery for wave third, and clinical of the new first second vivo launch programs; in CRISPR-based and ex

will gene we on promise Intellia, look the well our be on to continuing catalyst-rich you progress. We're and editing. forward of updating This a for way year to realizing our

now the for questions. the for open now call Operator, may open you will we that, call With Q&A. your

from Maurice Yanan Wells with Zhu Jefferies. question [Operator Instructions] the ahead. We'll The next Raycroft Fargo questioner with Please first to go comes Securities. go

this a of progress And quarter. lot

So congrats on those progress.

a have Wondering U.S. the on per And ATTR number III currently the speed about enrollment trial you that many your month of opened. sites patients. thinking at ex internal of number Phase you how about site patient the the the comment I have is enrolled in -- are of question currently the -- expectation. any And expect line, ahead So What's U.S. of what terms in sites? time tracking? versus [indiscernible] enrollment the completion per updated tracking

I lot think - of a questions.

that. Sorry about

going one to updates giving questions numbers you thank of sites, patients asked. not be question. that you Yanan, We're many site, the on et cetera. per just And for

around before, the and of will ultimate said said a X/X in States it's We've this remainder will where we study the the world all the is sites As actively spread world. study initiating expect balanced around United sites. be be still about of the we

see very well. the We've in that patients about we sites here the study. very to regulatory submissions to been see we're waiting the I the that enthusiasm We've able to expeditiously. come that and mean, would into we're I is I think takeaway process been encourage you this have early are initiate going things investigators. with and excited clearing been

morbidity state patients to disease of as the and a it's patients ongoing which think mortality with current and to what the We see therapy, testimony from you physicians of progression. approved with with drugs, current know, continue the suffer

But we're patient We're we're patient off So we by will not making. be encourage very strong would giving you updates very, to that to go. enthusiastic as a see we I progress start. about the

From going think approval your I their follow-up the may of in of gene that. you in upcoming does how What -- GIs with- you If how the make sites. quick from you hear impact about from and interaction thinking therapy potential terms on do patients a on that particular, the treatment. of silencers? what U.S.

early the the ability trial, participate the very patients you Stage patients for on that's et trial the presented proBNP patients in patients far, I'll about to putting and a heart look study cetera. put put seeing the placebo. thus and interactions next created impressed a are to encouraged that failure, reasons these people X:X the There's over I been our think with have favorable in of randomization for or results. thinking to how study. on In forward their to it patients. that in with I want remind higher data that we we're lots they're We've III study physicians very favors patients that it. to Again, -- question. to influenced we hasn't a drug level, and Thanks.

Maury with Raycroft question comes Jefferies. next from The

technical Sorry Congrats issue. the progress. about on the

precedented the was patients. at study XXX ATTR in studies their APOLLO-A and the XXX. just so [indiscernible] was PN space, HELIOS-A was For then XXX study, looking

what And how so what about endpoints? FDA clinical your XX imply conversation does Phase your study of And what if those are with just with patients. the III on about wondering on HAE talk Maybe align went and this endpoints you talk about III results Phase the can be? to size could expectations for

to specificity close the with with working you it David and we'll Drug let designed David going world. I we both address speak Administration so see. some study would read respect very animals. [indiscernible] point last to and trials, and specifics, HAE around is to and one certainly different as polyneuropathy me endpoints Food X first, the into I other the about But before a how we wouldn't the emphasize, we've And that, of the developed these hand Well, to more can take to totally think speaks over regulatory agencies get to other relationship I'm then the specifics just how of

I have for these ultimately a regulatory very well understanding and their good have expected understanding meaningful purposes a as own way to of of agencies how for show the effects good think in drugs very and other they the as around of them a world. physicians for

you things will we maybe say can why think about and that a study successful. David, how we're be it approaching few

An picture are big talked the design, that seen we the reductions is is part been very patients about that TTR with our a this we're the despite and recognize existing we we deep them. of and effect them that discussions on do positive And consistent think to data. drugs. progressing they our clinical and in that what that's getting First, FDA still have important

of patients. FDA TTR terms of show the In reduction the seen [indiscernible] that a also of leads number number has patients, studies for the

becoming fact. a that's well-established So

way in course, actually the disease, more what how reflect forward approvals the to to Of biomarkers happened a is go future. in but interested be may to the only FDA not

their and the with XX this database in much do trial Phase Phase also of have confidence the from in do working I I So a as but patients course, cardiomyopathy we as larger we're well to only trial the ongoing of think III. size the not the has how

Cowen. question next Langford Troy with The comes from TD

So with development more this for NTLA-XXXX. placebo-controlled surely for the traditional you Where dose refine move Phase you I asset? how with you have like do quickly this NTLA-XXXX, So with pivotal work can like to do NTLA-XXXX. after to dose think think think we'll try into a study asset you a Phase II do further take you the study pivotal through would

well from Troy. for drugs, of emphasized I'd as emphasize start question, our point in safety a the as by earlier comments especially an today, understanding from was Thanks efficacy we view. saying, these just as

guiding that as of carry we study those will our out be And principles the so XXXX.

what you with remind normal the excited drug established human regulatory able preclinical fundamental We're do. beings. we data, the we've I'll we're think nonhuman levels in as can in that primates to that that's the We think seen in strategy. to reach

is Phase quickly progress agencies Drug I to as way efficient see will we'll hope we that, and to approval. working we that most what shortest, the we related is that that show can, the in establish assuming and Food as with Our and other regulatory Administration studies

you So that lies ahead, proceed. and we'll as updates we give

with next The RBC Luca from question Capital. Issi comes

and David, your recent of monotherapy show rethinking all the cardiomyopathy, Are primary design overall for and they to trial why add-on the from the do any the endpoint Maybe have aspects optionality TTR on the you scenario you you no progress. did enrolling competitor. on changes hits between your where as HELIOS-B Congrats the given [indiscernible] expand just split benefit not in are all a patients TTR on to then in can quickly polyneuropathy, population monotherapy on similar pivotal? U.S. what maybe any and the the

So we've when some have specifics which data study, happen, the as trial. they all really as case to been we how gone in able the approached some of by well, about understanding before is TTR, been in. the there's that some rate an pivotal trial, of the fundamental thoughtful -- driven of In progression. events other we've us from by I'd been mindful we the drugs learn certainly surmise things gone moment prespecified and we've which differences rates may be in that of presented We've at by time out to not pointing endpoint-driven start just but of David the the speak

is, trials think the from I beginning. an approach. endpoint-driven the that's And we've what approach in standard fact, to these embraced

I at can think, current it David, our maybe time. few thinking does say words affect you how a this just not does or about HELIOS

been of The those first John magnitude assumptions may the conservative we've designed we confident, point the the as is as it. in all study saying, in design is made points, that in because we are you have

than Of is study course, it HELIOS. larger a

The I follow that's also event-driven we important. of these we think disease proBNP very patients. patients time took upper trial. amount limit an that. limit this on more and some don't of higher, advanced in We don't the have that And we in is these

be Of in reductions, can before, because drugs course, we and we -- consistently in deep studies. class as to TTR think we we've can durable get and stated other do best be we the better

be to -- So we will trial. We from data that be paying it very comes positive obviously, out attention expect close very a do the to HELIOS.

modify out whatever point at potential You in from But it the we the the order point. will add to -- this whatever results, found to how address know don't trial we those comes to drugs. on other is [indiscernible] have our much out

insight important where also think on that conducting have it control and I it's looking why? So FDA, Phase placebo was discussions control it to arm. polyneuropathy a we're III for into our with slide for the a the [indiscernible] some clear

patients are good is these to access U.S., In access silences. have in the to do So silences. the there world who very not

So satisfied patients patients. U.S. be having the of for would the reason main need in not those gain we the is because

aligned do that study that They we're similar we and note treatment we it. should this doing think that to the a with And to be important because are it's would information readily how so approach. the United understand we'll be practices applicable And States. where

Barclays. with Wang Gena from comes question next The

for Harshita Gena. is on This

a but understand you is these completed range when each you're more can't agency to on preclinical this that study, you the speak a least and now on NTLA-XXXX, able take us can long from data? color submission that how on And you the I able Is that if to for plan data? granular embryological provide provide development unique, color -- you on us at on submit any lines, agency provide And you be Just situation one the quick will are would time to to the helpful. review it imminent?

for and processes Phase things not is each don't go through you question, and of remind gating the procedures even program. Thanks submission the Harshita. III report-by-report this for those for We I'd and that

position The getting first because seen strong think I start enthusiastic the regulatory well, data and FDA, think quickly is to position expected. in year. to patients that poised we data, to in we're this hopefully, with enroll point respect position is we'll we're as we before really we more that to readily about. And to reminder, the Phase and the those, end going the have our What's expect interact Phase during to the be start and make have going in said take II designations we those submitted David the this issues in of a to program that proceed. we're we'll be very, III actually no allow FDA which us There's with and we've year. to really begin a the really something a very III, as drive And to Phase some to be to data the that's comments sharing was referred good a as later to by we when year, very it's earlier,

The BMO. next question with Biliouris from comes Konstantinos

Congrats the or question great XXXX from One us. on progress.

the the lower post may higher be. treatment, survival that You types have amyloidosis from suggest TTR previously levels residual sold the that data the other of

show NTLA-XXXX collected year, data I'm survival. far you across and are TTR wondering whether so presenting you from ultimately can this more the relation you between the doses trends to have that different be sufficient that allow Given to levels test data

far and speak might to comments from I know extract provide? it's you any David, what endpoints. we early, to additional decreases to so you want TTR can but want

Yes.

other So that the you the been -- protein extremely what's say lowering -- with the found is diseases important. is you're right when

response, In normal example, with a fact, essentially have patients when the complete for survival. you light disease, chain achieve a

why so about we're results reduction. To and deep because our consistent important we're the the TTR and patients able we our this So actually excited see level be between we an data, as reductions very all in relationship goal our are reduction see to of getting very -- do a deep won't own do because survival are getting. that

example, reduction, But to improvement along and data one really finding back, as do other you be successful you These already. us you findings are can important the trial silencers for what just greater It look will trying the of a the that cardiomyopathy neuropathy. this that see, in the go for really do. goes and with polyneuropathy deeper from with types it's confidence to to gives in example,

The next question comes Gon from Stifel. Dae Ha with

templates This your is with Benazir all? Group discussed FDA, noticed repair the NTLA-XXXX they such introduced. phenomenon when on just with Can have for interaction to of Dae and recently comment are at a in kind Stanford detect what a was that there insertion employing [indiscernible] on concatenated Gon. We you published incidents this that on methods you're

Laura, group to might in cell? there Maybe, was that techniques And a are to that for the academic the employ extent if which question insertion. occur has an you found -- can concatemer one understand

the of range part different [indiscernible] That's a single clinical of course. sequencing next-generation to sequencing types whether of including or insertion long have you use Yes, development concatemers. understand

up about Excellent. Has that the information? FDA brought of anything wanting kind

The that. FDA hasn't asked about

question with Cheng The from JPMorgan. next comes Brian

year? tee [indiscernible]? allocating with you're you are moving later set years cash balance pivotal you're I you about this X and of always focus study off as prioritizing your our runway and HAE goal forward the to you burn thinking from the sheet. X when of how us a have conversation, resources and on give recall How sense the cash about is prior Can overall like TTR, to

for the Thanks this Brian, Glenn. is Yes, question.

having priorities been we've strategic talked now So laying cash. basically, that we've late That's investors. morning XXXX. to out X-year this And being contemplates all years' that funded the we about runway X.X -- worth for of about is we're what

like we in we're So shape there. feel pretty good

The last in other from here are you've consistent forward operating as to thing expenses I would say go stay is the quarters. couple what we just seen going of pretty the

Securities. Guggenheim with Chattopadhyay Debjit from comes question next The

on is what XXXX on this Robert from effects is for This [indiscernible] Intellia's potential and of us synergistic morning, view knockdown Debjit. stabilization? Two

how insertion, does like plan Intellia maintain and to gene therapeutic particularly a on And window, [indiscernible] in Regeneron a within disease expression

you a do word XXXX how aiming And therapeutic the for being follow want you primarily as pursued we're levels about we tafamidis? between that and reminder David? can that thinking hemophilia Laura, this is or pursuing with X point. B say maybe, to by up David, about then [indiscernible] how the a we're interaction

that only protein, protein the the we the abnormal So protein in then not enough, is see amyloid, we start of effect the the will remove you think And it of -- disease. about that reduction anymaloid don't discussed low keep this way accumulating actually if organ. the but might fact, gets the the that better precursor the on don't from of we already, I amyloid what the body expect lower amount the

lowest levels. So the our talked that in some about TTR work. get of absolute We've goal is recent our to

some fact, ask, number? your forward. the some at people In That's thing may if point, got TTR people important what is moving

tafamidis. with happens what So

get of then low still be because protein abnormal remaining could levels stabilized. bit tafamidis valuable could If enough the you do the be

with drug somewhat a go tafamidis. make to with from you even it amount better is our very what already perhaps low So

low interaction. might be it levels. valuable interaction So It synergistic may you a very these get as through a be

how much So insertion [indiscernible] levels. therapeutic much what's Part characterize involves with matrix those and deeply protein doing that on you and plan. In therapeutic of of of range expression across regards And insertion development the of data the production. for package right? question does template, to the insertion way clinic, how and level in what's And those of that going preclinical gene maintain allometric the clearly results part how escalating taking you is [indiscernible] into account humans, safe purposeful right, a of cohort. be your understand and development is patients of there very how to to that then the multiple in a clinical the as do translate

driven me remind by Regeneron. So that [indiscernible]

number move translate into time, valuation appropriate of insertion data, but they're important to insertion the a approved it's seeing mobility. the clinical program, data of expecting XX% is we're be gene So the diseases for and that platform other going already which to is a that not due clinic, will which for most we're in to and only

to say of expect. about Part stability Maybe the maintain you levels? that question how could word was a the we

stable for have key into proliferation where could of a genome, you difference alpha you other traditional and gene when time, very, by silence inserts sorted cells over from very just an Well, [indiscernible] shown the right, be the episome have therapy for This see or maintenance, can insertion. for we the that time. lost X is [indiscernible] be over episome yes, that liver

persistent So those controlled. going and achieve stable well you that we be once expect levels, those to are

question next Mary with America. Bank Davis comes from of The Kate

writing cystic maybe with guess DNA your with looking your at the and your utilizing of talk earlier and the you pipeline I collaboration medicine in editing gene opportunity fibrosis recent potential this market address technology stage could to opportunity with [indiscernible], about also indication.

why with start about technical we fibrosis excited and we address that Laura successfully approach. cystic the of I'll we're our some talk about things maybe can believe can

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Yes.

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The with next Rick Cantor. from comes question Bienkowski

all the on progress. Congrats

for different tafamidis for a top potential how on I hear from XXXX to be follow-up versus commercial on and in it patients magnitude. hoping I thoughts treatment benefit question on on strong effects So on implications your showing for XXXX show not earlier treatment had to a baseline in important TTR, tafamidis those the the is of was Lucas would treatment? what

you do address want importance and to benefit thereof? David, the

so as single -- be that the think than XXXX drug we a believe can better what I could tafamidis be agent. is And the

believe We of a there also will top that on tafamidis. be benefit

to on and Of are this What we still literature. Patients best better from progress patients for and from disease. for as proven. the course, investigators that's virtually looking continue be we the are to understand hearing from that all can is investigators they tafamidis physicians drugs

tafamidis what study. could that receiving obviously, So we single the -- This tafamidis is patients be do doing. seen than better think role we're is there in the who a in not are is for agent a

important part So analysis be this will our an of work. as

whether with program. represent a we've at to it's beginning or what outcomes tafamidis. the drug. I advance the the drugs mortal end in marketplace the which data and that taken significant we that nobody from very and think we've thus satisfied offer in sharing the that investigators a and and a state think what we real treatment, on that's to combination the that development be best far, to know the with trial seen used is remains the we I the currently all Phase over that. illness, alone, we've patients prevail that is of our important is show been going that's in disease expect will excited III state approach We're of The mean patients progress advance to And field. for of patients.

follow-up just maybe you Very would on about thinking Since here, Would the to those hoping you those and X to to there's had quick Or if just was in quick parts I X. great. the a dose vehicles? Alpha I on extended components escalation. an you And a follow-up. you're LNP the moving be tandem? X AAV. escalate comment dose different escalation control for how do able have delivery could

I'll we clinic. to turn work do as preclinical of the before possible just variables the ever lot try many to as set but into of the David, to table, a to entering address

with we think on clinic. that work want terms anybody just we of dead complete we as our our go the where with essentially in earlier, to a in what saw So don't in shared and was I modeling XXX, XXXX unknown

the apply XXXX well. the We as expect from work will insights preclinical very many the we've learned that directly of very,

maybe word of standard a checkerboard what sort to of be about say So X or David, could XXXX. you some of a would of just the contraction study

is every parts, LNP, the and XXXX targeted that from target this AAV, up so a the we contribution that and we've the AAV we've achieve lot make. open the with and essentially that particular site XXXX that can site being to program we allele that learned the for both Right? a can will learned and to So about what with LNP ability the these go already X

more So variation idea good a get will -- be LNP optimally as and how this of more is in dose expression we much we antitrypsin. of the AAV terms have the of to think needed alpha-X

goal achieved That's that's is clinically goal get what our and as to levels. well. preclinically, we've normal Again, our

Are next question from you Lee with ready Securities. June next The your comes question? Truist for

Staying June. This ATTR. on Mahdi is on for

a June and positive from late HELIOS assuming update So possibly July.

the change a does a in once Europe. third-generation could how year dosing So candidate landscape with treatment RNAi potential

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be for setting we the to we important it's will different So options. that think patients nonetheless, standard. But have

The next question comes from Silvan Tuerkcan with JMP Securities.

further tissues. little your like of strategically. cystic and fibrosis. within touched are today, are which studies, you've foot But give bone towards to of ones couple bit the marrow XX,XXX closer on ones more about years that coming you view which know strategy us over how new I tissues towards should these a Just you And Maybe those moving in we the can a think programs IND-enabling fruition XX. mentioned, on to all along? next on

It's Thanks individual relay and don't run. races these for think lapse But you that important question. the horse the I updates one. want race, it's give we an to a on

and been first intent delivery are the It the editing important. a technologies. set has tissues and company really that since It's we goes the very of out. these deliberate of strategic back about thinks the very to the approach basis very, philosophy that All

you apparent, with program XXXX started is So into the liver, we gene see which a liver. as with knockout the out as to insertion

take writing to has technology have the liver. in editing these outside to been goal to the the that gene add of form diseases in And various We that them to technologies was imply liver. the

We're that able places us that to may collaborators, ourselves. our own take by we sparing the we get doing not and supplement And with do where vision. work as with with people Laura We're there has work to mentioned have about can excited earlier, that ReCode. technologies be

earlier important as were of X collaboration, tissues, Each you diseases that have of medical with know, large have very with We delineated unmet need. these with Regeneron. them populations

liver we opportunities current Some in time. the what the see at of those surpass

point And preclinical those from so benefit all editing of the resourced of from development candidates. get an common to being very, of approach and them to very all are them aggressively view,

The Seedhouse with comes James Financial. Raymond question next from Steve

Timur for Steve Seedhouse. is This

in is II the who what have enrollment would who expressing Is rapid the treatment. patients study. interest study? couldn't any like we Phase it magnitude are patients Patients access screen tafamidis to on screened, no some other thing of will in the rates clarify on congrats So type patients more XX to One trials patients you I'd that on do is or other enroll or is what are the of to one failure what like out think be category? And proportion clarify dosed? the thing

any And as to going the all do, a said screening not in not our insight for terms there can to provide details work number that's Thanks go and into and question. of the earlier, for into of failures. that, we that's sites coming or patients this as you We're But and appropriate of I sites of David, as the general patients coming point? study go forward. share at type the we'll is

all from We with but of investigators these investigators week and disease for meeting stages. just affect patients, were this can at the excitement their there's all this earlier actually

disease can the progression their disease, you they they're want patients, at want even doing of As reverse early to for or prevent well least imagine, stabilize patients disease. with if they to sicker

of the the around from investigators So is them the access about screen the we are we expect families failure in all will and than be from continue. that's hearing them. expect The are already forward XX about but more proportions with piece XX well, we randomized cases. heard, any to patients more of they patients. gone with half some going in their the have coming half What world, you've trial. trial we That's what really with low, than or healthy right as behind obviously rate who patients it's might too very some too sick and is But

question last from Pickering with our And comes Bernstein. William

said I And CTA in you to quarter. what AATD trial follow-up filed believe have the application is you feedback underway, duration include any once old of received Could you Congrats all in the that application? want response or if you on progress this would initial December. presentation? that to any you share the data

status want you do speak CTA program? to of the alpha-X to David,

Yes.

of with that that. CTA, any we've day back some gotten status the So expecting straightforward hear questions We're to about addressed. the we've

to In body terms have of follow-up, principle will we data that have of a on report forward. bringing significant be follow data the we that always the we -- when we will

the has IND Regeneron approvals Europe of As a reminder, the and gained both cleared.

itself, So forward application that in the world. the of ability go in have the around terms to platform, we high do confidence its

Ian turn I to would the to At this time, over conference back like Karp.

our As session question-and-answer any for remarks. closing concludes this

continued everyone, And for and progress. to your us continue so a thanks, to as Great. you Drew, much, questions updating joining today Intellia. forward we great look we day, interest Thanks your for everyone. and in Have great for

you participation. for now has Thank your conference The concluded.

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