Intellia Therapeutics (NTLA) 3 Nov 222022 Q3 Earnings call transcript

Good morning. And welcome to the Intellia Therapeutics' Third Quarter 2022 Financial Results Conference Call. My name is Andrew 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 at the company's website following the end of the call.

participants in all listen-only reminder, a are a currently mode. As Instructions]. [Operator

now I President over to Intellia. of the Ian conference Relations will Corporate Please turn and Karp, proceed. at Communications Investor Senior Vice

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

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

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

an open financial Q&A. then John remarks before Intellia's the review concluding recap company's John call the highlights. up David And recent Laura will will then our the for for with of then will will will begin some on third business R&D overview programs. we provide an offer clinical update results Glenn quarter. progress.

to over the With that, now turn John. I'll call

solutions medicine. we're the At toolbox. vivo editing leading deploying genome broadest in with of company the novel delivery for Thank each to deepest joining you, all industry's harness thank to the Ian. therapeutic power us potential applications, building for CRISPR-based pioneering immense and ex revolutionize and technologies this and the editing by vivo morning. We're And Intellia, you

third our pipeline the milestones to we NTLA-XXXX quarter, execute against includes full During we've modular first-in-human our across spectrum achieving of clinical recently, studies and This as continued priorities our strategic NTLA-XXXX. and platform. and landmark more advance significant

were we clinical to with potential hereditary recap one-time to living interim datasets angioedema. be will or which Both shortly. for both data trials, treatments the David for from demonstrate investigational September, amyloidosis In positive either delighted therapies share transformative, ATTR people

our By showed we data levels evidence impact. meaningful successfully Further, for We're doing reduce move platform first clinically of the these ongoing foundation forward the coming modularity which powerful of disease-causing will on studies can of and and a editing looking CRISPR-based gene, pipeline protein, a NTLA-XXXX also therapies. interim that so offending provide additional data not these only the strong time sharing weeks. highlight the NTLA-XXXX to elicit we the in from with forward but in significantly a of can we

it David to turn now I'll over

data reduction X.X were John. Heart remarkably mean of of And with XXXX at adult results both show protein trial. Phase consistent kilogram amyloidosis, XX% with These data the respectively cardiomyopathy ATTR cardiomyopathy from reduction heart XX% dose the from day interim Association Thanks, to we with milligram TTR At X.X ranging and failure. achieved, levels, or XX. I XX shared levels class to serum the XX%. ongoing X were amyloidosis New interim of III XX% the doses from positive transthyretin and These at York amyloidosis, per for there ATTR treatment of welcome, arm recently clinical Beginning with patients everyone.

No observed and clinically at level. adverse XXXX which were either event. treatment-related was the abnormalities was tolerated. XX laboratory generally dose reactions, Two transient well reported significant of observed quickly infusion only patients resolved

of the best agent. protein of We presenting data, and to We be forward durable for Session excitement oral extended at that the investigative deep, look has an for up reduction time breaking interim cardiology Scientific community to The the XXXX's a these TTR our AHA the including a audience first continue levels these cardiologists. leading selection of novel underscores lowering for presentation approach. consistent follow believe potential abstract to to support late

part today the eight initiated Part in XX the that arm portion, expansion the include cardiomyopathy milligram and we polyneuropathy announced a a kilogram XX fixed dosing dose, dose study. corresponds X.X the per is dose to which minimum two, patients of We in at which milligram the of patients arm. will in two

study this US both X clinical selection previously As steps on pivotal arms XXXX. This of forward for by sites. expect of sharing guided, decision inform planned enrollment the end we include will subsequent look dose details complete We additional next studies, of year. to we Phase will early in the which to expect our

vivo treatment Turning the hereditary for now second to XXXX, of our in or investigational therapy program, our angioedema HAE.

at study of in shared Bradykinin reductions and in patients to that interim we Symposium resulted attacks from levels Here, thrilled ongoing HAE. swelling were We both the Phase XXXX in the robust with positive rate Berlin. the in X/X report kallikrein XXXX clinical plasma results

eight, dependent with mean A of single cohort milligram XX in and plasma milligram dose dose and XX reductions by XX% led in the kallikrein reduction respectively. to XX. week dose X%

period. mean of the measured rates observation XX primary analysis period. the a the at the being HAE of to attack in XX-week single first XX% XXXX milligram addition in reduction kallikrein the end plasma In in through study, with week are dose attacks resulted A of observation prespecified XX levels, also HAE occurring

the all the through have of two free XX up. single attack not have Additionally, week since follow treatment, and patients presented had a since been patients three HAE three attack

prevent mark been vivo a characterize potential and that of clinical the disease. for debilitating history, swelling fatal single And attacks These underscore XXXX systemic potentially the chronic genetic CRISPR-based data the therapy. for to in dose lifelong data time generated early this has in second permanently importantly,

safety and attack rate kallikrein Later reduction data dose kallikrein safety forward additional initial we data the look this XX dose month, milligram and at from XX milligram XX include presenting that from reduction additional ACAAI, milligram data and the cohort, and cohort. to will

portion dose next US expansion this the of year, part sites As to Phase the we previously, clinical as including placebo-controlled of in of we've the shared anticipate and trial. half X, study expect first begin

over on platform now CSO, I'll to updates and efforts. Laura, hand our R&D provide will our who

Thank you, David.

Looking a NTLA-XXXX, IND-enabling beyond the the of continue and and manifestation NTLA-XXXX, candidate NTLA-XXXX we alpha-X liver to for for wholly gene two candidates manifestation. candidate deficiency. of progress includes We're treatment insertion disease, activities both candidates. for lung the the NTLA-XXXX, knock-out for development This conducting owned antitrypsin our

overcome is vivo of we to Intellia, ex by At our developed allogeneic designed a host [indiscernible] durability rejection allogeneic the investigational to to now therapies. have proprietary current the efforts. platform Shifting which

By which create and novel of implementing with be matching cells anti-tumor the our activity, approach, LN-based in persisting we patient capable high maintain uniquely may T durable to cell engineering responses. allogeneic

genetic first and of cells, Last avoid novel II models, host previously the the proteins. HLA-B in The and tested preclinical allogeneic while in a the month, at knockout for by NK involved importantly, shared edits which HLA when Class resulting by [indiscernible]. C T cells, destruction time we our to approach, includes cells, ESGCT, host were and HLA-A, retaining able

the we healthy to donors. patient population HLA-B only patient small can donor majority addresses a with by T therapy of HLA-C, matching that Further create and the for set an off-the-shelf only cells of

host continue partner engine and propel of wholly research as our such these Class to and owned programs. I a innovations robust

AvenCell underpins platform collaborations and allogeneic already Our Kyverna. with

an TCR For this the WTX wholly candidate NTLA-XXXX, allogeneic clinical efforts, to as currently targeting technology person. also owned for hematologic are a NTLA-XXXX our IND-enabling CAR-T for an of CDXX to in ongoing applying expressing activities allo transition development former We're we our treatment answers. candidate,

With forward leadership at to our genome and of platform the the CRISPR-based revolution. toolbox, we're of position depth forefront the editing breadth maintain driving innovation our

over CFO, provide who the our of quarter our Glenn, call hand to will results. an financial I'll now, overview second And

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

as Therapeutics equivalents used Our was cash, securities were XXXX. decrease $XX million million from at raised plans. the stock the million. decrease $XXX $XX.X of in equity to proceeds of and market December as approximately Rewrite compared of from agreement XXXX cash cash XX, employee-based September part $XXX $XX.X offset by acquisition The well as proceeds The driven approximately by million of the operations billion of fund as million to XX, $X.X net marketable in in and was company's

XXXX, market to period XX, ended cash Subsequent increase raising an we October to our net continue by at through million $XXX equity proceeds in QX, agreement. position our the additional from the

the to Our increase third increased third quarter to $X.X during by by collaboration compared XXXX. AvenCell during and The XXXX of Kyverna. $X.X the with our collaborations of was million quarter $XX.X driven million revenue million

XXXX. of of of quarter $XX.X $XX.X programs growth Our third advancements lead by and during to R&D quarter by the support million driven increase third expenses to million the million increased programs. these XXXX compared to was mainly during $XX.X the personnel our This

to by XXXX. increase $X.X million stock-based XXXX of quarter the during increased compensation third million was compared of the during $XX.X expenses related including $X.X the of $XX.X primarily third employee-related G&A This Our million expenses, to million. to quarter

to expect beyond plans XX operating we Finally, balance our fund next months. cash the

I that, to With remarks. over back now will John turn for closing the call

Thank you, Glenn.

see, and this quarter can we the aim major milestones Intellia for marked to As serve. you patients

expansion manifestation. this from set regardless of portion trial. the of interim cardiomyopathy we a completing with life standard closer for study as the move a engaging pivotal results to the arm underscore study For new rapidly ATTR We're including disease, the regulatory US dose with NTLA-XXXX the patients in potential of the amyloidosis. of and treating to agencies, threatening

NTLA-XXXX's by HAE of debilitating treatment. the initiate study single data first preventing potential burden For HAE, current dose preliminary permanently We address associated the Phase of to we portion in believe half to the X swelling the speak following year. treatment a attacks the these next to plan with

vivo spectrum of in second both presentations we're by data the sharing for the lead revolution for the advancing NTLA-XXXX clinical do. proof editing in next the systemic of platform early weeks XXXX concept additional CRISPR can continuing expanding year. to and forward priorities the genome what editing, gene full NTLA-XXXX in strategic hand our coming We our and in from pipeline look to With horizons

Operator, that, to answer any questions. open be may the you now Q&A. With for we'd call happy

[Operator Instructions].

Harrison question The comes from with Greg first of America. Bank

Mary Greg. is on This Kate for

into cohort XX at subsequent your pivotal milligrams. So you dose moving for program, escalation decisions studies? additional selection you Can mentioned dose priorities for for an the discuss XXXX

think dose we how to speak about to want selection? you do David,

As with What information treat the reduction eventually to we the the in to milligram going pivotal the the closely dose. see. study, more of in patients our pharmacodynamic gave did same TTR, announce at on same aligned time, and are XX is at we we X.X think dose saw the we're and efficacy endpoint we and X.X doses which we're main very that we is what really milligram cardiomyopathy to designing started that collecting patients

In two those looking the safety you terms of that would, a into always dose, chose X.X, the doses, as dose. in translated at getting XX we best lower and milligram

Phase Now, that Phase is this good. do everything part that is X now confirming to likely a be Phase But is X dose. in And the X. still anticipate we looking This dose. quite it's

you that. we learning Phase keep things Of X, in and to have course, confirm do

The Joseph Company. Cowen with next Thome comes and question from

any In patients start versus has I for as of here terms seeing field on an prophylaxis is what guess, the a attack of term Obviously, we bar prophylaxis? if sort what rates, represents and sort in But significant follow-up, the is for result. after breakthroughs? everyone XX prior response that to HAE weeks zero advancement are taking longer of amazing anyone the

more one think David, what for How to shooting you we're attack we'll rates. turn respect do to time. with about you

work treatments. prior we're therapies getting patients of attacks, – them it's breakthrough group. to seeing would what are more getting this There likely prophylaxis, And most their that advanced It's extensive the as prior obviously, be prophylactic on in having isn't isn't attacks. we patients to move the not as with that so studies,

often However, reduction achieve about XX%. as therapies with XX% the that's standard for everybody, not overall. working But see, – you a the with can breakthrough, these patients current that even

we see it's XX% a of is – starting down. dose. a get getting now, with pre-kallikrein significant we advance first what So pharmacodynamic to will think endpoint can Right with milligram to the XX getting we be

can that that levels So to we get other we therapies. been know have achieved lower with

and and using We'll looking doses, at decide the safety two to X. picture the Phase picture the rates be in best attack what is at X, both in the whole Phase obviously, that looking dose, looking at

when possible occurs in advances what that to is zero if patients get significant trying the after weeks do as did or think to that all treatment. attacks, get-go patients We XX as the get one that or right the to they first patient, we're occurs as get many from it whether

to thought one add that. to Just

One would Obviously, is is of just additional burden the of think care. the mind primary of very to one be the disease. patients that to up things that we freeing the things I that's be think rates. not But manifestation of borne needs brings significant in attack value a really what it's

One learned in involved very to need the of that barrier and a many success. the things talking patients is to their take themselves we've is treatment KOLs patients relative that to

And very the going about, very to I think rates, taking able excited deep really those up a will very, so, any think in actually to from constitute which all, achieving a space. these in we're patients at we're addition free advance to be may position where which we therapy be attack in we many significant of in reductions

question The Morgan. Cheng from J.P. next Brian comes with

ATTR like HAE Phase seems in you on the US. the Any and from for in stand confident FDA will It's great can start your sites pivotal you're lining if with things of elaborate up progress. pretty super you where to It at be interaction the We're to for in US. terms trials with the to start helpful. X see the wondering insights

speak want to that? David, to do Again, you

had interactions around both programs. with We have FDA the

do of idea a what they for looking in are feel good very we the IND. So, we have

X Phase we've HAE, be happen for half, X that through we going going that stated in and trials. As first part already Phase that's said those are of will US the to the

study a what Again, Phase case, We a guidance. want. in those imagine. giving case, they you going on more so X is and We'll idea study, we're more that of as that good clearly can more for had advanced that likely TTR. X, haven't application have given be Phase a it a the to guidance extensive interactions, In we've we

with agreement. have we'll good out And us But we to about with prematurely to design is. don't and we what our top supporting feel want it a be We talking that more. want We consulting design do with do get approach. experts, very come

question comes Credit next with from Suisse. Richard Law The

Phase the what on the collect in is so of pivotal? that Based X to data study? there positive need you data before pivotal could would a HAE, to not, posting if become far type any possibility And

go we here David, again.

of a study XX three small including X now. we where is X in Phase The XX total patients have different right patients, It's a patients doses placebo.

small believe to do pivotal a So, that we too do is study.

or total that you an much than less get about be are this of that. needed not we'll study, pivotal the For approval larger in to Often have disease. a patients XXX even

pivotal the future we will So, X be Phase we obviously using but design that BLA, same data the a to as to support will study well. support

from The Sandler. with next Piper question comes what Swapnil Malekar

Two at experience AHA question. expect US? first-in-human then the collected of like, the there the now program initiate And and upcoming data can a data at AATD a is we lot additional what and trial One is meeting you that part weekend. is the in on XXXX, to for the XXXX possibility part have second

can of we information going too literally presented days. that's in couple I'm be to just to? sure far David, want not a do run of you ahead

encourage lies do to little to more speak the would I other But about for the some bit a you get ahead to of wait programs? want picture. what So fuller you

What think, we world, to will cardiologist in front is the we going is important, of setting be in the I AHA guess. largest

as cardiologists of have data. hasn't is high know, gave where some we abstract. you very the does an important It been reflect we in it interest the So, It program. this of late setting breaking since our initial long,

extend So bit this of extended presentation will a in data. we that

the the the US in plan part to always of antitrypsin. alpha-X been as have It's

in CTA said IND we or of will the going to haven't or What the half second a We we it's said have have both. is year, start. where exactly

our XXXX, program. knock-in first For that's

excited that. very about So

so, in And is that XXXX.

guidance XXXX, yet haven't which of We knockout mutants, given is on the the alpha-X.

even So, don't the in behind did that more for we as or plan meaning But behind very give take to far new it's the that some year. it. other one guidance that

Wang Gena question The Barclays. with comes from next

and Any is We would primates This morning. on LFT were in could Hi, remind us preclinical for I here you saw both models, color non-human good elevations guess XXXX appreciated. any if XXXX. be you for Rashida wondering, specifically Gena. [ph]

I class That quickly. cetera. et toxicity, an of to actual as think companies, is every And to that to single LNP, industry tested lead work, part to therapeutic in pretty by that across we index is determine given quantities, sufficient effect which literally ever one a as in regulation FDA as that sure speak everybody know, increases. the I been that's aware you we're well. routinely the across getting of LNP I'm Any will of, can seen else LFP field different from us that's the preclinical as levels follow goes

yes, increases, is thus The in little program that can can index characterize successfully to have which to LNPs far. so you no goal that's with to see does, So, LFP the wide, patients the therapeutic elevations. administer a you fact, clinical

The Goldman next question Salveen comes Sachs. from with Richter

beyond Congrats This cell you in versus How on the making for HAE [ph] further are thinking in on decision is about ex programs? TTR the pursuing programs therapy and vivo progress. programs Tomi vivo Salveen. and vivo more for

The work, the to about think we get considering benefit way the very we need, say, to And think think nature we general, set all unmet apply that deliberate try risk. we a we about of about to information filters of we do it programs add our we think we about particular fashion this edit, the medical where clinic. in in as of advance, in let's

clinical sort and far these of look target edit approach could of to relatively So us of manage genetic lead that biochemical the has served out be thus knocking were we that well. effects where clinical would TTR forward in HAE, straightforward that results a formats. standard straight trial monitor to well-established would at if And and examples we very clear

ahead, As on. think work just a we that that non-functional. shared the a we done move gene And may thoughts modalities, actual so That our insertions. gene is This alpha-X is we've on that or additional which look program few we reconstituting far. to otherwise deficient builds about speaks to to be

there's we there and about a can think fashion? apply, prevalent be therapeutics rare conditions. also do can conditions little same as will we've out, those made. on. that some need? be more for more we And laid year working can Do therapeutic beginning a advances we'll this ideally targets that reset be that We or But in either believe medical of that the talk the the into we guidance bit move sorts at reset as the is the this of competitive will rules several a bar? the vivo into additional to been about advance there meaningful space that on more we've so, speak we and time disease goes And in that we've when in we way of And patients unmet some

where in from vivo to is work Our far take able we're in it. and targets done we're excited about

from The Ha Stifel. Gon next comes with Dae question

you know the two dose ask the XXXX, in we the where full X you have very is a give probably goal milligram being doses the relatively you there first XX given the profound about follow-up the half fixed TTR, milligram of cohort The progress to where but expansion. safety XXXX less proximity what a Phase obviously picture ACAAI, to the to the the side start as on on were didn't timeline. short some But placebo quite follow-up of term in arm. here Wanted effects, and explored with about given XX we'll

Hope doses of think the you being Phase end the you before do of confident year how sort about forward around get makes that So, going in sense. X? the deciding and what

think, that term, on dose a instances two that. or first aspect can aspects seen us with I four everything for great with we foundation safety the about think of within that in of taken quite respect within to deal I question, particularly Virtually set in rare One short days of which first ultimately I to weeks. that to target, is your any would place what respect can learn safety is not the has the think we confidence safety. And either gives pursue. the the we've

from, a first TTR us to see to weeks. much very respect aspects, us clinical settle out and extrapolate can both a follow-up, able With in case agents studies, quickly, we sense of for knockdown up of advantage have to patients HAE out which very, the in have four the few guideposts And we that we we're the out. There's the did really as this just these exactly weeks within added where XX other pretty good these give settle where activity there the things knockdown in the case within been of point. seeing to of the

we're team meet So well is to a good moving way its we David's on guidance. forward. think that to in position that And

we as you tuned stay more details. So give

next comes question Terence Stanley. from Morgan The with Flynn

regulators. XXXX, the any was On that's additional like know in have wondering you're with I thoughts share the guys on more you conversations alluded I to if something arm? can past. you But potential control having sounds

So any just can wonder color if you there. share

the David that so as to want are in clinical That that's speaks be address set respect a We the in, available to them. doctors currently. study And regulatory progress earlier. and to requirements. with to agents will of who going patients that to Well, treat work the meaningful have referred

would And I that paradigms out very to good that think the terms look to in want to already have there's approach. add extensive? anything for David, them line But, of guidance general the you I bottom is there. you do

relative Yeah, of main will being we're I which this to very drug area. levels we make tested difference the we some get think of the about, deeper is can other drugs are that of in points. are in that TTR think a the terms to how encouraged reduction, effective is those of What course,

those very in in we are endpoints, important our our cardiovascular trials. what thinking So, the in John design most mortality, events, will pivotal we're reduction But important was saying, of study. that's also be as the those benefits, reduction and as the part clinical

more you'll come we that. forward, about As hear

with from comes Jefferies. Raycroft question next Maury The

point, do on based where type what X know Phase be it four attack Could HAE a you on X the on of based this it duration of help can narrow amount far, patient Based see the or experience month so at including will three to enroll into And study Phase study the want you portion, severity? time you the does a the baseline be? efficacy?

Well, patients, of just to with will of to could drug type set bring applicable. that objective, generally anybody would as develop certainly, be for who approach respect virtually something the HAE. we the these some possible. be believe that's think, expectations from And that X the our treatment patients to therapeutically relevant we be suffers set type We X to the broadest ultimately, or patients,

with David, you thoughts to the of study? Phase respect the any But, want duration to X do share

The period. in week quickly The we the follow-up should is XX Phase on XX-week for get once that, to as go these you the X standard know, answer studies and studies underway. fairly based is quickly our

Olson with Jay question from comes Oppenheimer. next The

on and balance allocation talk $XXX and about in risk? prioritize of to and how about your order any your million you runway partnering have commercialization cash cash the shared you development sheet Can of you extend thoughts

we a It's the design doing try various the only our that to in sure counting are as for to company. deliver about best something who a balancing act how priorities that we we talk a reflected that we very return. shareholders Not are across meaningful much guidance, because think can about. It's but we our that think us to about we're make want we lot

the think platform as both not full proficient, ourselves because this we is opportunities spectrum we a that. characterize But all want in evolving you vivo significant editing critically rapidly. and leaders in a there's As be very is that ex of important And but vivo to know, that's only the in we aspects genome company space.

always ability on any genome do think here. that's we space. is we So unsurpassed focus to that And and will company editing the sure by in making far, thus the of platform operating case any certainly type our

advancing clinical primary Clearly, importance for of is the us. programs

do work on side. platform validates in and XXXX the we the work XXXX Our that

important the So, of but that we that, forward said, to work, bar are about that programs it's as clear think available programs patients. us very for will to these move nature we the give the signals our reset also therapeutics

We're you think broadly comments that's wholly think serve we've and also here, space. the which basis those. very thoughtful applicable We the not Laura's about industry. to important in the two that ex ourselves, of programs established AvenCell across significantly own think but platform only about a allogeneic work heard advances We've to as we Kyverna. is, about vivo And across of we that done what again, how the sort for we already capability space, collaborations referred we will the

that those As we advance with think do that think, able about provide advances or I've as perspective. can the bring it we we're the us genome referred able things to about we that But partnering, from what have, look to technology biology partnerships, we're it's because tectonic we of company to think of less funding balance them to already the cash the we to. in terms editing that a of

all approach. we've on, look we'll of time partnerships done far, up. that to And how exactly are those would that, goes set take of kind And I thus if that across you that's as basically continue expect them

but So company, as top It's resources to out on it is. there have we our at that going think what a ample the carry priorities. we've lot we out set

question comes with Issi next Luca The RBC. from

for ALT you TTR occurred, think, Congrats the that XX. day Any progress. a on polyneuropathy I on Grade reported for Maybe the David. in one etiology X previously update quick all the of

of a you hypothesis being we including explored, time alcohol number were viral in last mentioned think that I or nature. maybe spoke,

ALT wondering or not was if drug just at whether So this was point, suggest we evidence enough to related. have that

to outlier be patient do appears unusual that, David, to want who [indiscernible] you much so speak that to far. an very

anything of terms in new We don't viruses. have

We the haven't identified virus.

about it we for. been usage. So, alcohol virus is, it's don't we able know test if more to haven't a And

great, John as very doing the Patient said, others. TTR has is, outlier. perfect like by a the is way. The it So an it's reduction, good reduction

So point. don't come much expect that's more obviously, at in and to we are, where this we

Lee comes the And Joon question today last with from Truist.

for alpha-X the for therapy with Any alpha-X start antitrypsin being There's and the else a mutant that deliver. gets you micromoles on of normal produced caught metric when something that trial? there gets plasma failed the polymerization XX by the success AAT the AAT copy gene that. And – do view out up because thoughts or

protein going you. form? producing presence we to do Laura, I'm versus therapy. on the How about differentiate of important mutant in I think ourselves call people normal And you standard the think how gene remind it's to

inserts out any be two have in allows candidates. separately XXXX a mutant or combination, used We copy. those knock the XXXX order. can form. And two to us healthy And in

other thing that keep Z right, manifestation. who to manifestation the mind no lung no heterozygous are is So, and liver in have they allele, patients

expression and – the may heterozygous just is phenotype. enough, be able if allele your right So, you phenocopy using high – conditional language, normal of to may

So, that's mind. in keep to

goal And normal yes, of alpha-X achieve is our antitrypsin. levels to

XX would not levels. So would it be micromolar. It normal be

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

updating coming Great. Thanks our We support months day, progress you so a to look weeks interest all your us on Intellia. and much, Andrew. forward and great and And in you continued Have today everyone. thank the for joining in for ahead.

for you today's The conference attending concluded. Thank presentation. now has

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