Intellia Therapeutics (NTLA) 4 May 232023 Q1 Earnings call transcript

Good morning and welcome to the Intellia Therapeutics' First Quarter 2023 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.

As a reminder, all participants are currently in a listen-only mode.

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

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

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

Sepp-Lorenzino, will Chief business an on Intellia Medical are Chief Executive up David available. Dr. XXXX an Laura before for Chief Scientific which Officer; the Dr. begin Financial at of be Leonard, provide John John review our we Officer also Lebwohl, call with and our clinical the overview recent programs Glenn Dr. time from open Goddard, results will financial first will will and David Officer. quarter update Glenn Officer; me for Q&A Chief highlights. Joining

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

first are a an novel this deepest continuing the platform. pipeline us vivo both editing we for regulatory IND editing important broadest to of and delivery we in our ex had and productive to CRISPR-based and therapy. to Intellia, solutions. clearance and vivo that and revolution you, thank genome Ian, Thank all continue At with and joining start across you our the recent investigational in accomplishment, Alongside lead toolbox vivo for We the the make to year successful progress morning.

announce to of Phase pleased angioedema of the we're has treatment or the NTLA-XXXX HAE. dosing in hereditary begun study Today X for

based and the IND in acceptance patients, our introducing complete of investigators clinical second enrollment this to step FDA's the study the one functional for Additionally, care living Both move rapid to application NTLA-XXXX half of of closer interest strong we with year. progression and on from HAE. expect a perpetual goal us people our of

people pivotal submitting working This of NTLA-XXXX vivo our for manifestation Simultaneously, of ATTR IND amyloidosis. trial in we're with IND. the next through second cardiomyopathy planned towards is

estimates pivotal global suffer many Some disease indicate this XXX,XXX who trial end. expect as to year by initiate Subject there around regulatory may as the to be world. people we feedback from a

the presented supported shift NTLA-XXXX Both by NTLA-XXXX paradigm that's last treatments represent and groundbreaking in clinical data year. potentially initial

As and Phase year. presenting meetings company date, to demonstrate we systemic each the study medical now to from only editing successful humans later forward interim first data to new upcoming in this CRISPR at look X gene

Laboratories. Committee from record over strong Chair CFO three AbbVie leading Chase very of and of management its Board Audit He of a from decades welcome the as and happy to Bill brings the spearheaded Lastly, we're formerly for Caroline spinout our companies. financial Bill Board. was will retirement track to following successful expertise Directors. biopharmaceutical succeed Dorsa Abbott building Bill her

success growth for this Independent in dedicated like to her Caroline and opportunity long her Director. to as We Dorsa Board her her company's future on service and also many would the wish first its take to well thank I contributions and Intellia's pursuits.

detail. over I'll will to the Lebwohl, Medical review lead greater programs so far call made we've hand year, progress who clinical excellent the Officer, in the of summary Chief that our With this David David?

update an with for single John, candidate Thanks with amyloidosis disease ATTR and people the in dose. reverse to after XXXX, I'll vivo and in CRISPR with living halt based an everyone. a potential welcome begin

the shared the dose expansion part Data have polyneuropathy back of the we arms dose will and in cohorts inform As February, pivotal for we study. cardiomyopathy two used planned subsequent enrollment Phase studies. now X the of to our from portion selection expansion decision completed these be in

ATTR-CM For mid we IND an track year. to remain on application submit this

and end. of package. currently IND are preparing XXXX robust the extensive are We together initiate preclinical in data. the including to putting plan a to regulatory Subject global we study feedback by to work the year Similar available the clinical filing, we process pivotal

and for XXXX are in a ATTR preparing study. study amyloidosis regulatory portion patients mg/kg re-dosing the polyneuropathy, the who hereditary received authorities. also recently X.X of X future escalation with X dose began Phase Phase with of discussions dose For we the we Separately,

As the first first relatively you the in patients received may human study recall, three dose. low in a

XXXX low in mg/kg. XX% or the was a the lower cohort testing in XX. patients dose this will fixed on the profile. comparison of of day to XX greater achieved for follow dose in who subsequent dose mean TTR who is dose reduction mg this primarily safety receive TTR objective in to dose, elect escalation reduction achieved corresponding activity to than and near of the Our by cohorts. receive bounds first Patients the X.X a to This examine XX% Patients cohort patients

ability as based LNP our XXXX that are potentially delivery based treatment, an to advantage we our is developing candidates a the of believe system. with one-time re-dose important we CRISPR While

for feature this validation including re-dosing therapeutic our other such, clinical applications, As those platform liver. valuable in outside the provide may

this arms plan of we study year. data from Finally, to present both additional the

to For include ATTR-CM, well expect endpoints. as clinical longer as data, emerging durability and term safety we

activity second with to HAE. kallikrein plasma the burden we angioedema just for reduction Despite treatments, experienced program with of by in attacks. availability single With now many people investigational XXXX, dose. prevent our treatment a attacks therapy aim XXXX, hereditary and or chronic lifelong turn HAE vivo treatment our high I'll of face the breakthrough to still of providing living

cleared This the period was application. the Intellia. initial work IND We were in the extraordinary our here the very vivo at XXXX team the March to first clearance FDA's the submission pleased from speaks and that to announce IND XX-day FDA in within review

this the the vivo IND time cleared based using in CRISPR delivery. a an Importantly, candidate has marks FDA administered systemically LNP first for

to data indicates Shortly expedite after need HAE. treatment designation for unmet HAE. XXXX The development early and and FDA we RMAT designation review. received includes clearance, also for FDA serious for the and D IN promising to RMAT medical of the people candidates address our therapeutic living It certain is XXXX for important recognition by that potential has to clinical benefits confers with

More provide dosing durable this patients of in a early following year. Today have doesn't We've after study. announced and Phase The begun significant alike swelling the attacks recently, the we progress we've to XXXX suggesting global seen second made enthusiasm strong clinical program there. our we and half with the investigators XXXX patients from stop X interest single in prevention enrollment may we that of dose. data complete expect

to the to trial. future a the Phase into the dose is X forward move reminder, registrational a As of identify goal

safety, the We look year. Phase to new expected the measures from three forward cohorts. study include of to all presented rate presenting be Data additional and first-in-human attack durability, new from X this later data portion

first productive very a quarter, especially XXXX been it's mentioned, John for program. the As

to living with we believe to progress, for new XXXX serious we closer XXXX and these diseases. continue moving are people that a setting standard As care of

this programs the of to a program advancing Notably on remain an XXXX, of the lead pipeline second vivo year. towards in our ex two INDA our in and programs, clinic. Beyond half IND in we equivalent submit first insertion wholly we're or owned to vivo track vivo

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

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

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

by $XX.X XXXX quarter Our of $X.X million $XX.X to quarter during to first collaboration during XXXX. of first the increased the compared million revenue million

of million during decrease was by This R&D these Our to driven Rewrite million decrease the quarter the by an $XX.X first programs. XXXX. $XX $XXX.X advancement acquisition Therapeutics during driven million in to growth and This lead of compared by support million XXXX. of personnel million by XXXX offset $XX to decreased increase mainly of expenses the quarter programs first our of first expenses related of to $XX the during the quarter of was expense

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

cash our additional expect to months. our remainder key Finally, of the and balance look fund XX number to beyond the and forward There's of been milestones presentations year. a the start very a we year, productive certainly data next throughout the operating to we plans

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

from The Joseph go Instructions] TD ahead. Cowen. question comes Thome first [Operator with Please

has you. maybe the what and to Good development means you obviously the a you just broadly, bit Do Maybe for impact that an in indication and been you terms little for question. us XXXX? all? my accelerated your U.S. for morning of this more Marks of program Hi gene fits Peter think gene development designation thank Thank And of editing favorable more therapies bit there. this does terms give for little a taking could in ILAP at in strategy approval through

FDA's to in see more David, we outlook U.S.? gene do editing want the to broadly the you and I question ILAP guess as the it for speak

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innovative they're So advance this program in which trying to a is therapies.

in have talked program moving the They've I and about helpful how authorities. gene worked in been closely with we been past you interested UK as think have very we've class very our a forward. to editing in

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positive noted has generally gene our these programs. therapies. more in gaining for In towards have we of do that cases, of we ways very to accelerated using approvals positive talk studies. need at the think general, because certainly study about randomized Marks data been will talked we registration we also We've that But more, Peter towards we response the the are approval them diseases. the nature accelerate these these assessing least about to to the In

helpful. Perfect. Very Thank you.

The next Jefferies. Raycroft Maury ahead. question Please comes go with from

Hi, question. good my for morning, thanks and taking

X ATTR how near with are Alnylam the you file. in the For you to Phase wait cardiomyopathy, have mid-year BridgeBio's file IND adjust But in you the plan and for to I'm inform wondering, key strategy? be? you final events place study likely have and two for midyear thinking those will space about when you a for and IND outcomes are guess this if your need events there timing data. or outcome will filing I

I'll Maury. question. the for Thanks Hi, take that

that to to get going guided and information agreement on need is We we're to think we have the priority the the IND protocol an is in move FDA. U.S. plan the with actually Our this year. of filing us program we've going forward it's be highest that to to for the much mid move to that with as the what forward

move that better sooner So we the on everybody. for the

question. Got for taking Thanks it. my

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

for question. Hi my thanks taking

been So interesting which to high global enrollment Phase made HAE this X that the XX, I study. comment is having has think interest you for

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the question. now broad of interests, for Thanks. Yes, based thanks We're irrespective yes, really geography. seeing

think brings there's we've why obviously points question really the the in We I of is be? are quite been IND developed very The by your to markets. U.S. the really program. speak we've of might that I here seen clinical investigators. guess gratified fundamental the interest program to a from core the that and that couple

for far frequently to for But seen program perhaps one, as factor we've been see they're underappreciated what translates young carry lives. that Number the uncertainty that as regimen, out put and moves when surpasses what And into efficacy and to by contend with the diagnosed has lifelong is think least competing what HAE them, infusions and go a they're there very at equal we'll companies patients that treatment or is they need the that with view it's on. about that whether they one patients with I remember their as they require therapy. that they burdensome daily thus materials

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stay So tuned.

the those and it's patients. goals, how As but alluring see work achieving to we've its goes far learned to certainly we on get we'll

the hear. update. Thanks for to Great

The Stifel. Ha Gon next Please ahead. question go comes Dae with from

may. Thanks taking our I XXXX, I'll morning. to Good question. for switch gears Great. if

requirements regards in experience, XXXX your with be to or that program I IND clearance? considerations talk eventual IND So, looking the as to IND geographies? soon other by certain of standard to of just IND availability will like submission overall you I decision about back filed guess of opposed Thanks much XXXX equivalent be can regulatory confidence the wondering, care in driven I'm so how your guess, and XXXX, this at much. plans, for IND and

want how we're speak work approaching you to do with to clinical David, regulatory the XXXX? to filings begin

the, able normal so XXXX the non-human of like get knock-in recall what levels that to first of antitrypsin, with primates. we're would Yes, looked of

this we I this So to normal motivates enzyme. we able of start lot what's there. also that's this a dose, test use would say, together, single have that to of able should be enthusiasm IND regulators, because with that people and to in achieve levels potential that therapy of be Putting seen important from the to investigators to the therapy the so

albumin able And of the of gene are are terms we XXXX things lipid that really two doing editing. at target the FDA In One in, aspect pieces, that we've is IND, pieces what the to the interested major nano-particle from is this. the the in there is site. that learned the application of

but well FDA The new is second forward us, new And is virus to program. all. very piece of AAV is that part of established. the That at for to that's way it this INDs not the

terms don't chance that putting a will has IND So, the We IND hurdle together, the be, two good a think or equivalent of passing. think we that's going in forward. major of

with Chattopadhyay comes next Guggenheim. ahead. Debjit Please from question The go

share data you dose for were anticipate data just later think a secondly, said equivalent information with NT-proBNP about X.X you'll that that around everyone. mg/kgs. morning, set? on year, clarification I is one And XXXX that providing this for Good those Thinking patients Debjit. This [indiscernible] to you re-dosed, the fixed do

think said I that. but dose, you flat I the also missed

you the the biomarkers. that, to information we're re-dosing the not share going the of that XX David, this some the can has specifically expect weight-based. receiving following program. some What guess the clinical some dose cohort everyone for to maybe the we expansion was the David, same flat say of is about I we phase up year? XXXX of yes you share. dose. meanwhile, been clinical speak can we the be information the speak these In It's to doing expect with is that to in to first do want that patients else in with question

the the information polyneuropathy we're this and and from going year. to the What enrollment cardiomyopathy get, early that show think completed What Yes. year cardiomyopathy last from is you to I the we is at trial the of polyneuropathy we both end arms. know,

have have we up patients will had those some year. the So as had, through follow move

be be, but it for late We be, haven't some in exactly that period. will will will that who patients started presentation that when said early

were Of course, longer of one in part on the the who the patients in information have trial. we the trial

In quickly come results. TTR and general, we the Those very present trial. will safety in

other the will come various up we we follow will presenting available we're but degrees general, report proBNP to we longer. patients. patients. that that the do like cardio course, the so substantial So in be all want now, say varying data be when But can't of MRI take there'll the do follow complete with forward we measures different really right In up for and important and information, And of what exactly find. of there's heart information with

Thank you.

The ahead. comes next ISI. question Liisa Evercore with Bayko Please go from

really for you looking question. specifically is it for, this if taking it's you re-dosing for you're you, are Thanks a know program about other if strategy. there. you're I Hi safety is programs? your there's curious anything the was particular for, what evaluation what beyond doing Are Thank will looking more or How you, there safety? for expected, anything

David, that? speak do want you to to

with -- the XX% appropriate to at in XX% re-dosing a so the in we is we've the in elimination results either we think in events. dose for we that complete seen the or general that HAE And think general. the is doses TTR biomarker what for of program. reduction can the TTR achieve that XX% the program of single reduction doses the higher want don't needed HAE, greater the We than Yes,

think don't on be know. Those we everything permanent based So need effects we they should re-dosing.

benefit the first who people world in of CRISPR really The joined patients as done therapy. vivo the joined re-dosing trial, patients a receive in being our based that those few to particularly for being who the done is is

to had get effect that to, of that we would them wanted, them we re-dose. that a we they if they, committed So through didn't we wanted them type offer

them is the primates. that we had first feasible in for very went do patients to dose that editing that trial doses. situation mg, We be the XX result of these we and good by preclinical if we in nonhuman to in the other proved type patients, the at achieve giving think full have this this we'll the able would be But to higher achieve able

-- you re-dose found haven't haven't indications? not yet. type to did But We in will that good you it's an useful that give But titration where necessary may a perhaps it's that if some especially we're we're this, to where the could be that is of safe dose. but thing. be -- to want mention drug this imagine liver We yet, that someplace other indication outside again, found know could it be whether this additional and in interested the to might give we

We you've the seen haven't seen of far. liver that, as course, so in

you. Thank sense. makes Okay,

comes with next The Pickering Please from question go William Bernstein. ahead.

morning. at what stated into share my in Could translating Good duration? for be conference cardiomyopathy could gives David, Thank pivotal that do seen. an you. that how smaller in that you question. Thank your than more trial taking you see have we about confidence you investor you expectation others March, and the you that study

we're that first translate been better types indicating into that seeing what the of I TTR, seen been neuropathy in reductions so efficacy. see has that already seen These we we the for Yes, deeper expect that's reductions correlation was to That's could deeper in cardiomyopathy. and other is efficacy. have better because fact

statistically a that you have to significant. do to achieve a don't positive endpoint endpoint people you treatment as a When is establish many effect, greater primary need

are patients. from very we example, HELIOS-B, course [ph] for be now at do XXXX trials other but there's the Ionis like will trial not of So that think different the

effect to small effect that could You'd as you a go very have treatment very small size see a when a such that. trial you treatment

So that's, I was what talking about.

Thank you.

Joon comes Truist Securities. The next ahead. from Lee question Please go with

This for Hi, [indiscernible] our good on morning and questions. thanks for Joon. taking is mainly

capable ex and of like you very multiplexing, So safe efficient. editing that based is showed vivo

and that's case, this there if the the in expect for are you notable in vivo domain? Thank would editing you base next efficacy And So or choose candidate you. do in if any same considerations would your similar vivo?

question. the for thanks Yes,

I think the here tools been to disease to we platform have approach aggregating where that our just specific think we we any a should provide how state. think basics the capabilities us go necessary Remember, we've gene particular to back and through address go editing programs. after we

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much. very you Thank

Please Richter The Goldman ahead. next with Salveen question comes go Sachs. from

This and question. you're thinking you regulators what Can design FDA on from study Shrinatra provide you about some Hi, study? like learned pivotal Salveen. is that the the Phase for thank my a to see do X front, have the what taking ATTR-CM? discussions on And for you [ph] on for with think in how would trial you in color your

David, how cardiomyopathy? are X about thinking for program you Phase the

important what's We can you that thank think the that the for trial, benefit we about question. you payers, achieve. important the of firstly endpoints and to the is think what's and to to clinical doctors the Yes, think patients, primary that

events, cardiovascular cardiovascular reducing mortality. reducing is this So

what So this can foremost, patients. for first do and

trials do that terms trial, of we to are think design In out the of the it will the other be there. similar

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So is want what currently we out to there. benefit a see over what's

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those are to. looking main the we that So features are

on gain later this agreement the to then starting forward and will that regulators We trial with go year.

Thank you.

go comes ahead. JPMorgan. question with Please from next The Cheng Brian

question my taking morning. thanks Hey guys, for this

that Thank more In those disease see X would exclude Given words, lower hanging the clinical associated variant you XXXX phenotype, that the for you. in [ph]? you for of also other fruit have and your specific is AATD a mix XXXX? study there Phase planned in genetic of presentation liver of basket a heterogeneity

the for you do how David, of to talk we're about about program X the want XXXX? thinking just Phase basics

varieties and patients right both. some you're of younger there, have of the the really loss patients issue, predominant a dysfunction, some patients, say, of function lung very there some particularly different lung. antitrypsin and so predominant have have a that liver are Alpha-X Yes, in patients Other

put the a X not Phase would when you're treating in patients think disease into because it avoid new we in gene. study, you have who general liver Going we

rid You're gene in the not getting initial of mutant the program.

this the of -- the XXXX, liver initially, for who disease which our predominantly out can manifestation their course, disease also as patients Similarly, well. allele, be knocks those second program patients for would get as lung the have main later on Z

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David. Thanks,

The next Rick ahead. Fitzgerald. Cantor question comes Bienkowski Please with go from

morning. Thanks good for the question. taking Hi,

given the safety the for from that cohort, program X space dose Phase could will the for you more the help you given of that U.S.? is be of required U.S., Will data to of here? So or dosing just time out XXXX, of the in each Intellia generated X with was the you dosing the the this U.S. dosing initial the outside rapidly for first the patients the Phase patients could be in patient us expectation

and discussed in very actively outside I'll earlier study. program the high take that. in What we're seeing is, U.S. the we the We're interest here enrolling in U.S. the the as call,

finish we we program. patients, X think typically a with space to this that's expect that year. go will the pacing And We in is of without to very, Phase done going respect the very to as fast requirement

study to and expect quite this program we'll So unfolds. as overall move the would quickly I update

on Great. the Congrats progress all here.

next with from The Harrison of ahead. go Bank Please comes Greg America. question

our for [ph] question. Kate is Greg. taking Thanks on Mary for This morning. Good

could you conferences, how at abstracts what Thanks. and are product of upcoming with at indication? looking this medical Regeneron hemophilia have have a some in at early impact Just ASGCT. this multiple looking the collaboration Maybe you for gene stage editing program program in the preclinical

You're asking about hemophilia? specifically

Yes.

Yes, way of aspects we a the I'll that there's take couple is, that. to about think program. The

lifelong it's with deal manifestations the as disease a they starts and the of of that hemophilia, unfold. they disease as children First morbidity obviously, all, lives in accumulate their

ultimately Our population. the approach curative pediatric a to able objective is get be to to for

that establish here, the progression would think of programs able want is of are couple we at as do got that. out we're that gene studies So We've insertion. looking we what about to carry a to

to as as And addresses getting se. available we're the toll. discussed are to for would factor at collaborators its really as Alpha-X able be adult David think reasonable best before our into antitrypsin, that Regeneron. XXXX potentially ability just therapies dose quickly that we ultimate are, achieve get the takes per suited are objectives The the what the of single are with applications objective pediatric X And if the population. population there our today with disease to that kids that

think And an grow as with with patient develops. that case, that's so or he sorry, advantage gene stable the pediatric can a this that she is the or patient, integrated important there that comes he in having we

from question with next Luca The Please RBC. Issi ahead. go comes

Hi, Luca. broad-based irrespective question. Rena for interest commented wanted of on just I to HAE, for Thanks ask been taking geography on my this regarding you enrollment. earlier there's is that

the So U.S.? trial add patient proportion was are a this just on Thank what I or based dosed? in more could ex U.S. of if color you. trial the -U.S. you at And sites wondering the site? first Was

correct that are You we've begun dosing.

talk is individual What that we're do about individual broad-based not correct You patients. going there's interest. to sites and are

and tuned. aggregate, updates in information we'll and that provide stay doing that, Our we approach done to all date, is to with the as programs we've continue so collect

Okay. Thanks for taking my question.

Gena next comes from go with Wang The ahead. Please Barclays. question

a is one. quick questions. for Rashida you for [ph] taking Hi, We on good Gena. had Thank our morning. This

gene for your for components to how with IND program, you'll in half nearing that both you're get XXXX, you're it and this to wanted your AAV comes just given on equivalent insertion? we second XXXX, when you. dealing of dose thoughts selection insertion thinking Thank about So program, LNP IND or the be as

I that need that. can a LNP mean, couple there's the as be I things as both as say, principles the of you well AAV. address are, to assessed, the

So our dose number lot Phase pre-clinical a reduce greatest the we've extent program is X thus to the XXXX objective those variables. in with to program addressing And of and far. Obviously, to possible. about LNPs would variables learned be the XXXX a how

we but have haven't probably guess program, share a finalized on how of understanding point, side details. good that this the this think we are And that equation, there's behave. would to obviously My more yet, explore finalize we we be likely pretty at those the to as more we'll and AAV so of

Great. Thank you much. so

from with comes The next Raymond question Seedhouse Please ahead. James. Steve go

just sounds program, in seems and knockdown the at do Thank theory and liver. the the both mutated it insertion you SERPINAX morning. therapeutics you. In to approach locus, lung and the that you it correct addresses into one combine to the for have gene Good want optimal ultimately AATD like

And gene hurdles just I'm here So prevent what a are would the correction correction making limitations are of specific single Thanks. AATD in curious, apply they mutation? those the technical that to for or therapeutic general?

out the Well, One has the we've knocking is call. is way approached it referenced been looking the to two deficiency at of antitrypsin liver primary diseases the as earlier manifestations gene the problems. Alpha-X you on causes

circulating The antitrypsin. other a normal is of providing one to levels basis replace essentially Alpha-X

in we've our With able the the been complete that done achieve can and as to two point is see normal you you that rightly we've knockout. out, circulating preclinical essentially essentially protein of work which on work levels relies what approaches,

far that to have offers thus patients what normality. looked approximates accomplish able with have opportunity think we many we're So something program what we've an and to that something we this at for

as in in an possible solely correct many appealing program, system. patients some a gene in our addressing achieve with the single what's achieved, as potentially maneuver LMP own using road one And maneuver based can about for a say But think it antitrypsin we we currently imagine which down at of technologies it over if can may steps It's simultaneously. we've do this see we work progression starting our both all what already that one as in to point, Alpha-X you I with think be today. is advance writing available very a, both

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next go with question comes from Tuerkcan The Securities. Silvan Please ahead. JMP

and morning, Good thanks question. taking for my

any can there one following the results achieving trial? Phase U.S. your is of specific HAE engage number of versus a goal that trial, so ex-U.S., regulators you. patients specific percentage a Thank with you or quick on Just of initiated first that X

we at dose to can into something the We're studies quickly the do I'd is we've think registrational what committed in at right said we not that for step. But to now that objective our patients answer, as very, No, to in when U.S. right proportion interested get we absolutely is be we can many the having the this will we that participating to the any year. believe dose we got move trial. and of by this, us. for the so next this say program -- very patients is in pre-specified so have a many, we absolutely getting outset, priority particular Right patients geography. primary

Thank you.

from The Richard Credit question comes ahead. Please next with Law go Suisse.

morning, my for Good question. and taking thanks

timing ATTR-CM for that then then doing that's before on the possibility case, be right completion? mentioned an would run thinking analysis? show positive analysis if Thanks, your and there's the just possibility in to a you early of outcomes to and of not and And is based So a a [ph] preliminary file there now, that charge BLA CP XXXX a what possibly an interim interim

were might the interim what information? and with steps thinking you what do David, you about

Yes, question, the elsewhere. so interest in new to for approvals it back therapies the important and goes in greater U.S. accelerated

positive place analysis know, at have be study. So if you we, in a a what -- But on have as do there exactly would, an we based purpose point, But the of we but result? to for could learn BLA. trials come interim at achieving details that's an other an the say potential be analysis be can't the that we it, whether Would interim this would it about We'll they earlier valuable this it more is to of see would point. forward. people's the timing be we a again this

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

Drew. on but now. day great you wish to joining thank we'll today. then for a and us you much We we you keep everyone look all continuing so our Bye progress, Thanks until forward informed And to Great. talk again soon.

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

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