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NTLA Intellia Therapeutics

Participants
Lina Li Associate Director, IR
John Leonard President and Chief Executive Officer
Laura Sepp-Lorenzino Executive Vice President and Chief Scientific Officer
Glenn Goddard Executive Vice President, Chief Financial Officer
David Lebwohl Executive Vice President and Chief Medical Officer
Salveen Richter Goldman Sachs
Maury Raycroft Jefferies
Madhu Kumar Robert W. Baird & Co.
Gena Wang Barclays
Joon Lee Truist
Steven Seedhouse Raymond James
Yanan Zhu Wells Fargo
Call transcript
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Operator

Good morning. My name is Drew, and I will be your conference operator today and welcome to the Intellia Therapeutics Third Quarter 2020 Earnings Conference Call. At this time, all participants are in a listen-only mode.

Following the formal remarks we will open the call up for your questions. Please be advised that this call is being recorded at the company's request. time, Li, like Relations Associate this over turn at At Lina To Director Instructions] I to would it Intellia. [Operator of Investor proceed. Please

Lina Li

you, we press release Intellia's our welcome progress Call. everyone, replay and Good on discuss outlining today, Quarter the on quarter topics Earlier intelliatx.com. today's call. a XXXX live will Earnings to Thank to issued website Media we this operator. at plan morning, This Third section our is and be website. and This release also a the can on archived of Investors be broadcast and call found being our

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

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

will update XXXX. by begin the today's John the of quarter review for call, third Glenn For will our on progress, will company's provide highlights. and discussing R&D Laura an our financial results

turn the to we remarks, prepared over will be let open call their me With CEO. Following Q&A. John? for our that,

John Leonard

with providing curative quarterly Thank us towards to we've to progress you, this patients made move joining us morning. Since Lina, thank call, life-threatening our last for and you all closer diseases. treatments excellent

pipeline strategy both is includes ex vivo Importantly, our vivo in that therapeutic spectrum a and generate robust applications. full

causing lipid vivo proteins. or utilize approach genes normal selectively we nanoparticle precisely disease knockout our a diseases, genetic delivery genes insert produce system For to to based leverages that in

vivo utilize we our ex engineered approach therapies. create cancers, to For self

we products product cell the news to to transthyretin for cancer begin is cell to the Kingdom's robust multiple ATTR. a evaluation own momentous natural immune received patient's from is fronts. the Our system produce Medicines based NTLA-XXXX and homogeneous that Healthcare TCR investigational United receptor In epitomizes approach approval of regulatory or eliminate therapy October, This treatment designed the T on of amyloidosis our agency or clinical cells.

as an First, delivered the systemically genome reach be CRISPR/CasX human first of advancement clinical therapy to in NTLA-XXXX editing, field studies. the will

Second, validate a for trial forward and to non-viral clinical mission. is our our Intellia, delivery provide significant this us will technology opportunity the step proprietary in

first-in-class importantly therapy that course for potentially a most reverses after ATTR halts our patients, product is of and and single Third, treatment. a

our we've or submission and angioedema NTLA-XXXX also Both for an in for progress our each pleased to IND respectively. track wholly-owned studies first of We advanced and is hereditary programs programs patient track NTLA-XXXX acute that In will design today end as go of into she on year. shortly NTLA-XXXX, reviews the we dose the through are to this on and equivalent myeloid are Laura to detail enabling the by IND to XXXX. regulatory leukemia more parallel reconfirm trial. continue

We manner broadest to in realized possible. being progress platform are potential excited make editing is the the continue our by where the we equally genome across

XXth the diseases. our Annual that should preclinical our presentation platform effect of variety One approach power data at therapeutic genetic the compelling modular Therapeutic Our Oligonucleotide our engine is in pipeline knockout of and of of update was insertion a of recent Meeting fueling the both candidates rare for Society. for technologies have reflects vivo lifelong that

ways for that paper modify Emmanuelle launch are prize we Intellia. at Their conditions fundamentally opened today. editing awarded CRISPR/CasX who of that patients XXXX suffering poorly We for therapeutic revolution new treated Charpentier her the the breakthrough genome to Jennifer the of this prospects believe biology technology. and from Finally, Nobel jointly options and cofounder, for XXXX to of the celebrated achievement DNA Doudna are work our chemistry science collaborator, categories thereby in to scientific discovery improves the were door a proud and of part many we in disease be creates research

based honored Laura? on the I'll genome to Laura, to the will our bring are provide this chapter, technology promise to evaluating clinic. as on call next the over Intellia pass more now our details We programs. of patients editing embarks in who CRISPR/CasX therapies

Laura Sepp-Lorenzino

and for TTR all lead is wild-type John is protein development to an or the a disease circulating There gene and you, It in ATTR everyone. of organs. to of XXX,XXX wild-type our disease by in and morning disease NTLA-XXXX which wild-type cardiomyopathy. vivo starting buildup with and protein. the treatment polyneuropathy source and NTLA-XXXX estimated ATTR, the good liver, are update People hereditary in applies and provide living hereditary most with frequently form the candidate our of manifestations which fatal XXX,XXX is patients worldwide results of the of patients a diverse can approach knock multiple progressive our clinical TTR have pleasure out Thank manifestations, my progress TTR with ATTR. ATTR the in in on IND XX,XXX either caused between an [indiscernible] ATTR.

disease that deep NTLA-XXXX's and one on and data potential treatment Our halt TTR long support showing lasting to be eviction reverse progression. to a robust preclinical

John our our to CRISPR/CasX enter the delivered As now NTLA-XXXX, we noted, is systemically trials. with for Phase authorized clinical which X CDA UK's [ph] therapy first of are clearance initiate study to

polyneuropathy study It is efficacy. path efficient as For this most approach TTR correlates protein the This clinical modality with study we a provides defining optimal first-in-human a dose. initial to ATTR validated novel in that reduction of population. hereditary selected the with

we in NTLA-XXXX's with dose, plan have active us provides safety patients. and patients patients optimal established clean likely ATTR and it conditions a evaluating Additionally, expand than polyneuropathy obtain cardiomyopathy. to the to best cardiomyopathy NTLA-XXXX Once are with we since the tolerability less polyneuropathy to opportunity comorbid read safety and have biologically on ATTR

safety amyloidosis NTLA-XXXX is trial XX X NTLA-XXXX and study label, open pharmacokinetics multicenter and IV X+X Up to Phase receive transthyretin hereditary a patients following ascending design at expression with further point. single the active of be polyneuropathy. up Part through to serum dose design all dose. gene X and We PDP global optimal This evaluate will we at note, a as biological dose levels biologically biologically at activity liver includes patients two-part, a at will single the optimal with of infusion. disclosing of safety, and the to neuropathy additional with TTR the an study will clinical cohorts anticipate quickly of tolerability, a Our move this knockout. aimed additional NTLA-XXXX, enroll Part measurement in X functions trial dose. which patients measures Of pharmacodynamics not including the identifying of X, a plan dose including to a [indiscernible] starting single traditional levels to on be of initial neurologic of assessment cohort direct are to X data active Then into obtain dose dose levels dose. we biomarker

on an development for enthusiasm submitting end are in with the actively once be have you screening in We track the and forward regulatory milestone are patient, are year remain the to great and by look strategy this to expressed we program. We applications who to updating our We countries patient for NTLA-XXXX of for genome who other dose ongoing first our for we first field patients, as dose editing, Intellia. field global additional exciting part of for experts and working patients. delighted

therapy to on on in patients experience moving tissues. in the vivo wholly programs, updates treatment NTLA-XXXX, Now NTLA-XXXX. NTLA-XXXX for painful owned multiple our in recurrent, and angioedema of HAE. hereditary is and development our unpredictable or second two swelling HAE

completed therapies durability used are is acute data NTLA-XXXX non-human in significant same KLKBX are the prophylactic leveraging treatment there LNP NTLA-XXXX. for there designed gene Today the and knock system the opportunity to patients efficacy. for from and a year-long pleased liver to out primates. we in additional for delivery the HAE, study is While is clinical [indiscernible] share

This debilitating advance NTLA-XXXX to free regulatory lifetime highly on a disease preventing effectively protein attacks. from on GLP reduction continue optimistic application these study of half remain NTLA-XXXX XXXX. our to the XX% effect, and levels a dose, track and and to in patients serum activity. initiated We the of toxicology efficacious up we its single a clinic sustained HAE we’re symptoms. first-in-human for and for in could towards we second quarter as approximately Following Based expected this submit the that and a patients of to achieved consistency studies be kallikrein durable translates of reductions

now to efforts. our ex Turning vivo

process toward based We to approach a potential are Here we create of of tool that a editing and the cancers. self T as engineering our to cells strategic engineers employ whole by variety groundbreaking arming enabled TCR [ph] CRISPR/CasX of harness therapies. using

a that improve use proprietary translate robust quality simply risks edit put, the tumor. receptors and objective, cell and system their T a enhances function. This ensure We Our T approach reduces is replace patients TCR. tumor patient's receive should high engineering mimics cell their cell product with natural to potency our precisely against and own to defense process safety and to targeting

multiplicity enables memory desired edits efficiency, the of viability and technology phenotype. high Our with view cell

in with T feedback T develop treatment efficacy. NTLA-XXXX submit will We of we with standard improved next IND Society program on the to strategy. translocations the from that that we Hematology equivalent process. and regulatory for benefit achieved for of of forms development American early characteristics introduction guidance for T data to AML, remain are December half window reduced continue Meeting such the from safety additional patients Importantly, shorter leaders first the our time favorable of products [indiscernible] Annual preclinical lead activities. cell the With of track development studies cell of therapy XXXX. as to with our We in profile progress traditional generation the a engineering in at is potential in an presenting times translate NTLA-XXXX recently in candidate, unlocking look or and product Essentially, to the submission enabling should received forward cell regulatory without all IND compared upcoming field form steady and and and of make as cell our TCR

utilizing our platform, of leading modular At to validated striking and and made we evidence efforts team editing, editing vivo technology. of in our solutions and insertion our vivo that approach the ex generating in OTS and transgene recent impact applications for genome lifelong strides the our Meeting, on has expected further Annual broadening consecutive therapeutic research Moving presented targeted vivo. great the

bonding genome For murine proliferation. hepatocyte rapid liver, the After a partial a the the and and insertion the knockout full cell unchanged a both, despite close of of model technology conditions of after treatment. we these permanent or resection reduce the edits a in were to ability of and of under in of effects evaluate The edits liver supports causing genome hepatectomy protein approaches, durably a course single employed magnitude disease nature subsequent edits restore persistence our of regeneration, proliferation. to functional two-thirds protein

protein this seen further to NTLA-XXXX. deep in assessing and year approach our NTLA-XXXX NHP of on the built durability long For and we in that knockout, have permanent support reduction

based functional our approach promising significant best the expression. results compared a durably expression early a be to in programs the working AAV further targeted hepatectomy in loss hemophilia in therapy, study therapy. XX% well that, gene including our gene With forward insertion with vivo would these in patient's ability Glenn our on over hand to as life. insertion, now We transgene to restore to overview loss data For demonstrated potential I programs. third will AAV when our editing opportunity in as observed a progress highlight to platform look joint whereas quarter financial consecutive believe owned and results. to with who our of We no our we the the XXXX yielded to intervene like wholly In an gene the protein traditional call class insertion partial provide of Regeneron in

Glenn Goddard

good for upcoming In morning. is Intellia Laura you, and milestones. positioned well short, Thank our

fund million $XXX $XXX.X cash The XXXX. compared million. and as increase operations the of million cash December $XXX.X cash, XX, September Regeneron $XXX.X to from to million by net XX, proceeds Corporation, equivalents, XXXX, activities in mainly as used of expanded of approximately by part and driven of marketable were from million Our was offset our external securities $XXX.X financing

hemophilia $XX.X completing A million related revenue collaboration to Regeneron. to program deferred XXXX quarter was the third $XX.X of quarter increase million by recognition in by million million Our the of collaboration mainly third transfer the to the during revenue rights driven XXXX. revenue The of increased compared $XX.X $XX.X during our of of to

$XX.X $XX.X Our of million XXXX. approximately compared research advancement the the programs, lead personnel XXXX as of of to of R&D quarter programs, by increase driven This third as expenses the the well third mainly by quarter these during support increased to was the to our development $XX.X million million expansion during organization. growth

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

to Finally, we expect for summarize fund milestones. John turn XX upcoming briefly to the months. at our our cash now, over the I operating next to balance our And least plans call back will

John Leonard

announcing of patient productive to and CRISPR had of we We’ve Thanks a Glenn unprecedented therapy received the the the summary, In and that systemically year our authorization X NTLA-XXXX Intellia, initiate study Laura before challenges. first a regulatory with year. we've looking to treated very we’re at tackling for end forward while the have updates. Phase delivered

progressed have continue enable us the and to to the of pipeline extend to progress. could CRISPR to application that we’ve address each broad an team cure a variety their following a forward or of NTLA-XXXX and program for to of year. we unwavering our have genetic rare building will on rapidly those Further, I cancers clinic We my to Intellia Thank advanced realizing to been patients, treatments to innovative toward sincerest offering you and diseases. equivalent deep I'm and the you thanks also for promise entrusting genome want technologies NTLA-XXXX bring commitment platform of entire track power submit and next IND gratitude remain families, and who patients. the us my their for delivering editing. that at regulatory

pipeline we months We focused retaining final are platform head as into beyond. momentum and across the XXXX on the and strong of our

questions. We open will now any to the Operator? line

Operator

Richter first from The comes Sachs. go Please question Instructions] [Operator Goldman Salveen of ahead.

Salveen Richter

thinking or be as you you in on one provide indication? to study take progress with another and geographies about separate us the will Good or into how the you're U.S. details would more in whether a of could curious kind latter, morning. and TTR will cardiomyopathy, I'm trial a about to trials you program if U.S. polyneuropathy trial? addition And just in various incorporate progress wondering Thank beyond geographies you. starting these how large other

John Leonard

The taking And want clean, polyneuropathy get with that the cardiomyopathy some The the in reasons some question. now. safety. the from some at should this went more very although clear But of patients into We dose we patients experience us population. perform morning, a that's get for study Good of just clean patient we beginning some to with very, to which Salveen. patients be Thanks more it's patients at background somewhat primarily drug move Laura gives look to insights the quickly, fully for very through, we'll we're indistinguishable are that that same. study, so editing degree that who in It's a clean be cardiomyopathy with that degree the want move we're we very to is complicated. polyneuropathy, that should study a as to have the peek get permitted how approach either into with detailed point. expect and in will background are

cardiomyopathy, is which need focused this bring quickly, very, unmet best hope we're of ideally and very the the idea where potentially which course, Phase along much then be think I to then hand, very with is, in that would we overlapping, it. the with polyneuropathy globally, on or to the go thinking greatest begin, even heels on through of So will study way follow I

program primarily of shouldn't here. stretch any point of this of departure imagination. just the polyneuropathy you think So as a It's by the

mentioned to with Now, we've goal we other in comments, there. respect this the up as running sites particular have made progress is our to great and trial, and

course, that well. other And subsequent in go geographies. the will to as foundation be work we of So into is

quickly. on like be we'll and fingers this to progress going So very, able crossed, very

Salveen Richter

U.S. Thank in difference the of gene trials might could I Europe see, is want start in you. to order followup, cell in what the And, terms John, between to and a there if they editing?

John Leonard

decision around take a as the terms which the the in best it disease the difference different tried we've and at patients, information very geographies a the good so the then as depending much on wouldn't to the progress. efficiently. I look and how make particular opportunities indication, what programs with is at how proceeding you perhaps can And most do what we environment is, characterize our are regulatory of rate for in one world get of can all

we'll is and very the come the here critical US. would efficient. I running mean, an the in getting collecting of information and up But very, objective UK quickly. quickly, for that definitely more back - purposes be it made that So related this and be is the to geographies we judgment

Salveen Richter

Thank you.

John Leonard

Okay.

Operator

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

Maury Raycroft

[Indiscernible]

John Leonard

if I I you to It’s know can't Maury, could might me, hear want you. don't hear you another connection. try -

Maury Raycroft

[Indiscernible]

John Leonard

to want I you, I can't. No, hear but

Maury Raycroft

[Indiscernible]

John Leonard

not… I'm it's sorry,

Operator

Madhu The of Please next ahead. from question Kumar Baird. comes go

Madhu Kumar

Yes, taking thanks for our questions.

of phenotype think I of dose you the really one in clinical first in have that that mixed do parameters said polyneuropathy, patients just patients you asked. cardiomyopathy will allow just the is considering Phase So about escalation? something kind Salveen now, who our John, X/X into the, How cardiomyopathy a of you think assessment kind

John Leonard

I Yes, think it's important.

cohorts, you, dose. we many study, from what the in to opportunity physiological expand then and and nice and you a assessments biological these this get Madhu X think limited to only our X optimal it's all, of is to patients. question morning. physical a X+X of there's detailed Remember, get thank get First design Phase we It's

is What get is The which TTR. assessments sorts circling in about, descriptive the would So looking departure more levels. detailed of what a would TTR asking readout be we we're study. on primary think that that's subsequent I would in go safety point the effect assessments. of be get those done to for And you're

Madhu Kumar

of a practically, NT-proBNP biomarker say test of find if more include enrollment, get effects? like, surrogate could amend lot mixed a you cardiac kind is you blood guess you for to I protocol patients, for getting the phenotype

John Leonard

and think don't our to would the be I go protocol. intention it amend

to tests exploratory particular measure. got be be I Looking to study that asking or and mean, we've of a have not on the remember is ability many will so of polyneuropathy. the into patients trying patients biological with study large the to two the get these And patients bit to of more requesting suffering to symptomatology will the will a assessments opportunities collect, we're expansion kinds opportunity all from be very, of phenotypes may know, real are lab that to severe you're given But degree. again, dose. coming the that information an all, primarily backwards, cohort, as cardiomyopathy derive [indiscernible] and optimal we the at you about, we There little going think that part called that the out limited, very, overlap to particularly get I insights,

the physical we're to to but don't So TTR that reduction, conclusions draw level terms out. get of reductions of description the many be going primary detailed TTR should again, able I some able and consequences we read is think it in just be of to

Madhu Kumar

kind in partnered the like having cell CMC disease? and question kind non-gene program plan vivo When like, one of test are And how at from you like how you either and how your for how about ex look cell cell up one guys competitors then to the CMC of you sickle approaches, more it the say, do are different in viral that appears, regarding sickle with you therapies? for you both last ones of both that of and editing is blood about of we kind Novartis? How are cell progenitor cancer kind setting need envisioning thinking and plan therapies ahead your CMC, a travails or programs, good internal considerations? for and kind of your Okay,

John Leonard

that in they've that speak our some their lot substantial program, own responsibility HSC progress. a think big on the our shared That's again, at developed and is of the ASH which of the making respect TCR vitro we I with at work the terms our work in to how case of own been cells they we've can that in a which proceeding a about partner, some Novartis and highly cells. and is in bit well of Madhu. those vivo kind for in this there. very, talked transferring question, and who directed information approach perform is settings third period With against in There's very way a that's given that WTX, they've in to to work, that for XXXX the in they're We've little is in to this In manufacturer that And well. we're the settings, our space of very, party adept all very on experienced time. taken space

guidance and we think shape meet next our we're currently, move this into excellent clinic the So begin, to to year. in

the So been has going well. I entire quite think process

Madhu Kumar

last you, level going one, TTR of TTR got durability do I'm what lay you And there. convinces is durable knockdown you've to really of What apologize, One think a I out winner? silencing? Awesome. it just level

John Leonard

of we very the done for as very, people been have look been TTR at paving talked this validated space, in set with way who have outcomes. previously, a carefully clinical Well, by nice that. who've reduction levels benchmarks job certainly of we've the [indiscernible] that And

time. and very that is obviously, now promising, that. fact, in that There's So our preclinical to to it show. won that very, reductions and evidence, done. and and see of look we think very, very our through at that knowing data, And, think of started to one to of with match ideally they're the got rate With this that translatable Laura to patient cells pre-clinically lines comments, and are ability which evidence a approach what single we're very to has which their we've time, just liver, to that's done goal of but model respect is work best her have in means and - extensive two the to clinic. is over our the watch surpass that And take ways at that, go bar assuming the systems. to in this, which overcome, least explore And on divide. a are beginning went the we there as we the We high accelerate you've very, ways is, line

this the by she there So a referred liver, is at that partial it study is And hepatectomy grows what to, grows where was what you the back. reduce and what can look thirds, take first back in two place.

as waiting of no with every animals loss passive we've single species different studies accelerated as well. evidence effect. line of So There's we turnover explored, of that far as can and tell,

be to is lost So in effect be the quite I'll frank, here. if surprised

Madhu Kumar

All right, much. excellent. so Thanks

Operator

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

Gena Wang

or escalation line reach regarding there assets you just dose program, one Thank are saw to to the regarding rules, WTX abstract have how wondering knock CTA. remind is already and data or to impressive can down program two, that also like, that second taking data, you ASH moving the first looking stop NTLA-XXXX you or will if you my full reach is file clinical if cohorts you questions. we will will and ATTR determined clinic, dose ATTR make the the then my dose? that And any at wondering you the say, that question XX% say what top the for complete all or stopping IND us, for the at third is the try going, I are second you was very the work, program, that

Glenn Goddard

bit is so Glenn. this Gena, with to little I'm his problem your of having ask, audio going for headphone. a questions. you Hey is John thank

address I to our think have in those. going and jump Laura, some of CSO I'm

We're Laura apologize. bit you. questions. can I little I a function, of address just So Thank but having think those audio

Laura Sepp-Lorenzino

Yes.

that, I'll cover And good Gena, I to ASH. wait rate for So, of presenting the some question. start for will but you, NTLA-XXXX. John thank be morning, with I’ll at the and

into TCR we fact in and put that that's that had the NTLA-XXXX that So, is in, WTX with interested process clinic. we're the the

another that OCCs there vivo data AML sharing the of more primary there - well AML of of performance demonstrated piece those is on WTX, blasts to using versus going that we as well no there in normal in be data as the vitro So, a that, TCR in activity is very there TCR in sourced model aggressive the graph as cells from on modified were people. this reflecting important model. of as blasts patients, signs was nature And the from the from of

Sorry, what were questions? the other

Gena Wang

is You Oh test so, down the that knocked this it for is the works? question dose question, down further the stop or knocked team more? dose irrelevant And to the, other do know, hitting? or or will how you you the the file test I'm program? you and escalation have say then, IND efficacy CTA to when just making program, up safety? escalation for XX% you ATTR do a like numbers, bar the like, Is

Laura Sepp-Lorenzino

Yes.

clinical pursuing strategy. Okay, going as we're we're global for so with ATTR, XXXX development a

with TTR And, engaged trial. through X incorporating we're we NTLA-XXXX in human clinical our Europe. four regulatory as guidance, U.S. regulatory the clinical, move agencies that the cohorts our have quickly With trial. multiple firstly, Phase in to in and we intention So regards design is to

our preclinical to with data in through the the the regulatory from starting dose try And to escalation. doses. quickly fairly have move work Right? select We consultation used agencies

had others back. TTR benchmarks the before, surpass RNA saying the John meet to been goal believe and with is was by established John who, the that is silencer maybe As that compounds, I and

Glenn Goddard

want you, interrupt to I has you. reconnected. thank he didn't Yes,

John Leonard

died some reason. I am for My back. I'm sorry. connection

Sorry.

Laura Sepp-Lorenzino

have like you do Gena So, questions? all your

Gena Wang

good. you. Yes, I Thank think that's very

Laura Sepp-Lorenzino

I appreciate it. you. Thank

Operator

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

Joon Lee

the taking the accolades. for congrats questions and our thanks all and Hi, progress on

of history a in the any countries think key what oligos? any ATTR have you could program, you targeting, of important? could on you. genome you're terms germ patients? seeing study, soon program? then editing do piece so, And evidence particularly approve And like off enroll you something hoping your Regarding antisense which to of following the if think work is initial your willingness failed different in that and across as cells, it’s you up Are patients [ph] they will as concerns delivered, what ATTR that those different that are program I as a Thank about attitudes confidence approach global this this gives -- be to the systemically

John Leonard

but I've the hope that, thanks all I got for question.

not that last part, the first other treatment for with will of the into part are coming therapies, to nucleotide. they gene have and patients silencers route First, study, preached the receive prior --

this not? responded that of that notion would those We're So, patients. somehow this they or hadn't excluding trial if affect setting, in

you So respect in many the what have With requirements? place. the question, never if are received I to have first attitudes, understood treatment to

Joon Lee

Yes.

John Leonard

no, Yes, no.

we're have fact, been, treatment that gene patients prior cannot received In silencers. with have saying cannot

not is study are which based on means silencers. there , asking that no So -- we come patients into the

So based there's not prior that we're no, on come patients a a so to failure into study clear. be the asking just with therapy,

Joon Lee

Yep.

John Leonard

and our with patients, with with respect pretty regulators, systemic in much around so the to KOLs, interactions Yes, therapy, attitudes world.

therapy that the looking is are chapter written the reversal therapies in truly doctors well, look I of to I with the think that of from what there's amyloidosis. levels standpoint believes reduction been no at has perhaps halt anybody and to made or here. Curative unmet terms doubt disease lead think has of a space progression for. of and in all final that of patients the been bringing improved they don't need. progression progress patients And,

we to any that think case in at them in program. information the I And this for the they it's plan drug. would the so, with mind. that that's look particular they're this when And, assessment, And obviously, as preclinical bring we as any outcome interested be modality,

patients go So in quite great we shape about to and think and study this are and it. we're enthusiastic doctors

think the in expression. of In where tissue terms target, at, all bulk of it off look question, doesn't And to exactly to LNPs or I really that, that's think of want we with place. where we'd LNPs the to respect tissues preclinical assessment, where TTR the part where matter we the a first the that's them go go

Joon Lee

you. Okay, thank

Operator

Please ahead. Leerink. go from comes Mani question SVB Next of Foroohar

Unidentified Analyst

in the good Rick morning. is Congrats progress. This Hey, for Mani. on dialing all

of or either developing on prepare get the was manufacturing the for there the in background the cell some need to get steps you that detail process completed IND characterizing as gating program. AML cell therapy work some I the Are IND that's hoping submission? any first, more the to products or major So in going before on for

John Leonard

would I this characterize way. it

meet are which by to to is of comments, transfer third we've guidances the a her work approach is We pretty much much Laura the offered. characterized on very is and party. manufacturing track The

the and product interested that a insertions is assessed appropriately documentation agencies that the step put look all into are requirements. of the and knockouts those nature we're I are we interaction, are think components cell very engineered go sure filings forth. it. in And the in so specifications and that in in there there we is, so and excellent It's what meet we is that to and think lot making things at and regulatory those to when incorporate in for hand release you on characterized, the and an And up of those regulatory of back that regulatory and as and shape can place these products. and so the open right that's have all information

Unidentified Analyst

do right, have and All follow great, a up. I

So but editing. get antitrypsin a hasn't recent I a hoping I know the program target for as to just was that bit, your on this in Alpha-X been thoughts most in deficiency gene forefront

disease. data that down Now validated some RNA have we from this knocked clinical for

John Leonard

the question. for Yes, thanks

we've a her certainly some call the what lot there's a at about of an the talking moving But in for of spend going company. certainly these we in insertion about kinds excited time comments, said that Laura We we're lot the three of with big and focus work our the gene As that's discussions. clinic background programs very area of are the on that done work. into our

for investigation thinking the Antitrypsin. express other Some to including an has data in about presented how been well it's presented data We've active Alpha-X models. targets, of human And us in that interest area space. for some of hemophilia with proteins of Alpha-X respect previously can we Murine

I an that you So, to this encourage is stay area with. would tuned

Unidentified Analyst

right, our great. taking Thanks All questions. for

Operator

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

Steven Seedhouse

I time sort part in Can can or get treatment by just weeks study part. relatedly, you you expansion what therapy? enrolling important naive percent ensure dose TTR with some diagnosed patients Phase dose of on like down also therapies, you the later. think then it's couple knockdown knock Thank data morning. looks and curious Good study anticipate launch market addressable to population about but tafamidis treatment enroll of to I The for patients an would the tafamidis you you. with respect of just knockdown being join whether with a on already and to switchers the potentially the escalation, talk the switchers And tafamidis in that's stop example,

John Leonard

different know, your that's the so TTR Yes, mechanism tafamidis stabilizer as part action for levels. question, of to an a you agent of opposed that a as lowers has first

would interactions concurred experts So, actually have levels. that of with our in it KOLs influence judgment and was and that other measuring terms little really TTR

ask our or come that they what patients to assessments need able who for receiving levels, at not the are we're doesn't that the we TTR Phase X in then and point tafamidis study. carry wash present tafamidis body, that is, the that so screening, so that are we And do have affect in effects out out, received as

patient it really about that say me to years talking Gene a that the data and I now. and this get thinking down is be, for respect market -- to patients the a the us are it's enroll we we of as different this available, study of It's makes population of available you happy could that experience situation them So outcome study. the it switchers, a It from be to include to in although you just particular guess a tafamidis the larger necessarily we maybe get kind reason silencers, will see. makes not been to will the from little speculate and that on in have road, point quickly, need. at that that more proportion about a We're on time. tafamidis permits will that's few With the hard

that's come think that future that we that I not how impact So may date that at at back study to time. and our or a think about

Steven Seedhouse

thank you. Great,

John Leonard

Sure.

Operator

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

Yanan Zhu

taking questions progress. and for thanks my the on Hi, congrats

thanks the the beyond provided you clarity the the for for development on program, long path TTR study. first, term Phase So X

have the ongoing trials XXX pivotal ATTR the to treatment RNA cardiomyopathy therapeutics arms. XXX the patients in Just of in curious,

trial? So genome what XXX trial required size do trial. would patients you editing be editing a think therapy I unprecedented or XXX for to genome guess any gene be would for

biopsy as to to the on X like then begin also between dose And efficiency the would your dose patients, cohorts? liver period sorry, Phase X trial, lastly, Phase biopsy there thoughts editing in look a the portion, do you patients study, Phase there? tissue portion look I between you. a the adherence in at amyloid is TTR at of hear expansion I'm to And the you plan for So or requirement waiting X in for tissue? and to Thank

John Leonard

of information reduction the achieve agent. well, Okay, of provide biopsies one to be that don't doing be that not do and what need that We a pathologic We're think here, of that Remember straightforward. very the protein last would first. any kind. really is value. and let's the trying limited the to That's is to would going tissue we're we

would been a you size previously. the important is and a therapeutic endpoints determined So reduced. that's just certainly getting. gene modalities because editing small determine by And the biological circulating case optimal you which measure to And is editing. This of doesn't in nature, the degree that With direct surrogate clinical technology of is you're respect about going that can measuring certainly you and study. the extensive effect, we're trial, going guess what's a do to think to the the best objective the type which be of of we're a my at point frankly, this study be TTR can almost And our cardiomyopathy. information think mean really tested levels be is good what is TTR and size that will the that will for to about for have be necessarily most

will particular in I related learn, to population a a TTR information be and measure outcome. we et extensive had of consideration taken effect of that's to of related the those a terms will us think, that and then, before and the physically what's what from that's questions particular size cetera. into to drugs, as are in the lot terms We we study measured other reduction clinically here going or and some earlier And background

to we've they can done pretty think then I figure an we standard where patients a think that brisk X+X Phase here. of up at as pace. there's that of But quite should And set that so terms in a All of you you work this get in thing quickly, study, are that cohorts, have cohort watch move the this fill to how assessments the It's I do. go we assessment rules and place there cohorts get in design not those next finally, is and and think the through this going advance. the I to and

Yanan Zhu

one just question more you. I the it. may, Got If Thank program. on WX TCR

target And to patients that leukemia you've your blasts, burden. is Leukemia In too certain first perhaps level first population, thing but much, surface trial, patients the T with you're ongoing various product, human different think the terms drug, I the indicated in trials, not looking study high first of from the AML. just or population that of in blast CAR study targeting wondering of antigens too similar in a

John Leonard

prior call TCR David we're Maybe Lebwohl. program our our and trials, responsible direct doing on with very well. I'll David the in CMO, work CAR experience is as T for has that

you relates to how our prior want to some and that do talk patient initial about population David, of work? the So

David Lebwohl

John. thanks, Yes,

had prior study this generally patients in who patients be will therapy. the So

who come as burden very or similar patients disease blast So in AML. But look lower may they to patients higher might you some or have have other a have had separately. after and prior as them we back for who therapy, burden at looking their last are so mentioned, a studies to

we're targeting what's is which think from antigen, CAR normal that an therapies other important present I and on different is T tissue. not highly this

the we XX. So in with studies CDXXX or been expect see the a type that's to severe don't one of toxicity AML seen

Yanan Zhu

the because TCR T. a terms after a in in question, T Does antibody. and also that, more curious much you've which WTI CAR T-cell, a and one looks antigen Got TCR memory is highly to the implications abundant TCR, maybe opposed as the persistence not of a because Just TCR it? have mentioned that it's cell it's also, in product, like

maybe some So, Thanks. on yes. formation, the perhaps implication memory

John Leonard

David, from comment if want keep to you going through…

David Lebwohl

so will Yes, you know, patients, these have blast. as

obviously, due the is to antigen initial tumor itself. So the

tumor, thinking is life. again, blast at some studies, be the will the eradicate do advantage which cells of can left but that Of course, there that the is eradicate the return cells T you our present, be often to if T is will goal antigen cells completely there a these have the long tumor. present point, if do the the, of if persist And

Yanan Zhu

Got it. you. That’s helpful. very Thank

Operator

from Oppenheimer. next Jay question Olson of Please ahead. comes go The

Unidentified Analyst

you. did the Hi, not And maybe this have just our planning not back to question. in trial U.S.? so start quick is XXXX, directly the then maybe follow can for in or us euro, taking one in Jay. XXXX are on [indiscernible] wondering Thanks you you we the a for us, if move and maybe are to trial that's Thank

John Leonard

looked what of the We well we've vivo very, learn And the do that for We're with seen around the and as subsequent laying out that what we've world in geographies. from a And we as is certainly influences programs. the advantages exactly still lot U.S. regulatory TTR. And thanks very our our question. Jay, different we've. Europe. carefully strategy

But as certainly So synergies all their more selection, have will way we from that will many TTR. details say with we'll And that interactions find we've advantages with this, program. final this we've you. had them and those on I the share into benefited

Unidentified Analyst

Okay, thank you.

Operator

Roger go ahead. The Please Credit of [indiscernible] question comes from Suisse. next

Unidentified Analyst

for everyone, morning Marty, on for as good Roger time. and thanks your Hi,

expect range initial And you intentionally cohort? regarding should there's under therapeutic Thanks. quick One, And How an are low two dosing plans? the to escalation study. are year? update? the have of data you the an design dose low thinking update about looking secondly, then we before dose So are expect next you how disclosure be questions to you this within or I've XXXX just in do much dose

John Leonard

you ascending we first much in of reduction. the as zip human, Let that But at code, there if as think would where possible and But me preferred and to you'll reasons, that's that and to house reason higher bias Anything, that to and to we for where though. is to start side, be overdose the the of will lower idea a this necessary, think efficacy, opposed obvious safety then crack something escalate. the optimal David think, start TTR can dose I measured as as side, no can de study there's a somewhere have biological, in or to say call escalate you involve here. the we start as single is efficacy, it take by I

this think, in, first And notion So how is from we're that's that the where to as work. logic really actually a I kind good extrapolating preclinical is get made, when that its are. comments going predictive models we as, the applies we're prior in of those And this to the program. this and

that something seen So activity, four to is study. biological extent remains assess. that's will of and There's there cohorts our be in this be the only thinking should we which

mind. we'll One a of mean, get CRISPR/CasX, primary think is for systemic that date. optimal I study With information is biological which we do dose, its guide that that two to to pretty different course in the time it's any And a many domains. respect where quickly very, to not in we'll that that there's been get first of approach data the assessment is this this something suggests dosing viable insight the enroll patients particular we're then we we're first be think done. to kind when that's will important as very I So to kind, of it's a think And of I it. any doing in of or there's the time systemically. disclosures, editing obviously objective. to going elements bear is,

remains think it and enroll information, is we the that it small determinations make again, meaningful we'll accumulate at about we and that when I and is, appropriate disclosures to quickly. a But is, how look as but what quite where we make as this should interpretable seen. in So venues, and the be study appropriate

as So possible. forward soon we to look answers getting as

Unidentified Analyst

thank Great, you so much.

Operator

And ahead the Please is from question next question. your Maury with go Raycroft from Jefferies.

Maury Raycroft

Hi, thanks for question. taking the [Indiscernible]

John Leonard

with check be back May you can sir. we

Operator

time, to this session. conference our back and concludes answer closing this remarks. At I the would turn for Lena any like question to over

Lina Li

to everyone look and and has call thank today's forward for updating for day. joining continued nice on a operator, support. Thanks, interest progress. you I our your and hope you all We

Operator

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

You may now disconnect.