NTLA - Earnings call transcript Q4 2021 | Intellia Therapeutics, Inc.

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Operator

Good morning, and welcome to the Intellia Therapeutics Fourth Quarter and Full Year 2021 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 on the company's website following the end of the call.

As a reminder, all participants are currently in listen-only mode. now President conference Corporate of Intellia. will Karp, to Communications I Vice Senior Investor Instructions] turn over the Ian at [Operator Relations and proceed. Please

Ian Karp

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

John. full remarks, then that, Sepp-Lorenzino, the with Officer; for from for Q&A John will financial Officer Joining Scientific who Glenn, will we'll open to the will Financial Chief the today's provide the With Dr. concluding an David call. Dr. Officer; followed Intellia's available Dr. Chief Chief John and be of overview Intellia Goddard, Lebwohl, highlights John recent Laura are Q&A. Officer; turn and fourth over of me and quarter Leonard, Chief XXXX. begin business call will results Glenn up Executive portion I'll call for Medical also year and by review

John Leonard

promise program Intellia, fully the possible we're morning. screening it's XXXX We platform within therapy. year two candidates, of first building for deficiency initiated Intellia with shared in disease thank patients edit to broadest body our offering to AML a patient precisely programs for our for genome Thank editing antitrypsin and vivo landmark we during for first company a joining the nominated industry's first-in-human showing a vivo you systemically B the for clinical which led applications. medicines dosing realize by a additional proof-of-concept partner and us the targeted CRISPR-based spectrum our NTLA-XXXX alpha-X We for and study. all causing toolbox and and both you, full the clinical CRISPR-based At ex for a two leveraging new data development platform, this hemophilia Ian, with NTLA-XXXX deepest NTLA-XXXX’s and through was insertion delivered gene Regeneron.

proprietary example Blackstone We of proof-of-concept a is product future and strategy and CAR-T allogeneic editing in and vivo finally, attack of also a bone immune capable presented cells evading And novel noteworthy platform several new a to for engineer advancements, technology based launched including we marrow, editor. to allogeneic to with collaboration develop cell therapies. rights AvenCell called company Cellex innovative therapies. platform gain of the in maximize This another our of partnership universal value to our preclinical

core this upon on in achievements three these priorities. we building year, While we already focused tremendous XXXX. accomplish amount we're a are specifically in XXXX, More

data ATTR NTLA-XXXX HAE from the looking And to vivo clinical additional in presenting for accelerating of lead present later validation this to amyloidosis NTLA-XXXX we data from initial candidate, we're this for our our pipeline program. year, week. next First, front, plan the clinical and forward clinical

both the And opportunities. of business by expanding advancing clinic on expansion nomination against along as multiple candidate vivo candidates of and innovation key ex vivo strategic vivo driving and enter these to our forward actively are NTLA-XXXX, XXXX, our with is in leadership pipeline far through in via scientific ex wholly-owned through the we genome cylinders. robust capabilities. Next, well the our new in Intellia settings, first objectives as platform on building cell so all we're firing development execution engineering our With finally, editing, delivery development

with to Our event Phase continues interim and It will the We portion. company present single data feature arm TTR all NTLA-XXXX X, include from early next all patients include cohorts. cohorts Part with data, polyneuropathy. data recently well event we as an cardiomyopathy amyloidosis The across hereditary of week, reduction a look X forward ATTR additional an study safety serum in will in dose patients at hosting ascending from sponsored with look four progress. to to which expanded as to plan dose additional durability

year. results the initiate X, to single track we these expect to We selection this the of for inform quarter expansion Part on dose remain cohort in first and the dose

of to portion of directly we Phase likelihood we've we X/X Based continue in program. gained as are NTLA-XXXX, the patients insights increased this which the the success NTLA-XXXX, dose we NTLA-XXXX, to the from For dose escalation believe on applicable study. advance

the Looking from of this interim data ahead, presenting of half first this year. the second cut we trial anticipate for

the eliminating the AATD. the This we're development disease, protein a AATD. the the designed nomination associated liver disease of halting deficiency candidate liver, today, insertion In alphaX-antitrypsin candidates NTLA-XXXX, to expect NTLA-XXXX, knockout abnormal in need progression the a are And or an the lung for gene next of inactivate or is manifestation candidate of to to announce gene the liver NTLA-XXXX, SERPINAX pleased advancing addition aim we transplant. treatment we to for which now for liver includes alpha-X year. equivalent for of a with application file of AATD responsible IND wholly-owned NTLA-XXXX for in for two of production

to our AATD, disease particular platform manifestation. relevant modular us treatments, patient for optionality tailored provides For the the

positive as cells Hodgkin’s cell shielded in or expressing is important expect an designed critically Today, weeks. study also allogeneic our first we and NK is attack. dose NTLA-XXXX. Moving on patient X/Xa persistence long-term cancers, such in studies, of T both classical cells. of announce of preclinical is for NTLA-XXXX our developed we've the nomination the cell the to was patients therapy the approach treat excited enrolling allo our coming using and engineered relapsed/refractory to to are CAR-T NTLA-XXXX, from our allogeneic cell first platform. allogeneic AML immune distinct pipeline designed rejection engineering development proprietary ensure and Lymphoma. from ex hematologic T-cell TCR were to the Phase T vivo for It others In host CDXX begun by candidate, host This the autologous

each the employ The Rewrite existing allowing biotechnology our towards toolbox continue therapeutic present to leading the NTLA-XXXX for maintaining year. this a at editing data of revolution. strategy, of innovation, genome this drive the our capabilities, February and best enable company acquisition currently editing Rewrite editing platform program by IND-enabling range position application. technology scientific base our additional we announced of the private further parallel, CRISPR/CasX activities of Reflective we Therapeutics, acquisition in the genome are an expands thereby upcoming to We our writing to plan technologies leadership In forward to this and strategies. advancing forefront complimentary conference a preclinical of development of us tool whose at DNA to may leading adding editing

this an occurring proprietary against cells cancer agreement. naturally lead platform product with platform, application this derived we our can of cells which cells. traditional diseases immune CAR-T collaboration. play With we recognize agreement including focus vivo NK In for activation programs. NK an five just the autoimmune who abnormal engineered genome our rights treatment we’ve breadth the and treatment agreement cell to a platform critical and option our immune expanded of reach commercialization others with allogeneic beyond valuable in investigational edited to have and candidate proprietary already announced role hold our CRISPR core areas we rights with U.S. with indications, capabilities. strategically two extend based up options, specialized cells, commercialization the that from transaction the importantly, This develop lead the CRISPR with strength a Therapeutics is co-commercialization us therapies ex including of compared of to possess In January, commercial editing co-commercialization collaborations, our The for targeting This NK technologies CDXX a for completed select Similar for to platform of to we Therapeutics provide to announced CDXX to a for two when under to Kyverna a rights grants complimentary that to future of strategic agreement a that Kyverna global diseases. year, to co-development is cell candidate non-exclusive oncology and for allo leverage cell novel partner ONK develop both and KYV-XXX, U.S. approach we therapies. our inflammatory to up are collaboration license month, cancer. ONK co-development therapies cell collaboration

over year to medicines patients. of through Altogether ensuring Glenn, capacity deliver with ultimately quarter manufacture next forward a for to to GMP transformational earlier ensuring years key lease ability our a and hand to the who commercial fourth will at and The financial XXX,XXX our to is we’ve and for positioning cutting results. facility of the crucial manufacturing square XXXX candidates, an edge come. a supply and capabilities the develop components clinical readiness advance recent agreement to foot we to Waltham, This carried the preclinical stay while in expanding call compliant provide With Intellia efficiently to the I’ll medicines. Finally, week, full bring our these new support to strong of commercial this pipeline. year and the our that, products past for reliably offer for mission now announced announcements, momentum our we facility be CFO, Massachusetts to of wave overview CRISPR-based rapidly will

Glenn Goddard

Intellia you, is we Thank everyone. aggressively morning position pipeline. in our expand and as and John a strong advance good financial

fund December in as XXXX, from and increase driven from by of our as was proceeds $XX.X million to $X.X equivalence markable follow $XXX.X cost in million employee-based December Regeneron July million. $X.X million cash to operations mainly by offset were $XXX.X stock XXXX. of from were of on million cash increases cash, The securities of offering, agreement, compared plans and net company’s Our These sharing. net proceeds use million ATM $XX.X $XXX.X proceeds part from XX, billion of the XX,

fourth collaboration increase $X.X Our million to in $X.X quarter fourth the venture $XX.X This recorded in increased our the compared to driven million XXXX. of was with million XXXX $X.X by quarter joint from of revenue by during XXXX during revenue AvenCell. million

increase was of $XX.X by support the to during and these million of fourth Our the our the $XX.X to driven million quarter organization programs. million of This the mainly R&D during to expenses $XX.X fourth expansion programs increased compared quarter lead by XXXX. the of advancement XXXX R&D

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

Finally, will now back turn our drive we balance to next expect XX for Intellia our positioned the John With current months long-term the fund well call beyond to plans I operating cash growth. remarks. over is closing to as that,

John Leonard

Thank you, Glenn.

already facets well against executing priorities business. way on towards the across are of our We our strategic all

advancing including partnered program. and wholly-owned both We’re robust pipeline,

candidates, are the clinic. development eight with Now which in of four

on of patients. We’re impact against accelerate the expanding rapidly reach to and our objectives our delivering the corporate platform

in-house continued progress, our own later Despite are our accomplish clinical and the we’ve to network this manufacturing our we the to to innovation much have propel continued in capacity support leadership We candidates. all adding by wave And year. scientific expanding to still manufacturing growth. next GMP support

to not Before you the we questions transthyretin, we understand, call NTLA-XXXX questions with open readout the many data be or NTLA-XXXX upcoming any our we this the our given data to proximity and Operator? progress regarding answer call. to planned topic. interim you from will in pipeline we’d addressing of on on interested the ask platform. sharing the week to that, call refrain in data this are with requests kindly to any We questions of our be questions look additional rest today’s about and With that using forward happy you next

Operator

Truist. Please [Operator Instructions] The with ahead. first Joon question comes Lee go from

Joon Lee

taking next for and questions the data updates Thanks looking forward the for Hi. our week. to and

you elaborate not strategy also as program, plan why mean, pipeline program? can for do and on that. programs? for I follow-up other you two XXXX? a XXXX screen this therapy And patients I combine these single And these the have quick a to how after On

John Leonard

not gives independent. in point, patient. Thanks, to we’re point a the doing a manifestations because doesn’t patient. one sure, mean that Joon. programs you. different it But that type it, type other balance intervention view for you intervention think the from may I’m that us, as the At patient come there’s together Good just where for way maximal hear Proceeding know single of the this tip and in another and we could favor flexibility more we disease they of of

So think understood that quickly. point, the we it programs me. like the about I’m for at keep not question your best advancing simple sure we interest elaborate this maybe If patients. most to in that bit could I that’s and screening on you a little of

Joon Lee

do such medicine they patients disease liver manifest single I should deficient Sure. driven both the alphaX-antitrypsin both – separately. same both. have mutation and mean, by it lung patient Isn’t manifest that should

John Leonard

understand. Yes, now Yes, I I understand.

helpful. that’s So

the And same underlying of important in problem, different the to mind etiology however either plays that in manifestation organs. the gene. is in it’s out bear correct differently You’re nature for the

that So without it’s have liver later where is want abrin local in in local on lung that disease in problem case, it certainly development appropriately the out the of needed and doesn’t to Whereas focus inflammation. do is the didn’t the liver manifestations, this typically get is pathology and protein the you what and that out can prevent causes lung of liver in empyema therapy. liver, In protein most case occurs of life lung. patients of replacement to the the a disease when get

primarily proportion later other. patients in oftentimes as have be it’s manifestations predominantly time with Can the primarily a gene So life of and early underlying small patients the But frequently, a a will have life, manifestation. liver on? problem mixed particularly will phenotype pulmonary one typically or goes

for can’t to case, for this causing the knocking does able the is doing particular locus. the which we in tailor different in together not same bring we’re way manifestation. out therapy targets that albumin the patient. what Remember its any that them the so program in And with a mean that But and gene is particular insertion XXXX disease gene the we’re again, expression,

And so patient, different resigning there can you interventions at get can have these same they both times. the of but

this it here simple least chance as us, So learn at and the beginning good at and gives therapy. again, we to think we for keeps tailor a go us

Joon Lee

like $XX they to quick and based Got tool a of tools. million, recently Rewrite that it. a And more it the you follow-up, on for them find announced acquisition this founders, seems do have Is XXXX you see to that did XXXX you things Is there that what a eventually? like would the and you. deal? – something something attracted single that allow next-generation in Thank step you combine to allows

John Leonard

Well, of our strategy. overarching Thanks. is Rewrite part Yes.

the which full that clinic, in in the is platform, modalities. all clinic, the vivo kinds say, when ex in of ourselves a a be the that So spectrum consider but a critically having company, leg programs essentially means we important toolbox genome of of really is spectrum different programs third we legs The three stool. full want to a vivo and to editing

genome one approach be want that is one to I particular it’s mistake any might think a that editing all address particular definitive to going and pursue. to targets think

a lot modalities. toolbox And lot kinds truly and time so different editing we delivery in a of about building lot of that thinking has a spend a of all without effort time of – spend also invest

up forward. open that we So, really opportunities things we to going we that is is toolbox of already think full are as go where beyond

base so the multiplex capabilities there’s in and we’ve we want genetic permits it we built single know knockouts problems appropriate as we’ve vivo of some then case ex been that’s do sorts with think And powerful we gene form presenting that in is we we the might technology can There’s instances writing, at one ideally particular the There’s have data but think there technology CRISPR, So setting. that address. a interesting which writing. sequences for add generated a varying to of where setter, of canonical the where be scientific this and that to a on sequences us data the there’s the able Rewrite, suited forward make we introduce as that. gene we progress the insights And will use forums. we’ve including one of lengths, to with that to to over think That time, short and of medical the at present can mature that, years, that nucleotide. it bring we’ll I immature and that as is

and view where think another tool that one keep of scope. us we have again, So where that But more that sense are we is you as lets just broadening as implement a serving our this we well. that get

Joon Lee

Thank you so much.

John Leonard

Sure.

Operator

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

Maury Raycroft

taking dosed Hi. an ATTR point Good expect commenting questions. update have if year thanks cardiomyopathy polyneuropathy at for this morning, elaborate this can patients many on you’re not my this And you we cohort? how ATTR on everyone. Acknowledging today, from that been and can

John Leonard

Maury. morning, the we’ll appreciate on XXXX, we about lot and and together program we’ll when say broader happy the rubric to I stuff of a at that to XXXX talking the into your about be Monday that falls let’s devote But persistence. Good large. come talk session of

see there. to hope So you

Maury Raycroft

another half all what definitely see for little more be you And Got bit of wondering I’m a that data question can it. to to dose cohorts? talk this about I for Could expect just if HAE, year. three update from second we We’ll guess there. expect

John Leonard

that We of consistent, data you us. about that’s of of playing and we XX said information think with one that guide is apply in the similar And very the remarks and the in exception have, the say we guides program things, to structure, think as principles data And this premature meaningful, like same anticipate half this about some very XXXX. that same much earlier to last what essentially that, we’ll moving interpretable, goal same did to particularly nucleotides. having share cargo, progress good But very, in that we June we’ll the We should and it I it’s lipid our second that we favor. of is mRNA how much our with is forward. earlier, said same making with the we’re out modularity as about year. believe

us a cohorts, given that cohorts move XXXX the into ability that’s the the to little we began So with. further

to we XXXX. year with had that think later And really information of that our we’ll that so the some hopefully we findings we this position get share corroborate have

tuned. So stay

Maury Raycroft

Great.

taking to Okay. and next for I Thanks questions my update look forward the week.

John Leonard

Super.

Operator

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

Unidentified Analyst

Hi. overall And Tommy for you on taking This you if development ask of partner? to Thank deals for in your see strategy we business should in XXXX. in question. what are is about and you. whether Salveen. for Thank looking a more to the so, expect Wanted terms

John Leonard

Yes, thanks for that question.

timeframe So any think should a do about what to you able so we’re – get be a for timeframe. start because have out, to to already, based you on we the commit let’s work. But is particular some particular asked I any think notion our I seen within and how strategy deal we leave what you’ve not timeframe, going to of

on control much laser and participation. are foremost minds. we our a significant to those will remain we absolutely It that to we is that. programs that pipeline that focused or very And first us focused allow guide on So our have on

try or that products. biology, competitive if an of look applicable what in go maintain find that those and is And partners ultimately insights the and which immediately deals we two everybody’s we participation those or patients we’ve make think better the we best significant insights now us more is And the the participation However, that importantly, as complimentary – because some we’ve you to in practice. serve gives to case more are those the first we love that we outside believe and I And like groups, by it technology opposed one. does perhaps broadly significant think of work cases, can combining in able do we of What at the opportunity just it’s our gained what we like that. compliment reduce realm are to and interest. some products research complimentary more if we’ve every AvenCell year is retained our in one, we partnerships that, last the from done area find commercial in since some to can

by parallel extent will fullest end, substantial not opportunities capabilities idea consume that work our can and to possible. we those we but their with. leverages arise, When of the the them do So a pipeline resources resources have that others essentially is insights catalyzed does complement the that on are

which if just own So our other back and have cell partner, pipeline excited at about. to we step you in one NK an element it, is look now, really addition we’re

universal about. ophthalmology think we’re now space, editing patients. We’ve have when way we think I it We a be opportunity. genome which for And you meaningful wonderful about how that, position CAR-T is And the wonderful into step thinking space. in a moved look excited back is have ways are autoimmune also in at effort, that a we and deployed which can of a really really

you and as watch always at So we possibilities. always looking confident we’re looking go should that we’re be us and

Operator

with go The next Mani Please comes question from Leerink. SVB Foroohar ahead.

Greco Song

questions. with for questions, the instead that go everyone. approach preclinical there with separate question, programs? the did for observed AATD off combo one. one in guys go that you taking This you programs, or morning, is questions program, of Mani. But of I studies our on Thanks decide was for for going two combo advance not other guess by Good you we I the but something have can first Joon’s Hi. made to sort Greco independent NHP the and

John Leonard

that to is answer the no. for Thanks question. The

back if really two we steps, that presented, two that it look independent at you is fact, study In and go steps.

the the in candidate we that case that primates demonstrate produces the of doing remains like is did the show what that But demonstrated human we’re And obviously to clinic an generate now. we exactly be beings, And those important insertion separated could non-human in causing the in what was and and we human we human production levels the into protein in so high in which of those out key what gene would think here. and inserting the be protein knocked the liver separately of I was with disease gene and target a high humans that’s SERPINEX point went then levels offending an mutation. in for time,

us identical so, way at we is essentially think the we And the doing it’s opportunity into strengths weaknesses, a that patient before again, though, we’re always any prudent. single which have into programs we to think the two bringing is will humans. patient. it to advancing road. learn we’re down if the What same And that we we about in investigational the time, think about agents taking various a not and have the single it possibility gives of together But

Greco Song

profile And AATD scientific coming insight programs? risk risk differences and around provide will the profile have from program target and a suspect the Got these between some both color these different each population. I approach and Can it. clinical patient you

John Leonard

is they in of yet in XXXX if different clinic. example the a another Well, the already are a that sense knockout programs knockout two

now so, And for principles XXXX going and XXXX. be we to to learning are that immediately applicable the think XXXX we’re

just to is The clinic us a gives we more think of a so perform. course. clear in on is population focus patient drug how the virtue likely And just that that different, experience of having – by that

an program. insertion And have we this in mind so XXXX to as the proceed. that we case is bear In of

combination it take where to both gene going you’ll that an territory the non-human work. the template presented And the programs these then data hybrid the in the target for doing provided are the of is AAV terms insertion Obviously, primates. we LMP that’s already remember us. to of requisites is in And new insertion So place. where we’re form preclinical of clinical the is

continue our with don’t build We package much in pre-clinical as on purposes. But humans that we yet. to know for just regulatory

will there’s have that. programs before principles uncertainty we program, other same we that more the applied the to and logic that with comes And apply that say I’d so that with but just

well. studies will our us So preclinical hopefully serve

Greco Song

from the me. That’s you. all in back I’ll it. Thank Got hop queue.

Operator

The from question Please next Malekar ahead. with Piper comes Swapnil Sandler. go

Swapnil Malekar

taking question on for you Thank morning. One Good questions. my XXXX.

AML VENCLEXTA be company’s given to case talk not in you competitors about unfit differentiate limited may to that be some population? that might space? patient address and a AML strategy that So broader similar patient how conditioning applicable you regimen. XXXX, the could versus in is to population which the due Can of patients, the with And

John Leonard

last think a everything, that? – think. of hear Could quite statement the agents, that made the restate other but Swapnil, you quarters the just your three comment, I I at about couldn’t at you beginning we

Swapnil Malekar

Sure. Yes.

just VENCLEXTA unfit requires XXXX regimen, be So is that and applicable it of patients it’s given and in AML not conditioning number so in like patients. smaller approved might a limited similar

the population. that go to into in like what’s how So XXXX strategy differentiated could broader patient space? be a And

John Leonard

ongoing clinical is data we’re early stages here The David, is know, with trajectory the data the in I study regimens will levels. how And something levels to of talk David, that, that conditioning strokes, broad safety data affect that about early the if disease determine patients in we and collecting to affect safety program, with thinking I here? go. you think want extent about? mean, we to is is don’t might where and anything I want counts higher and and and add disease the I think where you the response path by PK Swapnil, just low profile blast the that question that the

David Lebwohl

is Thanks. David. This

intensive bone are that patients to patients receive transplant, unable remember I unfit cetera. think concern would not regimen. to receive therapy, your unfit be marrow et conditioning the And that is able

TCRs, of that’s is but used receive that the who are or and receive then this tolerated patients older, expected type for CAR-T group that more So preconditioning it a they of therapies. CAR-T themselves be to was are therapies, TCR doesn’t necessarily than mean can’t that which

considered we treating be be transplant. marrow point I some for patients to So otherwise that think able unfit who might will be bone

Swapnil Malekar

I then the about given part question concern like have And expectations the like remains won’t failure. to But ask the one you. be Thank on regarding I it. XXXX, trial, some Got data BridgeBio the there cardiomyopathy next of week. for

thoughts updated an any on your outcome like aligned BridgeBio? to stratification or of that in patient Tafamidis more get or there like than are selection terms – is like So to like similar rather endpoint strategy

John Leonard

on it happy to about and it talk about a think Monday. we’ll We lot be

Swapnil Malekar

you Got Thank for it. taking my questions.

Operator

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

Dae Gon Ha

our non-XXXX. and Thanks you stick morning. there's With the good I'll all Great, AAAAI also on release, specifically, questions with for your in meeting. regards mentioned a to congrats XXXX taking press progress.

second we talks it given as your second wondering cut there, pertains preliminary and the of So what biomarkers release the expect also half in interim about and announcement to half, proof-of-concept. press update, can the

you've So our should we should be for. kind you but else And that what prior related. disease of frequency, in drug much. we that out eye attack calls, I as related of anything very keep an versus kind safety mentioned won't also necessarily Thank event discount think in reductions it

John Leonard

the And focus we attack the advance. is have from some anticipate expect collect to get are of guide much on protein And way with observation a purpose you're in knockdown. for we on information We'll for more for done kallikrein, information, be to observation we're should advance sets, that data able should this to Yes. about in do the likely XXXX, as utility. What others, the that the that respect I for of thinking if terms is Thanks similar of benefit place ultimate this that. as program period, in guide determine this question. the mean, we've we on presented is and PTR to offending the a speak look we but in which protein, all half clinical use what as be I We're for on benefit, And said first collected it about particular will the you're the for what to you that the time we'll do the clinical agents think case. sort the the we what XXXX share rates. study. guiding in we're looking the effects earlier, work a that very to not then and extrapolate way looking an is as obviously to therapeutic be, for would of phase and attack be should the doing focus over information the with the is, target that, any is effect second of give what XXXX, not primary notice in appropriate to how rates we I you and to after longer

more So year unfolds, about we'll would talk but that for the as now. about think a it way that's I

Dae Gon Ha

regards with anything to And AAAAI meeting?

John Leonard

we've don't what at I talked yet. present think to which meeting specifically we're going about

when, available greater and provide how on focus the that we'll about as additional to at the information to website. our point the specifics going exactly and when information our permits this but again, we're about, that's So meeting website beyond to place on available the meeting guiding into that we're we but once it talk anything time are and we'll not meeting, present that, make what talk some comes will

Dae Gon Ha

Great. See much. you you Monday, very guys thank on

John Leonard

Thank you.

Operator

[Operator next Luca from The ahead. question comes Instructions] RBC. go with Issi Please

Luca Issi

Maybe the Monday. about but tropic last not comment and for you a much. can question, Great. a to marrow question I that what's ask if Wondering next Thanks on tried Thank program. year. flipping for to particle you. I’ll on one limit bone data exciting so behave

John Leonard

work next species. And do scientific when research important well, engaged translate an view to more a it's us. One that the level where that in. that at as that is We It after the the very liver LMPs one so we'll will we team the of have tissue actively and present, appropriate we're very, target meeting. for to working service into data is actively

Luca Issi

so much. Thanks

Operator

next The go Please King H.C. Wainwright. with question from comes Mike ahead.

Mike King

morning will some and of wondering ex vivo with clinical how should with modalities, we programs, what to thanks. differences to think of recently with challenges respect your been AvenCell about and in the proprietary also I guys. space. you wanted have respect regulatory that in only light just bring just ask Kyverna. Hey, to Intellia the but seen Good Not to

John Leonard

Mike, you anything general question. to that want very to in us Do speak it’s have a mind, you or

Mike King

guess holds clinical challenges some that's framework in have my also Well, placed there some others, been kind been I of there's programs, on, based just on been with durability so I'm – question. there've

John Leonard

Yes.

that up Remember thanks. So, running. XXXX is and

speak have have what case package may the they've And preclinical to, U.S. it. we've that have an that work our been the goes done. IND, with the and We satisfied can't with preclinical despite I been work seen with the which others, regulators and the in all

patients. we're so actively enrolling And

to which approach the fundamental this the material translocations. And think very predictable to go what’s preponderance no that questions the starters done that we in to in You space. use programs, is about We lipid something editing, way the unpredictable into a just that from of to tried take genome use introduces nanoparticles we work for different presented – And terms that. about with matter but this fashion, back we've with beginning how lipid et modality asking of just using nanoparticles. that cells how the all the genome overall that now in breaks is we've with electroporation, editing electroporation get is biology in and your We've results. in is happens data back contrast cetera. And gone

first have essentially You themselves are transduced levels in have indistinguishable been the that breaks of never place. of translocations

So in we've and of that the off-target is our others low effects, is go it starts B we judgment carefully the have work what effect. levels quality low genetic different and very distinct disturbed very, architecture. what measuring of it And of A presented very we nature guides, sequential you monitor there, that levels the as we to have seen And we from changes. the then the genetic do of that introduce relates

a So good in that with puts that basics. we just position, think us the

we what allo other well to I they're presented not of surprising that we're it's that treated because reconstitution taking durability a lack programs And show not those more pre-clinically it. way we'll data spades, to We've NK XX-K cells, programs short in outcome attacks. with think for that in by allo cells stand happen. approach. known We respect to one. And we've system, as that seen on susceptible a we to lost share media do with year that different falls where that's program, going the we're our about remove cells, the don't our trying our goes do, immune fact, find of those of seeing matter that with with Now, judgment class where in weeks, graphs in that

for patient persistence So will we really of presents graft, think that the think but just is is which think address having which goods. problems we that of the a the that one fundamental important not material we ultimately, space, in the also well when of for having augers cost the ready efficacy. And

clinical allo have those and data forward, addresses it altogether, out moving we're believe will. programs, that play that shows are the So we with we'll hoping in points the does we're thinking fact way

Mike King

Okay. Appreciate the answer to question. the

John Leonard

Sure.

Operator

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

Ryan Deschner

house Hi is for Ryan on NTLA-XXXX. there, this for seed Steve Deschner

AML you You an therapy with this given about vein Could you anticipate noted that is cell enrolling prepared? patients the therapy. talk being to time? began while you’ve patients the problems is progressing autologous vein Do

John Leonard

and three find That's will vein proceed, expect in time all translates we as the and we cells effect them. from And been that preclinical in light aspects think transduced work that cells preserve the be remember, of programs directly returning removing considered of around of the the into our grow We we've quickly we time it the viability. in our weeks. we've well, vein clinical that we our but shown to the benefits – terms shown LMPs and between that light have benefit and

it we be pre-clinically more clinical So actually we but speak weeks. as we'll directly to data, around three what we get our would to measure, judge

Ryan Deschner

any Wonderful. had wondering progress Thank for on for of or quick timeline indications if you real editor an very you technology? your update much. targeted potential base sort Also

John Leonard

that, the not we And in setting. we to the vivo complex And space. vivo in we got. the one is what suited think that's as have mentioned like Today the guide we're I earlier, to position that ideally ex about more a base editors of in ex things think to but we've we multiplex edits knockouts

So little more as programs talk bit we'll advanced a about that.

Ryan Deschner

very you much. Thank Excellent.

Operator

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

Jay Olson

for comment Hey, XXXX related IND XXXX? the congrats gating talk the factors to progress. the for for And also question. you taking and size on commercial can the thanks then the for and Can opportunity you on XXXX that, filing about the of

John Leonard

parts. of into break couple a XXXX we So

hybrid The application first, is have remember a the LMP we this with the where AAV. coupled

respects many follows advanced in program LMP work previously. is data. that know it the the And we well is we that generated we've So that done because

that we're with of AAV goes with lot do the So that. of down the XX, template some work the a the road this and the and of case work well in to has tax

get nature going the but underway. anything not dates I'm the of that's so And work that, that's or to like

Jay Olson

commercial also opportunity Great. can XXXX comment you of size the the and And please on XXXX? for

John Leonard

this substantial it's in And of space excess it well we with opportunity up depending going we're that alpha-X $X you not billion supplies, at generally. Well, we in a plural And measured there's particular the read, number. believe billion. but on it's reports the to point think come

drug and that's available better value. almost in ultimate there's going I have that's available that where setting, get And which line I on to we really terms patients plays, of is and actual going think effective important it's to more that to think case is existing to is patients therapy the in no essentially it of therapy inadequate, currently. the perform. point a and the we'll to progress determine out therapy, But data is how of or what die for how And liver that's patients all these would replacement for them. sight

So that we think a space it's point. this wide open almost at

Jay Olson

Great. for Thanks the questions. taking

John Leonard

Sure.

Operator

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

Yanan Zhu

Hi. I program. on taking questions. brief question my a Thanks very AML the question a HAE have on very, and for program the

is going, year? question your to are brief very arm about on arms higher the Phase arm the noticed disease is into selection maybe arm. any there the that in parallel And could we first? clinic the dose Are reason? is, data and And you. burden or two other expect levels, contrast a some going little AML study, Thank in different lower this this for is the burden going they disease two dose to there one either from X study the Is in you in effect I the anything which ATTR of X program. Phase or NTLA-XXXX portion makes on HAE target had cohorts particular that designed different So

John Leonard

on But the crews ML We're the Lebwohl And the David apply they're what data. of and study to which program the going really not same short our in on function will, in patient second disclosure, data said and the we the about logic we've the the a at rate to to focused we back can, first that a sorts function as of be two what parallel enrolling really is address data it's and cohorts but getting it's question we program. we've of and now. those of we're to patients Well, can disclosing the a answer progressing and committing principles HAE we place. programs, look will at had other that to as have soon that

word you So, David, program? say how HAE cohorts just in do want you about different think a the about to the

David Lebwohl

Yes. thanks. Hi,

So we program HAE about do the in somewhat dosing think and differently program. TTR

There's reduction a see as So possible could in that proportionality to be as evidence getting beneficial clinical TTR and amounts of the TTR the good program between patients. for benefit is that patients. in low we'll that good

data higher. that. with XX% just of reductions, an you saw where HAE or going XX% the that even major reductions lesser as we the upwards pre-kallikrein, So there's with get the in XX% in And on program, you'll data reduction attacks very of more we're see Monday aims

the have will course, we go dose a the two have of very looking levels. we level also that different X to at dose what single what we on with Phase go portion. to with, we dose need So potential dose during be depending forward careful escalation find Though, does could forward

Yanan Zhu

you. helpful. Very Thank

Operator

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

Silvan Tuerkcan

and taking questions XXX brief I question X. morning. for had just progress. a the thanks my And with Good about congrats

changes But that. allo you table persistency changes consider manufacturing. of changes, you're that if your touched could with dosing would potential consolidation seen like have in trial we've initially, you tell exploring a on potential that and re-dosing even kind if about this you You you. us will in initially off may or great. that's thinking the Thank That CAR-Ts, you're in be

John Leonard

X are for if in re-dosing trial for Phase the any what XXXX. your David, plans

David Lebwohl

Yes.

autologous an program. that So we XXXX is call

don't program. anticipate So we this in any really issues with persistence

a now it as right is planned single So dosing.

Silvan Tuerkcan

you much. Thank very

Operator

one for Chattopadhyay will Okay. today Debjit questionnaire And go ahead. with be Guggenheim. there I that that is believe Please more time

Ry Forseth

in Hi, on on online albumin for questions, consequence Debjit. secondly, the the XXXX. albumin levels And Two gene levels? tissue this XXXX, Ry what's facility anticipated capacity GMP Forseth into comes and when what's for of quick is knocking albumin utilization the circulating

John Leonard

every most first virtues the one single in doing do the of watched highly carefully. preclinical albumin. the we're done. albumin work probably We've And expressed the is knocking very, de of I'll we've into not What XXXX that very the which something Well, had on effect a locus. it's the we've albumin in gene minimis is That's body. locus

protein lot need, of And of so drive primates, levels that at same circulating humans of of non-human preserving least the the locus. levels normal that that can you you in albumin of while protein achieves what function a get produce gene the high accomplish introduce still exactly you you so there,

really talk about ideal think to resource. an excited a the road. that's capacity facility to that yet respect particular a to in the that's position it's facility, that that and we're place we're I'll utilization GMP drop square say insertion. It's down the we not So about With XXX,XXX substantial in feet.

only going we you to space, position collaborations on, efforts think well. what our those as about you it as and some our as commitment If supports to working you facilities increasing forward the us go that, our out that of important And provide put appropriate of an see some carry research realize think in judge on a not that and resource but part. really other in we substantial a excellent is it's to we're

more something a excited be all in I'm facility. and position about plans that about just know, our we're it's you that to proceeds, you as So bit sure a for tell work we'll little our

for thanks we watching So and that. appreciate

Ry Forseth

you. Thank

Operator

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

Ian Karp

at is your the PM for to week on at look everyone and Event, you not, interest our see Great. And February if of joining support you hope many, continued Investor Time. on in thanks to much. updating for we Eastern forward all and us next XXXX X:XX which we today Intellia, Thanks so XX progress, NTLA and

great So then day. see and you a have

Operator

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

may You disconnect. now

Intellia Therapeutics (NTLA) 24 Feb 222021 Q4 Earnings call transcript