Earnings call transcript Q3 2022

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Operator

Good morning and welcome to Editas Medicine’s Third Quarter 2022 Conference Call. All participants are now in a listen-only mode. There will be a question-and-answer session at the end of this call. Please be advised that this call is being recorded at the company’s request.

I would now like to turn the call over to Ron Moldaver, Investor Relations at Editas Medicine. sir. ahead go Please

Ron Moldaver

corporate Q&A. Good of conference And the remarks, we for call. providing after on After and press Maria. call completion. of third be its website will issued to will approximately quarter we call release available recent results financial XXXX Earlier Investors our prepared A morning, you, open replay welcome today’s section everyone. the our a update. our Thank our this hours morning, two

As remarks statements future various this plans Safe subsequent statements various by XXXX. recent the a most about and on call which these results result of in file of Act a SEC discussed under those with purposes by Actual XX-K, forward-looking from important during reminder, updated including Private report Risk may indicated make Factors we our that of of Reform materially the the Securities is constitute filings. on Form the those forward-looking section factors, the expectations, company’s annual provisions for our Litigation Harbor prospects as as differ

representing required In our views subsequent disclaim our change. forward-looking statements as as of views if update forward-looking only today as any should by or as law, any to not we relied date. even obligation upon views Except and any be any statement, addition, of represent our specifically revise

O’Neill I over Now, Gilmore our call will turn the CEO, to

Gilmore O’Neill

Scientific good Michelle morning, Officer; Mei, the our Medical Ron, Officer; I our and of Baisong Chief Editas Financial by Officer. Robertson, Shearman, very Thanks including members our and Mark Chief joined team, much everyone. am today executive several Chief

team this am quarter. with made I has the our pleased progress

execution. we a on continued on technology foundational company a transform focus company to into have company's our stage therapeutics clinical We from and platform build as

ways we evaluation therapeutics, leverage update our coming We in the months. developing provide evaluate continue will editing this expertise and for to gene an to new on

priority programs. top highlights that, would is on pipeline advancement, I some focus clinical our going with like forward. to execution a clinical Operationally recent our provide With on

cell provide unique to mechanism of high with transfusion-dependent patients First, designed gene in thalassemia. TDT disrupt This suppressor. the reduction is for the thereby, and BCLXXA gamma-globin EDIT-XXX sickle and severe to hemoglobin in levels cell and beta in the EDIT-XXX disease binding action blood added and of of of sufferers fetal promoter that reduced durable and in utilizes sickle patients. TDT, sickle red cell sickling resulting cell anemia disease a

our in that editing I EDIT-XXX program which higher our data suggest would nuclease, and uses note results higher fidelity AsCasXXa also preclinical our efficiency engineered proprietary CasX. than

reminder, the a drug nuclease. initial time represents As using vivo product EDIT-XXX with edited ex that our first AsCasXXa was patient an autologous engineered the dosing

RUBY have the disease and the year-end. would These Phase on first initial the to patients. X/X patient data cell cell as from release data patients, from treat safety the first track trial second as EDIT-XXX data In include dosed treated disease, we to well RUBY twotreated evaluating sickle sickle the data remain from trial by efficacy

have completed we and are on In and first the the apheresis patient dosing. thalassemia, for scheduling transfusion-dependent cell study EDITHAL CDXX+ X/X beta Phase editing currently

of optimal the consider a or who to identify by study and retinal visual photoreceptors of X/X us physiological BRILLIANCE study. that for retinal talk to benefit-risk the EDIT-XXX EDIT-XXX molecular, objectives. dose achieve end inherited patients, autosomal that The LCAXX several identify a to therapy for devastating is safety by We is recessive turn EDIT-XXX and to caused early CEPXXX cause the Phase likely which clinical most Let determine about subpopulation baseline benefit to designed from the are a parameters, registration and identify now LCAXX, delivering dystrophy a severe mutations or of optimizes to study. to balance impairment characterized points BRILLIANCE blindness to

An update readout Data efficacy XX on the and and CEPXXX That include natural through month company-sponsored on all on BRILLIANCE LCAXX our press safety release this BRILLIANCE history high-dose will webinar. mutations and provided pediatric the will patients be a contextualize be study dose trial the adult in data. of with from cohorts. patients gene to one-year used adult, mid- and the would

In will to would treatment see for registrational provide study, his viable Baisong order further forward to to move segment. details we patient meaningful benefit a need commercially a in a remarks.

the CRISPR-based retinal the our have side, diseases. safety utilize for we programs the on platform and been drug in trial, well use our in as as at reinforcing far, BRILLIANCE tolerability to provide a able laid Thus presented will Mark inherited demonstrate recently potential from retinal the an EDIT-XXX, subsequent foundation data meetings. EDIT-XXX of preclinical the that Beyond summary pipeline delivered safety program therapeutics. AV-based development on of vivo AV scientific update

to cover or CasX turn our and the basis the therapeutics sublicense exclusive in at our in now portfolio, a exclusively non-exclusive University our an either license, IP Harvard that exclusive me US. use for discretion. the Let patents of Broad is Institute With licensed on which granting human includes from

Broad As for parties know, PTAB, known CDC. Circuit, twice Institute the Federal you as once and earlier third collected time, with against this prevailed the at year, the

Circuit most ruling the was law decision the the determine PTAB recent anticipated, the review and to Federal As will CDC whether applied. properly appealed the

its are product that a evidence, need commercialize Editas's confident the And exclusive US. in to prevail. and the new CasX decision expect companies a court CasX favor and licensor plan that in the US panel in a Editas. from of in decision Broad not will would once product We the the hear position that deliver as again the therapeutic Broad license the utilizing that will will court XXXX. court mid-to-late reaffirm all patents Thus, developing will The remain we

about It addition EDIT-XXX is remember things on IP important the two in to front.

development. commercialization. IP to CasX believe our proprietary which the any providing relates need a driver CasXXa for our We therefore, be uses forward our not not the late-stage is subject We including look with license updates. in in of strong for and value additional IP development significant it nuclease, will to CasX competitor CasX, the several First, therapeutics AS company to clinical disputes, using numerous that potential EDIT-XXX a products position US in human has

I to Medical of turn review the over the now Chief will our clinical Officer, details Baisong, call programs. our to

Baisong Mei

you, Thank Gilmore.

Let's start with EDIT-XXX.

As disease Gilmore noted, recently Phase we dosed second patient RUBI the sickle in a study. cell X/X

patients. data month. added patients Data study's test and Beyond dosed is two the Monitoring RUBI several XXX Independent available review first Committee, The apheresis expected or EDIT-XXX, have this sales completed also with we positive additional the from IDMC, successfully to

to dosing we parallel has committee the protocol, per dosing. endorsed Once further patient then can move

accelerate sites. expansion of our including trial multiple taking to patient measures are recruitment, We

able the data be RUBI will include in registration treated We the all to from efficacy patients package. trial in future

released and data from as data both what will of an fetal hematological to parameters one We patients, platelet expect patient key update by So end from those provide to cells. F on cell total as safety and engraftment and plan both the neutrophil erythrocytes, this year? dose hemoglobin with include tolerability hemoglobin RUBI from hemoglobin, measurable fetal patients as known that construct from well

fetal after the the Fetal sickle we're Taking Therefore, at Hereditary complications patients a HPFH five a XX% occlusive dosing. usually In hemoglobin achieve the gamma-globin is in when vessel as damages. end-organ is level or Persistence least have to Hemoglobin nature promoter, of mechanism threshold, or cell XX% back, HPFH. step no months four of with limiting to level EDIT-XXX fetal aiming targeting patients at hemoglobin

expression. and EDIT-XXX, can by high Engineered our the we targeting promoters with objectives, If would of and and in this AsCasXXa meaningfully gamma-globin these specificity our increase provide vaso-occlusive to achieve product. efficiency editing fetal clinical cell and differentiated this competitive confidence a those X, hemoglobin being By using leading disease. X we expect Gen long-term Suppressed robust deliver with benefit sickle crisis will

preliminary development transfusion-dependent EDITHAL beta efficacy designed the EDIT-XXX. Phase our safety, efforts assess The our tolerability to study in of Moving EDIT-XXX. in to thalassemia of is trail X/X and

the The enrolled and completed been study first apheresis. in patient has

We editing date patient, for scheduling patient. to of cells CDXX+ we are the and into this have infused dosing be the complete back the

studies over turn part planned The LCAXX. EDIT-XXX of to normally meetings. me recently met Let for the as BRILLIANCE IDMC

a has profile. satisfactory that EDIT-XXX pleased are We safety maintained

endorsed its review available the continued cohorts. the study of active BRILLIANCE Following recommended enrollment all and data, continued IDMC a has clinical the in

to data update clinical from will The two the from pediatric BRILLIANCE we an adult including results available the one patients study mid-dose month. efficacy are mentioned, track of and was on year update XX data The available provide cohort data from adult efficacy cohort. data adult safety on months patients. and six adult Gilmore adult As and update include on this on high-dose

disabling visual multiple These demonstrating meaningfulness clinical The LCAXX optimal significant endpoints measures efficacy-related patients, suffered from visual The psychophysical endpoints. for early outcome identify to has offset BRILLIANCE is best-corrected who goal in impairment. measure visual life and study BCVA National VFQ-XX acuity full-field outcome, visual Eye including navigation the including that function including sensitivity, measures outcomes quality visual including course, outcome, instruments. and functional of that Institute of or

progress making their with the clinical the these contribution I'm new advance support that members are our site patients, the execution the at program, for we and helping study investigators our to want happy and thank and in of us therapeutics. in we staff improving

our now Chief With I to call preclinical will that, turn our programs. Scientific the Officer, Mark to discuss over

Mark Shearman

with simultaneously start aberrant knock I mutant Baisong. pigmentosa. with one. and gain two a virus correct while to the redoxin adeno-associated rhodopsin EDIT-XXX like function, autosomal-dominant toxic uses you, to EDIT-XXX the would Thank functional for that out retinitis vectors gene of replacing

XXX gene cause than data. mutations different employs highly RHO-adRP. mechanistic previously EDIT-XXX preclinical The promising that approach a reported and potentially This address can have we approach more program in

the annual which editing & productive Last over highlighted effective month, during to an oral replacement, redoxin presentation that we Gene nonhuman primates meeting, study. generation Society Cell XX% nearly NHP functional of and be in therapeutically European data demonstrating XXX% gene in the Therapy proved of at

year next testing. We expect following IND-enabling the initiate of analytical to vector completion studies AAV

our cell programs. Moving Ex-vivo now therapy to

program over existing EDIT-XXX important is This for several tumors key towards offers studies. iPSC-derived cell approaches. advantages NK program NK Our cell IND-enabling advancing solid many

potential EDIT-XXX antitumor a substantially has cell preclinical and we've production, introducing cellular exogenous activity increased utilize models, material. of feeder We shown mitigating system potent In cell-free INK thereby for persistence. risks that

able cells. knock-in select abnormalities and And believe clone, such and editing NK editing differentiating avoid which development platform into technology, NK cells. our we sleep engineered the process, provides capabilities we the are us our Through the fully proprietary characterized engineered to program finally, a superior potential in iPSCs AsCasXXa utilizes helping nuclease as

the CDXX cytokine supporting killing regulated showed cells of new data and Last thereby had further continued well cells edited presented using in-vitro serial membrane-band in development to exposure, data CDXX, as continuous persistence SKOV-X enhance preclinical the of month ESGCT, knock independent prolonged tumor cells. expression and after tumor significantly that cleavable cell as The EDIT-XXX. at SLEEK IL-XX of the to EDIT-XXX enabling we

demonstrating survival, with model, rate SKOV-X reduction overall significant the combined intravenous days using tumor a cells to in after no-survival EDIT-XXX X% an when the XXX% and solid compared tumor XXX In antibody. burden a in antibody, resulted just the increased

As has Cancer we continue Immunotherapy next an development therapy approach to activity at with tumors. Annual medicine of off-the-shelf solid Society data of week. the the EDIT-XXX, potential on our additional enhanced We presenting plan against NK believe preclinical Meeting we cell for allogeneic create

the Chief hand call review Michelle, financial our now our I'll to results. Officer, Financial to

Michelle Robertson

Thank press I'd morning, release you, of for review and today issued earlier quarter refer our third our good everyone. items. to briefly you XXXX. few to this opportunity the summary Mark, I'll like take to financial a of results a for

to securities as quarter. of equivalents $XXX were million prior September million cash in cash, the compared and marketable XXth Our $XXX

expense We our runway with continue cash management our into to extends and disciplined XXXX. be

agreement. recorded we out-license minimal third an For quarter, revenue the from

same the program under year, opted in we our an additional into revenue $X.X last BMS During collaboration. when million reported period

$XX strong support G&A year. XXXX flat the our This third quarter the we third in of to by expenses as compared of quarter and Editas R&D last million advance for the programs. CMC our were of to with trials. quarter million progress financial capabilities million $XX compared for Overall, investment in driven remains were approximately a expenses clinical $XX of the our was ongoing increase position and third manufacturing

that, With back call I will to Gilmore. hand the

Gilmore O’Neill

the pleased past Michelle. over Thank am quarter. I you Editas' with execution much, very

edge therapeutics operational, company in leverage to a the continue transition cutting capabilities our of best-in-class on innovation. manufacturing our technology, We and into

our active platform, efforts, editing This further underpinned advancing discussions transition enhancement development our to to foundational and related IP. gene is by business

more of updates providing forward pipeline programs weeks. our regulatory We the in are months. And our a of probability We the clinical in the coming we EDIT-XXX commercial information that success. plan technical, focused our on look maximize in share robust goal of EDIT-XXX for strategic addition, to about building and coming and plans to assets

that, With happy are answer questions. we to any

Operator

you. Thank

Please from will with Barclays. with proceed question-and-answer first be session. Instructions] We now your question. conducting [Operator Our question a Gena Wang is

Gena Wang

have I later update the question. questions for the year. you data Thank my two XXX taking regarding XXX and this

should clinical be the for be -- be benefit. wondering XX% regarding given sufficient mentioned competitive be seen show that the your so will that fetal-globin What profile Just But sufficient goal we've will clinical to far, to that you fetal-globin be level? what would XXX, competitive?

collect And BCVA give the what considered is logMAR visual then will considered magnitude data like us a terms in several EDIT-XXX, sense clinically you you will that improvement different be for meaningful points. and Could navigation of meaningful? what clinical of

Gilmore O’Neill

result to in Gilmore Thank you of very hemoglobin we use of have based have of our product the actually enzyme, that our as XXX, a a determine the you robust we differentiated ultimately here. expression much, correctly durable regard believe to safe a stated in target a potentially We differences product. well on Gena. With competitive with some approach AsCasXXa a we would that fetal as as effect. the what to our in minimal threshold as see critical

future, actually hope a be for years, and itself -- of evolve we slowly. And we differentiation profiles and approaches, as move things other space that our that is belief product plan actually over the among therapeutic as around number the competitive see the few will actually, because stems in believe the there is would next space space that will that this and Nevertheless, to we declare forward.

slow very or will We launch, owing the a we factors time the the present a what but of are the vast hesitancy, confident untreated number arise around potential that evolution majority that anticipate from to our importantly, some around of uptake at will landscape. be the the of population result and payer of remain

what as our the study. and we on With based actually clinically have determine regard history to contextualize is a done that the we XXX, of around to we will sharing meaningful, more lot be work natural data details

to a important know work real that for of it's around this lot done parameters ensuring determining have we think change. reliable I that we on have

meaningfulness that remember dealing with to to inherited actually The functional improved an clinical visual important it's in be would to for that severe disease related ability by will onset desire see to change loss. and also, And early with participate because outcomes driven our society. of be we're patients their of and to determination mobile

Gena Wang

Thank you.

Operator

Our next Greg America. from Please with Bank is Harrison proceed of with question question. your

Greg Harrison

as on the the Are commercial to potential is you What it EDIT-XXX your itself just taking confidence maybe more could Thanks apply technology earlier viewing And in a level around comments for you the of program. the question. a in opportunity the proof-of-concept program? future of elaborate you maybe the the where as or other indications? could of

Gilmore O’Neill

Absolutely.

I first was really our has experience actually that technology in develop all, program the statement. correct purposes. in EDIT-XXX two that our designed, And to humans. applying So of It think you're with

the most things, this one, population second think or treatment. the of to other be is to response the I remarks, BRILLIANCE meaningful are necessary segments clinical be segment in And patient determine my things commercially a likely patient that subpopulation I've obviously, is will are population. that, amongst segment viable designed, or as with patient highlighted respond that to a that one opening what study of

Greg Harrison

That's helpful. the Thanks Got for it. taking question.

Gilmore O’Neill

Thanks.

Operator

Chardan, Our Livshits. with caller is call. next Please your from Geulah proceed

Unidentified Analyst

on this the a Hi. if Chloe design for submit pivotal be year. you [ph] equipments taking [indiscernible]? Thanks remain I to This an FDA and track question. is the on to for to Geulah. alignment wondering clinical was the trial from for And

Gilmore O’Neill

to but you I I answered the your said, Thank the I I Geulah. what question. think sure afraid was question? you, poor. very I'm sound know Could be want you restate

Unidentified Analyst

Answer better? question… Yes. that Is the

Gilmore O’Neill

Yes.

Unidentified Analyst

Yes.

to the trial you're feasible what [indiscernible] and FDA [ph] in to year data So gain the submit get to left on this the on a package and question design buckle feedback before FDA whether alignment check the the to is track was to package some

Gilmore O’Neill

data submit poor. like a very was am the you're what track is, take sound FDA I on are the a actually Julia, – that what Is to think correct? regulatory going XXX. would we clinical So the for to But study question discuss I to asking look package to

Unidentified Analyst

right. Right. Yes, that's

Gilmore O’Neill

Okay. Yes.

the XXX will actually rather, from actually to that objective Are And outcomes. finalize very we forward. use there outcomes should should go sharing design FDA the is that or go that move can this size require, meaningful obviously, data only of well, in to population to amongst obviously we will a determine trial should registration subpopulation I go be the with data with negotiation our forward the we sharing we and be our in things, With be determine, And based be interactions other -- of on that that we importantly, there a population decision. able could say, the -- clinical So as that the that month, or study. forward. is a will we cell FDA, would regard trial we would

Unidentified Analyst

Got it. you. Thank

Operator

is proceed Please question question. Securities. Truist from next Our Joon Lee with with your

Joon Lee

questions. Hey, thanks for taking our

For you the you're you. that EDIT-XXX, excited sounds rate versus should which and for Thank How using what clinic? when in it is IND to CasXX, And transduced step what's relate about? XXX% are transduced CasX think, area? claiming you're retinal the space? devolve area, expect why we for like limiting it And in editing but that does that the you

Mark Shearman

Thanks, Mark. Joon. is This

And rhodopsin. using conveniently as in because and rhodopsin particular mutant the the down are knocking a in human codon-optimized then a this we product, this with are know, approach where CasX with dual it you is replacing a nuclease we single So fits CasX this AAV. AAV

a non-human study, we in typically terms microliter In admit of the administer blood. we data, primate XXX a

elevation the expression that in within average the is change So XX% region. leg

the that untransduced versus from area tissue calculation. take the we area and transduced So punches make

more as testing analytical and research setting a to the requirements. taking in and we start. longer detail studies, that's you toxicology for around completed typically, there's a give we a this script, timelines explanation out mentioned and from study that IND-enabling when pushing had don't than the And study GLP specific is setting, the the rigor until we Regarding initially IND-enabling the on little anticipated, we've submission basically And work. start IND move

Operator

Harrison from with Matthew your is question Morgan next proceed Our Please Stanley. with question.

Unidentified Analyst

question. Hi. [ph] Thanks for Matthew. is taking This Gunjana for our

is two trial. the RUBY have we questions. So One for

be think into CRISPR do other directly So, or in patients or data could other should there the to you think SCD take we things you do comparable are some differences trials, account.

off following your the about is second BRILLIANCE comments. The

package potential in in So in to patient to adding for FDA what adding of terms of registration trial specific upcoming of trial segmentation endpoint terms should include update clinical update expect the submission, trial? potential we enrich registration in or

Gilmore O’Neill

Thank much. you very

with in So plan data we RUBI we and will regard be that reading that data to would be and to the December. the believe that show competitive. are will to trial data, And share we share the out data EDIT-XXX we able

As the therapeutics said and believe previous that in technology TDT to I patients. spaces for competitive the have and believe we actually require potential my sickle remarks, product have very CRISPR a the accept provide we that and and cell we multiple using can

With data Baisong plan to want we'll next that or the steps add? regard be later set actually, to this share in month forward. Mei, be share do will to That able you data, – talk of to actually our we about November. to And XXX able this is month.

Baisong Mei

Yeah.

that RUBI earlier. study, on about question add the bit also Matthew, and related to to Tina's a Just

fetal target hemoglobin of the of level. the observation HPFH it's of is expression the not XX% hypothesis level hemin our on fetal my in comments, clinical the So based really about of and

we with Gilmore product, the mentioned, this we we looking year. forward data and a differentiated As later to share and believe you have competitive are

Unidentified Analyst

thanks Okay.

Operator

from is question your Ha with Gon with question. Please Dae proceed Stifel. next Our

Dae Gon Ha

I on of efficacy BRILLIANCE. data good congrats data before I end heard the progress. taking think one. Baisong RUBY, but Hey, all go patient by I Thanks RUBY clarification for questions thought and I On year, two Quick morning. I to heard from on our say

if can great. clarify So, you would that, that be

BRILLIANCE, two-part of I In more of terms a question. guess it's

data this of say be be to the what at comes presentation earlier month, it about number that for range pertains that Gilmore, later we we efficacy. are had ProQR would enriched thought So, as talking it when the might turned year, out this what efficacy to completely you viable, kind population to when of And prevalence? signal opposite they commercially see considered

think see population? future handicapping month about you. Thank of So that later how how do you outcomes be and what sort of EDIT-XXX/LCAXX and could you predictive in this

Gilmore O’Neill

Thanks Dae very much, Gon.

the RUBY. So, let me clarify

from be data two safety data will efficacy patient. want you expand? Baisong, presenting to from and patients may We one

Baisong Mei

Yes, yes.

clarify just the So, to -- patients we reported the recently last these second first during that, on updated August, in update first we we patient. the the have dosed dose have then we patient

the cell to gene duration have observation longer it program, efficacy will that expect patient and of will given first time editing meaningful data. So, take this gene a see therapy sickle we

data the be that all we first feel the share Hope will meaningful efficacy perspective. from patients, We data two helps. may for an available more just patient the

Gilmore O’Neill

population, patient very first at of determined highest Baisong, at intersection the of turn around factors -- the segment, commercial of essentially effect actually in let determining and to meaningless to and clinical me BRILLIANCE will and addition a Thanks magnitude And of size by of the size. number is an the that regard will question viability segment Essentially, actually the these population when the that level, be data, the the I the we with question. the much think viable. of look definition to

your this disease. patients a unfortunate very second With with around that to question, regard was outcome obviously, ProQR, devastating for

were it's think genome an between they we're to approaches important tool, and highlight RNA that are antisense-based significant using that editing differences delivered and AV their chemistry. there I an using

actually -- go I it around you. a that. Thank you, are more robustly And to very we detail we being in patient the at our into and share able we look But segment population we'll be predictive think as a importance of of with able conscious data to manner.

Dae Gon Ha

to forward Thanks. it. Look Excellent.

Operator

your Please question. with Co. question from comes Phil proceed next Nadeau Our Cowen & with

Phil Nadeau

Thanks Good questions. for morning. our taking

us. from two Just

I being First, that measures. to able any you the have don't that that evaluation? going on crisis that, efficacy that that you. mentioned remains Will heard on about you or you you early on – is say data to first patient's be patient you're that with clinic anything what Thank on part second, on XXX, believe RUBY release in any events I too efficacy before the data then enter of you've -- present program. it just And or data can from

Gilmore O’Neill

the going ask question. So to answer first I Baisong second Mark, the and to am question

Baisong Mei

Thanks, Phil.

data, we efficacy I most will But I RUBY be safety as hematological mentioned, may well meaningful, VOC. the provide as of certainly report report earlier, a parameters. duration mentioned system key For but a as We will as observation not will short-term data on that.

Mark Shearman

Mark. Yes, Phil, it's

at think necessary -- IND-enabling – for support the have some perhaps completing in mentioned I year. that's We're any we discussed earnings pharmacology as time conduct call. animal tox and script, the the the design that's data planned next to and last we study

Phil Nadeau

Thank you. Perfect.

Operator

Our next proceed is from Olson with your Jay Oppenheimer. with question. question Please

Jay Olson

you the group. Two for taking thanks started from the us you EDIT-XXX, remind us dosing questions. can questions Hey, if for pediatric high-dose

for you And pediatric I you. cohort you any on can provide Thank last think just year? you'll IDMC about SITC. data is an And there of the then update you that be was at presenting there mentioned review had previously mid-dose EDIT-XXX, can that? that talk

Gilmore O’Neill

much, very Thanks Jay. Right.

XXX, that So address. the with Baisong, SITC, to regard I'll I'll to ask XXX and answer Mark question have

Baisong Mei

was IDMC it mentioned. months will planned, a to also although data and satisfactory not the impact it the as study in We about that plan safety continue this recommended as And they will cohort to not And mentioned, profile. adequate the a existing but we decision this about clinical we program. have the your I IDMC for review previously high-dose profile, they at data all did impact will regarding our pediatrics. previously – discussed safety IDMC. they normal program. And the Thanks additional meeting, month cohort at planned, as from the satisfactory have decision we with pediatric the the And of Yes. meeting. data high make patients are meeting as to a our about question. release three see recommend question just dose Yes, we before as the feel data reviewed scheduled This starting

Mark Shearman

to Hi. in yet, It's give Mark Yes, embargo. under here, really data not given the about position SITC Jay. it's we're that a specifics

ESGCT, in examples. mentioned did gave As at in some on data the I the tumor [ph] model CRISPR update an which killing script, as pretty present two vivo interesting and we

So, we'll -- data the track. on will continue that

Gilmore O’Neill

Thank you, Mark.

Jay Olson

very much. you Thank

Operator

next proceed Beatty is Please question. with your from question Our Joel Baird. with

Joel Beatty

Thanks.

gene you expected presentation and replacement would and Could editing why is be enough? levels. XXX% discuss knockout also For XX% to and EDIT-XXX, XXX% XX% to the it lead XXX% described last editing levels month protein

Gilmore O’Neill

Thank ask you, to going it. Mark. I'm to answer Mark

Mark Shearman

setting only clarify so can wrote mutant So, is So, disease which of dominant just necessary in the you is it's since up XXX% part of code to XX% allele. editing optimized the the the remove second that? XX% this The this and of the replacement, is be a a would and disease. essentially rhodopsin XX% of endogenous rhodopsin

showing had at and a replace may as the that XX% knockout-only that a a arm you the is model, loss correct NHP we photoreceptors with recall, replacement. arm, we were And able data which knockout-only arm. phenotype reported we well reduction had the as knockout occurred in In to basically

was encouraging. overcome very that of phenotype. particular rhodopsin loss XX% some a that know, Data could that are, system model that dog I'm in There data published So, particular particularly was the to rhodopsin. arrived you conclusion somewhat show thinking of the as a that around similar at we felt may correct [Indiscernible], model in which model you from sufficient

Gilmore O’Neill

contrast LCAXX, confusion So, program sure any In and XXX, or thanks for make down makes that addressing XXX generating you the an that. very want highest is no LCAXX, is be knocking to RPX the misunderstanding. to XXX to disease, which clear, why Mark. possible there's the efficiency or is room in just much absolutely the Mark inherited of toxic point have which gene function, that's gain the Joel, that

as of the that replacement of nature the toxic the believe sufficient. we're replacing we I codon-optimized allele as healthy a with knocking that therefore, well both that heard the and why are Mark rhodopsin. to replacement rationale the Owing we is allele approach, give you down And mutant think

Mark Shearman

just one And other maybe comment.

high such editing program, getting is I think for we guide. are one productive a it's single reason the RPX why

and frame so of where we're dual introducing That's loss percentage. reversion guide productive a simply or indels, the transcription the editing, this different have shifts which is a endogenous caused and is you the deletion in the XXX program low rhodopsin. So, therefore, which in case to

Joel Beatty

you. Thank Great.

Operator

Our proceed with question. with Issi RBC. next Luca question is Please from your

Unidentified Analyst

Hello two platform. Thanks One Lisa NK the taking on our Luca. This have question. for everyone. cell on is I is for questions.

you there. seen any updates for press -- your that here partner you have the We suggesting to line some are pipeline the oncology have wondering NK Just if lately one-yard program?

number two, the measures any on additional from besides you to I comment CRISPR is you Are questions. in or just risk, And competitive. differentiate like thinking others? about Thanks on further cell given wondering failure the renal also product if endpoints help your failure? stroke for looking you're sickle [ph]? Just space sickle order into heart could cell, taking very was

Gilmore O’Neill

executed. kind has And sharing very our to full Yes. through keen very regard With unlock platform NK past have NK been Lisa. to have partnerships. with updates, only Thanks forward we are to platform, open the we the we that regard but partnership and much, signed updates, talked or we so sharing of to, the and of look over any potential year will when

readouts later RUBY regard our our approach -- study. XXX – the as our our year will to initial be from With differentiation, and data that this

determining product. differentiation of of Going approaches are for to potential the considering actually forward, a we our number

opinion of discussions from we obviously, those leaders, those There in the in are details with actually them. are share future. with considerations, regulators key some we'll and are experts and ultimately you've I future, considering others touched we the outcomes many think on that more and our

Unidentified Analyst

for taking Great. questions. Thanks our

Operator

Fargo proceed with question. with question next Yanan Zhu Please Wells comes from Securities. Our your

Yanan Zhu

Hi. taking my questions. Thanks for

So level of think, you meaningful, that mentioned the cell HbF first level. target which more Baisong, HbF considered is sickle Could the program, bit on clinically minimum necessarily on I the level? a not XX%, target elaborate is the you HbF

-- you terms And the plan the month cell And where for update? make you venue do talk program, also patient, secondarily, in to follow-up for for about where the Thanks. the data cut for follow-up? this data of, length month first of of the in sickle terms could the the

Baisong Mei

Thank the you, Yanan, question. for

fetal based For mentioned [ph] sickle design clinical about patient as just cells. observation the the I patients, of cell that's molecule with that, hypothesis our sickle the hemoglobin HFB regarding on level, was the

we these how So XX% that's to patient, of get right.

clinical of So our have. we certainly not It's that's kind what target.

we aware to data were with later of this you looking field. this our year. fully share forward And We’re

Gilmore O’Neill

Baisong. With venue end for very Thanks to be much, on. venue the regard later letting before we to the year. this share of we will the data And the are update, the planning

to data, early, would space. patients, we this parameters, with have the disclosure. disclosure, for the Then regard the the phase safety hematologic as follow-up length outlined the has the with efficacy data that would XXX the plateauing for to regard occurring on will that two should in of and and based data, experience our for be in cut to on we be But certainly anticipate data close based cut

Yanan Zhu

Great, thanks program. the for And LCAXX the on color.

only. I heard pediatric for meet be data safety think those the the I patients, will

from data months' at to have So, two three when would expect least cohort? the pediatric questions, you

and of BCVA those the the tests And results? the measurement patients patients these reliable also Thanks. could perhaps navigation, -- provide and and could also pediatric FST perform produce for

Gilmore O’Neill

Yes. much, Yanan Yes, follow-up Thanks for correct. very those you're questions.

share give Our clarity to duration mid-dose cohort. follow-up disclosure you plan the is on of no specifics. at the the will the and time of we And safety pediatric from

bit just the or behavior don't outcomes want to the BCVA if not Visual one to it I And actually is but courses, that. comment on but to long-term History the amongst that, Natural those carried in other of with multiple add Study important may across we was regard know things, Baisong determine I to that actually what note a age VFN, reasons including progression using, and of was, that BCVA, the groups. have of to Baisong, and of that you context the Navigation asked that was disease, the disease we're understand out

Baisong Mei

Natural Yes, History yes. question we Gilmore Study. about Yanan study mentioned earlier why endpoint, good the the a well this as as actually is the about of that sure. conducted That right, premium purpose as

Visual adult fully be to is actually use will for out it we challenge Study for be FST Navigation patient, from could as not understand you Course. only to patient that. the The other so the able for too, contextualize, we data Natural So, pediatric History or pointed

those of retesting and testing So, and Yeah. we are reliability variability endpoints. all the analyzing

Yanan Zhu

the Great. for color. and thanks all Thanks, Gilmore Baisong

Operator

Madhu from comes question Kumar next Please Sachs. Our Goldman with your proceed question. with

Unidentified Analyst

we [ph] two have questions. this for here Madhu, on Hi, Mario is

the oncology hemoglobinopathies should how for first, programs? X-ocular therapy about versus we development external or for cell So, particularly pipeline think internal partnering programs,

then And second, Inflation IRA candidates? or what expansion impact Reduction Act pipeline could the have on on indication

Baisong Mei

work that we said are Mario happy have continue portfolio the prepared and across in little much earlier sharing the I've remarks, the and in executive we months. to forward -- With as the a looking we regard with actually as and my strategy with Thanks questions, internal -- I've been team to very for excited [ph]. our our I your done evaluate coming pipeline, looking very in

technology. think, we the by robust ways our a full as that been unlock partnerships thing to I the of well want focusing enable can potential will has share of believe that the I to to to that expand months. able its we find very I technology internally, said to fully more we in our say be say, and consistently seeking one is bandwidth as appropriate have and powerful potential But coming us the about But we as pipeline realize to exciting

a to need. has it unmet looked important there their high technologies struggled the us regulation pressure, to with the certainly IRA, thank talked restricting for you human at been We a target, know and is and approaches, new And develop legislation difficult IRA of that. to is there think I -- we bit very and diseases have that believe that that it, companies designed with and around actually some I and we -- enable actually has it's regard that about to challenging

optimistic context technology And innovate, of that so to feel we continue operate the innovate actually the written. way very or within can and is regulation the into our IRA the

Unidentified Analyst

you. Thank

Gilmore O’Neill

Thank you.

Operator

comes with question Please your ISI. question. Bayko with Evercore next from Our proceed Liisa

Liisa Bayko

insights process. see question. of you I’d from Hi. marketplace how through cell the the just Thanks in progression can any maybe positioning regulatory fast about getting follower, through curious you're Thanks. be read or the for there? you taking And the then describe a as

Gilmore O’Neill

we're who's Yeah. the developing the an as editing Thank question. everyone Liisa this and very for much, will very be important as space, this specifically, in along for your you through therapeutics genome pathfinder process. regulatory pattern approval with excited, moves very Obviously,

out just the worth learn share us that as it's we exclusive for with for of there their addition, holding thinking the pointing regulators CasX, regard our beyond upside information space. In potential the license to evolution is the to an additional that

because the the timing progress we very uptake, payer high need of that progression very years actually it's prevalent and that over be With making launch. new of of might hesitancy very importantly with will we the and actually because still evolution the said by tempered what and of position the the believe couple space, and at be program positioning, landscape have patients unmet therapy regard that would XXX the to we our just the next good a in of in will time with the be we're the some awaiting majority

see. additional potential with the so we upside And there's differences our differentiate, and approach, as to our in

the substantial space Thank and those for and So product that space, XXX there our product as TDT need unmet product. that patients a will and of with to space we believe cells competitive our sickle with meet we you. is for say significant have product I be our

Liisa Bayko

you. Thank Okay.

Operator

reached session. for our We you Thank have end the joining the question-and-answer of conference.

at You Goodbye. may discontinue time. this -- disconnect your lines

Editas Medicine (EDIT) 2 Nov 222022 Q3 Earnings call transcript