EDIT - Earnings call transcript Q1 2022

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Operator

Good morning and welcome to the Editas Medicine First 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.

Ron Moldaver

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

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

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

Chief Now, Executive the to call Mullen. I will over Officer, turn our Jim

Jim Mullen

our Michelle morning, and This first Ron ever Counsel. Robertson, executive meaningful vivo dosed incredible the members We of pediatric our BRILLIANCE team, for joined Financial CRISPR was accomplishment. dosed trial Thanks, editing, the General forward good everyone. Officer the this using today first Chief to and the and Editas time an Chief made more our EDIT-XXX. including with am gene LCAXX I quarter this milestones child in And year. by Stern, look Charlene a patient we Scientific several marks excellent Mark progress Officer, Shearman,

yesterday immunogenicity EDIT-XXX. dosing at treating pursuing a that leading And is of we conference, presented this forward critical the supporting step to LCAXX patients the the inherited childhood profile of potentially one younger safety disease. data of further and causes Given blindness, the ARVO is favorable

ARVO We the the providing conference, XXXX expect we'll year. of in second data later design initiate year. dose our EDIT-XXX IND-enabling knock expect demonstrating update We're testing half on planning mid for pediatric of our out program the high our half unique cohort EDIT-XXX Later cohort pediatric towards at in advancing with we complete the also and present clinical and of That this in end of vivo the non-human to dose to of today, the candidates year. retinitis for studies primate replace retinal which start by the function. is to preserved pigmentosa, first

trial program disease cell for beta screen to thalassemia patients. Our sickle and continues EDIT-XXX and enroll

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our dosed first for EDIT-XXX study BRILLIANCE for we LCAXX. pediatric mentioned, As in patients

the we've a significant many will field. second the we editing been diseases. The viral has continue the that disease scientific the and of to patient for sense later initiate gene first This data apply EDIT-XXX. and of the favorable considering most can months data been young is tolerated cohort data exploring we the development, clinical cohort. we will entire to sensitive year. the as for level ARVO other data and registrational assessment information, To the the viral trial safe we indicating living CRISPR we least the And the the viral with to evaluate the including of have monitoring to profile. relevant using AAVX. while will those dose comprehensive medical how enormous levels said support at This also to past, safety shedding, their months vivo complete individuals cohort, endpoints and no administered therapies, as our from EDIT-XXX EDIT-XXX dose data, as a including gives all ocular at committee The on administration. milestone no the patients. shedding six as continuing gene on we're families, least in and at transient, safety was interest provided medicine begin on adult presented least genetic independent lay viral supported well evaluations, and for dose year. had editing first The trial anticipate child. cohort effectively of up trial pediatric the adult what which shedding high yesterday to as in assessments There's that to time the patients on meaningful this and program's follow with of of months high who of capabilities mid dose The the community pediatric dose between expected foundation received interpret data Then at the XX This we persistence safety patients. conference half is other four correlation systemic EDIT-XXX registrational patients following six data has trial treated mid clinical ongoing expect overwhelming, year. in date, before we of gene enthusiasm and update following it will well the most which the of of A update second based efficacy plan expect We BRILLIANCE be and dosing more demonstrated us the on in has most is support always to half been the these the include

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trial is results enrolling study cell The to track XXXX, we're in disease RUBY patients initial for sickle Phase EDIT-XXX year and begin to first dosing Moving by our end. top the vivo ex expected clinical of platform. with line half X/X on in

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like to CasX. I'd the to over Charlene update to surrounding turn IP patent foundational on decision Now, recent everyone the the regarding call

And some Editas continues the one instrumental join have in in to people to her first this developing Charlene launch be of was the For thrilled IP and was to to company strategy. company's the Charlene. join background, I'm XXXX. morning. she us

Charlene Stern

with the intellectual interested companies development the Those have Institute substantial serious placed We intellectual means were and for exclusively Patent that importance Thank to Medicine was morning, Editas, recently reinforcing US position. patent in issued the using Trademark for Since for discovery. foundational to This licensed good favorable that Editas the property cells. was are Broad we interference scientific pleased positioned of in patents This our Office by securing to with the medicines in decision decision determining everyone. covering of property the favorable the make first diseases. second protections, owned use you, from human uniquely Editas's therapeutics. founding ruling eukaryotic Jim. available robust Editas to people those CRISPR/CasX licenses invent developing of CasX Broad editing and living And is

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Mark Shearman

We're Thank you, making compelling we've our data progress and with announced programs. strong just for our Charlene. preclinical several pipeline, of

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with survival against Society wild-type identify important vitro New to limit EDIT-XXX, single for in editing of gene It's and know desired, tumor complex when editing resulted of combination compared actual eliminating lines EDIT-XXX while or absence the ADCC tumor we and in extensively vitro in edits that reduction those an abnormalities, scientists is Conference. will in unwanted with and cell presented exactly we risk the off-target significantly the data, to therapy upcoming persistence show the at ovarian having of exquisite Gene We solid off-the-shelf types products medicines preclinical model vivo iNK and platform Therapy an which demonstrating chromosomal An can burden edits the have antibody prolonged We'll associated a process mice Editas precise prolonged could single be cells. ASGCT the that process, editing in greater tumors. the or allows cell to with active multiple against data cytokines. believe iNK clone cancer what characterize no plus on potential the for and selection the having therapy cell used Cell multiple approach clone clones because results. for American create highly we trastuzumab exogenous has want of and our example. augmented combined NK in our of cell solid be treatment in element

our CasXXa addition could potent [ph] this autologous platform BMS collaboration advanced opting nucleases Bristol-Myers into forward immuno-oncology. look our BMS and to The technologies, T create most to develop that therapy proprietary In new novel we allogeneic including The We productive important guide for cells BMS leverage program continuing is advance design Squibb cell with result partnership in-house a to CasX around of oncology unique seventh medicines in collaboration and our to the several RNA alpha-beta for our programs program. approaches cancer. cancer. with continues with IND-enabling programs, from believe our approaches programs in T in to and studies, these

FDA words expect, taken editing products. The the at Finally, gene as see and editing of gene that as few ourselves like bioinformatic Editas. the development see the our very to development. to human to already the on and guidance recently industry were ensure highly of I'd We our consistent development as for of being gene-editing pleased steps part human rigorously characterized and therapy vanguard incorporating is target we important of a we've gene would preclinical computational editing. issued is, are foremost on therapies biology on to say activities, products We software safe you the that off guidance with safety FDA

pay We've particular ensuring and editing to in our that, stringency genome components I'll attention to our always the also our recommendations With financial review test for priority product being and review and safety the FDA's call development follow development. over closely design, plans. required to and We updates. the patient and highest on to the manufacture quality turn considered placed Michelle guidance

Michelle Robertson

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

cash, XX, cash December to securities Our and million as marketable equivalents were compared XXXX. March $XXX of $XXX XX of as million

We which mostly similar same BMS. with by offset $XX advance the were last that, operating triggered it quarter driven first the management this million relatively back this runway for our expenses in period we slight strong was clinical will payments driven as by G&A XXXX. to increased XXXX, decrease from XXXX. year-over-year. be manufacturing position And and I compared investments. million Overall, programs. continue collaboration Editas flat Revenue by With first year, was the Jim. and was financial expenses remains early our This for the the slightly last expenses hand quarter of quarter R&D in increase of to million that first same during there were $XX expense compared the a was quarter quarter for year. our the no with our million for cash activity year, Revenue period million $XX for success $X.X quarter million $XX with into first to disciplined and extends were $X.X compared with

Jim Mullen

our looking We're of XXXX. set in and for an Michelle. exciting forward you, company programs Thank to milestones

our We're half update of patient we the and also the therapy We'll programs expect that supporting advancing patient in cell the high-dose to we in we pediatric on as IND-enabling EDIT-XXX enables cohort our and top platform further clinical that BMS mid-dose technologies, expect year the in initiate programs. EDIT-XXX cell move their cohort efforts our And We'll have anticipate well. for anticipate the With year T-cell data the advance future opportunities. additional our we dosing and a by internal cell iNK And pediatric platform SLEEK EDIT-XXX as goal. provide the innovative complete objectives, towards We year-end. for we second first the continue as of therapy and and alpha towards for development dose beta program. towards first the our expect XXXX half of advance such sickle dosing to the program top for studies, the data first business we'll innovative half development of clinic. first dosing commercialization as line preclinical to provide they release disease, TDT on

robust engaging answers. to with and We're these questions intellectual And maintaining pipeline, up a finally, plan interest I welcome and open by forward And We that investors they property all have secured it across of tremendous on productive I'll your the to we'll potential still the Gilmore stakeholders, pipeline by including programs our and the can from affirmed recently and our for of the was you broad support. with dialogue. bring to of ruling. to and once We look advances we that, many USPTO by of platform thank company. a again excited so technology a aim our benefit gene patients editing. foundation to

Operator

Thank you.

session. conducting Thank be [Operator Instructions] will We you. now a question-and-answer

Our with first proceed Truist. Joon question. from question with your Lee Please comes

Joon Lee

questions for cell We're from of on investors taking for update. a Thanks Hi. getting our lot recently. questions and disease the sickle

And I asking you follow-up. sort as any discuss duration, is So Can to size follow-up FDA the EDIT-XXX XXX - FDA have that? you get a and and information with steady approved. a for the what your around share the trial the of

Jim Mullen

seen to mean, point, different trial. trials haven't few the of will reason the dosing any competitors. patients we've have this - the any our than I At we believe - FDA through have from our engagement is don't after be next to the what in probably first pathway we we with that clinical completed Well,

outcomes looking whether anticipate XX plus efficacy they're for patients, bluebird the it's we're to or they're and probably that still I and or minus looking same that XX CRISPR else. durability looking anybody for, outcomes that So or

Joon Lee

Great.

so the U.S. most - question given strategy, position licensing your second is And your IP in BDN favorable the around inbound [ph]

of yield do you. in and foreseeable types what have BD licensing So in Thank mind you the future?

Jim Mullen

Charlene?

Charlene Stern

of going doing to these deals. wide do CasX, to of This true We're range that licensing. arrangements, both expect CasXXa. a continue licenses. both straight would partnerships envision and and will we I licensing I exclusive strategic for maybe be would well non-exclusive So licenses, as expect some as would collaborations, where others be

Joon Lee

disclosure have by of which patients data that you. to the pediatric the dosed strategy you're Thank first we Okay. I put a if disclosure. been until April first Or Can October? half, one, disclosure the wait such data quick patient expect prior can. EDIT-XXX for four is Since all something requirement And of of around end dosed towards will to you X-month data the your going pediatric [ph] in the year? and follow-up end

Jim Mullen

Yeah.

the that report the So readout, that adult we adult patients pediatric we about of expect we've disclosure a - you of from a more on out the we but unlikely have about. bit the we'll we safety - at said data rolling efficacy would October, of update that part, talked information. probably frame mid-dose, safety, the a outcomes that high to previously, the first to have obviously, which would November, that's little data October, cohorts we've probably thinking know, would try But try - complete about talked then sort on And actually to dose. have time is going the be forward before we'll we reasonable

Operator

you. Thank

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

Joel Beatty

Hi. Thanks questions. for the taking

agents happening the you And entering time are that that year? like some BD, next For of for if the you'd clinic clinic points have or or do there more that catalysts in could the within that to make before your potential sense? are lead see certain so,

Jim Mullen

question. in bit. the And tried past this I've Yeah, to a address Joel, good

get term, potentially U.S. the view a become inside U.S., sickle my some for to a - thalassemia. outside development and that partner So longer the let's particularly - that with a maximum have to the we because, ourselves. probably those and where some couple clinical that's are partners that been start we conversations once years of and can probably It's engage disease that look we I the of that that the XXX, in time better about of over with share want create going view, was my value partner concluded they the cell for avidly in we product, the always stage. comes would thought what late data of to bigger the on bit for ago to, a but to commercial always that's start beta sort

our So well second as depth the partnerships - look to I I enhance area bit not would more to everybody a create but suite those particularly, those. a value run lastly, to programs to That's it's as some There's vivo later. question I/O said, potentially to And enough can little to The as continue enough we're but some of and any targeted what to that's I'm own and iNK it's the a areas there's the Charlene period partnering ability that space, about we then some those We'll say over And and the for a in those those one. than platform. trials think both expertise, complex, we'll I'll you trials ocular a what of not our need, is space. on that those unmet work look And could year. know, current [ph] with commercialized. would particular area is it's thinking just certainly rather of expensive. we there, small sooner for we that finance to high and and have we later in, we expand not time play thinking longer clinical programs. - an clinical in and up likely where just space, hypercompetitive yet start we are talk that ultimately have like In done But programs see. in in about disclosed, of we I thought about targets. accelerate call by time

Joel Beatty

you. Thank Great.

Operator

you. Thank

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

Dae Gon Ha

Two Thanks congrats me, on the our progress. XXX. all Yeah, XXX for for and morning. questions questions good taking and one one on on

really space, So in using gene analysis read competitor, II/III sort the I and terms recent comprehensive find of looking disappointment anything misconduct. following Phase your out their XXX, ASO, at of their trial in couldn't editing but not

Jim, remarks just end your prepared far, your FST think to thought comes I take of it on seen we've only additional on so history trial. looking And your when at is wanted BCVA, study, what the points derisking Navigation. your so to in and commented And you the on get through natural process when

So of that's your been presented differentiation year, a competitor be there kind be are some since pretty the differentiation seem towards of the question to any of seen content here. that's or recognizing HBF XXX, year-end set to Thanks. And you've expectations is That's end the one. collected specifically, it just some you're competitor? you hasn't endpoints VOC some based point rubric going history since of already analysis. curious, from about on for that compelling On data data? to of can point But you we've leaning the additional by file the natural following that that the

Jim Mullen

Yeah.

Okay.

ProQR. let's So the really see, anything, if - and in XXX first do we make so was ProQR what, from question the endpoints

is time sensitivity We've What about that perimetry used FST, other about in with as and visual you about are have we we retinal So talked mobility. measurements that talked trial and - we other haven't as talking outcomes. things. we've OCT and we've also about There know, have such patient-reported spent talked acuity.

we in would other what are the and which data, making physicians sure more connect visual interested reporting element be up objective sensitivity, to retinal acuity, cetera. that match to the the patients that performance So be we're probably may would et

So to our those of doing we not for the looking patients is that be that we the we ensure get sentinel are information patient groups and the kinds natural is at we're light that one are study after sentinel dose go, perception would of from ones as the thinking to patients, the What things trying so plus past to use spoken outcomes. much remember learn reported only. probably the always the about we've history and each we're

poor vision using. with those are patients likely to we a that to visual retinal sensitivity, measurements. ability that it's be And - that that, very, sure make very a particularly, endpoints incoming show select So to acuity, we're most and response after mobility able

actually that's before. for the So the about logic cohorts we've that talked extending

we'll least at another add probably dose cohort. high adult - in we're the four patients So

on did I So all the that answered, looking get one? Mark, questions did I over... I'm

Mark Shearman

to they track, it So the for other ProQR data patients then a Phase got data and II/III. the unfortunate very us they the thing, valuable the the was think right? that did for set is I surprise with

the response we true rate to trial. decision So the we and focus know registrational the on on against performance make before that have endpoint we

think helpful. I going So be that's to

Jim Mullen

on something And got Yes. this sham think don't ProQR but got everything event, we've that pass probably control. as natural speculating, into based from any I'd say, on that seen even pushed a into study also I history you'd I'm in IRB. warranted this it's actually think don't we I'm think the fortunately, pushed that's I or - guessing, I could procedure the get do it's

So start what's differentiation. is, editorial Dae, well, comment. question we year-end? point data remember, your what an if by that's our will have that And XXX, I to for expectation just to we'll

sufficient see really by sometime There year. will and will to or to make for data the be patients dosed have data VOCs levels. that expectations is be for next fetal anything year-end going any we the engraftment, we'll start then by on hemoglobin until set not So conclusions to

from whether to able then to clinic to actual want to how think be at about you you extrapolate, Mark, we on and that? or else either add preclinical data in decide should data have look people that the will, that anything you'll differentiation if - have So efficacy to durability. perspective, do

Mark Shearman

the data Only we're on making to good very progress the respond that that study. the on the set had on generating to questions FDA they

So really that's going well.

Jim Mullen

Yeah.

Dae Gon Ha

follow-up quick question. one may XXX So just the to if I add

release you one the dose here of to On the or press next as expand steps your this cohort. be and levels adult would in previously, said more goal

depending of dose I then expedite mid whether decide So might seeing starting second dosed kind at you've since expansion, the make to we low again. and some dose be in pursue would sense. or it makes and and hope a whether high that mid-dose on that process low the that on by mid-level high? response already sense least the to on Thanks,

Jim Mullen

Yeah.

a question, you that difficult Mark. me over now really So gave to Dae. I'm looking

Mark Shearman

a question. good It's

So we're out. as it looking at the the date - we're data going to rolls look at

cohort heard, first X. the As pediatric the we've dosed mid-dose in you patient

of taking on emerging and cohorts as really as that drive final to adults. the the look sure want both the the we well comes we at both efficacy for close a that safety we decision available have or that in make information think one data, assessments expand the data, to a use I any from whether So

Jim Mullen

would data Yeah. roll But is likely preclinical Mark effect. we the just high react to in. the as best say see to have the said, data it dose most we to have The as

Mark Shearman

dosing low recall, data next. as we I data on finished on December year. in we the X last patients that adult will mean months dose And mid-dose report have initial the you of then the XX-month and four we'll

stage that in well. come the third So quarter through will as

Operator

Thank you.

comes Our Chattopadhyay Please your next with question question. from with proceed Debjit Guggenheim.

Unidentified Analyst

on Ray is This Hi. [ph] Debjit. for

you For through us and subject dosing EDIT-XXX, engraftment how for safety walk timing account the you evaluations? necessary between time and the could

Mark Shearman

Sure.

by their we safety going with step, has approximately first XX-day we infuse, That's period which patient the evaluations general once neutrophil So over monitor expect engraftment. cells followed clinical patient chemistry. engraftment the are we and to

engraftment, the a So we point, similar patient go for second have evaluation. be proof of trigger when to at dosed through then will that be they will that and the

Unidentified Analyst

you. Thank it. Got

Operator

you. Thank

proceed your Seedhouse Raymond Please from question James. next Our question. comes with with Steven

Steven Seedhouse

one Hi, first and good one I question. taking sickle morning. on Thanks cell LCAXX. for had on the

the from - or at you it six shedding there's and ARVO, the across Just the looking like look [ph] in of viral data patients, as copies virus below week to it's And there's six. then out x-axis, doesn't is the detected patients to but most of the shedding, that related look on one limit dose. detection, viral have difference the seven magnitude like orders

wanted engraftment around safety of versus whether difference it's the lends preclinical safety what appreciating that expectations? heterogeneity anything expect safety term. of in is And of long what if the ask to I taking tell on And to the question expected pharmacodynamic the So are time just drivers, Thanks see questions. your But I BCLXXA differentiation then you are Does for just hypothesis an expectations or if your data the about cell the the time implications? sort or it curious guess, approach? just sickle here? events I'm early transplant, suggest to you'd

Mark Shearman

on So viral I'll one first take shedding. the

vector. So variability on data variable. experience, volume correct. I based the is prior blood slightly viral different. way with be may the you the virus expect get retinotomy. is of in you're what The the somewhat of the sometimes administered, this and say, that reflex AAV would my are run There administration Occasionally, exactly is meaning subretinal the you'll

So a kind just data generally, procedure this to on a get viral shedding. of is messy tight

that what it's the I The pretty overall to eye therapy. relationship what really response response. much but line finding. you'll And in Albeit amount. or no essentially administration just expect. vector dose in that's I administration, clear a occasion, from an we get message and general, would that's any think expect we're think with appreciable expectation in given the some to is it's general, rental we is, on what AAV-based benign would There's get medicalize, the beyond doesn't the

Jim Mullen

That's that anything question is it going what the to was point And watching and fact of so about about and preclinical. engraftment to is to seeing our product your do data, what how we're question of the we've mimics in likely seen a early in XXX which differentiation.

the this to high mimics we other that have - have levels, high we'd with very preclinical, expect indeed it be if So we've the quite seen cetera. seen and expect what we've editing to et to we'd what in graftment programs similar

- also to know higher correspond anything better. of a and knows I see how hopefully, will beyond to that don't to Mark, that? don't threshold, much we'll have once that if high certain relevant how know HBF actually I you production I that add is. clinically and get So is don't you anybody

Mark Shearman

which [ph] or efficiency be know very HBF that mechanism of we response are we AsCasXXa I here. of think cells, don't important is from you're could We nucleate getting are viewing any get the general of that high of editing. We we know pursuing, a No, we the good that correct. the progenitor think editing the following determinant data durability editing the preclinical and the with linear really

the longer factor So I think too. an be term, could that out important over that plays

Steven Seedhouse

you. Thank right. All

Operator

you. Thank

with with proceed from question. next Please Our comes question Jay Company. Olson your & Oppenheimer

Unidentified Analyst

I is to mentioned guess more taking Canon for This are on you endpoints the Hi. for Jay. the question. EDIT-XXX, looking also responsive treatment. that Thanks the and [ph] line for you're for more sensitive patients maybe

both the patients? Just patients? Thank are if same you. outcome also, collecting you the if applies patient wondering to pediatric And from reported pediatric

Jim Mullen

selecting pediatric the yes, So logic just same the going how want I we to how describe would to about we're And apply forward. patients. - patients careful be

you it's have useful in, maneuver not to the token, coming too actually not the vision in, very sort what through have is more really even a as the ability - about incoming enough criteria, of endpoints range going how of incoming a and right? product for can it's Do baseline that. trying or will. right? works will there's target I pick - kind the these at doesn't we and already good scale, we by examples So that's eyesight that the as we're information just from range they work, more Berkeley, inclusion less about very the get you're endpoints the to sensitive over they we low it's do So to So probably ensure more top are of have questions? maze their site if is you same about those coming the in they what if up these where think a And appreciate, if - to much BCVA here think to in is very patient the very, use of work the difference, adjusting

Operator

you. Thank

proceed Our with next question. SVB Bienkowski comes Please question with Rick from your Securities.

Rick Bienkowski

Good morning. for Congrats and on questions. all thanks our taking the progress

The don't I this is for of second program. First, But filing. and many completed major recent a before question. that's have highlighted any also guided to guidance is. into this we an just of parallel know milestones? discuss ongoing IND how hoping exactly assumptions expect where the clarify of in your scientific work presentations or what an dependent advance be Could maybe clinical release into could cash filing? have I and I believe this some runway dive not number, for press I And you EDIT-XXX a including was have you to IND early whether steps some for development lot any program Could you around in incoming maybe XXXX. need the on EDIT-XXX programs then you

Mark Shearman

this the Mark. I'll I'll question. take XXX is - Maybe

you're correct. So

given differentiation clinical produce clone studies. preparations for selection the to of of preparation IND includes necessary are imagine, which We haven't an includes the all date you guidance specific towards - study, side also conduct IND, toxicology filing. all and package the But the expansion IND-enabling toxicology pharmacology directed of file GLP the to as will both - that we be and an to can sufficient the of on and sales completing work, yet efforts all which that our data the pharmacology but cells these of then

we're And more date plans guidance as the in later the as could right when we So that's IND now. think on year, maybe a to give available. going we'll narrowing solidify, where be the

Michelle Robertson

question, Mark. second take And the I'll

clinical as cash not our so about, or we've that does based is and our incoming our spoken as plan, - BD. it assume milestones programs, cash programs current includes current So preclinical runway our on it well any from

Jim Mullen

So assumes about that talked pipeline and preclinical everything at forward it pace. clinical moves discussed our essentially or we in

Rick Bienkowski

in on work the for that's that mentioned Or ongoing perceived well? bottleneck it up the external Is press Yeah, mentioned the and is manufacturing release with just work? follow to the tandem. be as – that I preclinical a wanted Great. internal program. parallel other EDIT-XXX filing just capabilities to advancing IND in so

Mark Shearman

just that for methodology a differentiation the Mullen develop to to approach we're CDMO. it's latter, taking transfer of expansion the of external Is and Jim methodology then the the an

Jim Mullen

limiting view view ahead. business, and moving program we go But to really normal to that as don't don't we that as this

Operator

Thank you.

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

Unidentified Analyst

Thanks. include homozygous is for XX This guys. does I wondering, cohort just high question. the on Madhu. Rob Hey, Thanks of BRILLIANCE was IBS any taking our for patients? dose

Jim Mullen

I this in don't believe at so time. point

Mark Shearman

Yeah.

the haven't patients of those. we're the entire in so that know, you and population, the homozygous focusing cohort. of about We constitute as included just XX% But on probably population, patients released not specifics the

Unidentified Analyst

you. Thank

Operator

Thank you.

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

Luca Issi

Congrats much so Thanks taking questions. progress. for on all the the Great.

a one here. few Just

on about the CVC the to candidate that where this update come all is is ideal in us IP, pitch we given how which spans just Would given to ophthalmology, can between the lots as assume so you a can that LCAXX? be a LCAXX, of give the FDA So is lastly, ask second maybe just you the long-term the depends bit little much. on the and will who about fair on maybe the down today EU. the breadth But And of questions, understanding understand the in economics what parties the as cross-listing agreement be will us my geography? that Officer primary U.S., And remind And you then the the obviously more circling it stronger directly. hematology a Medical the for maybe Thanks any moving but talk is then position back end should on think the search? Chief in on oncology? endpoint pipeline, upper the And few still will I have prior endpoint you implication, the parts data evolving. now

Jim Mullen

compound question. with… start Do remember Luca, to you're a you want can we assuming that

Mark Shearman

XXX. Yeah, with

We yet. that made decision haven't

there's coming still But will in both pediatric based what on like and guide indicated, data-driven and the hopefully be we've under a now, data that choice. the As our decision adults of and in in patients, the that final will lot evaluation. looks right

Jim Mullen

do you that? take want to Charlene,

Charlene Stern

Sure.

to decision think technology. that, decision have we will see and we Editas USPTO is Broad, to that value we So that be upheld very in how license the we favorable a longer-term significant about opportunity we and The believe our and think tremendous IP, on appeal.

in believe commercial drug that the of and will majority We a play sales out here there's development also U.S.

And IP and importance we the so have. underscores value the really that of that

Jim Mullen

of I've for looked of has at it the everybody or that's no there's CasX FTO of is by to programs put is the mean, need always might an which And Europe, And like together two I is commercialized. all. the we did, models anything market. get come the saying in Yes. of always revenue that companies underlying that actually all more presumption thirds going it looks means then out for some clear at also U.S. are in what and that

using for particularly our sickle - program, remember, cell So and CasXX. we're

completely point other we our program. outside Mark that's other else CasX time Go just program for at sickle the programs so in was U.S. anything the ahead. irrelevant that the our not cell and – XXX for this adding is uses And And need question the AsCasXX as any of it's FTO for clear well.

had I another anyway you - So so forget... sorry, - I'm

Luca Issi

you. Got it. Super helpful. Last one on the Chief Officer Medical search? Thank

Jim Mullen

you Chief get Medical asked Office, question, I me it. a I told you if wouldn't compound

So I've right, need that successful think scientist the Because that's said be perfect develop. clinical and development IV, overseen have part in initial fairly targets then approval. team be only are and to to quickly. have to able right, we to as with that we up highly a that's need this the relevant very starts see we're is, sort of know that to and It about match your sure have why the Phase in pathway Mark's they They not foresee clinical a they you had targets, before, that can to to obviously, next clinically need technical to development of somebody from been the a so but program closely as of part moving what interviewing doable, they are end. they develop important here And candidate. what's beginning the area be are get is technically make reason partner real is, needs we they scientist. they is after - to how clinician of the process people, And a the

sort and high had the overseen and has that have building has medical functioning And that of somebody a be knows and it So experience. expansion to to organization the have second. that's of the lastly,

people So the I the we're think those are from interviewing, we're we're that getting for looking sure, profiles those. for. And

real here. I'm going be is a So That candidate get building optimistic here nicely going addition now very team. to we're to quality to high what's very a a

experience, has remember, preclinical XX moving and a in the So done research The than for much drive clinical was. programs the come. Mark into really part to and team to added not yet we've of Mark, of a these we even got CMO necessary great getting of now months XX it years not had focused starts right, all moving months, to I development. technology Gilmore's getting business this better it job the think really And

Operator

you. Thank

question from Please with comes your Gena next Barclays. Wang with Our proceed question.

Unidentified Analyst

Tom for you. This Gena. is Thank

and questions, two have for We XXX one the sickle cell XXX. for disease

everything data-driven. for further And XXX, for be it's selection cohort the I update? cell patients assumption to design should can your pivotal pediatric how additional So guide engraftment dosing for for know we sickle I and disease and But many to to earlier just given get trial expansion expect dose trial? wanted the for the interval need valuation, year-end the or safety the

Jim Mullen

year-end first by on number of was The patients question XXX.

Mark Shearman

year-end, Yes, be our we have expectation dosed by patients. will that will two

I indicated of is start monitored. the carefully earlier, trial the As very

six week reconstituted. is being first So out system to the be confirm engrafted to the patient, edited in product at least has that able before will we to that patient immune have get

first that dosed year. us patient indicated takes the of get we've that first the will So half the - by

to So we of that the then least one hope takes before us year. into sort dose at patient of the third end the quarter, get and more

Jim Mullen

than can then is that, we think I'm... data that for to Mark's discussed to fish. we XXX, have expect And have people is we've may really but question I And your what will referring more what of

Mark Shearman

Yeah.

So cadence it I the of dosing. additional was think the

year-end. by of the adult And of we we by initiated completed will expect the high-dose that be patient I with and year initiate by pediatric to the hopefully cohort one cohort complete So cohort, mid those and think expansion similarly X have pediatric guidance with dosing the the mid-dose we sticking that year-end. dosing we're given

Operator

Thank you.

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

Liisa Bayko

Hi.

questions most of think guess I one I my been answered. outstanding. have just

vision we're occur these loss For obviously to and ocular in diseases, later makes endpoints of know kind about what sense I it's kind of thinking EDIT-XXX, in tends as very life. complex track to disease? endpoints this

might - you kind Just think used or by metrics. in one that that endpoints easily other commonly some more some of thing measured the require are of

curious about regulatory here? Just you're thinking approach how the

Mark Shearman

Yeah.

to use rhodopsin indication it will of we most the same XXX. our intention So be do endpoints exploratory as for the for

a is rod As photoreceptor you this disease. probably know,

So decline in vision. initial the peripheral

will of be ideal primate that. an looking way So at

all And take so But OCT includes which typical anatomical will do this doing right look of this and been there we've now, measures the which those already. we for a therapy evaluation a is XXX, types is of that indication retinal monitoring by for because is an things outcomes. not of an so patient-reported all on, we'll at sensitivity,

the the this patient treatment. is in the have of most population, see So endpoints to we'll to which responsive

Jim Mullen

coming of and these and with central collect are a patients make to that will the don't [ph] little XXX. easier endpoints these they groups of a in the eyesight, a the be have least conduct things of because good some to on you stickiness some at certain in vision And hard patients with data

Operator

you. you for again today's this your and Thank Thank once presentation. gentlemen, Ladies participation. concludes

You may disconnect. now

Editas Medicine (EDIT) 4 May 222022 Q1 Earnings call transcript