EDIT - Earnings call transcript Q4 2021

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Operator

Good morning and welcome to the Editas Medicine’s Fourth Quarter and Full Year 2021 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. you. Thank

begin. may You

Ron Moldaver

be and completion. press everyone. results release A will X call call corporate Laura of the Investors we website, open our our and this prepared issued updates. approximately will today’s of a providing after replay Q&A. on good you, the Earlier section its morning hours morning, recent available After Thank for our we financial remarks,

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

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

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

Jim Mullen

highlights everyone. vision. a children. Michelle team, cause with want for providing trial good am in today Editas. demonstrates members few The EDIT-XXX Robertson, joined successful morning, vivo I improvements of Financial and I upcoming inherited year Shearman, Scientific last Chief for achieve We milestones in Officer. off in with from clinical editing Mark some to including executive initial Thanks, in delivery our data by Officer the our Ron Editas blindness important proof-of-concept a leading LCAXX, by several start Chief of and and

pigmentosa, half pediatric the We the of initiate to finalized of high-dose cohort dosing track construct XXXX are pediatric form mid-dose We which of and year. retinitis data the dosing another first sharing studies. treatments. has expect progressing EDIT-XXX IND-enabling plan dominant spring. on to this autosomal ARVO of complete the to conference the preclinical This on of leads in program no towards for the at We additional this and later blindness disease is approved retina, cohort

in products now vivo of have and expanding programs pipeline We ocular Our X quickly. is more ocular wholly-owned opportunities.

successfully and to on eye, in cell well-positioned dose We also explore sickle preclinical of our ongoing work. this potential sites disease, are pleased we first outside half year. to we clinical clearance edited transfusion-dependent with of have With to for ex for thalassemia indications first setting cells vivo, and the EDIT-XXX process study the program patient obtain up EDIT-XXX And the we are subjects. are are also to in the in additional of IND screen track beta beginning

million has investigational the edits. approach programs we NK and have dose our with tumors. have create therapy solid royalties the for beta into against announced gene To-date, in over data potential studies. to We off-the-shelf potential a patient declared in year. future. allogenic activity this to with highly sometime expect We cell alpha further X opted how beta demonstrating presented advanced differentiated NK-cell IPSC-derived We IND-enabling plus Squibb with thalassemia with first in BMS enhanced And medicine EDIT-XXX, Myers Bristol an our medicine, milestones payments candidate has collaboration finally, we development X received T-cells. $XXX in one

to treat quantified for at maneuver by I’d improvements in best meaningful editing delivery clinical vision. different acuity. levels, in demonstrates important full initial the Last visual field proof-of-concept patient’s light were successful our and to a in programs. sensitivity ability Now, EDIT-XXX vivo reviewing with data BRILLIANCE like spend clinical improvements through few The corrected we LCAXX. minutes trial light assessments, in mazes testing a These with several improvements including year,

strong very patients. on by dose-limiting surgical year the one reported Any adverse far been of procedure we we been to toxicities the dosing as breakthroughs of and cohort was resolved. events. last across and have as not has three very progress and events thus EDIT-XXX encouraging We all dose for At attributed are advanced trial levels adult the Advancement top by our highlighted results in year, seen The have any the pediatric serious safety end we adverse American profile excited subsequently the is Science. or these the last Association high-dose of of completed

half Our with milestone first is mid-dose cohort dosing the IDMC safety of review pediatric year, of high-dose in pediatric the complete cohort. next dosing the the and the the initiate of

data high-dose include will we cohort be Thus provided in patients. with are trial which additional registrational cohort. the cohorts. We the adult of in appropriate data the and far, latter expansion most endpoints. design and explore importantly, completed also we in The or the have for very from received of X-month one clinicians, us That this mid-dose provide as patient EDIT-XXX and XX-month the and clinical help feedback the data expanding year, more previously the dose on responses enrollment investigators, encouraging half will trial update select of the will further

participants to meaningful a outside simple study including their more bit upon doorways have walking improvement through lives, the walk of around daily being independently. Some like or things reported able

what information support most meaningful exploring to to and most the trial, further the is to are endpoints continuing We evaluate development most interpret can sensitive of how we patients. considering and relevant effectively trial

data data, that identify measures we expect study looking the at and history also we addition data to trial the are to this natural later have reproducible year. most our In available to

one-time thalassemia. disease vivo EDIT-XXX to beta sickle treatment on transfusion-dependent platform, developed potentially we for Moving in both a cell our and ex have

crises, more persistence binding mutations protected seen genetics, a or is exhibit human we generally this of experience Our organ natural life hereditary safe who or mutations unique and indications. to consistent EDIT-XXX in HPFH. of to the and occurring hemoglobin, pain have is be of these of fetal fewer longer result validation don’t site the treating approach approach durable approach spans. disrupts believe Patients damage these in naturally and what hospitalizations BCLXXA with Because of one both co-inherit for of likely that

the to CMO a clinical up As IRB with disease dose durable RUBY by search the successfully This generate patient initial approvals also therapy. disease, enzyme. of in the is eliminate patient efficient a afforded expected clinical half are sites, wanted specificity EDIT-XXX, data last sickle reminder, ex symptoms beta clinical year. XXXX, hemoglobin, potentially trial that on was and we our utilizes are lead first end. for even and HPFH. beta have we operations, In reduce editing FDA thalassemia and and vivo patients the cells and our December TDT. changes and everyone AsCasXXa editing sickle by efficiency enzyme, could finally, setting promoter process increase similar added safer We of and more highly the I our believe while the the or with specific first disease And EDIT-XXX protective choice by individuals proprietary cell combined site, or update to IND a of in globin of cleared screening with in tracked The in the are dosing year patients fetal TDT editing underway. that we expect cell for by to unique should

reviewing retained has has current The been candidates Our initial extensive experience, top which hematology and team, agency coming on trials. clinical our clinical continuing the tier in and search us months. is to operations ophthalmology the to plan in advance we assist

clinical Our clinical our organizational anticipate who work preclinical to our to trials, in look someone for do and closely made. any And objective spearhead is and a multiple Mark’s our With over you me upcoming call let as technology. milestones. a I We to pipeline. strategically turn updating forward not the to of proven program is Mark approvals programs build or track someone review disruption who platform bring regulatory medicines, complex with decision once can that, team with in out an we leader record will

Mark Shearman

Thank development including in stage been excited Jim. announced year. this very have progress early making we new we our pipeline, the am that you, by I candidates two all advancing

replacement productive are for the pigmentosa, gene for address to a currently only replacement than no rod is means that patients for onset treat of EDIT-XXXX effective of which early expect of non-human in gene occur rhodopsin have achieved of dual the a mutant The and gene therapeutically approach. also blindness be accomplish to rhodopsin-associated we symptoms protein RHO components The gene virtually this that same experience on necessary knockout expect and mutations XX% typically AAV leading primary replace the EDIT-XXX to more photoreceptor editing, Based in human There a adRP, if the dominant able retina this then RHO corresponding a very adRP this to significant later their data, we the number therapy knock of optimal necessary in the we we delivered cell corrects which cell. our gene sensitive function. The in disease are First, autosomal functioning resulted disease. unique disease gene protein although a replace replacement approved production primates proper And retinal light the retinitis can To showed of RHO importantly, the with the case it’s XXX% knockout at that this preclinical to life that of XXX We in level. in one. gene. treatments. with gene years. caused approximately out in and dose, to been knockout causing the tested

Notably, earlier gene the vector With editing AsCasXXa guided negative the Usher to significantly our Syndrome be our gain a and under an for has construct expect anyone advanced have needs RHO with the first proof-of-concept adRP The As the for used And optimized This that where different clinic we to a time editing of Conference. AsCasXXa construct. in that present increase be rapidly. well moving for an approach IND-enabling to improved dual productive knowledge a disease program, treatment in to EDIT-XXX packaged This AAV editing end resulted construct alternative towards we and compared autosomal dominant dual initial ARVO using that will Jim plan enzyme the XA with data approach other configuration. provides our we refined also the productive potentially an additional delivery. using RNA also and is by a a indications, in of corrected. increased studies. by at in XXX% these are of we Medical program XXXX, function is pathway the mentioned, we AAV

diseases ocular also our are and delivery We in working large advantage the in eye our indications of other on range taking of experience solutions.

We an expect indication new in additional vivo this for candidate an ocular later to development declare year.

on iPSC-derived our a have therapy EDIT-XXX, differentiated development for we tumors. a cellular NK-cell medicine programs, announced solid Moving to new candidate highly

develop to an is objective cell and engineered NK approaches. overall important existing potent cells persistence, Our many limitation with increased anti-tumor with NK substantially activity

engineered NK-cell only tumors. the X believe dependent the the overcome to one be to For could multiple currently active of edits an treatment potential improve improve are off-the-shelf proliferation of To specific resistance, make one microenvironment types highly this knowledge, improve that edits, this construct, has to allogeneic cytolytic antibody our for one and the create one medicines approach tumor activity, that cytotoxicity, systems. has combination to cell. best company and of used We of this we solid therapy we in cellular to the activation,

strong this improved expect cytotoxicity demonstrating data to cellular in and this enabling IND even than week. significant one antibody-dependent persistence reduction presented without year, Earlier program year. EDIT-XXX initial We or exogenous advance we support, studies cytokine preclinical clearance less to tumor this

We more upcoming locus. the selectively for vivo With receptor wanted GMP SeLection spring. I of which SLEEK editing. increase the anticipate Exon and us follow-on utilization EDIT-XXX, may this Knock-In in antigen product going We you highlight to will a be may or EDIT-XXX, year. short selected SLEEK a for have knock-in introducing streamlined. solid further differentiation After AsCasXXa selection or to customizable the have for describe Briefly the tumor for Essential-gene high heard chimeric back last of anti-tumor many activity, development technology continue specific we significantly first is the the presented AACR technologies expansion that to the can nuclease at specific and while clone process there by at number of of key utilizes we It’s program indications. which and that CAR. programs engineered potential the and cell additional SLEEK to some well NK vitro view experiments, a types, ensuring underway in follow-on the as that that cell a advancing, configurations technique of We also be high be tool transgene controlled as and Essentially, efficiency will edits be meeting of number such therapies. transgenes knock-in with get allows accelerate efficiency expression. robust integrate into development gene-edited us different to to a of a anticipate

knock-in nuclease We various have the XX% iPSCs, AsCasXXa relevant T-cells than target using efficiencies and cells. NK more demonstrated clinically cells, in including

to also edits. with chromosomal fine-tune We with for of no once for medicines. important another why a anticipate cell clone next cells, the safety the platform. the therapy SLEEK population cell we a final I’d an have cell of This we is intended off-target further attribute attribute point example, us of edited editing. gets single also have ensuring precisely transgene in that clones development exact heavily using delays That the abnormalities, out we clone to levels expression characterized and has edited any generation of important so cells invested cargos, derive our that elimination reason select extensively therapy

are They our BMS ongoing and IND-enabling our good at turn review are proprietary partnership development of nucleases forward programs with Squibb studies. in BMS Myers immunooncology. create many work results. important to our to around opted to allogeneic With new CasX is X will Bristol and I colleagues with leveraging cancer. over to candidate continuing with that, Michelle into We and progress financial AsCasXXa including to alpha-beta develop our T-cells. closely RNA one Finally, and our technologies, we make it design for approaches guide to to continuing autologous look medicines in has our

Michelle Robertson

I the advance will take our issued position like in our Thank press for morning to few this we fourth of you, refer opportunity quarter our briefly I forward. to and release results would good remains financial today you Editas items. year Mark for summary portfolio and everyone. of strong financial earlier review full to a as XXXX.

supporting trials also We of pipeline. our are enabling our manufacturing the advancement well-positioned clinical preclinical continued and and execution of to objectives the

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

million million with was in decrease as mostly decreased driven Our in Allergan recognized of by XXXX. XXXX. the XXXX to our by as to revenue The in Sandhill over our million XXXX in and expenses $XXX for agreements $XXX XXXX. extends our Revenue XXXX. cash in $XX $XX million Allergan from terminations agreement EDIT-XXX million runway collaboration see was R&D XXXX to Juno, we part we a EDIT-XXX. termination This advanced and $XX our offset decrease compared and increase is with Factoring did as slightly in related G&A the decrease is increased costs through in expenses in collaboration $XX an revenue out, $XX million now from last related driven XXXX $XX million manufacturing success BMS. payment which expense license by million in and of expectations clinical were those consistent year to agreements. from with well from

expenses the clinical just to primary We by programs, With hand expect XXXX. full-time clinical on-boarding and expect end last our with that, and developing the approximately under CMC growth supporting of Jim. be XXX ended spending on it will manufacturing of the back focused year. this of building have be employees in employees year Most out to our We to and XXX drivers capabilities. will technologies new I

Jim Mullen

to for XXXX we year year. Michelle. be and exciting you, was XXXX another a Editas Thank transformative expect

adult half previously initiate dose or one in data more dosing clinical a EDIT-XXX, expand the year. cohort, the and provide the completed pediatric in of update of we will second cohorts high For

have declare to EDIT-XXX dosing will with thalassemia candidate expect And for of also patient year series in they cell the a programs clinic. indications will cell our ocular disease studies In into beta continue construct therapy the EDIT-XXX, of vivo and We a T-cell BMS first fourth USHXA programs. data of in finalize be and advanced iNK and indication. new product the cell the towards as sickle initial sickle in well clinical IND-enabling supporting for our by program begin disease development platform, their initial for studies will end. our both program we IND-enabling EDIT-XXX first we

the And with mutually capabilities to expect For platform on development development new it long-term we where value when in finally, always creation and believed plan technology we and will innovation, and techniques as editing and an continuing, have be improved developing leverage facet delivery. important our technologies enhancing as technology beneficial. partners, gene as for business well I of

addition in your focus up our core that, plan to interest pursue areas we efforts, you support. of we and for partnerships. strategic outside through will We opportunities to internal commercialization all our future it Q&A. In With development thank of open to

Operator

comes first Our with Ha of question Stifel. Dae Instructions] the [Operator Gon from line

proceed may your with question. You

Unidentified Analyst

Hi, good guys. morning,

Just on us. Gon. This for Dae a is Jack couple from

I guess minimize LCAXX what you on you program? Just the are general what your or/strategies and taking a measures thought, are such the discrepancy in is BCVA steps in to gap and your

Jim Mullen

am that? but completely am not you want that not take Mark, sure maybe I to you – question, I do. understand do I

Mark Shearman

least to BCVA of quite course the the on has LCA obviously a of the see patient this so At is we population measurements my time disease. understanding, and range Okay. get the them. time the at trajectory based yes, And that

addition you self-sensitivity visual standard retina We maze. function ways their have as chart, acuity the ETDRS of and to and instituted in the BCVA to the mentioned of in additional measuring addition the

their the assess function I of think to measurements of is to the to really approach our try so and to treatment? patients And response those all use

Unidentified Analyst

the drive will what your you enrollment helpful. then and you are And contemplating PR, in cohorts, what your the mentioned expanding adult Great, between is thanks. more? determination one or end That’s in

Mark Shearman

on have the So, tested doses goal with we more in or the of is expansion the already generate really adults. to the one data that more

as dose across be and the think some patients to best I there optimal we response is the decision want the patients. make variability had to able profile and so indicated we of on

expanding And as through. We flexibility so on up numbers what comes that’s the those ourselves patient the into based the actually give data driver end it want to being numbers. for

Unidentified Analyst

Great. Thanks. the Appreciate taking questions.

Operator

of comes question next Truist Joon Securities. a from line Lee Our with

You question. may with your proceed

Mehdi Goudarzi

Joon. Mehdi good for Hi, morning. is This on

of we to programs is you relates SLEEK And using understand potential and question technology. Our on technology technique are SLEEK if that elaborate powerful like internal these could would collaboration if for this any to there this sublicensing vivo or do method technology very you application potential of for for technology And tissue is specific the end, SLEEK out. different genes or at Thanks. in genes? see interesting any

Jim Mullen

will for Yes, take that. question. And thanks the I

the So right two its the constructs. iPSC technology Bristol to editing highlighted now CDXX the knock-in we the with we one, in where addition, have in we areas: have membrane Squibb. internally in for Myers our T-cell use alpha-beta bound iNK on used that collaboration for utilized have program, And then IL-XX SLEEK technology

in systems. to itself. continue powerful guys and and in that different in we additional its control continue It’s to advance sites, ways ways We which for into technology SLEEK example current the can transgenes look look which we expression to for for

technology Essential not do so is Exon. the for die, knocking reconstituted those the essential essentially that of makes an those cells because theme. will One you the that total homology SLEEK do a that which And cells directed proportion successfully of that powerful and that into conducted you have repair enrich have are cells, properties is and

potential of its technology. but in of does that So, is powerful many aspect because to use really vivo, that provide in settings, the property limitation it

in, cure nuclease. technology. as lastly, situations the to as the is So, know-how then it. everything in very knocking it we powerful for a possibility are There proprietary this of a terms use the don’t as but is we lot of open in all the yes, in we AsCasXXa which are using see of And well licensing of

licensing yes, And we would potential opportunities. entertain so

Mehdi Goudarzi

for much. very Thanks you our Thank taking questions.

Operator

Nadeau next comes of from Cowen Company. Our with Phil & line question the

You may with question. your proceed

Phil Nadeau

Good our from for taking morning. Thanks Couple us. questions.

timelines is update be as there EDIT-XXX, we completed? essays, to this the any curious if efficacy on when are on any First, could new

Jim Mullen

start it. to I want will you and that, Mark maybe finish

using potency few is So the you Phil maybe efficacy, different and be well needs there a referring to is assays, efficacy/potency I qualified to developed. patient samples. it that Ultimately, assay, the think

So with couple quarters that undergo of if of don’t patient we know that to is anything process there the will samples. assay to next is you Mark, on? that be over a qualify actual want course I the elaborate

Mark Shearman

is performance add would confident that I assays. those thing of of only are we The the

We lot as of internally to have good as effort can be. put them they make a

it’s to able really so the getting samples the And Jim mentioned to confirm patient as specifications. now be

Phil Nadeau

have do have the And that verify of the getting to you can to us the you remind clinical FDA uplift your to that you presumably findings have essays have qualified? process and of is been the submit they to agree you

Mark Shearman

saw cleared That’s on liked by easily, that were they the essentially run contains fact very I relatively should so there. will correct. FDA encouraged same how beta these and thalassemia the We the assays the what that got because IND information say be

So and I them their matter information think that of a response. to providing getting it’s

Phil Nadeau

then in from And you notable the us challenges Illuminate designs recent informs in you studies? prepared discussed learn question conducting that results on the in anything from your the trial, Did second Great. some anything of your is condition? LCAXX remarks, there LCAXX studies future the of from

Mark Shearman

unfortunate at that the have LCAXX later ProQR was information line think secondary, terms the is we is outcome community. of patient do released, right top essentially for comment than very don’t and the of further insight primary can’t any this I in anymore point. So, the I mean, so the now, which currently data that we on you

Phil Nadeau

Thanks my for Great. taking questions.

Operator

question comes Barclays. Wang the of Our line Gena with next from

question. your You may proceed with

Unidentified Analyst

am question. Gena. the taking I [indiscernible] for for Thanks

us in to the late the multiple long-term? one should from about think preclinical stage, prioritization program in we how mid program with Just pipeline

Jim Mullen

we the about Interesting that is think pipeline following question. going to think the we to data. probably prioritization? I How should answer are be

that’s investments. meaningful on leads where double that down to platforms to the we strongest or drugs, areas data we drugs, those the so actual that start And will see

well the is data data, don’t data. it data we follow the we that we of So, premature, sort the ocular on great sickle – the sufficient Probably thalassemia. question second sufficient half because – just of will into as and have yet But don’t matter, a proceed on cell XXXX. a programs or it’s for data probably beta clinical we have

Unidentified Analyst

you. Thank

Operator

from next Luca of comes RBC. line question Issi with the Our

You may proceed your with question.

Unidentified Analyst

Perfect. Thanks This my Luca. for taking question. for is [indiscernible]

Just or And for LCAXX. that safety you to me looks low, present LCAXX are is update going a shedding. couple I on which on it noticed a like going to look viral at

wondering aqueous behind the eye? for? possibly mean highest the dose well? Just decision the safety most you. efficacious expanding this also you just is about color this based wondering, in adult can the that cohort, maybe more the is as dose, the something look you Thank the in Then if been or enrollment at provide has shedding this, is the another to viral how be question partly on does not also around going rationale assessed? this FDA fluid of And asking will Are

Mark Shearman

those can two I So, take questions.

into set introducing way know standard it a requires a subretinal It’s of precise really retina. beyond and of viral shedding concern. assessing based virus the is this studies, that and anywhere and go tears data assay seen that the So, collection I based is data requirement AV And have it therapies. from similar on on it the and no of And for a injection relies doesn’t blood FDA the of standard is we the that. very

answer, have the are that my limited escalation. Phase number just in mean question. to a yes at still reiterate X previous we that’s point And patients of I this enrolled. So, we And dose

be his just which plan insight the the to two best the as earlier can the is information remarks, and in as efficacy we registrational we effective. trial. we to that to that mentioned And – as this And want so any pivotal other make to acquire for of opportunity with dose data use may the decision doses the more so to Jim different important into measurements more

Jim Mullen

high in to some But it’s really did QX. any perspective, early the from to complete Yes. just meaningful so And just too time readout cohort have we dose that. add,

open expand. just from from the data to we decide data to more plus going we where our options see So, keep we mid-dose as the extensive are that,

Unidentified Analyst

thanks questions. that, the with for And taking

Operator

Our Madhu next question comes Goldman Kumar from Sachs. line with of

proceed may your question. You with

Unidentified Analyst

for RUBY my just on data trial from Hey, the was initial question. at to year cell from see for the I patients guys, Rob looking It’s what sickle Madhu. thanks wondering, end? are taking you in clinical

Jim Mullen

– beginning Well, will – be that’s the basic we probably anything the Mark, expression do be at safety point time. it that? profile, – Mark to want and about you will have first it that all engraftment add to and in hemoglobin. if fetal will

Mark Shearman

key later factors early They are engraftment HbF the engraftment, neutrophil platelet production. in and potential of signs

Unidentified Analyst

the Alright, milestones thanks. question how should of other about is activated the And one collaboration terms T-cell like just adds clinic? we to in think Bristol

Jim Mullen

guidance that have the don’t how much guidance Milestones, I specific know royalties we mean they would I commercial. in given be pretty classical milestone payments that past. and if became once they Ron,

Ron Moldaver

the We can about progress. just talk current

Unidentified Analyst

Thanks. Alright.

Operator

Joon question with of comes line Truist next Our Securities. Lee to

You may proceed with your question.

Mehdi Goudarzi

mutant like a reintroduce recessive said Hi, eXXX it again. adRP. they ask as is Mark, should the allele Based be the wanted on I to is understanding adRP this Mehdi are that this mutant a to sufficient that to question you my is removing allele. related copy. functional of stop necessary

So, wild the what is in of type necessity patients? the reintroducing allele these

Mark Shearman

So, the type. is discriminate to mutants endogenous mutation approach between and actually mutant not both – because sufficient the knocks guide and and the rhodopsin the the down wild

get knockdown So, a you going of both. are to

And require so replacements. that’s you the why

AV be a to replacement and in copy provide second serve rhodopsin epithermal the copy that the will cells. will rhodopsin those functional of So, generated from an

Mehdi Goudarzi

you. Thank

Operator

Our of comes line the next Jay question from Olson with Oppenheimer.

You may with proceed question. your

Unidentified Analyst

the Thanks for taking on the for morning. line is Jay. This question. Hey, good Chuck

safety IDMC if from Just cohort any is for pediatric wondering adding will that Thank happen? when cohort, data And so, this separately, just there XXX see could ongoing we pediatric you. And review? year? if chance that

Jim Mullen

middle of will be mid-dose cohort. IDMC as pediatric the further the we enrollment complete the are mid-dose our hopefully, high-dose of pediatric guidance mid-dose we the cohort has patients that’s of as And and cohort. the yes, year And to there hoping end, review the well, well we So, the an adult pediatric short is the if guidance is given. answer goes enrollment see IDMC by all that The from following by high-dose will of the completion data year. have

Unidentified Analyst

Okay. any pediatric the we year? the cohort data from Thanks. And see this chance can

Jim Mullen

a yet not for presenting timeline to have information. that We committed

Unidentified Analyst

you. Thank Okay.

Operator

Our line of comes Joel Baird. from Beatty the question with next

You question. may your with proceed

Joel Beatt

taking question. for the Thanks

been and the of of could made approach editing enhancements editing have type applied Is newer EDIT-XXX? to XXX%? older XXX And how of versions that by something editing to does be compared increased With EDIT-XXX? EDIT-XXX the that the that to that

Jim Mullen

Okay.

the a EDIT-XXX approaching guide. Exon the differently are So, little or EDIT-XXX, guide single an and terms editing dual all it’s a of whether EDIT-XXX Intron, and a in

So, back would and a a what I possible that advance you we Comparing what processes. both potentially really or and I there the numbers, is one programs focused maximize EDIT-XXX AAV to in is with I is have on technology dual AsCasXXa, CasX across two. consequence a think essentially we going say is editing with single that really format. to that the are But is because the all right generalization make do, don’t to thing different three not know as looking can at

that the nucleus, the configurations. molecular features of at the that expression around of gene the the looking stability expression So, all guide, introduction that of biology a other may lot sometimes in cassette, enhance the involved of the design then is different of guide, and of

system given maybe in of system spending approaches by different Each plasmid optimization. all of that contained that in kind but need from as system a And a configurations. generalizable, editing them have the an lot perspective. are some said looking kind at self would of rules, those I of quite XXX% is these time based at of a how that’s we arrived So, movement

Joel Beatt

Thank you.

Operator

Our Bienkowski SVB line Leerink. question the with comes of from last Rick

proceed may with your You question.

Rick Bienkowski

for and Hey. our morning taking thanks Good questions.

CMO like would first the company need all those and assumptions focuses execution, disrupted trial are could the question clinical is clinical So, CMO is Could projected while my a readouts? join when maybe who for clinical So, execution around of really trial it current And to hit a just base what on case or planning any around transition charge trial in while currently timings? to new order also, search timings ongoing? the in searching of around CMO. the elaborate looks a bit you of projected you the starts clarify is hasn’t

Jim Mullen

will I question the last start so first. Projected, with

when the are will, concludes, do So, those if in nothing projected all CMO have motion. a may search you because to balls timing with really

experience hematologist. And ocular We in physician second to the look that in there multiple programs, have MD cell as disease, who with we the XXX with a decades and of oversee beta clinical executive the of team, running, operations. with course, several addition senior She we trial overseeing this that’s sickle is clinical trials. a that of space. on a sort programs And preclinical those thalassemia, an have at hematologist the is area, collaborating of staff is the Mark’s overseeing experience trials managing overseeing, and

We trial also and data management scientists have clinical some the in data as activities. clinical the the about that think design organization some well analysis, look that and of

and the blocking just and reporting we new the will and look short-term collaborating CMO. I for report it, of team a piece it together whole will to operations with clinical more the call in I pieces of The he are tackling as it medical and as Mark we forward to pieces are scientific me. move so And that

Rick Bienkowski

That’s we got been deliver to few have in-vivo editing, off companies gene a a in there technology have are for That’s ex-vivo helpful. topic. But Alright, I a little second LNP and editors applications. it. competitors to look question. that embracing if some

on really like and I Editas’ While pipeline. incorporate to of not most be get delivery. why appropriate into Editas’ around would or gene vectors delivery in and are to why LNP AAV editing thoughts some technology this electroporation focused was of it looks your hoping just programs

Jim Mullen

perfect give to Mark, for to that’s a one that one going Mark. am I

Mark Shearman

have Alright. And LNP of do across an in yes, Great interest question. we a the deployment number of programs. different technology

revealed but an We have of that yet, for the we details is area interest an in this sure. have not that publicized,

Rick Bienkowski

got questions. Okay, it. for the Thanks taking

Operator

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

time. this at You may your Goodbye. disconnect lines now

Editas Medicine (EDIT) 24 Feb 222021 Q4 Earnings call transcript