EDIT - Earnings call transcript Q2 2022

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Operator

Good morning and welcome to Editas Medicine’s Second 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 second call. And XXXX we Earlier update. 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 statement, any any as only if or date. not as to should and representing

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

Jim Mullen

our Chief Baisong Officer; everyone. as welcome Medical Scientific Ron, team, Financial of Shearman, I’m the Executive Officer; new today including Chief Chief I Editas Michelle who our and Gilmore good Thanks, Chief Officer. and by thrilled morning, Mark our Officer; executive joined Robertson, am several members Mei, O’Neill, to our

Board. became Gilmore and Executive the assumed of As the you role I X, know CEO on Chairman June of

ensure team of CEO the seamless Gilmore past two the months, Over changeover with partnered closely the and executive the to I’ve role.

CEO. appoint of to fortunate a as leader very are caliber Gilmore’s We

biotech’s for medicine to including his built leadership skill of on at nearly and leadership, has passion patients Gilmore’s are company experience, for towards commercialization. past Gilmore positions foundation and senior and development medicine poised right experience of years the call CEO, clinical leadership are years genetic very any pleased undoubtedly exactly With led the few I’m XX the Biogen, some few indication and that, experience successful we’ve his several to drug next genetic development lever most clinical success. proven over abilities, the he If first over are During Sarepta our in and programs. of Editas’ years focus O’Neill. to set months his turn lead the new

Gilmore O’Neill

And everyone. you, good morning, Thank Jim.

that Let am delighted be me to say today. first I here

a in to editing forerunner development am life technology opportunity As create continuing very want potentially medicine. the gene the also Jim to company over I you and a Editas of such of CRISPR to the honored strengthen people. few to I talented I’m know, work changing team to alongside and and Editas group the happy past dedicated is years have to thank the for leadership lead

the With Chief Medical we new leadership and in complete place. a stronger Officer, even now have addition team of our

has impressive. differentiation chemistry product serious of led pipeline proprietary make will discovery. Editas then These RNA expertise current diseases. have drive science my start Editas The is therapeutics continue and even seen create given drew a technology areas develop to a that enthusiasm Editas. distinct bioinformatics to commercialize transforming in with the nucleate assets new the untreatable and in since to two what company to assessment. enhanced me is including coupled giving high precision I from foundational platform into do technology arrival enhancements potential. company’s by is company’s And potential my me of will you a efficient What confidence has off Let the therapeutic reinforced my has numerous initial more edits. month me lead enzymes, and overall treat company previously that

addition, a very expertise, a our posing R&D have group, supported In discovery pipeline. innovators creative by found clinical robust in assets CMC team and of strong I

bench to place pipeline, happy Mei Medical with have on from is we registrational specifically, years disease Baisong multiple and importantly, in several him, us ahead. Sanofi, drugs started therapeutic of I attest Sanofi, working global experiences Baisong a both therapeutic breadth effective has knowledge Officer where and his areas, privilege record the disorders. non-malignant approval forward substantial, officially as welcome two rare track our blood pieces to proven Dr. to and and drug clinical the much had ago, disorders. and these very project Medicine. which of approval. of new as to can and leadership weeks for also trial head He’s incentive at support to to oversold to advancing Biogen, Baisong I’m including served we on from global our across With and Based his why work global Prior now where blood so more clinical and He to on through he execution, Chief translational, of contribution therapeutics focus months joined Editas the senior where hematology, he I personally bringing X the leading about development rare skills. in to look extensive

product my data EDIT-XXX provide disease cell thalassemia. on would our program thoughts initial I sickle with our autologous pipeline Now starting like for transfusion-dependent and to lead

anemia, patients in patio or sickle hemoglobin of a and is events HBF, know, a gene a independent In resolving and suppressor. high that the you edits of this rethread manner sickling turn, mechanism patients. reduced dependence binding EDIT-XXX thalassemia fetal the unique disrupt of transfusion that in of to many BCLXXA in durable levels action the provides resulting As vasoactive of and of promoter in cell utilizes gamma-globin and

that an ex In using CasXXa autologous edited is product drug high-fidelity first time this been nuclease. the engineered addition, has vivo AS our

prefer future registration us the efficacy from will by partial package, quarter. The cell clinical to engrafted. FDA, all in cell have sickle with a RUBY we and of cells continued edited hold patient disease, the patient allow collected momentum lifted and was which has for disease dosed We data trial in EDIT-XXX the successfully past build first that in have the patients to We include successfully patients. additional sickle and

We and continue cadence enrollment, we create help top-line to before track year-end. provide to to trial data remain on a steady sites of expand clinical patient

designation thalassemia orphan received year-end. by and TDT in remain drug the patient we for we May, to base Additionally, track on first dose

Moving by LCAXX, dystrophy now to for vivo CEPXXX inherited devastating mutation. our autosomal program, EDIT-XXX is retinal lead in which caused a recessive

efficacy the This into high support in pediatric and been been patients endpoint The profile occurring pediatric benefit safety most thus patients. into a morning, gene dose registration has the has Phase proof registration. to X identify therapy to the in in generated identify concept from of allowed and use study is patients up to dose to far and encouraging To we EDIT-XXX the very to achieve baseline identify for The ongoing move we can that date advance announced likely and has in study of that LCAXX characteristics using benefit us retinal Our our study. BRILLIANCE photoreceptors. mid-dose a to optimal of registrational data and segment objectives cohort. two to several that a balance physiologic have risk editing clinical optimizes designed that and dosed

patients to a to development. data of at we assessments update remain identify help and include we on on and all have evaluation. trial months on least this who the efficacy year, BRILLIANCE the clinical most endpoints to will safety later six registrational provide track support follow-up As had today, expect of which relevant These sensitive trial

safety for to the and for developing editing edit through EDIT-XXX a trip in have foundation we the that the of administration our within machinery programs lays CRISPR/CasXnzyme The success and the demonstrated photoreceptors LC proven of subsequent in subretinal platform. can program the vivo. the retinal effectively injection, capsid, of cells drug development Beyond popular delivery the vivo

in our For example, for than easing. preclinical of addition, efficiency program all these leverages autosomal-dominant has with much editing RP EDIT-XXX XXX% redoxin higher and demonstrated EDIT-XXX nearly elements

So both program vector EDIT-XXX and substantially CasX even programs editing use efficiency enzyme, and the EV-XXX the though of same the greater. EDIT-XXX is the AAV

as the are time trial appeal licensed ruled portfolio, these Rod and protecting Editas referred Moving Institute the intellectual California, therapeutic the a covering year, Earlier reminder, to for development. favor. CasX against Broad prevailed for Broad’s exclusively to co-owners in property the third of this the to PTAB in our University when patent along patents the collectively or board patents with CDC as

sole filed for will CDC an for States, confident is remain Federal appeal for anticipated, with again once issue is nonexclusively, the the licenses Broads, to exclusively Circuit, market indeed As IP either Editas that in rights innovative and which we Editas United medicines. the extension, dominant therapeutics retain CRISPR/CasX human the and will If by the case, or prevail. growth

not is We strong platforms coupled exclusive medicine commercialize new at Ex-vivo property Medical value our future right both to with to our IP CRISPR/CasX our will enzyme, stakeholders. Officer, is to reminder, our grant intellectual cell companies’ a a licenses position, significant Mei. driver by proprietary use believe group over which Baisong ASCsXXA to that, circuit to. any this IT Chief turn I With for our As encumbered desire

Baisong Mei

you, good with and excited very by Thank and be drew to you. Gilmore, sharing Editas. today, off start Thank I’m we’ll speaking morning. everyone with

CRISPR-based genes the gene closely by evolution medicine, editing. I in gene the I new Editas’ editing last would was was therapy by to its in disease years merchants of RNA working approach I I and leadership and therapy diseases. address its sign the Firstly, work of field. genetic in quality company unique the several of spent impressed triggered so and and monitor

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of cycle. XX enter my drug a had in have as of career, CMC over development segments Scientist privilege ago. work life industry the Over pharmaceutical all years course to the I I

will goal updating my an to look approval through subsequent I calls. develop to forward progress Officer, Scientific our for and provide Mark, and at who over is leading which myself assist responsibility a development clinical experience clinical took first-in-human While a discovery is approval. where CMC valuable been research, in in from in a has role I and my profoundly bring I discovery developer on With on right in development. on efforts. treatment. to to consider in I to general, will update heart study I medicines global development, and you leadership I to our research need opportunity clinical the R&D My that, to In patients turn the clinical has an to Editas help who working discovery direct work call clinical had have was I with team. After also Chief research CMC, impacted transitioning our a end-to-end stayed. be

Mark Shearman

Thank you, Baisong.

on EDIT-XXX Gilmore updates begin for As like Row-ADRP. with to EDIT-XXX, has provided I’d EDIT-XXX and

gene differentiated vectors EDIT-XXX effective data two a As rhodopsin EDIT-XXX different cell. reminder, same is the The replaced of in organization low preclinical NOCAs and therapeutically nonhuman with gene XX% at be simultaneously only cell productive XXX% presented over the while study. the address also that knockout adeno-associated more XXX gain function, primates meeting photoreceptor The components approach preclinical and can reduction highly we to replacing to the nearly ADRP. knock treated agnostic active NHP gene retinal one. with improved in mutations in replacement, that if the a that ARVO May, morphology virus improved correct demonstrated order the which At mutant mutation uses the functional group. gene delivered potentially generated and that and toxic in in from retinal a data. to the superior and data of photoreceptor and This gene aberrant to replacement editing are cause of functional also in occur can EDIT-XXX showed approach that proved than

product, we further is As presented. that also for potential already upon we’ve the there we data continue think to improvement optimize that

to and initiate on on the also encouraging remain work recently received this approach we our an Based on FDA’s the feedback We IND-enabling filing. input on studies and its year. we later EDIT-XXX program, ongoing FDA IND track expectations as

Immatics continuing apply In fifth Moving that world-class this cell research an egging June, to our adoptive delta we a announced therapy, where gene and cell collaboration therapy gamma Immatics now agreement, program. to CMS T-cell area will believe both platform. a are and pursuant to the to solid licensing with related about momentum T our we expertise. We cell allogeneic last technology to our opt-in collaboration alpha-beta strategic cellular to has sales multiple develop both this collaboration compared and our optimally which over XX months, T efficacy heme these program collaboration therapeutics The enhanced that study. this in incorporate their with was in opportunity and Bristol-Myers pleased T programs adopted is to BMS In tumor eight cell-based novel and that engineered will morning of the announce program covering therapeutics. in vitro in demonstrate cells, This the collaboration use all applications. edits clinical give earlier that we for potential were advanced Editas’ into recognition. characteristics us with an therapy optimistic was editing pharmacodynamic early Squibb, tumors both believe technology to numerous has currently engineered partnership. open The phenotypic the from most multiple improved we and type target that tumor collectively for engineered door and an antitumor IND-enabling We killing T vivo. cells and improved cells,

medicine are making with with currently to we nuclease to that NK antitumor preclinical AsCasXXa resulting for we medicine solid combination complete ASGCT tumor our NK Annual of EDIT-XXX an have on iPSC-derived wild-type that for over end lead development. an the an dosing models, focus over potential in tumor a solid development in significant could in frequency fix same potent data have in May, course pleased patients existing as programs, These our EDIT-XXX At survival preclinical the mice cell-based model, potential all program tumor lower persistence cell a the EDIT-XXX of and XXX-day approaches. therapy in model, lead developed cells this compared study NK and demonstrating very improved we tumors. solid INK burden antibody-induced dramatically cellular increased important reduction, in-house data with progress in at our cells which in mice treating technology, experiment. support XX% such in Using as the activity our clearance of tumors, are period. allogeneic substantially that cell vivo presented in we engineered EDIT-XXX Further, area advantage engineered remained the the program For Meeting mice the of impressive alive for and believe we many proprietary with the

are One to drug and key final that platforms a update. risk platform differentiators into immune NK contrast, of our the of expand typically INK our feeder cells call system using of cell-free and product. Officer, this into of GMP use edited cells, the the introducing scaling iPSC Chief that, Michelle system process is exogenous Most financial the the facilitate approaches getting into for mature developed is cell cells. involve use INK that program In cell-free of differentiation differentiate over of cell review feeder turn our inherent iPSC to segment the feeder in the a we With I’ll with defined components currently to Financial production, manufacturing. our an

Michelle Robertson

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

and to equivalents, in prior $XXX marketable were $XXX June quarter. the million compared cash securities cash, XX Our on million

and disciplined expense for We with under our continue is for $XX an XXXX. BMS. driven for half for last mentioned, announced XXXX. to first were the first million quarter of R&D be $XX the half compared in to primarily manufacturing increase to million expenses were year, second of same extend $XX the the cash our were awards to of The compared to Mark of the last for This the that in compared the expenses. million half and year primarily the with the period G&A expected same Immatics. was $XX was million decrease year increase collaboration program attributable investments, And same employee-related clinical licensed additional performance year. by $X granted to into the $XX for our year primarily by million was we runway period as collaboration in recognized with XXXX. increased was the a and recently expenses period driven XXXX lastly, million first Revenue management

an future development, Editas this payments on With part strong commercial Editas upfront for remains is and regulatory and products receive we payment Editas milestones. royalties net sales As collaboration, additional any of may programs. Overall, on based on cash position, also eligible this received the advance that, collaboration. I’ll continue back in in call and result that our financial to turn Gilmore. will to

Gilmore O’Neill

Editas. the reiterate for of Thank excited Michelle. how I Again, to future am want you, I

have technology platform our we are enhanced a simply capabilities, company. While no we exponentially longer technology

to our commercialization building end for we have Over the the is while patients, several clinical of thoughtful Underpinning our very innovative cutting there’s on proprietary and development, platform. operations we a manufacturing sleep thus years, our innovation. remaining life-changing out therapeutics technologies, further go, are on and sights gene-edit rapidly focused on medicines through transition of transitioning edge past and this teams to advancements our company have the our knock-in of which

look business continue activity to foundational to forward advancing to be discussions our also active with property. and We development related intellectual

forward, that regulatory, the store technical, commercial in in of success. This of is Moving this and current assets will future rest pipeline. includes year. our maximize prioritize possibility for Spot the we and programs

data, side, use and will of visual trial more That year. update for segment EDIT-XXX our vivo for later optimal retinal functional On focus CEPXXX photoreceptors, editing the providing future and in clinical on of identification the this the be additional will update registrational BRILLIANCE in POC comprehensive on the a safety the data outcomes in we study. patient

the for plan for studies later EDIT-XXX year. IND-enabling initiating from feedback on we row this ADR Following FDA,

which sickle and cell potentially in the to for to include disease RUBI patient. top-line later have expect we second initial ex-vivo, data patient from year, this data study will our Moving first clinical

the editing for first apheresis ready Q&A. Editas support anticipate before EDIT-XXX patients are have year. for product for all end of thank and the begun call to we beta-thalassemia we of all the for with Editas. screening you And study the We interest patient dosing and Finally, base open your thalassemia up of as of that,

Operator

with Please Our from question first Wang Barclays. is proceed your Instructions] with [Operator Gena question.

Unidentified Analyst

Thank you and This the on is third [indiscernible]. -- taking for Gena. two I our have I for think patient question. Tom questions

understand size be more I the estimate trial would the would with and when Secondly, the dose So few first, indications, do earlier, side, able products? but be on in Or a other you to of do rough to it required parallel? trial FDA’s the you be period now following both next enrollment able have advanced for experience that? patients given might you

Gilmore O’Neill

us second After first we his rather with Medical an patients when independent quickly where cumulative space clinical the Thank a closely those XX in afraid Baisong happy supply to hold of to us to necessary data are review ensure to to have size new manufacturing I to study is monitoring regulatory feasible couple and you. authorities be or to of execution. the to Officer, With for the on intention have why and this but recruit we have initiate in have one days, our enrollment, I’m I’m space, your double we protocol couldn’t this our really down available the days, and will enable monitoring the we move FDA a for and regards our third but as follow and safety criteria that of as we patient. that Chief patients I our designed experience the them in guiding of catch is set that, the XX and name, capacity so specifically for data numbers. current is reasons help while approximately Mei to the peers of this we us study, propose other are

Operator

you. Thank

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

Joon Lee

quick Thanks the to the On cells need have think you a the taking and Or management. the able are manufacturing with differentiate compared forward as have lastly, enhancer how with is Promoter I presume Right. using that and additions for questions. you EDIT-XXX, edit? will engrafted? to of clinical disclose Do looking benefit? lead the could show for edits And to two EDIT-XXX update more consideration? that the you a how biologic quickly better up to et follow-up. progress to bypassing our questions looking I Also, a from to And EXO-Cell? are new I

Gilmore O’Neill

very you Thank much, Joon.

will to we actually as gamma-globin end the the not parameters. be time, of when have your discussions, benefit our patients. we themselves as will over to be regard outcomes year does of world, around hemoglobin. as data there at we expression think increase in hematological disclose able declare longer-term we previous editing the actually I durability planning a I So the the parameters be fetal promoter to said in That afraid. believe engraftment hear follow-up but we believe not differentiation, anticipate over -- of displayed that actually will we’ll give the clinical say, may you the as that With third have be the of we clinical I’m well important hematological could for we which question,

Joon Lee

much. quick No, A on Delta. think to have delta you you that Mark. strict you Gamma Gamma you What might delta gamma multitasking. I answered the the so was your are missed program? and for Thank method it. I I as applying drew follow-up

Mark Shearman

applied certainly Hey, the the program. have program provider. Joon, used the we opportunity gamma to can support DMS be delta equally can Immatics technologies with that really I the We that. T program answer that to Mark of it’s here. Many like magic cell the the

can As to earlier getting they we looking targets have yes, that forward that list nuclease a the of to but is, opportunity for we number technology to program. that AsCasXXa press the from to and indicated initial had them, apply short the we’re answer up in program, release, nominate

Operator

you. Thank

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

Dae Gon Ha

half over Gilmore, point us. all I term. questions we June’s speak taking strategy guess marketed? secondly, potentially for up have presumably to Then dual Mark, the looking why wanted the I Gilmore walk and guess compared and dynamics CTXXXX you’re here sort on would when you the I a can differentiation. vector, just talking to at longer nice follow morning. would question using we Good follow-up vector of utilizing EDIT-XXX, being about questions single that from us or perhaps read-through get would progress back play be on welcome differentiate and Great. a board to and itself to it be our the idea see CTXXXX just IND through how given you EDIT-XXX anticipate that that is on of so about at superior update of from EDIT-XXX the the EDIT-XXX to on BRILLIANCE, looking anticipate anything congrats underway? we enabling long there to the a out as could second differentiation Two you seen that you as Thanks declaring that between the be before market would potentially differentiation studies as be again. much. Thanks

Gilmore O’Neill

Thanks very talk much, again. to Dae Gon. Good

that the clinic. something the read-through the on address the to differentiation around would question long over will actually That XXX be me and Let we determine term. first have

in to long However, see the say to hematological long-term with the through in I maintaining reading patients see durable will levels favorable address near term. at health. level regard market those hemoglobin the side that to the ensure we organ that reading term, medium benefits and could would is do see We to something on based beta-thalassemia would very dynamics. in you potential to clinical long and both sickle parameters on end the cell a we of could see anticipate persistent we through homologation term. and In feet here

challenges pull-through that, approval will we to CTXXXX I or large think to this treated while of commercialization market, at our and with again, here recent gene challenges too. I’d the CTXXXX therapy patients like reimbursement. our a time the therapeutics anticipate number be so-called gene access may site of remain experience based large a of and because the actually negotiating what not well to one-on-one as is launch around first actually therapies other seen address that number and launches, capacity, on one-on-one that as the patients or we’ve lead from observation untreated of around of and

for XXX, then those market of we believe believe product that this we For reasons, our that treatment our of is and do could the differentiation be there choice. with

Mark Shearman

size the you up sectors, the both probably by the process sectors anyway, reduction. And of knocking targeting where component the can can those Then transduce that for two-step data this The is way, really answer that for human with on happy from effectively are gene therapy all who we your photoreceptors photoreceptors but well FA both as injection, as but photoreceptors with similarities and dual EDIT-XXX, CasX pretty similarities as on it’s Ropotoreceptors of and gain why as take of down take both edit two vectors the we actually, which dominant it. the mutant rhodopsin reason execute disease the much actually photoreceptors, split therapy production particular, do with a removing endogenous autosomal component tumor lastly, so And those probably XXX that’s With the you opposed function at so of EDIT-XXX, which using it negative remember, and other we to only the is to the by we’re pectosystem our we’re of based question an then that the wild-type on between mutated the for because AAVX subretinal codon-optimized way that with EDIT-XXX. the I is with a moment. capsid I’m dual dominant the programs, end. to The any so them has a and can cone with only rationally know replacing think replacing start Vexosystem. up

Operator

you. Thank

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

Jay Olson

dosing the thank on to for Hey, on also can cohort introduction, everybody. for welcome of expect color when the second and the review comment and you EDIT-XXX, you IDMC Thank questions. patients, expanded the pediatric on thanks on there next any to see can also complete or I just update many we color taking a is the clinical and the mid-dose in the cohort. the you can pace you you. to the management of in just of any expect Maybe should enrollment data And team, want dosing terms the you year? new provide in when safety and how when in cohort, provide half

Gilmore O’Neill

Jay. Thanks very much,

and track start at will data So before to has end from of be completed data frame on efficacy the of end and clinical at or end October-November let the efficacy say the provide the of me update around just point the that view. that we provide an mid- year. in year will been we guidance from Prior the and dose data cohort adult time set, high the

to it we the from will have and actually timing IDMC, we those high-dose evaluate at slower as scheduled cut is point that is all patients, pediatric would approach be to data, that pediatric of than in we the initially mid-dose safety, to IDMC independent two given to including enrollment, enrolling regard expected. used us meeting start a enable was the decision Then the For meet patients which practice. quarter, with a regard cohort. monitoring be the committee this to good later the With meet to data data scheduled is would mature will the

really I’m here that double efforts is clinical One and down has on us, joined the to execution. reasons our that of Baisong

near will say have amended with amending yet, be we’ll we the future. but a and that in line and I of anticipate sight that are faster. I to have the complete don’t you have We share approach enrolling actively do that

Mark Shearman

to one upon adults be well. recall, on in the to as data Great. a and add adult evaluation high-dose that mid-and the to ourselves the option into as expand it’s used reliant comment possibility, obviously, Maybe of gave we you merchant the

Operator

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

Luca Issi

another to get How single-arm the and the approved on for Patel this the the is blue taking Can a assuming earnings on the on three concern? talk approved what you my organization getting ask biggest ahead Luca wanted you trial? Mr. decided about a hesitation your sickle for you Reena And likelihood cell, FDA question. ask Vertex or possibly Issi, about run are given trial? of to reasons maybe you you. and I O’Neill, you is just CRISPR of was just this head-to-head why join first thinking to and can call, Hi, thank top

Gilmore O’Neill

Gilmore Reena, the for to say, who one want time and clarify. company I’m company, been Sorry, the to some Financial. Chief has company question. joined I just just a based for I has as Michell, just the your on thanks is with This happy here. am the

me, the strength design enzyme the I the the have I its would saw So what what of particularly that science high-efficiency things, expertise that a forgive maybe and me addressing not importantly, CRISPR fidelity, evolution, to of and be technology, is of just the that number clinic. is sure company say drew were a just to the fact foundational that very want now in and example to the question I me differentiated in nuclease the history core AsCasXXa we have and which engineering, high of here nice but the actually one you’re

our within important for quantitative In guide is RNA. RNA, our capabilities a also in sit RNA. and us both design addition, substantial our there which in a particularly the guide Other aids around discovery design biology, of group, guide expertise chemistry

precedent was you’re and actually me and and be positioned as as well. very think question or the clinical the are around in And in to FDA well for confidence Michelle sickle we Vertex and our that on impact us And talk FDA see and outcomes is both would joining. cells. the and that field two edited that quite might which I technology as treating our our hematological autologous XXX. we clinical are design. platforms, on, that frankly, scale are approvals importantly which evolves, the for as the impressive gives enables CRISPR those quality things using developing discussed speculate patient actually of The a position, the finally in have cell did I well the as you some that tell design what as our the both not well the with currently, population. was we’re How designed that as other but there seeing more Then will us we of some will but approach recruiting cell in parameters population to described as concrete both confident, the the cash I and Finally, clinic way strong in look we the but CMC the with are important CMC, patient about delivery to that currently obviously, beta that other about fact more I the AAV is well have are examples trial capabilities, things to approach pants, want both are supply, at we as drew

that all It I other was enables to it everyone actually FDA. to a trials do efficacy marketing want clinical part that partial of hold as because by use our ongoing the does in removed the Why matter? of data application. remind us matters is The the thing

Operator

you. Thank

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

Rick Bienkowski

Hey, Congrats beta-thal. taking the good is EDIT-XXX on our two of There question My development from progress are thanks morning. for and first and questions. us. regarding the

that beta-thal for the US, thal would currently what about or in program Phase have upcoming programs challenges partnership get and therapy the setting some of treatment and the competing an agreements For Given beta alpha opportunity thoughts gene thinking a and the delta a be along can was question, make the is I for product? the keeping starting the partners developed program rationale for stage here cell date in right this therapy PDUFA not your with T in-house. sense. outside my owned to a is being license would with in development whether for an there hoping on of INK to fully X gene the both approved candidate speak second trials you gamma with

Gilmore O’Neill

Thanks Rick. very much,

when of will to believe number actually, value opportunity or we continue We patients our going cell treated first slow, we a both and launch, believe and that but as we very it believe be of EDIT-XXX, when it’s indeed own around to number look and actually ongoing product, access the the to space uptake differentiated would we initiate that we has into the regard for the strong, that commercial with recruit at beta-thalassemia we whether at time challenges for we So patients its right, the a on approval reimbursement. that certainly trials that the remains because believe. relatively sickle we believe challenges of and important, outlined, I’ve

for actually spaces. in studies. that ongoing Edita reasons as as there that able those partnerships pointed and in out regard you our We’re will program to this the RUBY With is some to be to partnering, confident immuno-oncology we also value execute enrolled our well and

INK said, moved has has the Mark As well. program

interested at or partnerships. potential looking the always in We are factoring for

well the maintained the of seen and great of of when opportunities value way joined for its that patients position the broad good very Editas think that a I technology I One stakeholders is a the to challenges said for one have partnerships is I that organization and and with applicability and maximize I platform. we technology our looking as to said maximize to the including other for our open that are as broaden execution open number partnerships bandwidth through and and so before we partnerships have at INK to our are platforms, of across

Operator

you. Thank

and with from is Company. with Please your question. Philip question Cowen next Our Nadeau proceed

Philip Nadeau

programs taking questions. for our from thanks ocular on couple Morning us. A the

It with experience that a pediatric you design us registrational end EDIT-XXX, the on the modest time. trial dose the only year. like think by to the of you’re have to I by First, pretty seems defining high guiding

data information possibly on you necessary that the and dose trial data? can dose design high When that the registrational important how So without endpoints? the provide efficacy high dose patients

Gilmore O’Neill

at by not for final do I of we’ll bring concept have selecting adult in selecting, year study history natural important entire discussions regulatory population we have and also the and Phil, set that that and to segment guidance. and and high-dose a potential have thank large will a simple that defining designing, and we seen way, at the endpoints we engagement. that adult the note will have anticipate data be will the a and we evaluating question. data registration in reason doing that performance the set our authorities, have of and good guided including be adult be also your we to data and in looking from by is you to that proof that What broad with population. also a looking helps patients would have you, regulatory meet pediatric of with we say, mid- those because that population end look at able It Good understanding one endpoints, at that to patient subjects, set spans us for interaction FDA. data, to to should our but given very the will we a the not is We looking actually

Philip Nadeau

rescue EDIT-XXX, very protein nonhuman level about disclosed in production is editing That’s and protein shows is that the XX% primates. disease? second XX% question data whether helpful. you’ve data production Then sufficient on that human of to there nonhuman What to suggest the primate

Mark Shearman

and cells, That’s approach knockout otherwise -- be the which are study Yes. Thanks, a that we that the in retina. rhodopsin that to statement. that eliminated arm corrected those that could were led that percent so in in the be make knockout-only sufficient able is to Phil. factors was replaced itself the there and photoreceptors lot two one. with rescue in would that particular demonstrate experiment, Well, from of us One to XX-ish

the from In and that sufficient to expression where in dog to are some The work guide second and XX% Beltran, system. expression is though those be us experiments model level UPenn Art William can two IMD&A knockout ongoing in we their the of that case did they What is There to and change Cideciyan that said believe, photoreceptors an a we’ve they in that approximate the also rhodopsin. are that, hands, was study. is level a rescue that still experiment model there’s that sufficient. replace the showed with modify production shRNA and versus information the well-published together, that way Group, lead-up Those was that will levels, particular things in that of pieces conducting the we reduction to replacement experiment. the about editing

Operator

Thank you.

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

Joel Beatty

to as using on the of natural registrational arm thought the opposed Thanks trial? control use any history in taking question. FDA the for cohort of gotten feedback a have the a On EDIT-XXX, you

Gilmore O’Neill

very Joel. point our within much, natural important internally the very history Clearly, in been that our development. we have discussions around of has clinical Thanks cohort a strategy fact a

authorities, a with and history yet not have BRILLIANCE be with well we trial and the design study discussions natural authorities FDA. the in optimal prepare will clinical both but through regulatory we as our as consider using We such had the interventional the those as experience for discussions the

Operator

Thank you.

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

Yanan Zhu

wanted to my Hi, question and appointment. to thanks new add congratulations taking for I Baisong my for and Gilmore your

in is methemoglobin do just CTXXXX the call. manufacture more data up how need we XXX where for for review touched you induction whether from when expect patients in to you improve Also, follow compared many study, you we RUBY have the first in question just CTXXXX. the promoter related think want data, at freezed those at in patient currently unmet the your year-end? XXX editing or Also could specifically data expect see could you. clinical further patients differentiation expect set the with I more and potential year-end perhaps million areas? data to earlier should greater and/or Thank you XXX, I available and see First CTXXXX, perhaps on to the upon the your those approach product update, data you with how

Gilmore O’Neill

Thanks to meet nice very and much, you. Yanan,

Let preclinical and to of address back the data. also some ask end Mark at and I’ll the work me and start site

we’re could With product, simply time potential independent so because and we the mitigated is fidelity, actually share highly hemoglobin editing targeted, to actually believe of durable, the two that become of fetal unmet possibly data are important our has is hematological are CRISPR important expecting year. AsCasXXa, planning given of fact differentiator, efficient to independent. end to vitro be highlighted of and what differentiation that the that, durable the Gammaglobin would it something as include a as and in an to significant it high expression which the and expression Then expression promoter the is data. the to regard form -- two the addition, we’ve data using the and a well fetal very advantage at with benefit expression I’ve patient and we that anticipate doing elements long nuclease the we cohesive, regard a over owing differentiation. drives believe independently increasingly be that is preclinical We to engraftment It durable clinical talk this a that there but it in confidence parameters. and in differentiated ask genes cohesive, of confers term. need, of by from one That of safety patients the just our hemoglobin to little to durable of fetal going to around our maintain bit hemoglobin, that Mark CRISPR will

Mark Shearman

derived the Yes, model, a BCXXA whether cells, the but with Yanan. preclinical editing the maintain importantly, levels, promoter or the HPG promoter preclinical some slightly and to the program, approach, mechanisms in least whereas in region gave HDF TCLXXA Early at fidelity line also there conducted queuing. was region that the from lineages the different team those direct the the the of higher itself editing we comparisons of

data some we have that the be and BCXXA which will concerns I that’s also the at editing be an on and there has this advantage and than think factor, we data then efficacy-wise, believe long-term, determined differentiation safety-wise, point by but transcription with time, in clinical could more is function, emerging. one the

Operator

you. Thank

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

Ryan Deschner

study, Ryan your note SAEs for you. is product? Steve. the any have patients is in sickle general there of wanted cell particular with in to cell and Thank for Good what been ask, and morning. for associated Deschner expectation I This platelet the neutrophil on engraftment

Gilmore O’Neill

Thanks, to Brian. Good meet you.

of Second program autologous the and for does but but that places around is importantly, has been more specifics neutrophils, actually specifically, the give product. this year designed we at but that the able but about and as we itself our editing, The progresses. patients, expectation will general, generic and actually Our end not have seen near beyond across of and to I we obviously, not of our adverse experience for the question to risks to share seen varies protocol in for it the experience very would engraftment be we obviously, more all not carry XX. both SAEs to that Obviously, that XX we’ve and day closely, very have capture the in experience procedure think date, and engraftment we in events general, monitor concern are that aware identified SAEs seen the us

Operator

you. Thank

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

Unidentified Analyst

This for Madhu. is Amay on

profile meeting what of my us a given do a efficacy question, what to separately? need sense registrational you For to pursue a of present dose you trial, and first give do you see a value XXX update or medical at then kind at could

Gilmore O’Neill

that we’ve our want to of adult stability we improvement extension With seen criteria, would POD regard with and what is the to in success some an potentially of patients. see the

the a criteria be data, will evaluating this our currently the for a will study will anticipate in our population be I co-analysis think or select And patient regard Phase are to by webinar that determined obviously we of a maintained study X where that interaction dose, at and selecting part therapy we it’s to and endpoints actually selected for will study later year. set the be success Phase segment of as natural interacting but improvement study. the And the And probability the that of the or being also and share success patients. actually by with responding other selection X of history dose. identifying be of likely say, of the has stability that that segment with analysis dose would be you determinants a that the maximizes of a maximum with also of determined to of

Operator

questions There call. This are further does participation. your no you. Thank this conference for Thank time. you conclude today’s at

disconnect. You may now

Editas Medicine (EDIT) 4 Aug 222022 Q2 Earnings call transcript