EDIT - Earnings call 3 2023 | Editas Medicine, Inc.

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Operator

Good morning, and welcome to Editas Medicine's Third Quarter Conference Call. [Operator Instructions] Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Christi Barnett, Corporate Communications and Investor Relations at Editas Medicine.

Cristi Barnett

Thank you, Rob. Good morning, everyone, and welcome to our third quarter 2023 conference call. issued corporate release updates. we results our morning, recent and this a providing press financial Earlier X completion. A available section approximately in of the after replay today's Investors of call our will be hours its website Q&A. After our will we prepared call open the remarks, for

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

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

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

Gilmore Oâ€™Neill

our team, us executive good and Baisong Thank and of new by Mei, Financial Chief today Strategy Deardorf, morning, Officer; our Earnings Chief Linda Thanks, Chief Cristi, and Call. joined Officer; Chief Editas Scientific Commercial Editas you our today Officer; for everyone. Medicine's joining on I'm Quarter Burkly, members Lucera, Karen Erick Third X Officer. the our Medical other

quarter, sharing and momentum and Editas' to in I details. forward look pleased third the these are with We progress

June company. life-changing company's platform provide untreatable to back help commercial in this the therapeutics quick to a joined development tasked to goal take diseases. goal evolution realize with previously or let's however, from patients to guiding company is step Editas perspective. a I with to XXXX medicines . Editas' untreated that, Before deliver a

know, programmable January Editas we shared you in decision to in of and editing. of year, gene Editas' As many leader position strategy a this as

As pillars our a strategy. reminder, underpin X

development to First, sickle accelerate beta-thalassemia it and our approval cell launch. transfusion-dependent vivo towards EDIT-XXX, and gene-edited of for the drive autologous severe disease clinical medicine and ex

discovery XXXX sharpen expand therapies; development technological of focus CRISPR-based capabilities editing and addition to and robust to our can the in start development following IP the maximize the in of third, objectives. partner know-how to activities our XXXX, vivo in At to vivo that we business advance our our out-licensing Second, to pipeline complementary medicines. use outlined

update data thalassemia based cell to of dosed have of trial to provide for trial patients the EDIT-XXX, XXXX. SCD, or the provide RUBY To to a of by by the by TDT severe RUBY XXXX. update clinical from And for EDIT-XXX end sickle trial total in for MDD-XXX end a For disease the clinical XX or XXXX. the transfusion-dependent end

for and core medicine gene in hematopoietic hire HSCs And complement business Chief to leverage capabilities of IP editing discovery development, vision capabilities. For and our to advance with vivo business stem in the to development, specific Scientific other and and expertise robust vivo to Officer of our development or drive editing aligns cells to to our value a portfolio technology tissues. new

strategy we in and quarter. how executed third So this have against these objectives the

start Let's EDIT-XXX. with

First, on data. clinical

call. later both patients beta sickle the ASH, our details trial plan XX from tandem thalassemia on share clinical month. is webinar about and in a will to present with share patients on in next poster That December trial. company-sponsored We Pason will in XX. Edatel X a the more We presentation cell the at presentation RUBI data

Second, on enrollment.

X and continues pace. studies, our at a and and screening respectively, XX sickle patients good Ruby enrolled into cell have We beta-thalassemia EDITHAL

XXXX in trial dose to present to expect the track disclosures, patient RUBY time frame on January schedules. of details remain in the And a of our and clinical will sickle of Third, study now set on share XXX data on in headed for with cell to dosing, data readout based regarding his we finally, we XXth patients the December considerable data XXXX clinical further XXXX substantial remarks. the clinical individual middle due in the follow-up patient progress in RUBY

at for of Medicine of also designation intensive front, including, With a limited BLA. previously review respect the we and rolling sickle On in the medicines cell ago, leadership not RMAT made Advanced another hire as we cell severe plans, to important designation review continue for pursuing their stem we in possible hemoglobinopathies. disease. the development Regenerative drug programs, include baked granted of shared, Therapy expedited priority and Track regulatory we EDIT-XXX to to, of FDA commercial the the Fast as weeks position or therapy the are through us efficient but benefits gain the pleased FDA treatment on just guidance momentum hematopoietic or recently RMAT Advantages designation and all X to

highly In prioritization Chief commercial Officer. successful value as and creation. late strategies that Commercial announced joined Deardorf leader and Strategy Karen into and assets robust business Karen with a discovery has disciplined has clinical translate September, ability Editas experienced we to and a proven therapeutics portfolio early new our

early help facility room previously patients. record and and make as track Additionally, increase led leader the will global into commercialization, this clean our CMC successful further reach launches. when shared move To we she to in July, product ideal U.S. enable we goal Karen's Editas capacity new team has expertise XXXX. our her in multiple [indiscernible] for the

testimonies manufacturing, capacity, delighted EDITHAL EDIT-XXX forward prepare to In step and in review and well new powerful alike, the as for very transformative addition increased patient for commercial readiness. for gene need by Ruby The disease. the confirmed we patients nature industry we of recent a trials our were to editing for as see were scale ADCOM and clinical sickle ability own demonstrate supply to this XXL confidence significant the The and for and incredibly off-target the both ensure the the With our medicines robust moving AdCom. focused for FDA assessments. treatment our cell our of and in

in vivo and to now our development. pipeline Turning

year, stated in discovery targets we Discovery head Spirit in appropriate efforts. time. looks this Group and And our Drug cells sharing work Linda As to other on began at tissues. Officer hematopoietic therapeutic Linda more Berkley in vivo to earlier hired our July, Scientific lead the stem Chief as these forward

of we identify technical, that will targets As probability success. will and The genome target to work editing therapeutic under targets maximally target standard that selection regulatory selection for serious the our to differentiate allow a of criteria, diseases. reminder, maximize current will select from care criteria the commercial our approach new

development, Now license into let's we gene shared turn ex an as hematological for royalties payment received VorBio, that related be intellectual future with a utilizing for to milestone malignancies. nonexclusive we payments business therapies Editas will HSC agreement, and entered and the well agreement CasX as and/or property. vivo upfront providing for treatment prevention this eligible development. regulatory commercial In August, Under of on medicines the an

intellectual state of Editas ongoing and medicines. international U.S. proceedings. human Turning developing property and as foundational the patents U.S. PTO licensee is fraction a covering Harvard portfolio exclusive a and Only large in CasX Broad of University the holds our to patent patents Institute small interference a use these of the CasX are reminder, in position, involved

uniquely sublicenses we CasX these medicines, and to are licensee the seeking any to positioned to vivo for make including to enzymes ex vivo company As issue human users. in nonexclusive exclusive use exclusive and

we patients in robust and our our specifically correction and limoglobal. With term, call strategy Baisong, now by Medical patients long disease our value confidence focus, over meaningful EDIT-XXX a our medicines beta momentum and fetal serious now increases and world-class recently execution, the announced future. in with bolsters deliver that sickle driving IP promising genetic clinical the the Chief drive portfolio further build and to cell employees differentiated in may our provides my we are diseases. and our our data thalassemia Our safety to energized scientists licensing onetime that to continue MoBio to taking sustained anemia To will strategic editing efficacy in to June, and medicine conclude deal the progress shared turn sharpened of we that with in I durable clinically keen to benefits life-changing provide can Officer. early be with differentiated remarks,

Baisong Mei

you, Gilmore. Thank morning, Good everyone.

[indiscernible]. Let's start transfusion with AD-XXX dependent in and development of sector severe disease

his cell mentioned sickle in edit for the in enroll As we disease and continue severe remarks, barefacemia. and transfusion-dependent patients Gilmore the dose trial for to trial in UBI

XX have XX in enrolled the As the is and trial in XXXX patients be of dosed expected today, the patients time frame. to the RUBY trial, January RUBY we in

patients. trial In date, to [indiscernible] for the we EDITHAL have enrolled X transfusion-dependent

of sites, trial I enthusiasm been and patients with have recruitment, lives. EDIT-XXX patient support investigator visit earlier sites. investigators investigators, EDITHAL [indiscernible], As from to studies. hear both EDIT-XXX I'm that visiting continuously pleased our clinical in study and the the with and I I appreciate our already in editing the their continue speaking dosing shared have Ruby excited positive dosed I'm from at and this to and the changes the seen in with year, momentum

second As we the have year. engage half with we FDA of in the will the previously shared,

we this the reaffirmed note, and and On editing power the AdCom a technology. found of [indiscernible] has it recent related potential gene recycle,

diseases to As gene patients diseases serious potential the living a of with treatment I'm and that patient the for lives. excited ultimately, has physician, editing the transform

to see eager first approved more followed medicine the companies, and a by including developer, medicine As I'm from Editas. drug swiftly other

EDITHAL excited More the at share ASH a we poster as December both announce as and to Monday, clinical XX. will well on that Ruby in presentation in company-sponsored webinar importantly, I'm data

show. patients. we're will set, we Ruby clinical what from So data The data XX including

vessel patients follow-up hemoglobin, these including at will neutrophil follow-up. patients of total data, months includes present platelet [indiscernible] and including X with and least X efficacy X patients engraftment. with data, [indiscernible] at crucibanks XX least months The safety follow-up an and additional BOE and XX hemoglobin period We

will period. set X-month X from patients other EDITHAL The have data will to clinical patients. a follow-up include X- The

and up will The period first after X EDIT-XXX data hemoglobin total the including We X platelet includes feedmiglobin full data present data, safety treated. and investments. least from months neutrophil and at patients treatment efficacy including

have to The X X other patient follow-up months period. will

covers dataset a first oral data month the also from Ruby our first Congress European clinical trial. Hematology first safety company-sponsored shared data in X As [indiscernible] including webinar, hemoglobin by vessel treated and EDIT-XXX from to or promising range treated, followed patient including we of from the this The XX early patient a physiological patients from data demonstrating we [indiscernible] of early drives past correction initial reminder, Ruby EHA increase the EDIT-XXX Association sustained fetal normal data possible an those remained data efficacy as in June, after at the hemoglobin and hemoglobin presented treated of presentation in and EDIT-XXX total robust X-month robust [indiscernible] patients in hemoglobin, of since the as months Ruby total X second treatment. in drives of cell EDIT-XXX All patient excess treatment. free sickle XX%. anemia X the

patients Ruby of X tolerated well including cell engraftment and profile X mobility trajectory by transfusion. [indiscernible] dosing the And of red was with and fetal safety across [indiscernible]. anemia correction autologous to of patient elite stocking month was Additionally, stopped [indiscernible] hemoglobin and the for transplant. has of consistent patients, time conditioning [indiscernible] X the the of same EDIT-XXX and patients AXX a patient EDIT-XXX peasall blood within at follow-up was expression and consistent successful and

the potentially to provide potentially thalassemia, patients to and benefit with term. provide clinical sickle cell disease believe in severe continue EDIT-XXX that robust can differentiation transfusion-dependent clinical long We

We additional to and December. of follow-up presentation look in forward longer clinical data a our

added red to other resulting promoter in HPG health capacity compared the As cells, and critical normal switch matters. target improve hemoglobin cell human EDIT-XXX promoted off-target [indiscernible]. the ASA proliferative used previously to when Editing have on greater and fetal promoter. ASKX editing choice production increases our CDXX significantly including and we to when sales the blood of HPGXX and expression [indiscernible] enzyme compared proprietary efficiency enzyme editing of anything XX to of replan CRISPR post HBGX stated, enzyme edit in to reduce BCLXXA.

could clinical and life. in Based potential function clinical differentiation be quality we We of of that a X data endpoints on level normal and trials. the patient sustained EDIT-XXX. believe outcome point look Hematological categories the differentiation at hemoglobin reported so far, for of in parameters of

We clinical hemoglobin patients. end anemia an significantly outcome can As a to is important sharing damage. and Ruby total month. EDITHAL look normal for including data of sustained life ameliorate quality correction and improve the a reminder, of forward as organ clinical next data the level additional

Now I Chief call over Erick, will turn to our Financial the Officer.

Erick Lucera

in X quarter portfolio be the this differentiated field, impressed leadership our strong quarter in to press players few science, of today I our briefly you today and gene and, what highly I'm for impressed happy my I the editing intellectual belt I'm Baisong, continue more XXXX, summer, for the be to morning, Editas, other under financial work our everyone. you, see. even speaking with of to like field. Thank was excited good that, you with and to our and the with to With quality this I from I'd join by opportunity at release third summary and a review take refer the a earlier our issued I'll to items. property results

cash, million at $XXX securities compared to $XXX marketable XX, XX Our were and as cash equivalents September XXXX. of million June

nonexclusive bolstered license Bora essentially August from XXXX, to million, quarter payment expenses for spend the expenditures cash, to third and million, which targeted including in of the third reflects third the our were R&D Revenue reprioritization quarter equity including precommercialization our Bio in existing our quarter marketable fund The sharpened and program, equivalents million of in this $XX recent which decrease primarily EDIT-XXX $X.X offsetting XXXX. million, spending quarter were cash CasX expense financial costs. relates related position, the expenses, focus, of including by for primarily decreases efforts, the is upfront the deal. remains was stock Editas expenses, legal XXXX quarter into We quarter and capital $XX focus head under operating to offset securities of the of decreased expect from for attributable $XX our and expenses XXXX G&A third to third flat XXXX. R&D our various affairs June raise by strong advocacy. increased to capital discovery compensation patient Overall, and in on out-licensing reduced decreased XXXX. in which expenses count medical

ample of EDIT-XXX advance progress third and and resources into manufacturing research our and discovery our runway support Ruby continued of commercial cash to the provides quarter the Our XXXX in continued EDITHAL trials and efforts. preparation

value your As work former I'll the investor, buy-side medicine. feedback. our . sell-side hearing I've gene shared of I'm from before, forward as shareholders We buy-side know our look to that, the And we hand I and Gilmore. to value editing a I advance to call back and knowledge. with

Gilmore Oâ€™Neill

you, Erick. Thank

We with remains and sharing forward an our continue exciting to goals, and our our the we year As for momentum Editas. continuing execution of transformation it progress you. look our

you, must As employees, your for our achieve always, Thank to much without community. our that questions. objectives be you. support Thanks happy not said Editas, of we caregivers, could interest the it patients, and investigators, investment corporate we're partners in very answer and

Operator

Our JPMorgan. from comes Brian question Chan with the first line Instructions] [Operator of

Unknown Analyst

[indiscernible].

Gilmore Oâ€™Neill

some background incredibly the Brian, there. thanks was afraid you're it to much. problems connection noise, I'm difficult I and very you with suffering from hear know

talk you heard all I think I about targeting.

you're assuming I'm Yes. [indiscernible]. So referring to

is was because the that if your commentary, outcome. I to it's heard. the say that really robust the [indiscernible] generated outcome it genome just discussion the And . What the answered positive represents to go [indiscernible]. and have off-target was can so CRISPR confidence I as great disease, the actually countable not in patients first referred also, very very cell represents hope almost step milestone us follow a an tone. as for was where living I've you for we about of towards incredibly excitement think listen editing we day to approval the about I think but battling me what medicine. But struck now and can let a this AdCom CRISPR-based incorrect. with I warriors, it that drove our focused It then a data strengthened But we've a sickle substantially actually in for a the AdCom package great week. that significant a for life, question

in a want think our significant which compared is and to to I of fidelity when advance reduction CasX. edits off-target that we I the ASKX have only thing indeed other higher chosen course, has to proprietary-owned emphasize, enzyme,

Unknown Analyst

were effects. on if of lot I'm there focus just specifically a off-target wondering

gene just that sure that happy players in that monitoring will that be wondering doing than to now regulators work are off-target these effects. editors different the way the you're that is I'm are that here with different the any So if make is the you're market

Gilmore Oâ€™Neill

I hope than the this mean that into question. it's details, think without more confidence to about into are was that's from. discussed details. going appropriate data don't the go I our of comes I doing And AdCom. robustness the time we at package was our where

Operator

line the comes Semenkow Samantha Citi. with question next from Our of

Samantha Lynn Semenkow

just touched you Just get follow on as the bit between detail CasX. that you seeing a difference risk? what when different a CasXXa you how this, show step? about to little of be up the question, and And Can you're regulators to more talk last that to the in Gilmore, you'd able off-target sort and on

Gilmore Oâ€™Neill

to. happy Indeed

just preface the again of published sort in me Let with labs high-level data.

substantial and We to a have when in have we CasXXa generate not we anything versus it that enzyme. place continue package the to CasX don't are shown reduction [indiscernible] in and to off-target data regard others I'm Linda, that have add. editing targets, off to more if generated know, With share substantial. have the . you sure I details. is comparing

Linda Burkly

we additional we just at heard -- would and and compared said echo method to our and [indiscernible] in more Burkly, I what preclinical seeing in preclinical just -- using we're Gilmore -- to And we editing target yes, data, are our the in not experiments. so the -- proceed confident very package as AdCom. our path our what as in compared is Yes, would that I methods just add multiple add Linda CFO. we're we additional this orthogonal Yes, as methods

our forward. we're very So going confident package in

Operator

Securities. Joon line from The is next Lee of question with Tui the

Joon Lee

where clinical do on the been see something room AdCom, Based presented during that And is we differentiation? see to ASH. update. you that's for can expect for that the at all Thanks

Specifically, reticulocyte platelet can and you on your count hemolysis? engraftment comment markers

Gilmore Oâ€™Neill

are edit key clinical to just to of to design more we're the So that promoter. excited CasXXa competitive about AS going our choosing thing I date is little can XX. at a we we ask fast and and of consistent December ASH physiological follower already, we that we've by what level what to the global data with presented you which differentiation anemia the Baisong what was have seen to [indiscernible] think I'm for and potential see range, to about highest share tell as correction going very is normal what we in a a on

Baisong Mei

the Joon. for question, Thanks

trial. actually So And -- per deciliter, months, per with is to XX the we're -- and target differentiation, successful have and that gram as we the presentation, outcome ASH and are female RUBY longer go normal and see for from using to in XX RUBY are molecule and also for And we -- we hemoglobin the data to correct for for case, fetal [indiscernible]. there. data of our patients to will hemoglobin back during EDITHAL confident we and have have that to mentioned, very the we range And share see, pleased I follow-up very at we are data the patients, are XX XX-gram fast reaffirm, to X technology least I to and trial that we'll in patients ASH we in XX to mentioned pleased that a in as period with we X MOA the months able follower, RUBY with sickle data see pleased pleased presented the and the additional the also patients XX.X And total going question we then be very web and from and anemia. to to your are ADCOM deciliter our a differentiate a on as we'll the more given believe, and treat disease betasemia. we normal -- X male

Gilmore Oâ€™Neill

we're too hemolytic other it's note only that with markers -- is And than engraftment we've had think thing happy say space. early do that I in we I very It's are. more time also lines. think very the our the where to successful

Operator

line . Our Harrison the next of question Bank America. from of Greg is with

Greg Harrison

attractive the Just we for thinking do what the is when indications other do in there editing commercial vivo to cell and should think in relative technology? on you And candidates opportunity your how or an update then tissues ex expect vivo in sickle think you vivo efforts? are

Gilmore Oâ€™Neill

Thanks regard that company With discussions a updates, make can future and wants very deliver to believe on. much, absolutely is who promises Greg. to this make philosophically we that we

make to Chief been our on against that doing criteria. Commercial high engaging tissues. our over and ability Strategic last We here now to months. the want X team remind new with just Officer, time and selection medical to, targets question, year. again, she forgive has has about we the led to We have Linda and the again your sure already me ensure based choose work With been second by targeted me. our that Yes, conviction progress let's regard commercial

have tissues. actually talk [indiscernible] So about might to we advanced I have Linda. target

Liisa Bayko

you Yes, question. the Yes. for thank

to excited in -- approach of moving very on we're sickle locus success that seeing for and Baisong in course, has emerging and of targeting so the success to vivo HSC for program we're that the the based very to and of we're well XX HPG [indiscernible] disease targeting the positioned clinic described. that, cell an in develop the considering so So our vivo the an approach

through so working doing external and in hard up with come both for partners. delivery the to potential And that we're we're internally and strategy very HSC vivo

that's And interest, about of the future. other that talking we'll then so in terms excited be those we're of about -- very And avenue. tissues in

liver, the about be but in tissues talking the future. interested other well as I'll are in We

Gilmore Oâ€™Neill

to vivo like your of who question, what driving, of Essentially, upon incredibly in the can asked unmet that need of see expand burdens then or thus of family fraction was therapeutics, worth healthcare issue can tissue and opportunity the and We're and the only years States Thanks eliminating XX the there third about to the very treatments. remembering population to but system matched you progressively matched find about actually extends much, tenfold. members. of economies survival or opened conditioning, again, the XX testimonies strategy the of vivo. not that at By that an impressed and use donor healthcare by in And which for moving in going as the Linda. us XX patients X with described system are can can about population as expands was disease the patients further you the and United developed commercial from is their into the regard proportion a severe, finding the those the door age. parents a vivo, actually we're actually to patient we going number to And patient in for this ADCOM risks median about by with use these address and no XX% autologous donors. that it's a increased indeed, ex that we've patients about the that part patients substantial is mortality Allogeneic it's

there need a vivo eligible unmet commercial So the increase the patient population. -- and in will substantial massively is

Greg Harrison

helpful. That's Great.

. Looking forward the update. to

Operator

of Our next with question Huidong is from Wang the line Barclays.

Huidong Wang

a differentiating your for profile questions think about EDIT-XXX. the of I asked lot the

to factors to what AdCom. wanted to during pay surprising, attention these for ask, happened there saw events you I your early [indiscernible] thoughts then way, clinical is there? and try actually, we And of monitor develop what profile, efficacy differentiated your can in to order Xcel. VOE the versus profile a in I'm wondering kind maybe So events was so

Gilmore Oâ€™Neill

very much, you Thank [indiscernible].

want I questions, sure doesn't recapitulate to just So be all. get it your

reported have what on in treatment our pass differentiation disease, surprised I'll you start interested to I are then about and want monitoring our understanding our start profile. Well, we're will [indiscernible] were the think differentiation I the efficacy Baisong comment I the can at of some the for, and profile. -- product cell in efficacy of sickle differentiation on post patients that You for elements for talk we're to or severe EDIT-XXX VOEs you see And focusing as AdCom. in then

But obviously, something is VOEs, the regard look understanding this were think that drive of With presentation. that the There data more for presented, of is elements that able will to the to full cetera, to BOEs, we I be which a explanation in expression, at very surprising at obviously other being et looking the a not can truncated understand. data forward us get [indiscernible] hard with factors point. correlation were many not

think upregulation does what of that impact true stand a remains I hemoglobin on VOEs. actually the is has and fetal controlling substantive

Now look looking a differentiation the for of I EDIT-XXX, to to VOEs, other of we're more its control and focus impact, the with that just And correcting as disease. the that. particular we range Baisong actually on but talk elements correction physiologic with anemia and not about beyond of ask

Baisong Mei

still that are we differentiated Absolutely, confident we Gilmore. Thanks, that very Yes. believe EDIT-XXX a is molecule.

physiological that with not range see look outcome. that correction actually to temglobian. And reported as Given that, of into of that hematological patient end parameters, what organ have only total we we've seen as we well and [indiscernible] to can so far, function we

differentiation. direction be will to So we demonstrate that continue to allow us

Gilmore Oâ€™Neill

is a significant highlighting One complaint. fatigue we is key thing think reported I patient about talk outcomes when that it's worth

In not patient of pain, but the we Lack of fatigue and the loss just testimonials. heard energy. of energy, fatigue, fact, and again, hospitalizations

And specific so . these attention are subdomains outcomes paying to. or important the elements that of for we're patient

Operator

comes next Allen Jack the Baird. with line Our from of question

Jack Allen

the to RMAT? RMAT prepared expected you degree differentiation mentioned total the made discussed in little throughout on was I the on about during in a remarks, what To then ask substantial the wanted the quarter. hemoglobin Congratulations And data progress discussions. set more to the bit 'XX. a team mid

any be around viewed hear you here to the the what have thoughts could about and any to data During pivotal conversations discussions, RMAT did if set so?

Gilmore Oâ€™Neill

your for Jack, much, question. very Thanks

premature I think into go about details the of to is the FDA. first it's discussions thing the

quite including that that package by does data, highlighted out, how you as the correction as However, indeed, parameters, the and include happened correctly because I clinical we pointed the to am glad also anemia. of RMAT nonclinical hematologic did demonstrate design. review included you said, FDA And which we

in the as said mechanisms about it are forward submission. prepared various agency both . the essentially the as through our the think available the to rolling RMAT, increases confidence the we other possibility with engagement us, that remarks, review and timeless the as review I high-frequency is priority well that our point in with going obviously, of

period. a about I if by a continuing in say what to XXth middle patient generating the creating enrollment validates tells additional it BLA excel the XXXX that actually you or was included with 'XX being present our FDA data January study, set dose of really the time of of to a robust shows we've data XXXX. is that set our accepted that line the XL and to line us our a January data that XX and -- to to substantive added when a of An sight And Ruby patients You track of we're that frame efficacy expression at an sight validates XX and efficacy of around benchmark, hemoglobin look set into the robust period seeing able correction the in substantive actually BLA patients think follow-up as fetal to and What -- you in would the follow-up. original asked present on with I the anemia look during review [indiscernible] by seen being set. that

Operator

question is with Our Dae of Gon line Ha Stifel. next from the

Dae Gon Ha

off-target editing [indiscernible] I'll and go briefly then back Karen. for Maybe, to the

the about you off-target versus a I CasX On differentiation lot BUs. editing, of AsCasXXa Gilmore, the the talked guess,

around based bit on Just given work I think more need on you characterization the bit more side field. the view, test little ran offshoot patients, that blood theoretical that, all really discussions how molecular [indiscernible] in a And in didn't CRISPR-Cas your a samples and more little a of analyses there think post. also the CasX used medicine maybe I being prevalently were pre as treated is curious, manner, an but does these that,

you hear a to vivo of or or talk So you happening and delivery, target blood is post an in conditioning. guys know for can samples I but editing about but about Karen, soft the the [indiscernible] question HSD was are intentional if you you about if expansion pre then confirm if not? opportunity, see I not, nongenotoxic talked to didn't doing actually that question the don't testing earlier And

your you don't view Do as more a forward? to going on see Perhaps you that. I can [indiscernible] bit So comment a on Gentex of little if that know part armamentarium from that.

Gilmore Oâ€™Neill

regard very reduction we labs So I that AsCasXXa think first AsCasXXa. we showing targets -- takeaway differentiated ourselves that significant the data published to With with other much, in a is Dae off CasX, and is from thanks Gon. versus the published there have have

data package a of the have CASX we very for believe we prevalence that actually the would generation. off-target Secondly, -- change requirements don't

saw using that but nonclinical package we what very generated to the evaluations as And beyond adcom. also go orthogonal data way, vivo, both our in date tox silico Linda said, is our multiple in evaluations by including, at discussed Third, presented we're and and that vitro robust. we've in at the

again, to So actually adds are of the we all testing confidence. product. drug finally, that our then And

that's So kind the old of target.

remains that This conditioning. If question I an of area or then is turn your around could to to interest us. nongenotoxic

work. internal external have monitoring We're landscape. done the We also

of quite additional One of she out, correctly join of benefits one very is that as knows, things, you the that having stays the well. pointed us Karen

So Karen, a further I commentary. do can it

Unknown Executive

you. how And am here to I Yes. these the excited just be to help I want Editas team Thank to therapies move forward. with comment Thanks the for question.

us really the of modality. Now this be something you all that's absolutely bring to treatment part up and of point, evolution and space will a market of important important

with of evolve we offering, overall this a better risk sure think to of I strong benefit to the does take and and make part are try severe TDT current the . cell an but be we're is moving to we to forward treatment. very today important benefit the sickle very lot work seriously, doing for remain it that

Linda Burkly

patients to to a like the of do with test patients sickle not of just larger wanted number -- to we what affirm said, Gilmore in I off-target samples. had to add have sickle disease lots from that tested cell drug and larger product cell disease editing detected that confirm have we -- number

Operator

question TD of next is Nadeau the from Our with Cowen. line Phil

Philip Nadeau

Congrats on questions. X And from -- us. taking thanks progress for our

think to a years then first fatigue know question. not you'll what kind differentiated. continued of How X follow-up follow-up whether medicines of year? you the the Is trials. clinical to And quickly? genomic differentiation. that That's recruit recruitment of that patients second, to be in Do following approvals able you'll for determine? something assume truly rate are change of the need in sickle terms determine do in you end will a first the on disease be the the months or seems the that First, to likely you'll cell hemoglobin Or follow-up the long by that levels of if any relatively take

Gilmore Oâ€™Neill

regard can continue no, enrollment not that who very do pass change. and you recruitment, to investigator. Thanks with With that We Phil. conversations will I'll expect to Baisong, and much, about robust. our his sites experience any personal to believe tell we

Baisong Mei

been Really Thanks, have feedback on patients continuously we list Phil, this as and study -- many really And site strong momentum have visiting it. very your we question. for our about trial. for that actually

not see impact. do actually we So the

for momentum we'll will see continue to possible we that. see on momentum. And year, see So only next continue but we the it the to not now, and

Gilmore Oâ€™Neill

that this still and said they regard investigator that therapy with the area are indeed, notwithstanding So [indiscernible] the particularly approvals, is trials. our to an

another And that. asked impacts more Baisong differences differentiation total some about question actually then of other got around fatigue time And be in clinical ask to to downstream regard see or to about with the I've the to outcomes. hemoglobin. would talk You and .

Baisong Mei

yes. Yes, Yes.

a So very important is question. this actually

you on of see X kind the fatigue in based for outcome, X compared as you usually the for longer, kind something mentioned kind categories takes when of for that looking you -- outcome takes taking But other months baseline. time recorded this example, that the . see the that X you more clinical to that parameter, shorter differentiation. looking -- mentioned, Certainly, for will be of points specifically I into my patient are the hematological we're We usually, for end reported see. to experience as And we you more patient will studies see some usually you and to for impact much CBDs of when you improvement. year,

optimistic So pulmonary, liver, end well something I direction the organ the the reported to we be other actually and is we and on patient able outcome in and as we'll damage, confident category And very cardiac, CNS for kidney. also that mentioned into are and that. the see as that look for

potentially quite for forward looking areas bone for blood new, example, are relatively So but allogeneic the the in marrow limited, for we'll some brand, to also improve that function. the organ plus sico-cell patients are there are allogeneic transplant publications the improve cell system or actually improvement. There vascular brands and see sickle the system. patients, see relatively see in in reports vessel cardiac These transplant for that could also

So we're that to forward looking direction.

Gilmore Oâ€™Neill

have start when sort developers that predict et say have disease, unprecedented number additional that in Yes. are potency I the needed of vary using time And of patients, benefit firmly work. and have Phil, that clinical that medicines to to absolutely you're cetera, and require an ability as be the an Bacon trials one of I can new the block are in been drug long absolute demonstrate to point, measures outcomes enough is you the potent around things would applying new know actually

with longer to we're see slightly take we as that enough realism we very parameters functional a know parameters we're will longer. have it I are with we term. we that organ then to physiologic doing quickly. somewhat may optimistic, while that that We to just hematologic the are will also may be, So pragmatic about and experienced And but say, in an outcomes, other the regard see here, temper enthusiastic that work

Operator

question is line Zhu Yanan Our Wells Fargo. from the with next of

Yanan Zhu

Thanks for questions. my taking

I in variants. that's genetic for was of with of patients enough adequacy to target number the a prioritize at was the wondering, focus and the So patient tested off whether samples AdCom the certain risk

you higher The targeting single-digit analysis a as again, I numbers? next work, than you differentiator. is think I was Or a significantly about number sample off-target percent that. wondering targeting, fetal-hemoglobin are question are in your

I lot differentiator. hemoglobin and a also higher patients on was your in hemolytic patient that total X whether and first marker higher better on so seems talked is I additional that than But also who comment thoughts on data think -- the was correlated X thoughts hemoglobin based at patients with being markers your And fetal maybe between correlation i.e. at I a level, is -- can and whether whether wondering, wondering confidence hemoglobin. had ASH there replicated of being there, you XX% like you was had far lastly, I total a about abstract your reported about hemolytic total patients your [indiscernible]. about you greater a wondering in and of your it a you hemoglobin. on have

Gilmore Oâ€™Neill

Yanan. much, very Thanks

set complex to set cariograph to diversity you going a I'm a if questions, So quite and answers of try or question. of will,

very shows some informed AdCom vivo, to silico do remind I iconic good. regard view, vitro, at even actually first So the go think shown with at management, the animotopisity will evaluation in I should data risk AdCom need off-target risk the demonstrated to from of and I -- say, would that of ourselves thing so point -- that using what very and organal in I that -- our in that really data or well which we the robust sample, that's an the in is presented vitro on the the the and just discussion was what adequate at the discussion in the silico additional was ADCOM the beyond with set say are we package. assessments a

I'm And it's time not of the that going say of specifics patients, in share the future. -- just the to go into at about in numbers to we'll more an detail obviously, more. appropriate

both other very I the the by the on reaffirm that was want thing just geneticists parties, the I discussion actually to illuminating. that variance, think again, the I experts the thought thought at I about panel variants the in very the can really and the we really associated how how and as identify variation, with of all for of that. sponsor humanity well ancestry articulated nature and the common risks as nature clearly manage the AdCom

that was in it confidence of there. think the again, risk discussion. if robust add great I And very our data gave to don't generating you we're a management and Linda, want know I package to us that. it

Linda Burkly

summary. was good that a think I No, very

. think was summary very good a think was [indiscernible] I -- that I there that yes,

Gilmore Oâ€™Neill

different, that? thalassemia the what the sickle hereditary of is feed it's coincident described regards fracturing fetal the hemoglobin, excited hemoglobin With from fetal inheritance about cell greater And percentage Linda. you certainly days It's cell who much, sickle of the do disease clear add Thanks But is experience hemoglobin or for disease. say that want with percentage to indeed for us. to band the very of level obviously, I'd we very of persistence for hemoglobin have early Baisong, show. data the to the a days people or higher fetal early yet.

Baisong Mei

question. Yes. this Yes. Thank you, , for

on mentioned, a this to, over looking XX%. with excited are very expression. [indiscernible] promoter the rational that. BCLXXA, stage. referred to of also forward patients versus -- data promoter approach to better feeds we the But compare the It's Jim this we as targeting we see for that have are more we in see more early global want HBGXX we're is EDIT-XXX. and [indiscernible] on see found this you design disease, HBGX And pleased because [indiscernible] and We we is to As are data, to actually approach We that that.

Gilmore Oâ€™Neill

much, Baisong. Thanks very

and markers. hemoglobin was question correlation final between your hemolytic around the think I total

that question to before handing X remind thing. an think that, interesting just is I'll Baisong, I

Baisong, While is erythroid I . of production. part total you a to critical want believe design cell know key hemolytic The add if by disease. we sickle enhanced driver for hemo anything driving hemoglobin don't is

Baisong Mei

we're right, exactly -- that's to say. Gilmore, that's going Yes, what

mastic we I to so I is what the our the related is very Then positive that. hemolysis, total data that that. to One of want about level [indiscernible]. are mention market think hemoglobin on is aspects one --

the better think we and expression. have hemoglobin high [indiscernible] the have better process value have I design, targeting with -- molecule promoter we HPG we So to also this in of our And protoproduction. designed

very data see So . we looking our more we biomarkers. pleased to forward with on are that, but are hemolytic

Operator

Cantor from Fitzgerald. of Our next question is the Schmidt Eric with line

Eric Schmidt

this Perhaps the -- and of for editing HBF you if Karen. much deals there level a overall hemoglobin? be gene Maybe progress. a follow-up the of the collaborations. might We cell production patients saw what large platform-type to you impressive. are in that obviously on levels in couple maybe the hemoglobin week. which have so, disease on interest pharma then first, too And Congrats comment quite concerned potential could above sickle start a total Is grow

Gilmore Oâ€™Neill

physiologic the Thanks hemoglobin substantial very of that's hemoglobin, believe is range that benefit. regard to we've Eric. what we With seen, and total much, correcting

I -- think -- polycythemia it's with an has it's to that population hard broader with speculate we're there about -- But our as I some data a been way the the correcting say, the to in conflicting of not sort we about -- Indeed, general to feel experience confident total physiological normal a in to ranges. and level regard is patient same. haven't it going high. that getting hemoglobal. too risks in are data we very of about we're we

look portfolios. to genome space that therapy both final CRISPR derisking some the critical great of the of announced. would think leaning and is increasing for it what I editing this that now I striking they just it pharma, first is been some to pharma a In week. ends you step asked of period I to . genome excitement with genome and seen is the deals is where say their we've approval editing other this say question CRISPR-based dirt sort deals good actually substantial words, has in I that view about editing near would see in of pharma week their looking about what say to What see gene a their very essentially is the -- towards around are value as recent up of editing for big space. now grow our a

Linda Burkly

I Karen. is refocused well so now, It's I is Eric. add Thanks, would the Editas think portfolio. Yes. having positioned What right

path it to continued programs the forward a are should great us. partnering, opens We very move in for the to our excited are right able place and for by be door and be that own interest, the

Operator

Our question Raymond Steve of Seedhouse James. with comes the from next line

Steven Seedhouse

ones. quick Two

share that poster then baseline on separately, lymphocyte . commercialize intent the your ASH. own? United transplant, something And your and presentation is going EDIT-XXX to you counts or at and our post associated are First, to neutrophil at [indiscernible]

Gilmore Oâ€™Neill

to engraftment in of our at comprise monitoring we that that neutrophil that's We're end with Thanks that part your do is question, at very a question. believe that With in to it's the present engraftment look on our important. which it something that we could XXX actually because because -- year studies. to did be our and is was much actually our building that measure regard of commercializing then we And we own. the the presentations actually summarized -- second XXX. actually towards safety would regard or for Well, commercializing around data, of

partner would was any have large that any Obviously, and the in depend share of then. in that that something an really kind would previously but upon with we're We It a footprint. details might only how interested agreement, U.S. . partnership indicated expand ex we those negotiations. such much very of

Steven Seedhouse

just clarify, to just was post longitudinally to Gilmore, neutrophil [indiscernible] counts don't like about I because baseline. asking transplant, lymphocyte they and in recover the data,

just editor. I'm given So different to plan same you it'd or be share know it's and whether different if the that's something curious the to target genomic and interesting different

Gilmore Oâ€™Neill

our we that. . actually well, not date, I accounts. So happy up -- our with been with on accounts can but to very have we've follow We're happy what actually very seen

Operator

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

Luca Issi

just like peers X who Great. trying to appreciated. -- a a here. scenario by -- any is those there Two your broad adolescent you much Are if so include on in quick label you're initial January color obviously also track for -- of a maybe any ones XXXX. adolescent get patients label sounds again, XX some will here. it enrolling there, versus Maybe based second And on adults just Linda one, also include that where understand your quick

to I think and before week XX during off-target further manipulation. the characterize this do was genome AdCom a the whole in sequencing editing genetic risk actually to earlier patients suggestion potential acquired after

you're I to if Thanks something do. so So that's much. contemplating wonder just that

Baisong Mei

take Yes. question, Thank I'll you for that. and first I'll your your question. pass

that's and kind I because all this of a program, genetic ages intending -- are starting disease all actually is with so have young be able we patient [indiscernible]. to intend for plan course, those dosing disease we all -- kind mean, ages think to general of to things, we to age with the benefit we also to mentioned the have discussion label will very patients this further from from clinical FDA. essentially to And all with go alignment model you I intention. that have of

Linda Burkly

for AdCom. Yes. question. at that Thank a very was interesting your you the conversation Yes,

of the up using in -- method the biochemical and methods subjected which orthogonal method also methods One came with genome is and the to and method your that DNA -- of a for this which use, naked taken a we our AdCom you of sequencing then editing. applied [indiscernible] one was off-target cutting DNA. make a at this the basically to method is to from do is selves And look whole unbiased

And is called so this Digenome.

And that product to before so use drug of drug at this is our we our the methods one product and look putting with patients.

one that of criteria robust that of -- methods I the we think -- robust our approach. So in so is we the one this use

editing to this So guess confident share reasons were to I at -- one that confident approach package Guide basically use are about moment, I'm and the also what our that to in very other make to our I the that there silico but in to us we the that that's we're that many approach we aspects prepared off-target described are think that's basically preparing. methods -- our addition in to approach other were of

Operator

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

Jay Olson

on and EDIT-XXX across these to progress. some post-approval was about just on a and Excel the Can Maybe setting? read discussion Congrats the share you follow long-term of monitoring in from question patients. just another AdCom term thoughts plans there long patients your since surveillance

Baisong Mei

Thanks for the Jay. question,

a will fallout have for certainly long-term we patients. -- So

actually with that consistent pull study who actually so up dosed years is anybody and the And EDIT-XXX for requirements. also to patient the regulatory designed to XX with that's

So plan. . our that's absolutely

Operator

The next of Bayko ISI. is Evercore with from the question line Liisa

Unknown Analyst

This is Julien on Lisa. for

questions. X have we So

expect XXXX. more to CEC as or substantial set the we the I inform to XXX. -- dataset you regulatory you Appeals for I'm do an if you more you the patients? third timing We're mentioned coming that should of that? then data know that? the that the with alignment for mentioned for of hear sickle the results in when middle the decision that what's of is And second, on seek the TDP First, just engage Court half, substantial second cell expect plan when appeal we for you has the wondering And filed wondering path so, in And will the the to to then Street FDA discussion? for know

Erick Lucera

This is Eric.

any those until in courses of is the We -- to Just front comment that. respect front in court of on rendered. CVC want cases final to like or don't decision with anything the actually really

So just of we'll for all we'll awaiting anxiously that be like you. just

Baisong Mei

Yes.

for over Then next frame is, for January continued those patients patients So a next part based just year course. question -- the will and one follow-up have dosing have as program. XX shared will the And about time by also we by of we of that middle substantial dosed your year, for of have the on patients, year. next XX second we we as mid that,

year of over disease. So we cell lot sickle middle next the for have data Yes. a of

I in year, next not continue goal dose. I'd that X saw cover both, make Regarding. is we actually strong the have patients on we we enroll how the And sure patient -- shared, enrolled, with absolutely. many what that for for we have a we very study EDITHAL momentum we and just just -- but have Yes, Yes, and also that. shared probably because EDITHAL patients dosing actually patients. already your but And cover question to

appropriate an that and third is your share . And engagement We about of question will your in time. FDA part outcome.

not with FDA And we We'll the as share just we us of time. these we yet. management time appropriate in with have the including the the to But shared share designation engaged well actually to lot agency, -- so have frac a FDA, unmet we with that mentioned engagement agency. more allows prevent a and have have outcome the we're senior interaction line that continue

Operator

question is the Our line of Foroohar Mani Leerink. next from with

Unknown Analyst

commercialization. to us And line question, on potential ask could side, other scaling the and you for This update for package on the for of manufacturing in I then on terms the if development the potentially is an clinical of for give [indiscernible] XXX, wanted for just progress BLA. Mani. second both

-- was that see readiness had from if might potentially wondering to think I you timing, the therapy with but I potential would program. mentioned how you potential So package post-marketing you then and by gene XXXX, pivotal a impacting design? have data we .

Erick Lucera

Eric. take This timing. question respect to I'll first with manufacturing is and the the scaling

As investments the the what a in we're for confident making and in commercial we're very doing reminder, manufacturing launch.

timing, on just a scale standpoint. manufacturing we're everything doing haven't reiterate on specifically We commented but our or the confidence in

Baisong Mei

This is Baisong.

Your second question. part of your

which volume, next As efficacy the XX like -- by equivalent patients next by January and time year from patients dosed we XX substantial which in by data middle cohort. middle have we that XXXX, packages is FDA, of middle BOA have of shared will accepted then by next we data of have probably will the to access

in addendum months X the XX added months data subsequently they So additional patients additional upwards. X with

[indiscernible]. we package by exactly that to equivalent or with treat will that the do FDA excited with you mentioned package of exactly as post expect validation and you that, that when action middle FDA we actually earlier data . But recruitment mentioned of molecules. Boat of And we commercialization we I cell the the validation of not very hemoglobin -- see a -- CRISPR sickle too. package have the And align the technology, to discussion. about of as have -- AdCom data the further feel that And you able momentum fetal next so are AdCom the further agreement mechanism that FDA perspective, will have shared that about these the on So be X the package equipment we're about we file we will what year. this to marketing and

So we have actually to see impact positive all a those EDIT-XXX. work

Operator

of Our the final Stanley. question Morgan comes from line with Flynn Terence

Unknown Analyst

[indiscernible] This for Flynn. Terence is

how Also, about future as and about going tissue-specific evolving bit, approach delivery programs, can you in a advantages So target expand think and/or selection, should the to you thinking to LNP? your to investing AAV for we in forward? pipeline future you on your are think your the looking how about vivo disadvantages

Linda Burkly

question. your for much very you Thank Yes.

our care. that are that have selection, differentiated already delivering we're So important met that is as as so that from medicines that far approach Very are of we target by existing really to apply standard needs patients meeting well criteria the are not therapeutic.

have as we commercial to as are have in targets conviction going and And as high look success. which well profitability that for as technical so targets well clinical we for high

this very triangulating Karen targets. have And as so partner can her having so I'm as to excited well that here and select much joined to I Baisong our with

[indiscernible] think where, we're cell we're I now I I about said getting your sickle have emerging really target. to be that delivery. well vivo think already excited to data target we these supporting disease the question Because very selecting in as and about before, positioned

Where our strategy, which delivery. nonviral is Gilmore described January in

strategy. working nonviral through approach HPGXX to targeting HSC we in amongst deliveries vivo partners delivery are prioritizing And promoter drive an an and as our LNP well external so internally with for for the as LNP

there space. but interest think As in with interested of also in [indiscernible] deal course, we're tissues. that. tissues, announced validated technology recent far showing delivery and interest the as the our as and [indiscernible] positioned targets well saw especially so -- continuing we LNPs with we're other -- that well we're And just for interested I And in and other are this

there targets [indiscernible] -- at to in out time. emerges are so and excited there an as be it many the certainly we're technology, as you amenable we forward, appropriate sharing And move and Thank with future we'll to you. information to

Operator

This conference. today's will conclude Thank you, everyone.

disconnect participation. thank for lines time. this may We your you You at your

Editas Medicine (EDIT) 3 Nov 232023 Q3 Earnings call transcript