Earnings call transcript Q2 2023

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Operator

Good morning and welcome to Editas Medicine's Second Quarter Conference Call. 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 Cristi Barnett, Corporate Communications and Investor Relations at Editas Medicine.

Cristi Barnett

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

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

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

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

Gilmore O’Neill

good Baisong Medical Editas' other and commercial platform Mei; Lucera; year members Chief to the everyone. joined second Christie of Editas with X evolution guiding company am our therapeutics morning, tasked Scientific company. our joining our today I development new [ph]. for Financial for Officer, X Burkly Officer, Chief by Officer, Erick Thanks, from Linda Chief and executive ago new quarter Thank I us its today you joined earnings call. Editas team,

beta as of for of vivo launch. Editas' the reminder, approval toward year, and strategy development a thalassemia clinical accelerate cell and the this objectives comprises EDIT-XXX, just strategy, we sickle set ex cell our of January XXXX. therapy pillars: and X to We drive In first, -- for disease a and shared autologous

technology therapies; group and our and by Second, our editing organization strengthen group translational to discovery a discovery in sharpen and into advanced activities development our focus vivo to third, partner expand IP therapeutics an discovery dividing in it of maximize capabilities out-licensing the development vivo to addition and advance that in CRISPR-based robust pipeline medicines. can our know-how business our technological to to complementary use so

update thalassemia XXXX the clinical transfusion-dependent from trial patients total EDIT hiring our end XX EDIT-XXX the EDIT-XXX trial Officer our XXXX, new of dosing portfolio and RUBY in other Chief RUBY we to for and the editing a Our end by are providing and clinical development by which expertise update of And of discovery gene objectives EDIT-XXX by in tissues providing of our year-end, technology for a capabilities. trial XXXX. to with editing advancing robust have hematopoietic Scientific stem vivo specific value business complement just drive leveraging done, or IP cells aligned and vision a

strategy how and objectives executed the this against quarter? second So, we in these

our to we strategy. side, the On X leadership hires drive made important new

highly experienced medicines contributing or and successful has new to we our Editas record candidates. Linda and joined outstanding of scientist Linda an Officer. as Berkeley, multiple of the week, late-stage Scientific a discovery last Just therapeutic foundations that inventing has respected clinical approved track announced leader Chief and

Officer. work and Financial we Corporate Lucera, Chief our buy-side tissues. Eric that We Officer ago, we on Also, in investor, new in other Linda's stem weeks build the Editas as highly experienced X leadership are and executive current forward had Strategic joined editing Chief finance cells looking former as hematopoietic to a vivo Financial biotech announced

this on hear call. from will You later him

standard selection target of that targets approach from of on XXXX we future allow trial select to identify to serious of year. and this our clinical of work trials will dosing webinar. differentiate in and targets trial We from genome parallel XX editing both our reminder, of these date. As we data current our Hematology European maximally diseases. the year, therapeutic second this our RUBY will the we and new under criteria at to the patients a success. criteria, care that about probability June remain a will maximize in of the Association's developments you patients from commercial end by our commenced dose a Meeting track tell and regulatory technical, RUBY quarter selection Linda target company-sponsored EDITHAL at RUBY In The Annual Scientific presented EDITHAL the data at for

year-end another provide to track by trials. on clinical update both trial remain for We

of share progress well on provided We takeaways and as months. further the regarding coming will roll clinical will XXX a as developmental details in data the RUBY remarks the Editas we further in recapitulate in update his and Baisong that June. share on

common manufacturing and stated, plan of EDIT-XXX from have build extending the year. our And in EHA XXXX runway previously FDA to to transform engage second million follow-on our pipeline serious our towards DXX we of we $XXX approximately treatment quarter and third continue as we prepare webinar, net as company-sponsored the of the we BLA, the of cash raised our After with issuance stock, diseases. advancement into half for commercial of proceeds our the in

quality for medicines towards footprint to support continue to expand patients, progress to launch. clinical of of for our preparation goal and manufacturing our life-changing supply commercial we we management our As delivering also and

[ph] clean RUBY and prepares new room facility increased AsierDevons manufacturing moving supply This support are trials of commercial facility. and our the to new capacity and program, capacity a will scaling EDIT-XXX readiness. increased for recently EDITHAL We clinical

position. intellectual to property Turning our

in Institute's of now international licensee in patent of small IP and a PTO a and provides the portfolio Universities CasX Only future. portfolio Harvard patents and a covering involved U.S. the holds Editas is of reminder, CasX Broad patents the foundational proceedings. use estate large exclusive As ongoing making that and medicines. human confident remain meaningful U.S. interference are fraction We these our value in

in that and with long patients a We sickle are increases be sustained of seeing promising robust that early that safety anemia data in driving can cell clinical medicine employees scientists our drive deliver our disease correction June, to year. or shared may fetal life-changing and durable genetic We EDIT-XXX patients and differentiated beta onetime our term, and and thalassemia our provide in energized hemoglobin. throughout to by benefits medicine the world-class clinically continue new specifically focus, strategy we progress our efficacy on progress look and our editing strategy execution, keen to build medicines With updating diseases. to executing forward sharpened the we you in with serious strategic to momentum to differentiated

our Officer. call the I Baisong, Now, Chief will Medical turn to over

Baisong Mei

morning, Gilmore. you, everyone. Good Thank

with sickle disease. for start trial DXX the RUBY Let's cell

As dose remarks, patients in and stated RUBY we his Gilmore in continue trial. to enroll

RUBY year-end patients provide of remain clinical As we we data shared, trial and and on previously additional by. dose total to UBI we trial parallel year this began to in earlier have by XX track dosing in patients

We in on enrollment will share further the updates months. coming progress

As the quarter. provide half to commenced In Gilmore edit We in by in We transfusion-dependent the the track from remain XXX the trial additive trial engage dose trial year. FDA second parallel in TDT, for also mentioned, we with plan an beta year-end. clinical second of to of to update continue we the this an additional invocation. dosing on or the thalassemia,

accelerate for a TDT of strong and done trial, have the XX development RUBY have also measures we we As [indiscernible]. to the for multiple are taking

have completed have dosed have We or prices. patients have or multiple enrolled the been in XXX additive process who and of cells

like the trials. our the followed clinical to X-month the first data patient, from The Congress, total by minutes shared and These European first Rubin following: treated of include the drives including take June, a from with the Turning correction December. feeding. recap in patient we or robust and XXX were results X the and takeaways our cover would presented oral Association we Excitingly, EHA, data the RUBY we treated, in presentations the segment months last lime patient to data XXX data. to shared hemoglobin XX or first consistent an clinical sustained to the the data clinical drives early data data from In dose of few a elite XX%. the also X free positive June. All EHA hemoglobin range some [indiscernible] in of where as X I company-sponsored XXX safety efficacy data Hematology key from RUBY promising editor the early from the treatment. normal in remain at VOE, of robust as since events vessel-cusive excess initial presentation of in data including patients from pathological increase hemoglobin in in RUBY patients months webinar of

myoblastic by busulfan X stopped [ph] patients [ph] consistent stem of All X better safety retail neutrophil transplant. of same and of on and time at was conditioning collection points. was with and profile patients, assuming showed and and of well That is expression transfusion. full and X the patients XX patients engraftment tolerated the autologous the have dosing within patients across dose fetal was month hemopoetic successful consistent of hemoglobin And increased patients treated cell trajectory secrete RUBY Limiar

X. the the as months of X hemoglobin more infusion increased physiological X after levels to hemoglobin follow-up. treatment fetal fraction increase RUBY after X data XX the X, XX of XX X's months X follow-up. at hemoglobin patient months detail; persisted patient study level the Patient in by RUBY follow persisted Looking during hemoglobin total and XX.X% normal X trajectory fetal X, during The reached normal and total patient and months these same

just For This XX.X% deciliter X AXX in achieved dive, the X that after first XXX X.X gram [indiscernible] RUBY treatments. of first patients. or experience months over dislocation with resembled of per patient

provide that the potentially believe XX robust clinical long-term. to patients, continue We differentiation clinical to provide can in benefit potentially

CAF. CDXX calls and our target enzymes health when red choice production, human of when normal previously promoter. matters. cell editing improve ASKA As of and HBGX compared greater fem editing including the blood editing we blood and stated, significantly to red HBGXX-editing CRISPR X to X added ASKA [ph] uptake the in in efficiency BCLXXA. gene [ph] switch expression increases capacity resulted uses editing cell compared on enzyme to CRISPR proprietary reduced to have other port

level that far, a normal Based on could point potential we clinical sustained differentiation so hemoglobin of data be AD. total of for believe

of a improved clinical of as the life ameliorate sustained total organ level normal damage. important hemoglobin reminder, quarter a As [indiscernible] is for outcome correction significantly an and and patients

sites trial [indiscernible] clinical have been I support and As I appreciate inclusiveness investigators. shared continuously quarter, with and last the study RUBY investigated when speaking we our visiting size. our and I

in the excited updates clinical investigators year trial of to with I'm We progresses, XX data additional pleased sharing I'm already positive in patient patients dosed momentum dosing with see in changes both and forward by the recruitment, RUBY XX as look year additional studies. to from the hear editing the including their lives. that end.

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

Erick Lucera

everyone. to morning good and Bison you, Thank

say to excited editing speaking field. first quality came strong highly to was the company's I Editas differentiated IP I'm me to by that today. Let because leadership its from the science, players and portfolio in work other be its the impressed gene I the its you of field, in

this results shareholder will issued now, this exciting a quarters, company second human vision I shared to our was commercialization our join and time, XXXX. data guidance. refer drive eager we're and our future leveraging medicines of to asset I bring for to quarter take experience for financial this like of for the returns. just a look the give and regulatory And to Editas items. With to today pivotal press summary preparing few I'll patients take more forward help to and briefly earlier strategy. you execute innovative review opportunity lead I'd our we I my that, In at to to opportunity our release for

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

slight expect from second program securities the cash a collaborator, Revenue $X.X equivalents is the our last Squibb, capital XXXX. occurred XXXX in opt-in to quarter support quarter Bristol-Myers million remained did quarter We expense occur in expense. services to that were third activities, into related our our increase expenses expenses quarter in partially decrease in million XXXX primarily same operating the in was the existing of a the of increased offset by for quarter of quarter $XX.X of the second for compensation XXXX. and year. to fund the million expenditures compared and is second license flat of from G&A second BD The marketable period cash, expenses that not attributable stock million $X.X professional decrease XXXX. $XX.X for to The relatively to which

following our in in R&D expenses by of accelerate representing our the expenses discovery raise were continued of clinical this extend efforts. our focus, on progress to ample and strategic $XX.X sharpened the decrease portfolio, provides XXXX Overall, and continued commercial our million, a further our coupled us the EDIT-XXX. financial our our from a EDI reflects activities quarter resources capital strong recent development million focus preparation Editas and $XX.X reprioritization concentrate remains and allow advance decrease runway talent to manufacturing which execution offset in The the second XXXX. RUBY from trials increased to quarter of with position into cash of discovery EDIT-XXX, the support

As a look value to as from know company editing the investor, and advance the of forward medicines. sell-side our with hearing knowledge gene former our I to and I working we buy-side shareholders and work

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

Gilmore O’Neill

forward EDA-XXedithal XX of patients total capabilities. of in providing thalassemia gene study cells in year, full look vivo robust exciting year RUBY to from progress our reminder, study objectives our clinical with by -- for been RUBY our It year strategic portfolio the and me from include the by drive Erick. IP has thus this clinical you or transformation business update our technology tissues our trial fulfilling Editas. data value advancing a sharing as as a you, and the an and development transfusion-dependent XXXX, continuing at by and year editing year-end, the of far stem of a We for complement Editas to other discovery for of providing and EDIT-XXX end editing first end hematopoetic dosing for EDIT-XXX Thank we leveraging a update

As must achieve and said of the our always, our patients, without support investigators, we you. it that not could objectives employees be

much in very Thanks Editas. for answer happy And your you. to now. interest we're Thank questions

Operator

conducting be question question-and-answer Instructions] will we Morgan from Terence Flynn Our a first [Operator time, this Stanley. session. At with comes

Terence Flynn

vivo wondering for update could And on me. lines then provide your program? the Two facility, time was just in any I if Great. for going the you ASR secondly, what's on in do trials? there make bridging you any FDA an facilities perspective? product or work the expanded And the any required from from be do to first changes to use have two to

Gilmore O’Neill

really in an appropriate point from -- time make the to With are engaging when in in And on we Linda has much, regulatory as and the questions. anticipate of facility, the is maximizing going had are we'll against with Thanks processes. view, update a time we of APRA very chance we work I've our is or continuing to welcome to is progress. engage to drive are a positive double our anticipate down I'm The help Asia year with don't essentially planning to forward. It's success for to obviously, really delighted vivo, us at Berkley your And of leadership and to property Linda FDA standard focus this commercial on in transition. technical settle said, regard mark. conversations. on clear really lines track Terence, particularly of work here this And and major care. and whose half the have differentiation the those significant you the the

I our largely as We with we're to the down. engaging are locked for commercialization we really say, have readiness just there. aligned were that ensure agencies on And

Operator

next with Our comes from question Citi. Samantha Semenkow

Samantha Semenkow

you going Now on stem the Linda vivo how of challenges the discuss forward? hematopoietic could mitigating that about some you thinking challenges delivery team, these you're to of cells have and in

Gilmore O’Neill

I've has got say, to question. just Samantha, to joined. has your Yes. Thanks much, very for say, Linda,

how if I and anything, feel to to going about and talk free. So please I'm want -- add you lend, she

our of aligned that and really approach things philosophically the highly this. are to Linda on One I

we to deliver hematopoietic functional the obviously, work -- challenges. we are X to The good stem feel The the we enzyme have that that that. certainly the progressing news is guides and actually executive some X have made have and cells. is We challenges, work ongoing on of solved

fetal and of our and that first be our you The in use at our an of there also, CasXXa is thanks the time. stem progress the being promoter, on they're targeting on hemoglobin we update delivering hematic currently to cell have validation targeting happy in we we'll the something have And our mechanisms working trials, that those to XXX to enzyme program. clinical validated human more specifics appropriate specific and of man

Operator

from Our of next Greg with Bank America. comes Harrison question

Greg Harrison

What the at program. from update recent you feedback the EHA, XXX have since had physicians

Regarding And in data response? from a hemoglobin cell, to respect what set with clinically they sickle meaningful looking for net update? especially are the

Gilmore O’Neill

much, question. very Greg. to to Thanks I'm Ben ask going that take

Baisong Mei

From And asked how feedback our with the and normalization as well have already really that's see question will say, Thanks, energy our feel too. the patient told they're actually of from Greg, the to they you patient of I community. about own results investigators, that Absolutely, and him the patient that hemoglobin. very for -- point positive value very consistency life grad absolutely the a to one see we important and as the the investigators do his to there, and change [indiscernible] in total received the question. of normalization specific our the of has and different their investigators he

So patient the over increased to from presentation, the inquiries period patient community related patient and presentation. of after -- that's observation the that volume perspective, time our likewise after [indiscernible] And XXx of perspective. clinical data June

to they we're find the but not queries moving and and to only see U.S. So the we enough. through that receiving also join us And pleased understand in visibility we'll on trial too. continue can Canada help very whether them and

very very, So good.

Greg Harrison

Great to hear the progress.

Baisong Mei

Greg. you, Thank

Operator

from comes Our Truist Joon from next Lee question Securities.

Joon Lee

progress. Congrats on the

that pretty in and for Specifically, and/or include quarter of the BLA programs into specific. into activities and Your is does cash that pre-commercial preclinical EDIT-XXX. of vivo studies 'XX third IND-enabling guidance What's guidance? baked

you would the to commercial Or a yourself? the heavy be E-XXX commercialize to intention your offer? with you it on help looking is partner lifting fact, to In

Gilmore O’Neill

Jim, I'll let I So Eric think, start.

Erick Lucera

runway including With in dollars we BLA as dollars were respect from studies, not We the into other terms that commercialization the that prepare comes of research the future within to to we'll what IND-enabling or programs included with that partner. of cash not. early amount to with and need to respect everything at we all have guidance preclinical whether we in to specific do guidance. to are point for But up the guidance. some and activities with Yes, respect see in for are the the this do we reallocate where or Linda some that's

Gilmore O’Neill

nice if sure want partners, potential the shareholders, a are that partner certainly we as partners commercialization co-commercialization ensure making we U.S. really footprint. talk we we could see for in And Obviously, United to actually States. value global to or terms in stated We where to development Joon. upside, expand we that up consider with as addition to previously for particularly global line our Yes, commercialization ex we the patients, and can to maximize we

Operator

next Gena Our Wang question comes Barclays. with from

Gena Wang

Two very questions. quick

it say in and liver is the delivery? Regarding to manufacturing, particle it And regarding then vivo be a [ph] will target, the targeted fair follow-up. with just

do you to sit the U.S. regarding you in for collaborate have plan capacity Or manufacturing supply? the in wholly wholly try a facility. your in-house? commercial do So the future? to CDMO with also a commercial the And is so you what manufacturing Will plan would

Gilmore O’Neill

cells with can talked liver of and and the gamma tissues of interest of ASCs XXa on on With of that announcement infer over one The year actually potential commercial for hematopoietic reasonably we've our essentially our and interested in would the is and of on that's Azure We work the our Gina. cessation for stem interest enables around activities through future. targets, Thanks validation think elements or in I this with in certainly regard particular talked vivo launch. and the definitely that area about towards With the to globin of something technologies plus. that list very we past With asking capacity working LNPs working specific have past actually is of working And at promoter. prediction, XXX that. regard thanks AAV and regard talk us you stem and we our are capacity [indiscernible] much, area the we're obviously, readiness to of manufacturing, deal, an non-AAV more -- build about been in our cells. about our to because is of human the the an about to and

Operator

from comes with Dae next Gon Our question Ha Stifel.

Dae Gon Ha

total XL. of And with point discussion a you kind for to as potential One, do a as kind AdCom the will that contention guess of Two well. the line? as and you approach or line promoter what follow-up your down of at draw at hemoglobin me I Vertex being the regards go guess night about for last that ADCOM from on there when as look as targeting And of Bison, I HPG a are sand differentiation. remark would that, potentially thinking or to we competitors? you that? what announced that debate then an in differentiator be as terms raising you another point guys see AsCas-XXa in how

Baisong Mei

the question. Thanks for

looking Regarding protocol, are already for to outlined I as we previously, is the in from actively hematological total values. And the things. come category we're X of other related looking are lab One hemoglobin and that. and we differentiation, current

is other the and second The the end organ function. one

words monitor have -- renal in as maybe multiple The the function. part protocol, we We And measure hormonary patient the measures current last we on outcome cardiovascular, third would key two. and the to report function; functions a such be life. few and organ

the and that hemoglobin would believe health. we of So organ increase life quality total impact the

the of regarding a And on reported very function. that end could organ be So that's for differentiation. focus we outcomes, patient why part reported it then important

to the example, for patients. to For complaints the can we sickle main really main monitoring [ph]. patient too. on of by that have reported on the more things complaint is other one have main anemia also another we And that, the that pain, already the share close of also and cell and -- fatigue that outcome. show fatigue patients those similarly, that's -- relate data -- can And a

Dae Gon Ha

want initiation? you Do for the to Yes. add

Baisong Mei

like the their AdCom is AdCom. more heard And which course, that to area about a us help how that data is will feel a action, of the FDA is the to about ASK of related additional about caused we about concern yes, a not is this from question expert the believe which, well. how FDA second And we look novel do and our not how surprise. news mechanism XXa own we mechanism your as Then of additionally And feel target action then learn committee or new the perspective. of

As I the HBGX targeting before, we mentioned our the promoter of is persistent actually mechanism mimicking nature of -- that. relator perineal on

of we kind of the too. that's So nature mutation are targeting

-- forward sales. the And excess looking to the we're that see come from information so outcome and but the from

Operator

Our next question with & comes from [ph] Cowen Phil Company.

Unidentified Analyst

as for Two well. me

recent First, update on in EDITHAL, RUBY and will thinking the follow-up year-end that about is for most patients and be trials the disclosures? in number of the disclosed what patients both terms of the for your the

Baisong Mei

Thanks XX question. we year-end. track on are for Yes. the Yes, patients those total of by

the we a are not provide for update year-end but specific the released studies to to the at track are on be We at clinical data year-end. X disclosing

Unidentified Analyst

then are question the in steps those on remarks. prepared they you us where could And when the Great. second conclude. referenced in that you the and next Can proceedings remind interference proceedings

Gilmore O’Neill

the the of stating PTO. Appeals Court we we that before, as judgment the to we'd in a will in to mid-XXXX. is And with front well Federal Phil. Thanks, U.S. as it's have be prevail really -- step. yet what Circuit next are oral of scheduling oral the presentations as presentations our early -- worth confident Those Yes. anticipate is both The we have is that scheduled. then

Operator

Our Zhu from next Wells Fargo. question Yanan with comes

Yanan Zhu

second to Do kind enough data of designation? do of your XX enough I lastly, you patients pivotal XXX a similarly in inquire forming the the wanted about breakthrough competitor have on Do afforded of well? the about focus treatment the What cohort ask could interaction to -- terms to you there? to think think a in think the path? request length half be of will as FDA pivotal the your hemoglobin to differentiation afforded be year. you have you And total to added basis you the follow-up for and also, the

Gilmore O’Neill

and year that an the by we question. Yanan, know planning regard add scheduled can be well agreement interactions in agency our continue start. on, about of including will year, this to what we our continuous of element has we that to multiple process I'll regard for talking Basin as half said, the on to want as certainly FDA as if that much, strategy. anything. Thanks very as this for detailed come of don't FDA, that CMC, efficacy we we this ultimately understanding think, end are With of clinical And the elements, AdCom path Vertex's use. towards differentiation, will based then aligning I the how precedent I've With very or acceptance an the I'm the months I patients with a I said, having to will cohort glad will be you of year also XX to illuminating guidance of clinical interaction. and actually BLA as can be our pivotal and the that filing BLA where with actually think you see highlighted which helpful regard about a follow-up With the is and XX data data, be we of

Baisong Mei

thanks. Yes,

the think is for I differentiation related eligibility your question about fact designation. to

designations is reference us the the are and patients to that the after multiple your package carefully it's We very we required. about into things and seen and AdCom especially the certainly, related will good with final -- that we for XL earlier mentioned a so alignment more, excited but on know the as XX require we carefully looking evaluate there. data the Gilmore regulatory we we'll are this whether still from the FDA on and far different question in path. have by And

Gilmore O’Neill

And have, dose by course, highlighted of the are on I should year. the of I end was this XX good track that we think patients thing to that

a with helpful see BLA the with for for is acceptance lining number we excel number [ph]. what that so And up

Yanan Zhu

you. Thank

Operator

question with Cantor comes next Our from Rick Bienkowski Fitzgerald.

Rick Bienkowski

Hi, everyone. Welcome to Eric. Linda and

given So that any get focused to seems any based potential Azure does clinical with clarification the group and markets? capabilities to manufacturing But commercial in come on The European on the U.S. to Or in partner? have expanded agreement would I manufacturing. markets manufacturing be another readiness wanted company have from some the is Massachusetts. footprint for Europe-based

Gilmore O’Neill

Rick. Thanks very much,

and we right all that we everyone a ready we think U.S., of we'd consistent when be co-commercialization partner prefer as regard and point that footprint. ex in co-development, that, messaging in U.S. activities exciting that. I view, from with for quite actually talk our necessary talk launch. You're obviously, BLA reminding get the for new our and the us have to would to help this ex we we about With terms Azure expanding commercial actually of with about expect And the in agreement

Rick Bienkowski

Right. I quick follow-up. Got do have it. And one

So, year back in this exploring stem development the that for cell of highlighted one areas use regimens conditioning the transplant. of was of of milder January was

was here how question be my folded the I regimen and use a conditioning could is, clinical into for So is trials what approval like milder once pathway EDIT-XXX? developed, curious this looks potentially with

Gilmore O’Neill

conditioning, extend -- But mild the TD the patients sorry, a much, by population. path and which milder development cell are for eligible to by you very Thanks factory disease diseases. see eligibility that very again, Rick, importance serious for sickle of or because, we expand obviously, can creating reminding or us the condition, that increasing and or

toxicities it's they grow as And would that their use. across and general We really transplant balancing see it a how see they expand that efficacy. the evaluate protocols. continue that's use sites of terms, enabling it with reduction In something comfortable we therapeutics. milder as ex-vivo actually it, And of conditioning autologous engraftment to will will we question the with

Operator

with Credit Our next Suisse. from question comes Law Rich

Rich Law

Linda My congrats for joining [ph] to and company. the Erick both,

partner BMS question how cell NK I programs for And can like the a So you then to expect when disclosed insight with Biosciences. be more progressing, XX beta alpha And the T and are have program shoreline cell the is second programs provide do so in I question. the follow-up details? you to have these

Baisong Mei

opt-ins, studies. progress. X and advanced much, have happy of most the DC passed recently have to With opt-ins very the XX [indiscernible], those we're Sure. Thanks is in actually XXXX; There regard two with the IND-enabling Rich. most in been

to with seat to any Shoreline, programs driving Shoreline. the regard that I more is would specific is from Beyond is we're think come updates Shoreline. specifics and our have in actually really to program. with provide And the would Shoreline BMS oncology happy going. delighted really that, more on they're actually where NK divest it to obviously opportunity And the

Gilmore O’Neill

see the of inpatient either it's optimal enabling an for we because then of that outcome. patients setting? it second milder inpatient up that question eligible combination But to at for following setting conditioning the key that reducing monitoring is and optimized the have as is key outpatient element. I obviously So one the maintaining and enable still yes, versus do the and the on And is our an reduce of or need we use the important the expanding of an conditioning. it I while transplant Certainly, engraftment as patient outpatient substantially evaluation as continue population. element to in risk because really think, therapy actually, very Would patients a that

Operator

Our next question Baird. Allen from comes with Jack

Jack Allen

move team start cell disease patients that the the Congratulations lot it on your patients? it have into first you progress. I your Maybe adults. progress as the some into with of wondering to I thoughts on younger had dosing relates XX years I to But sickle are like seems any to competitors one hear a patients of then made I moved plans side; was any follow-up. clinical you quick there. And to have XX if to comments to old. know love

Gilmore O’Neill

Bean question. take ask to going to Jack. Thanks, that I'm

Baisong Mei

for that question. age expand intended Absolutely, all to are study Yes, thanks this groups. Jeff, we to

actively more data, we comfortable are to on population. are the patient more that. tile much go And So beyond we with working

Jack Allen

think the I front, before been leveraging scheduled maybe I about position the guess competitors would you Could and do I not you you're our surrounding that a then of mentioned this we to Or but their advance have relates hearings some coming there? quite yet case love and come your after? interference IP you And strong IP on your any should about as it have potential in how leverage that potentially as think conversations to hear looking IP guess Great. approval? approval. settlement how it to thoughts

Gilmore O’Neill

in in that of therapeutic we disease used vivo therapeutics multiple multiple are want indeed, products areas. large there's I enable frankly, it is our CasX to IP ensure and ex multiple patients key areas. think is on development in our with number across multiple vivo to that really for across And position both and that a

to area. about leveraging we beyond think point or discuss vary in of of will view, going And As monetization that depending stage negotiations. the not development from economic that, companies an that, we're individual or the we see on disease that

Jack Allen

Is you us who Just sorry, to follow-up just on Can you brief to the has from perspective? licensor duty IP? litigate remind your it or one IP. that as

Gilmore O’Neill

key we we willing but will not So, details protect talk we -- protection IP about our our give and the will is legal going for to IP. strategy very our I business want to that are but I'm of actually protect Obviously, and of IP. thing about think licenses the our we ensure open to

Jack Allen

on Thank Great. you color the the progress. for

Gilmore O’Neill

much. so Thank you

Operator

comes Our next question from Jay with Oppenheimer. Olson

Jay Olson

patients can team the on on sickle trials an track to the date? then do thalassemia. for since thalassemia new talk on can in and how the for have thalassemia? patients you provide And number cell many? year-end, opportunity to update about sickle separately, additions goal a sickle cell if so, dose to -- enrolled in and you you're you cell your dosing And the Congrats by And of XX similar ex-U.S. and

Gilmore O’Neill

enrollment. Baisong to on question first ask your I'd address So,

Baisong Mei

Jay. Thanks,

in XX we patients mentioned, last quarter year-end. already have those great enrolled actually we just as are eased. patients at -- rolling previously patients, we been patients the dosing have on track have So we're announced momentum, to I XX And we

on months. an the provide will update we enrollment the So coming in

end. on we number patient goal And we so for disclosed not more that not cell, goal that of the information yet. And doses share have the the regarding going of can the dosing

Gilmore O’Neill

opportunity regard believe is value best we for to opportunity, see substantial frankly, real patients a a -- for to the with that there. us then footprint. maximize a large and, And see partner And Jay. partners large a want with optimize in much, the sure we a partner. there very which number is we to is are Thanks And that large say, us. why But that we delivery space actually ex the the and that that potentially way to and opportunity to that shareholders I as to seek make the interesting also are a very we're U.S. of seeking and our opportunity

Operator

comes with Our from Manny [ph] Leerink next Partners. question

Unidentified Analyst

Obviously, we trial want could. dig call into et lot endpoints, on a have I this questions been around cetera. enrollment, execution, if to for of clinical

some other earlier sort of on fatigue, is issues discussed pain call, potential important and quality this of of life You've differentiation. area

comparability any, differentiate that As has we clearly subjectivity. clinical element so are who that trial have likely prior an on ADCOM approval your some ensure any. data are you to made, pretty these if endpoints, sorry, of and endpoints between and notoriously variable -- your to What competitors differentiation, can these if demonstrate efforts have clearly

Gilmore O’Neill

to going I'm Baisong address ask that. to

Baisong Mei

Yes. Thanks that. for Manny. little bit about Very and end your points step just I'll good question, a maybe question back on

after manage the severe changed So actually protocol, is III one of And the that trials the of endpoint consistent in similar the we're year. a other Phase last actually was in complete to clinical setting. Editas middle the we as I doing remission joined VOE with thing primary what

there Then including points end many the the that's are primary point mentioned, I organ the expected. are and to in end PROs end kind So too. of functions that looking as we

and You have absolutely there, in very good point PROs about possible the too. especially variations

-- that is that myself direction have method But that understanding. [ph]. that have we an data be into area the looking this publications in and we feel we're So we on will that more

will those of have will to end and points. we us information the other X more about stand allow We have understanding where for an and data

Unidentified Analyst

I one follow-up. quick Great. guess

would of and long across statistical you have on publicly how clear literature versus clearly are follow-up kind You on of your those of based where talked the what where any need on duration a little to we or follow-up competitors other average to or differentiate in around on to quality you Like are? provide from guys life? need trial bit able components your disclosed guys pain, evidence to where about data we understand experience in companies are you fatigue, be compare would caveats to on endpoints. own notwithstanding how Do of

Baisong Mei

Yes, very good are looking That's something that. question. we into

there take some scarce First already the of moves are after multiple And published, information dependent how shorter and in patients to their the see of in patients. endpoints, transplant time. other those longer example, on allogeneic all, stem then in cell disease, that the publication will improve perspective say for specific will for for some terms we transplant take

[indiscernible] we of increase the already For ratio see time example, period. and

So taking that's wise. kind probably shorter time of

see system in months of brain. we'll X publication, XX that analog damage actually allogeneic vessel point learn. actually ago after the some may the are for say from nervous or example, a it for to They far to so that's transplant, And take improvement space blood central life, the for months and they and hydrodymic improvement and continue in

the new are again, we are that. will So we period there's into in over differentiation. see some looking territory, see But of something are a But this we space. that lot steering we confident of studies a to time

Unidentified Analyst

helpful. very That's Great.

Gilmore O’Neill

hemoglobin differences and on patient based course, actually we've very also it's the as follow-up physiologic that already describing and the meetings around excitement the in and also treating of of and with in to alluded June. addition at anemia worth the we're generating correction exciting feedback since in that within and community data I the significant robust had expression EHA that are our biochemical reemphasizing, prescribing to cyclic think certainly and presenting durable webinar the physicians

Operator

Our with comes from next question RBC. Issi Luca

Luca Issi

bridging FDA similar ask you REGENX? a Great more comparability to seen we've Is new analytical facility. on Or the from manufacturing study do to fair sufficient. just it you're what think process that the a assume progress. since will will run moving manufacturing study that clinical your you to be Maybe one to

versus material maybe quarter get pretty there quarters. R&D, appreciated. spend much R&D this decline So then And prior the color on any in

one if wonder we should any provide forward? finally, that and BD. And that what line LCAXX on Any update modeling you I So partnering how drove one could on think on about then additional last going color

Gilmore O’Neill

of R&D clinical regard pipeline. to spend. any he color to the regard R&D thing alignment details actually our wants what will meetings already Thanks narrow to running really start manufacturing the With our and Yes. our space. where spend, is the Eric our separate with Asia We're key We in then as XXX a if space. about we did additional This was and FDA, that to key have be new the add I is we of sharpened will would vivo specifics I actually in One required. -- With part focused manufacturing, on be obviously, we much, following to are the sharpen within ask outlined. The on and strategy the Luca. Asia supply of very of regard and a With and accelerating a rollout that our our focus spend, think points. asset. couple

on as an had that AAV as so impact assets Shoreline programs. inherited our of dystrophy And to Redland well with the spend divestiture the our cessation NK of

don't I to to Eric, if you add more anything want that? know

Erick Lucera

high to think that No, on into add R&D resources spend, we're aligned conviction be we I assets. and philosophy think from all you basis I would shares the we "depipelines"" covered just really that not pretty well. want really that I high want a high philosophical board. on our that think that's to goal. here on have across on a team I We're conviction, focused focus that shots few the and the lots We assets conviction of

Gilmore O’Neill

the a with and the technology commercial selecting clearly our work can reason I piece in just meaningful in targets have data -- signal -- maximizing sponsor that us. tell the differentiate standard we LCAXX, you if technical that of manner. when aligned Linda philosophically success saw that when there myself is give a to probity update But firm Linda some important that in advance has it an vivo. specialty that we is take deal. selecting interest And by that very has And not where specific or analyzed our think something or we hope with disorder are and targets update to interest. of patient in that about leadership we from forward. progress with set it key continuing the in I to will actually It's care and believe a take more joined and and would comes we have of a a team regard a guidance are on obviously, the I that's another we approach the we actually you been With to will a

Luca Issi

much. so Thanks

Operator

next comes Bayko with Our Liisa Evercore from ISI. question

Liisa Bayko

the I regimen little more -- wanted just had conditioning I to bit program. gentler a on down drill

program? else out So like if on guess I something that your think there. into sort market? to fold approaching could someone you of the you most And I developed that, about you do what owning working approaches think are And like your is in and to you it? someone something how that of are terms when access you What something versus your come else that do think guess, What something could promising that? thinking is like on

Baisong Mei

to cell to just indications, sickle for so that of my as as crucial the technology Coming course, Thank excited for I course, conditioning as disease. a I'm truly many therapeutic impressed so is first, the many therapy your foundational I'm the well want well this much parameter excellent question. really the have you Well, with as, very to of of Editas. other indications joined say team. question, oncology, such super

on various all And And regimens that to I'm be getting as landscape here internally. only to what so terms Gilmore keeping of sought this this active I for we of conditioning there in can going approaches out and active over so to years. of many up after as speed But been course, are, have it's are been well the motor aspect. additional for I've on comments. monitoring to are the very for course, team very we is the days, of root doing X

Gilmore O’Neill

comes which in, cell we multiple is I think mito-conditioning, to much, use how areas Linda to with is transplantation. generalizable very the And as when it on of Thanks therapeutic you've that say, it said, Liisa. -- stem across regard would or touched fold it actually I I elements your think

target the would therapeutic rolling can population. our cell would transplant of need the transplant is which therapeutics. sponsor conditioning patients cells. on looking health the conditioning, actually use see development And would in stem milder see and is in stem an minor Obviously, need in and in as an we see systems eliminate spaces then any expanding obviously, their systemic have use. hematopoietic we we vivo -- protocols freezing in for way at whatever And a that adopted across transplantation. step eligibility the the advances CDXX conditioning certainly patient the harvesting, well burden collecting an additional further so care impact it across that could multiple any And as that therapeutic and cells reduce on therapeutic positive mito-conditioning, beyond the having by result generalizing of impact the of impact eligible and centers eliminating stem for

Operator

next Our with Cheng question from Brian JPMorgan. comes

Brian Cheng

quick one me manufacturing. from one Just on

when fit there FDA On as the will need agency? on CMC facility items important any the And come the of portion on with online? you pivotal facility, ongoing this studies. to specific this do the aligned you that are with the year, in any insights have you timing get could that How second the does discuss potentially into half

Gilmore O’Neill

this deal and Thanks of to Brian. tied this the expansion regard very much, online, is to really timing readiness BLA our With planning.

to our going With to to go with happy time alignment, appropriate the the Azure our online BLA. at regard And with are be progress so the FDA and to not I'm we readiness that's support want therapeutics cell into our the pertains guidance it essentially, and around the we program specifics confirm with to [ph]. here. guidance understanding of the as to interpretation and and But their just agree

Operator

from is Steve James. Raymond question Seedhouse next with Our

Unidentified Analyst

measuring and RUBY EDITHAL Nick and those is on to Steve. plan data. or you're for if EDIT-XXX [ph] This translocations for edits or or you how whether share were and not wondering We off-target

Gilmore O’Neill

have for comprehensive. very the Indeed, actually question. Thanks a Nick, we much,

comprehensive that at assays proposed in seeing edits. and were off-target actually and to choose as supports actually have specificity seen. ASKX enzyme a differentiated of very that it low our editing. data really We're we have happy you as well that which, those CRISPR its believe of know, that after the of for We looking for reflected off-target set assays we're an validates published indeed, But agency saw both actually the Interestingly, efficiency with very set to a we're off-target happy very is was many undetectable We guidance submissions. its we the our edits. following remarks controls approach as our

So, all that of is actually moving its portfolio. prioritization the selection our in consistently and enzyme with

Operator

we gentlemen, again the you Thank your Ladies participation. end have session and our of question-and-answer once for reached concludes call. today's which

You may now disconnect.

Editas Medicine (EDIT) 2 Aug 232023 Q2 Earnings call transcript