Editas Medicine (EDIT) 5 May 232023 Q1 Earnings call transcript

Good morning, and welcome to Editas Medicine’s First Quarter Conference Call. All participants are now in a listen-only mode. There will be a question-and-answer session at the end of this call. Please be advised that this call is being recorded at the Company’s request.

I would now like to turn the call over to Cristi Barnett, Corporate Communications and Investor Relations at Editas Medicine.

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

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

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

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

Medical of and members Thank and the quarter today Michelle on Editas earnings you team, morning, Editas’ Officer; good you, other Financial for Officer. executive Cristi, Mei, first by Robertson, today call. Thank everyone. joining Chief our two us our I’m Chief Baisong joined

gene hemoglobinopathies. shared a in, position as of you leader and our January, know, programmable strategy vivo many in to we As early in Editas editing

to or life-changing to quarter, undertreated we patients successfully with driving our of goal delivering untreatable first medicines During diseases. previously executed this the strategy

EDIT-XXX stem medicines in driving increasing program hematopoietic a hemoglobinopathy. pursue our vivo are leadership for cell We position in as ex our we momentum

recap, are underlying strategy. As pillars to new a quick three our there

focus to EDIT-XXX TDT, develop editing genome have in sickle discovery medicines. to cell or vivo thalassemia, for we and sharpened transfusion-dependent continuing beta our administered severe while First, disease

iNK that to announced Biosciences effort, January. of our in cell franchise we had divested part previously Shoreline As refocusing that we

Second, capabilities. and technological we are strengthening our discovery engine

separate criteria. We have divided selection each groups, our drug target discovery and division the of our implementing research new enhancing capabilities technology and into

strategic an we our continue our approach IP to to In Finally, to and is in development. increase pillar business partnership expanded will portfolio third drive out-licensing tandem, discussions. deleverage

new X/X how data our Phase strategy quarter? and competitive promising anemia differentiated that a program in reviewing treat investment have to potentially first RUBY have our So, increased We we executed TDT. EDIT-XXX study program indicated we have the sickle in our cell after against initial that and

for in cell we we the unique editing investing POC have humans. seen treatment Additionally, disease hematopoietic and for of sickle an stem EDIT-XXX are to and TDT, cells the in already approach approach for develop that leveraging vivo differentiated of

ramp We end of are up have the cell dosing to the sickle XXXX. continue enrollment by and dosed disease and patients on total patients to in XX for RUBY of trial track

regarding and we drug the to disease, pleased in clinical his EHA and or our will presentation Association, in European company-sponsored data in readout and further webinar. share enrollment progress the update our oral at Congress, Hematology details Baisong June an remarks. our announce data June of We FDA are are to we share will granted the also designation sickle RUBY that in EDIT-XXX provide recently to that for treatment a orphan cell excited

initial successfully and by clinical EDITHAL engrafted the Enrollment we to the EDITHAL dosed neutrophils EDIT-XXX from our patient to TDT, patient first to on progress has that data quarter are in in X/X trial we share and On platelets. and year-end. trial the that first for track continues pleased we Phase the remain provide

tissues. HSCs or this lead work therapeutic and other discovery began our on targets group stem Earlier in vivo. vivo year, in discovery at to cells Moving drug hematopoietic

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

lead search on Our a drug search group to this in forward I our in and progress, for the work and discovery to updating new future. CSO to look you continues this vivo

foundational gene Since property placed have Editas, Turning securing protection, to patients scientific intellectual the for covering discoveries we to our intellectual our the transformative are importance medicines and subject we a enable development robust in some the in portfolio IP It in proceedings. portfolio confident meaningful of is value to property our that and in position. CRISPR/CasX covering human have of patents editing substantial large therapeutics, future. advancements are U.S. cutting-edge only novel founding need. will And interference important that international to which provide of note of we

to nonexclusive seeking University’s licenses any We vivo make CasX to issue for in enzymes Broad CasX uniquely ex Harvard and these are the company is including and Institute’s of exclusive to positioned applications. licensee use to therapeutic human patent medicines, Editas and and an exclusive vivo estates,

market, as a licensee this buying licensing any party size number patent the ensures of substantial market, estate position medicines of for that of position. the develop discussions CRISPR/CasX as companies which, unique responsible given Our is the the to U.S. CRISPR/CasX products enter the exclusive we are patient the

scientists world-class execution, the the We during keen XXXX. annuity to progress execution strategy our to our fourth quarter and to and attention year. continue our on updating readout new focus, the our we sharpened and throughout our milestones look forward our from of momentum on strategic the build you clinical employees With of upon

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

Gilmore. Thank everyone. Good morning, you,

severe study disease. for sickle Let’s EDIT-XXX RUBY with cell start

enroll dose study. the and patients in RUBY As continue mentioned, Gilmore we to

and study three XX of We number have double from the enrolled sites XX patients, activated enrolled months ago. almost patients

we shared, we year. this began patients parallel As dosing of earlier previously

by XX We RUBY year-end clinical data update year-end. patients the and to well provide track total to both an as are month on on next as dose

from RUBY Association, or months from including at data at June. The first Hematology the European in and hemoglobin. that hemoglobin, set data. Congress, company-sponsored data patients, oral and clinical webinar and safety EHA total fetal treated, XX I’m data clinical treated the will present multiple to include presentation as patient second our months Turning we including patient the will efficacy an data X excited for data

data share neutrophil on also events engraftment patients. will and platelet the We four safety, from first and VOE, vaso-occlusive or

a we As data initial the two from RUBY reminder, trial. treated in last December, the presented patients first

EDIT-XXX who had VOEs. showed after of first treatment all significant The patient parameters clinically and with hematological months across improvements five follow-up no

after patient total the per at that had five well Specifically, the months an EDIT-XXX of correction normal level range grams And XX.X%, fetal into hemoglobin anemia fraction with deciliter. XX.X infusion. to hemoglobin increase of

total independent These This promoter with preclinical that and hemoglobin clinical with sustained level gamma of which is consistent increases high clinical demonstrated of observation EDIT-XXX indicated initial globin enables data fetal hemoglobin stress. level has that of provides of fetal of normal data, patients targeting erythropoietic hemoglobin. and

gene-editing believe Given potentially action robust patients initial EDIT-XXX benefit the and clinical and by that continue can in with clinical preclinical differentiation and we of potentially provide the unique approach provide long-term. by cell to sickle mechanism data, EDIT-XXX, supported disease, data clinical severe to

the hemoglobin an life be could differentiation a We point correction reminder, of ameliorate potential important outcome patients, an significantly and quality of a of total can organ clinical of sustained a sustained damage. for believe is As as hemoglobin level for EDIT-XXX. total improve normal normal anemia level

X/X thalassemia. transfusion-dependent trial EDITHAL EDIT-XXX for to Turning beta Phase

year-end. As in the We successful Gilmore the dosed neutrophil data and trial provide mentioned QX patient and our EDITHAL engraftment. we track on earlier, from first to patient by platelet has clinical remain initial

EDIT-XXX done for have the development strong of As we we positive to RUBY measures also have are taking TDT momentum. for multiple and accelerate the study,

multiple in process We have cells patients pheresis. completed who their pheresis positive and enrolled CDXX or edited of have are the have

study the the from clinical been sites. patient EDIT-XXX of trial both around traveling RUBY I country I pheresis, studies. the in sites. and our much investigators EDITHAL and appreciated I’m have pleased with the editing dosing in support the visiting momentum recruitment, Recently, very enthusiasm and and

investigators to year study seen EDITHAL lives. in by I’m RUBY and as progresses, with already next positive have additional look sharing updates the data dosed year-end. that EDIT-XXX at hear from data year-end, and clinical from excited sharing including forward initial changes patients month their We study to

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

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

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

and existing our fund XXXX. operating our expect equivalents expenses capital expenditures securities to cash, We marketable and into cash

assets oncology completed is previously $X.X related for XXXX. compared first million January was licenses, announced which of XXXX Revenue quarter to was The related last in Shoreline million year. $X.X the to increase of to sale Biosciences and the our same period in the

first expenses XXXX. of $XX million expenses increased stock the with compensation million quarter The development were to services the a compared business expense. activity, primarily $X.X to for partially professional increase attributable G&A quarter support by offset decrease is in million $XX.X for

portfolio, to million, quarter reallocation decrease of offset the investments reflects in this strategic our of increased accelerate compared first expenses a strategic $XX XXXX. quarter This with by is line of were the priorities. R&D flat our EDIT-XXX. development was which This of the reprioritization in to following capital expenses

discoveries. which discovery efforts trials focus and concentrate financial cash extend our EDIT-XXX our to in in our Overall, into as advancing sharpened XXXX, as for of other strong our talent and progress continued vivo provides well in ample hemoglobinopathy our resources in remains Editas runway a position, and allowed our us both research

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

Michelle. you, Thank

almost the Editas. to competitive EDIT-XXX, transfusion-dependent the including the of proof has a treatment been of two joined differentiated which cell for and concept concepts, It disease one I has beta since time, In sickle clinical of a demonstrated thalassemia. be has and proof potential year for this product Company

January just our look this In strategy, tangible remarks, on in taken And our reshape shared we on early as Company the we’ve We transformation executing which begun number you. beginning. a stated with to to forward the that opening around steps my progress and addition, have is continuing strategy. sharing in new I of our

trial with our our discovery As for stem CSO business advancing new XX and end hematopoietic end to robust providing a other include tissues, strategic aligned study objectives of from editing June EDIT-XXX XXXX, the in to drive in technology and and a finally, by of our dosing vision, IP EDITHAL clinical hiring complement our the RUBY cells of providing year updates of and study TDT expertise specific development vivo and for the capabilities. patients RUBY in activities year-end, editing portfolio gene reminder, value by leveraging data EDIT-XXX total clinical EDIT-XXX from XXXX, our

answer to I employees your Thank happy drive interest much our strategy questions. the who Editas, you. to thank all helping Thank for are patients, very and our and in forward. investigators you we’re

our And first Lee from Instructions] [Operator Truist Joon Securities. with question comes

for no sickle Hi. terminated couple a taking to of after benefit. treating recently program our questions. Thanks Novartis their cell see disease patients

is have to Given to you. similar Novartis And strategy to a differences work why your what continue your some strategy Thank you could should point promoter said, failed? Novartis editing when follow-up. editing I

Joon. much, see we or some months Yes, actually very Thanks did event ago. that weeks

I are are there critical. that elements some think

I end hemoglobin at the in expression preclinical that think as year, hemoglobin the robust thing consistent completely out as saw total -- with initial hemoglobin actually first in that fetal that increase is data were a total readout very early with increase generated. we point the of robust our in data last a well to -- POC

associated combining that hypotheses persistence which of an closer, our fetal we which the area the testing in clinical region In HBGX/X encompasses our actually our AsCasXXa see of promoter, of the effector with different with deletions unique targeting in CRISPR much was and fact, a approach hereditary mutations enzyme hemoglobin. or preclinical

towards and I hypothesis. difference those our factors that think differentiation, indeed, actually we our that believe all data and data preclinical clinical point have and a supported key

you have the using, Having So, versus the any AsCasXX and comparing -- CasX what -- in that if preclinical? difference is can maybe? same you data remind editing you’re me region the

Sorry, Joon...

Can you…

Yes.

were you same and what is AsCasXXa, which used getting have or…? what target outcomes region you’re can CasX, is which using, comparing mean, think deletions I Novartis to I different of you in terms the if So

you This breaking Baisong. is little think I me Joon, are down, but a let try.

noise. me see is try Let whether understand background and I correctly. There

question region question? Gilmore also CasXXa your to compared and your Novartis do that the and you Is to a comparison think CasX I So say between just and is mentioned. have

get? you if either with differences region, actually, the you in what No, same CasXXa, or Yes. outcome CasX target

Yes.

editing. from areas kind -- just the comparison, did me the clinical -- Let a of We identify which large do a it. perspective, of did promoter take region we scanned to the to region we

of mentioned, Gilmore compared observation region between the find promoter covered these CasXX. and the we CasX So, but HPFH. X. that the many which answer And CasXX related CasX without for the a then is we and we and selected, to to just too And region we question? specifics, large difference in with found HBGX clinical that consistent your the the we also out Does

No, absolutely. Yes.

good, very back is what was driving Thank much. data impaired I’ll just but you just Your was that so queue. in the hop difference. curious

you. Thank

is Samantha from Our next question with Semenkow Citi.

couple a for me. Just

presentation that is at late-breaking I a wanted to the EHA, presentation? just For clarify.

Samantha. Thanks, Baisong update detail have there. you I’ll or give you just

It presentation is a that normal oral expected. is

you I I updated And and I heard you, data And mention, patients. first wanted say, also second have heard obviously, for then we’ll clarify, then, to I four think, patients. the

is VOCs, that get four patients six So VOE, will total? it or will or is on data patients total or -- that it be

patients presentation. EHA at four the be will Total

for And there? just it the So additional a helpful. two those then can making months, cutoff it level you follow-up that cut-off curious, incremental information Any I’m couple is provide patients, one when what month? was be you’re would of is of

Yes.

We are months -- we’ll or two have these patients, next more. two for

it’s understand obviously, in the Just the to the that, than will cut a important based was be more on I presenting EHA data abstract, we published later, Congress. data thing, because will will, which earlier, be at abstract follow-up, actually other one think we

Yes. And be the Thanks, just follow-up available May also on XXth. on of abstract Gilmore. will kind that,

question Gon Stifel. from Ha Dae with Next is

the so Thanks look for to month. great. next taking And update Hey, the much question. forward data

your just of was strategy wondering I forward. going about kind guess, I So,

more or programs? to So, conditioning if a priorities HSC, curious, But Gilmore, would kind an your HSC think advancing you’re And mentioned it’s in you tech of emphasis or next can I’ve walk And lineup, or of through it about vivo latter, organs? us continue other you programs if beyond stronger you vivo also it the EDIT-XXX? follow-up. thinking how I less you maybe in or think, about that in a in got other I is burdensome editing. is ex new do would vivo? Then guess, is case, delivery

much, Thanks very Dae Yes. Gon.

our part is vivo. on We are actually large the -- discovery in focus of

that maximizes was technology strategy a us. to very available it pivot because believe important our our of or to and the -- think that powerful ability exploit part we have the I maximizes that we

effector sort selecting the good elements, point in of a enzyme vivo CRISPR delivery, in if then of problem we with molecule have enzyme, promoter. we HSCs, those believe reduce of of enzyme, or that solved a HBGX/X From target and selecting problems, of CasXXa, that use three you the to of two very the a target robust CRISPR data specific our robust and human

vivo And so in to an problem. reduces it delivery that

further looking I we’ll give at forward future. group As time are other discovery And and I said in again, updates we more on beyond the actually also my is actually our tissues. look we earlier working to in that, to updating an remarks, appropriate HSCs say future. that the in will

during remarks. And I wanted just on up commentary Baisong’s to prepared question, then follow second

that sort their lining of ascertainment kind to for on thoughts up that much. or in be you motivation any and high you Are as field from into take taking on you those do What administer Thanks Any what And been expressed are your on guess, what kind as looking physicians’ comment, going were Wondering now of there has Like, would docs gauge could to CTXXXX? also visited CTXXXX? are if of CTXXXX? approach? commented sentiment have? kind they there So, enthusiasm. you be so proportion any of right you guess, part you on I gauging or -- would helpful. EDIT-XXX, reservations I of their the patients

I question. a of are the of Yes. trials. the Thanks previous visited in study sites. for participating -- Actually, quite many number few Yes, them being editing gene

very are are we and for they different the targeting do. enzymes the So, approach region different taking, to about editing enthusiastic the including

have a us for those is this of actually, in the experience benefit so, investigators And field. lot

Yes.

remarks, our our with of We patients I will on in the the the against at can launch, looking just the of in future investigators enthusiasm think that. acceleration you’re Baisong’s increase our the indeed anticipate vast dosing enrollment. the as and for into out go on that the that be towards background of details awaiting obviously I more patients evolution and building of the majority time market

preclinical as monitor the to patients subsequent But I I and important resonated the we think through will replication our presented consistent studies. see has with think, a in very execution really with initial is were very actually point, December, of the something as confident encouraging very we investigators that our continue XXX them data we’re clinical data, that in that

question Seedhouse with Steve next from comes Raymond Our James.

My I hope everyone bit it of requires a can actually a a question moment. bear prelude, for so

least independent as of at recent program made ICER globin in erythropoietic sickle hemoglobin stopped and exa-cel reported releases fetal that well, as indicated know, comment ICER that you in one being stress. phlebotomy phlebotomy, the uncovered for And on there but increasing some editing locus You thalassemia on a the ongoing earlier use an of -- data cell some moment. just at report

cells So know, was editing the causes. years important indicating And use sure also clear for prevalent and you levels at phlebotomy to stress probably this BCLXXA phlebotomy it’s of cooperates all and ago, not exa-cel. as because is primates ASH ultimately with there erythropoiesis I’m data how accentuate HBF is F because

if all confounding where So what you if you. I’m advantage data think a HBGX/X use you? Thank And the impact at competitive that been has is approach if BCLXXA know that study like you specifically your hemoglobin editing curious in locus, potentially phlebotomy approaches. said, agree fetal and you what is potentially for all your phlebotomy for this the is

much Steve, thank very for your you question.

you’re for sufficient approach. preclinical to referring published clinical data stress BCLXXA clinical the for hemoglobin expression. also increase data, stimulation own our require some the erythropoietic data, hemoglobin, and to probably did It from So, fetal fetal targeting the

we’re actually get that’s target that’s reason the and been a the validated treating we the treating perspective, actually preclinical targets from now, time so, other and And all to we did take are longer publications. study by

specific have regarding BCLXXA And I publication. clinical the formal for not seen approach, data

them would have their understanding. a so waiting data publish and for I better So, to be we’d

So published. comment on unless I their data not will

our fetal correction associated However, normalization it And we early known disclosure, a is very stress noticed anemia of suggesting pointing in in that that our on the as erythropoiesis or and combination based patient our on worth hemoglobin of in out actually a robust December. that that we was data, with not expression, approach hypothesized nonclinical indeed dependent erythropoiesis. robust the biology is first our

from question Wells Yanan is Zhu next Our Fargo. with

in the total mentioned to normal the hemoglobin prior the you first from Maybe hemoglobin a discussion line percent with fetal continue in quite patient the to competitor very appears that little much bit editing product. robust the of be is Gilmore, gene The range. question. for

that red level that related reason, So, total to I potentially cells? the the of be the for be Could blood could greater hemoglobin baseline or total to wondering, that -- hemoglobin number is was that a it reported due of patient, patient? is in

along patient second the the is patient? two the female you hemoglobin wondering, normal line, perhaps which was is us obviously to could Thank into looking patient baseline you. to expect a what number range from for patient, continue at EHA. And could we and remind little I -- two further for and number the that what just

So the Yanan. question, for thanks

part blood your regarding answer with to I question these. red of number So, the of and the the all hemoglobin start versus cells fetal first will

actually patients robust see these we for observed. what we So, erythropoiesis is

is that though because And high. and to like total we level we hemoglobin also well total see both can level around then of see also -- mention the blood I the fetal of number of want hemoglobin as the by high And ends both also you also have of hemoglobin of the cells. the increase and XX% on even as amount per is contributed that. red hemoglobin, cell fetal actually hemoglobin do their So, that is

in of that. kind that’s So,

And then will the and the soon. regarding present we second very patient, data

of of the normal course, not per different. So, female to the hemoglobin can the Usually, and to but XX-gram around around is on for XX.X% on level XX patient comment I is deciliter, will reference male the specifics for data, mention I up depending lab. female male XX, that, to are

too. there, in difference some there’s So

Okay.

of I Yanan, per that just other baseline the is increase. deciliter was we one. the increase think the what think dosing we gram total can occur or one I X.X months say question a about first comprised asked the a And thing, you rapidly saw few hemoglobin patient that of what the hemoglobin within very hemoglobin

on especially usually for patient, bit add Yes. -- baseline because conditioning, when for patient gene-editing nuance Maybe that sickle blood for prepare and transfusion. they just they little trial. a pheresis And cell the have

we X is set recorded. So, the have lowest a visit just We the time baseline actually we baseline. the not at as of level the

on that, actually around we different is just it’s maybe an baseline, it’s this -- about deciliter lower grams This could compounded, the too. a nuance. That’s it our over when many reasons a -- the for per example little just for confusing. So than bit be actually on -- patient. not record actually baseline it’s It’s XX

significance expect perhaps nice. you. Very major a Thank the all quick to greater a you a follow-up. manifestation TDT you of at because a explanation. and level of maybe anemia this for differentiator Do is disease? Thank also that be Maybe for

the targeting was the experiments and approach and would cell be the that was promoter marrow just be -- really the We HBGX/X better. that original or that be our or targeting output would erythropoiesis promoter that hemoglobin of the directly designed of not independent And hypothesis. anemia. a those designed design with combination approaching best And the to determined HBGX/X the would using erythroid of group empirical red but or for determine specific experiments CasXXa output bone to scientists what discovery driving empirical expression, of selected fetal set robust

of to preclinical first what we in other as sharing expression. that in -- or data of approaches our we cut The nonclinical robust have And fetal supported is seen hemoglobin output the as disclosed from as data that, data RUBY comparison indeed, erythroid study. our the actually data showing well robust

obviously, it data But which data, haven’t seen what supportive can has with were data -- And both, is biological original RUBY are I EDITHAL preclinical hypotheses. the that say enough that we and so, of certainly consistent patient. demonstrated the is therapeutic in our

with is Cowen. question Nadeau Phil next from TD Our

Rodriguez is taking for Phil. Thanks for my question. Ernie This

the On on program. second sickle on you TDT to with cell on the met have visibility regulatory better the And a program, gain FDA question path? then, the the

that patients on the disclose include update, For switch from you disclosing year-end you dosing that only the And would you when additional sentinel patients, planning patients? initially are dosed, will or you to be dosing? additional reporting will sentinel if parallel you

of I question. do track I’ve I’ll is just ask and -- you actually I’ll the Baisong. interactions. the to on regulatory if I’d FDA answer keep ask question So think Baisong, the what the

Yes. for your question. Thanks

have a certainly FDA. of engagement we lot So, with

have package be data which you efficacy supporting with on registration the drug we FDA perspective, we this recently, registration with we actually engagement further the the that registration. we As planned. assay which consider potency see And announced the have BLA agency destination. to will alignment on previously total can the the also from the submission, orphan that will FDA, is for align the And have

the part the And your data. about is beta-thal second of question

will we more that So, have study, expect really along EDITHAL we and patient. than data we year-end, sentinel are the by moving with

we’re to the forward looking so, data share And that by year-end.

continue dosing so to then? you when you patient, sentinel planning are to -- before disclose approve you are more parallel And going than

of we’re do is I key get actually on year. think of -- to are we that end for the planning at what the end thing track the data the So, year readout at to for the initial the readout is

the to that going data well year. end important initial if that. good we’re determined haven’t for disclose track we are I of is on to We think share point actually But the the

question from Bienkowski Rick Our Cantor next Fitzgerald. with comes

patients a I size last sickle for pretty XX registration on a cell bit cohort substantial expand on guess, the is in path towards the question disease. EDIT-XXX. I’ll

need data of for of you do many registrational filing? any a have patients’ So, you will worth how sense

Thank question. for Yes. the you

patients So gene to substantial agencies. the data. for we to specifics a will number of we And approach align certainly think with need have of editing that able regulatory registration, use be able that to the the with be we generate on amount the to we

are FDA. planning discuss we with So, to

I quarter. another I in one. transaction, And other just the collaborative revenues attributable on the Were other was Shoreline attributable have more some for million $X.X or the to granularity there? all are revenue little hoping of revenues quick partnerships a there bit to for the in

It’s combination small and sublicense some a the both then of Shoreline other revenues.

Our Credit Rich next question is Law with Suisse. from

I you for guys. have of a couple questions

of and So, that license filed the with as that Broad you such for company pending, or perspective. the outcome the They for your mean what does don’t it BLA litigation appeal from Vertex already IP CRISPR/CasX CRISPR a a potentially appeal. the exa-cel could utilize the before from have launch product we know

I have insight And a be So follow-up helpful. question. then, would here any

is anticipate judgment in mid-XXXX. early key think to the we thing of the that the I sort

about application by of that the what before, discussion confident is prevail largely not because law are have we are of application it’s PTAB. -- new We and under the the law facts, as we and

for the of think we this that using interference, are of I enable to thing that And, that actually the should application around that Now that. have forward, we not happy of IP say human to to believe licenses therapeutics. aside CasX looking a and grant product significant covers portfolio recognize interference any to patients, is to technology value we setting delivery important in subject development

likely and the appeal so know license, how going the that? to not before granting think of launch? terms in BLA the we’re also Like, about do potentially So, decision you of sort

important number are to points make. there a Well, I of think

to in-human some not the think applies that to again, the I appeal therapeutics first, all. our but IP is remind just estate, CasX of

to any therefore, believe it’s think have human case. CasX actually development. and therapeutics interference, emphasize in that actually use is that product that not in is appeals subject subject we around CasX to to not it I IP we And or important that covers

but not people is I that so, interference all. what think on of estate, understand around that appeals to is really some I IP And case want our

then, And for me. more just one question

therapies seeing you’re some agents. conditioning development already new [ph] So with in FCB next-gen

gen So, seem shelf it life Any first if doesn’t about the those for like exceed, last too these will long. this? thoughts therapies

to just your be I question. clear So understand

questioning of evolution are new much really depends the on the I are conditioning. it nature the if actually very landscape either actually of to You’re -- the that change conditioning think would products approval? for close the

it’s something As we out at closely. obviously, look looked at toxification, we’ve

of a important for to with approaches engraftment is balance the of reduction One or regimes And we not that for editing size patients the is milder able we conditioning are Many all of the toxic believe both actually will regime. increasing because the be more actually all. it’s conditioning think actually the tolerate non-genotoxic, eligibility population very some see things patients and efficacy. to and toxicities to They what grow patient of important a are eligible could less -- I evolution so expand of that the dependent. of the actually path

of our that think, is the editing that to further for very are also editing of we as but eligibility the just population for looking vivo I important part thing, will beyond, believe conditioning, simple strategy, vivo patient obviously, increase the that the reason not looking treatment. in we in

with Chattopadhyay Debjit from is question Guggenheim. next Our

of just map landscape the ASGCT Debjit. and what be kind thoughts, to that you your vivo you given just of want Is This busulfan. abstracts that it assets to of approach? get forward for kind around on alternative off and editing the with of especially of strategy. is for both I build would And too, Can Ray is the to CDXXX get to sort sort the competitive wanted see and opportunity in bifurcated out exploring the outline conditioning, moving of us? that conditioning sort competitor of of discussion Forseth an in-license in on sort your

very much, Thanks Yes. Debjit.

and point I think strategic I’ll will are comment. start a strategy. From then have Baisong view, two-pronged I using a of we

as enzyme problem And believe for that the well have investment believe in have I stem earlier, vivo on, we target. internally of we significant cells. a solved said as of can can as humans, discovery focus we we challenges to around We where directed in think, on of a investment, the this delivery, I the, selection a certainly focus CRISPR our we three that hematopoietic two editing where is

in continuing we CDXXX. I more conditioning for question ask talk a approaches. you about to about think Baisong parallel, -- I’ll milder just In have that. evaluations ongoing example, or of are asked actually importantly, follow-up the, And

together conditioning Thanks is gene is And can deep, were that cell have for in that I there antibody milder approaches. one efforts the editing. One with direction. this question. the internal say the and into probably that two wise. CDXXX modification And looking looked other we your space as as doing in well actually generally very

the the have -- So, infancy, and still that. previous may approach different latter in I if approach on is is say, exercise many with antibody

and want So And understand monitoring going not conditioning, I are only to be and closely the these. the is leukemia if successful, very successful milder for transplant, mention to also cell therapeutic be many space I areas. think for sickle different we to it’s that successful gene going

space. into this So, we actually very looking are much

with Kumar Goldman Madhu Our is next Sachs. from question

RUBY? about just This is transplants think into wondering long and OpEx, forward for should the how be editing follow-up? recruitment how will Rob We given we And particularly were OpEx, -- spend given on around R&D, robust cell forward Madhu. versus

Rob, repeat you sorry, your just Could had hearing question. I great it, I’m difficulty please? actually

Sure.

forward transfusions We follow-up? should spending be are we in regard wondering just OpEx, particularly thinking how versus about to

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sure, about want the Okay. execution around So, I RUBY you’re think asking Michelle? correct? of forward-looking you’re OpEx to Is that the asking make study. -- I

Yes.

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expect But current as not our just more, R&D quarter-over-quarter. will say substantially. up, current, trials. And I’ll spend enormous go dose one our patients, obviously, do an more increase our cash don’t our but we supports we So, runway RUBY

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

Keith for taking Mary is Thanks on questions. This our for Greg.

we could to to efficacy Thank So, cell EDIT-XXX, enrolled how have see plans this and represented with year-end. patients by how currently you. with readout treated expect update? XX year-end and dosed XX Maybe the be many by maybe patients sickle for the in been

have And X just have the and in to as have edited process dosing. then, completed been who XX have patients and among have schedule ready are patients we those, we CDXX and we have -- more And been cells pheresis. pheresis, of So, we enrolled. patients actually other mentioned, dosed,

by to And so we year-end. be dose patients are will very XX confident that we able

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

wondering learned pricing. on to disease, million. obviously the patients report can so if bluebird key Europe, bio thalassemia, wondering maybe cell $X.X comment suggests comment could beta on there? in lessons the on of Maybe plan from what’s Southern how you market. sickle And launch most about Obviously, maybe are if you capture the And unsuccessful that of some you’re on thinking then,

actually then, maybe is your that any LCAXX, if partner there? on on with update lastly aligned wondering discussions thinking. So, And

much, Thanks very Luca.

obviously, of Europe, disease From of Asia, dominates others. are we South currently. focusing beta amongst point North actually thalassemia our America parts view the efforts a forward-looking, So particularly our Southeast that of world, Asia, is on a

with of of the have to We would are looking open a view, sort partnering, in footprint who an upside as actually development certainly that past from a ideally -- ex-U.S. shared point partner in targeting collaborate and we commercialization. large global

with So I say we here point that talk the your I outside happy that say we’re happy beta the making look will would North -- about with are progress North that’s States within execution we thalassemia can I to United when about America. America. and very what

to we’re discuss conversations and market it’s This approval closer and approval. regard over be when and future to that evolution talk to us With Obviously, we there. about going would to the that around very early something very look to be is pricing. that to we’ll launch but I happy days forward time, to yet closer think launching, for we’re talking about to look pricing, actually we

a to we we deal into regard finally, a LCAXX, we with of have details And have really really practice executed. -- then, going until not have signed and

And -- sorry, maybe more point. just more add add one one point just

about and we to to happy community recently value to are therapy, certainly, this the mentioned gene very editing and are the we report So see stage, space. this we Gilmore happy is of looking are pricing, As in early right? the see that

pleased will this value recognize as So entire in community be field. we to medicine the the of this -- we mentioned, Gilmore but see -- that evolving, the are

with Jay is question next Our Oppenheimer. from Olson

Maybe morning. us. Good Chang for on two line from the Jay. This is

XX I’m that? capacity just thinking? you. the is timing program this wondering cell you kind So, Thank dose question there’s is year. second And what to to where you’re if are patients that on of if planning for chance And What have program, the partner than disease more you your the a do best sickle to ex-U.S. strategy.

much. Thanks very

to dose So, than patients? do XX we Yes. capacity more have

enable, the the to points, rolled our we of out was that actually sharpen to other to CMC the And or edit on accelerating side, but XXX. edit clinical when have acceleration One and important that we deployed indeed, ensure also focus support strategy clinical and capital our again just those, not have acceleration. capacity developing on to we capacity to

capacity So than indeed, to have do we XX dose that more patients.

I then, your ex-U.S. was partnership. of think timing around second and And question the

we as interested think are I I said before, partnering. in

development commercialization. the have timing, to a with signed a we actually And deal footprint the really and actually looking support on to We’re that timing certainly global particularly we regard discuss would and until with a executed. partner wouldn’t ex-U.S.,

Beatty Joel is question Our from Baird. with next

for for taking is Benjamin Thanks on This questions. our Hello. Joel.

sets it could other XX sickle of Looking cell data TDT, that and approval. late-stage patients in across products appears support

track So, do by approvals? on the support you. regulatory think patients XX necessary be able with data to to how Editas dose quickly you secure year-end, you’ll being to Thank

much, Baisong? very Ben. Thanks Yes.

of to in of required momentum. we registration terms total and to agency. the And progression have the of about As positive support regulatory of alignment I very mentioned are need the earlier, number required patients in the with then terms study,

able optimistic patients So, very we we planned. as are to be dose we will

obviously, to and we as key sort the you’ve like sit of is, So, they with would need identified actually see to what agreement I planned, regulators set think programs. an But our do come that for is to the the thing, on Ben, what a data benchmark.

from is Truist Securities. question next Our Joon with Lee

had patients as along of different the locus? of sickle the persistence question versus asking. cell locus the BCLXXA fetal hemoglobin a have What way globin I same hereditary mutations of percentage Steve, So, but maybe with

impacted specifics a it. BCLXXA in much from will have but the not that, globin promoter expression the the know expression. So, the the what question transcription other only, impacts impacts, fetal the have region And factor, we too. we different cells directly globin your on impact fetal is do all But have multiple of is which the mutation, BCLA on different mutation will

promoter for and So complicated the region mutation a optimization [ph] gamma the globin than gene more is much the the issue with HPFH promoters.

was another identified this some strength to of characterize were genome-wide change the sickle ago fetal to cell really prevalence pointing way a a actually and thalassemia, the to of disease really whereas its actually determined the because actually assessment. another little quite and of -- phenotype GWAS actually through it is this quite and ago, a identified assessment, actually robust effect because was think hemoglobin way, the time but of quantify, signal, mitigate Joon, this substantially of I some persistence the harder capacity the is decades BCXXA correlation to phenotypic genotype few hereditary

Thank you.

Thank session. participation. today’s of once ladies for concludes Q&A your that, call. reached and the have this the And with gentlemen, you end We again

disconnect now may your You lines.