EDIT - Earnings call transcript Q1 2021

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Operator

Good day and thank you for standing by. Welcome to the Q1 2021 Editas Medicine Earnings Conference Call. At this time, all participants are in a listen-only mode. [Operator Instructions] I would now like to hand the conference over to your first speaker today, Ron Moldaver, Investor Relations. Please go ahead.

Ron Moldaver

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

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

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

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

Jim Mullen

us everyone and thanks Good morning, for today. joining

BRILLIANCE for data to we had the results well very preclinical start to corporate our will addition broader over discuss it EDIT-XXX In and position. with our will our is a Medical I by Chief provide providing expect We’ve to Lisa trial progress, the Officer; The highlights progressing clinical quarter an our financial Officer. Then, Michelle to Lisa reviewing Michaels, clinical Financial some then start joined by will and I to Ron, year. Chief I’m strong our share Michelle year-end. Robertson, on and strategy. first cash and hand and from update

filed pre-IND all patients. screen clinical RUBY activated, site work trial excellent the difficulty] to EDIT-XXX recruit made has patients we begun also in side expect manufacturing advancing year-end. beta-thalassemia in RUBY first trial. in and is for to has We've The and been before be The [technical headway as EDIT-XXX in for it

is partner continue platform Shearman opting He drug financial month to clinical in quarter. has We pipeline. January capital track comfortable CSO. two and are program trials new our numerous raise Squibb will long our an from another be into that we an And organizational Mark discovery who standing oncology development will I'm led our There successful Scientific gives multiple past be Myers pleased successful us clinical this as preclinical through and finally, programs therapeutic next experienced brings with brought advance and modalities. Bristol perspective, Chief across executive leadership Dr. our Officer. why of position to believe our record progress extensive clinic as multiple into Mark us partnerships. and reasons Mark From ideation an for has the to announce achievements joining a EDITAS

Editas with in pipeline preclinical will expand First, indications, and potentially programs these medications. to platform neurology advance therapeutic in editing world gene in areas. other a overlap areas our utilizing have of expertise existing immunology considerable our class his technology ophthalmology, Mark while

is global Mark Second, and leader teams. more importantly, successful respected of large research and

earnings applied team, our to I recently our us addition to us allow in leadership call. our progress right facilitate Mark's on significantly experience to Editas support continues companies you the genetic right the Merck, help technologies forward grow, structure to having and will look strengthens like talent. join As and attract most next Mark's and

draw conversations first have class and being by These and to employee, what helped mostly Editas through what virtual time world organization during all as can I an In attract people months talent. addition a CEO do the to format. we Editas, every as meeting retain to me spent Mark, appreciate recruiting with

our intricacies on how of editing of the we further towards editing my has confidence existing people gene all have editor chance leverage organization, having our I understand gene better three only we the these technology levels synergistically development. platforms, and every principal By with conversations in in of Speaking had that technology our the these a across strengthened we're and assets Built can top technology, platforms. to and developing. platforms

in we focused have ocular diseases. initially vivo First, on gene editing

just to building cell our technology vivo focusing and beta-thalassemia. therapeutic we ex cellular finally, sickle that These editing with different own platform The us three we And gene could that editing three growth. -- problems. patients intrinsic serve have ways Next, can impact these have Once focusing vehicles provide proof-of-concepts. we a genomic And leader of their These being expertise demonstrate for immense are different platform reduce practical we each resources. use each our products areas way also oncology. three and solve of applications, then indications. continued for actual we therapeutic platforms not and will gene different never requires as to seen finally, have in of within the before. values, a platforms on in programs have on These are sustained lives blocks they to within these vehicles medicine serve

partnerships and commercialization. As to will our critical we be programs, for continue development advance

pipeline. global our extend our best Squibb, and a leader expand to leadership our been date Bristol Myers opportunities by with and pursue collaboration oncology. accelerate, believe we've We to enable exemplified will in We successful

is partnership an additional BMS As that into mentioned, program opting along with quarter. this progressing

our that marks the Overall, I'm months last X that still and There's our this our challenges let objectives. over In progress. accomplish year-to-date short-term place turn need With in in to me another people to have declared long-term of collaboration, a the confidence and further our call under the we right space. addition Lisa. overcome we'll have that, important technological extremely happy in to to milestones milestone the of expertise validating development with this at candidate end span a year,

Lisa Michaels

you. Good morning, Jim. Thank

trial brief BRILLIANCE the a Let for on me EDIT-XXX. start with update

As screen in patients dosing initiated the we the our we this study. earlier in year. enroll second with cohort And and you continue regained momentum know, to

our inherited LCAXX pediatric milestone cohorts. is cause It's around early the independent blindness necessary of the we required exciting the mandated an in resulting retinal meeting, actually this As planned next the common since disease, monitoring LCAXX X and children an significant data progress, part out every will in data most committee is the blindness. is review loss of summer. and begin of dosing a meeting decide degenerative And affecting to vision XXX,XXX for steps protocol existing with childhood two onset the world. the And

that report results promising future. the to be I'm we'll in able hopeful So

continue we're of all every the first for X data safety We months the effects and concurrently, the to beneficial patients expected follow of the confirm to year, collecting treated editing. primary for endpoint in

And a model retinal annual Research the Syndrome platform, BRILLIANCE Association off X two meetings the As from developed end trial. on ocular advance programs. represents organoid, EDIT-XXX choking cohorts Vision are applied the and year. Usher progress photoreceptor retina. we've presented retinitis Jim trial of in vivo Exon at to we mentioned, before selected ex me, of clinical USHXA retinal for initial complex human for deficits we this XX the data the and trial a effects to intent to USHXA rescued Ophthalmology. practical which being study deletion plan morphology. also the I'm restored to of other and for protein This pigmentosa the the moment, recent a that mutation, and have iPSC of include excuse in preclinical data provides XA share a editing of from And the organoid most data derived is our our the human first learnings from The patients year in vivo expression

potential the Now loss visual functional the provide USHXA and protein these to retina. function results evidence human in of of function restore rescue

relevant our year. clinically which As editing for strategy. for candidate program dual to And RPX by further to using expected a AV adds clear RPX, system, editing development therapeutic levels we've our validation we demonstrated

also specifically to Now our ex sickle and for transitioning programs, cell vivo beta-thalassemia. disease EDIT-XXX

which lead and patient and dosing. We our clinical to for been investigators that through very included training that patients of sickle and steps needed first for our trial And and consent from trial procedures We cell their staff cell the And harvest disease pleased have the has completed the end patient RUBY clinical announce activated. has for subsequent the screening. to the dosing complex the year. also approach on to I'm anticipate that first started site site actually the meeting protocol before enrollment now investor

Additionally, we are currently the preclinical in IND filing to beta-thalassemia. on for targeting And application the support track IND work year-end. are required we by still our completing

such killing our cells knockout allows environments. belief specifically most occurring persistence and a the future data so, proprietary cells. And program. approaches other fetal finally, NK edited our separate beta enhance And NK [indiscernible] function as the NK play exploring demonstrating believe enzyme safety, want NK that cells naturally TGF that for superior other demonstrated showed hemoglobin. cytotoxicity CISH certain treat mention to showed to hemoglobin We beta-globin medicine enzyme, additional of in opposed containing tumor can have The further BCLXXA. mutation that expression cells. of recently NK a enhanced presented and ability that edits which with the concurrently for improved metabolic our iPSC mimic CasXXa By when to EDIT-XXX recent tumor from will have activity We and unedited and at of best-in-class our proprietary beta-thalassemia. We've compared solid fetal to NK aim In cell Association cells support Cancer that continue sustained as derived Research. sickle editing with doing of been to demonstrates to targets I associated we the disease through role findings posters robust tumor to CRISPR/CasXXa edited American two locus and an engineered hereditary use cancers. in important we're to cell treatment These micro at differentiate we also

And will updating like to you turn financial things the now that more results. And over program before to on that, so look through to I'd to the I of briefly Michelle end forward the run year. with

Michelle Robertson

Lisa, and operations advanced as you, forward. and Thank position our strong continues in Editas good to portfolio everyone. financial a be morning,

proceeds equity million of sheet. balance Our we The cash was position $XXX the million have at raised $XXX our March end XXXX. our from offerings strengthened of compared January as XXXX to XX,

plan our will fund expect XXXX. balance current into our We cash operating well

preclinical position of executing and across BRILLIANCE and leaves to while trial, us advancement also manufacturing our ongoing continue our capital candidates clinic the additional strong Our into the clinical pipeline, funding enhancing enabling our capabilities. poised RUBY and

compared million we the quarter of Bristol issued also The $X that first for our results strategic the period revenue majority today. have attributable quarter Myers Squibb. and Revenue was and revenue Now turning $X summarized we expenses, XXXX for release of this with year. to to alliance which financial last was our in press million same recognized to the the earlier

by for $XX $XX development million last and Our and remains the by This year. result operating clinical spending to million. well Overall, mainly in related $X administrative expenses success This approximately same a million, million licenses to expenses our $XX compared collaboration the manufacturing approximately of was capitalized. was Research year-over-year General to very charges year. by increased $XX as two expenses. compared increase related quarter period across to first million and to million well Editas expenses to by of as increased last $X for employee and increased $XX clinical nonrecurring million payments programs. driven increased operations higher

we capital sufficient our into operations well to XXXX. sustain have As mentioned,

be to hand important value series continue back will inflection confident cash a position strong advance to We our fundamentals of Jim. expect that, points. and With of technology I'll help in our potentially the business through our it

Jim Mullen

been the you, months company. past Thank Michelle. very It's few busy a for

human us its an us for nearly editing. As allows conceivably genome. last advance the doing foundation we've genetic been This in with to mutation work treat it's science, incredible I new for our in programs on possibilities generation of truly mentioned our every call, the explore exciting technology, gene to and once time

small capabilities. fraction addressable of Our represent only existing diseases indications technological by our a potentially

novel a to technology the progress into and genome pioneers a solidifies technical basis, challenges practical a therapeutics. editing develop among place gene of we continued of order on Editas class discover was originally daily Our continue formed and clinically transforming leaders numerous of gene the overcome medicines, in editing, to and

platforms future We the and continue additional to across forward developments. updating you gene our our to of expand reach efforts editing on look

support. Operator? As always, all up you for thank we'll interest and answers. we open that, of to your questions And and it with

Operator

from Lee Instructions] Securities. Truist question [Operator of line much. the so you Thank from First Joon

Your line is open.

Joon Lee

Thanks Hi. for taking our questions.

knock you RPX, for than at RPX editing, the and is efficacy XX% How Harbor do which set XX% for there threshold at looking you LCAXX. poster test strategy, the out, higher So for a this the therapeutic is given get a through editing negative in, us knock can follow-up. efficiency condition -- the knock that just I dominant vivo have you And and in out require walk knock you in expect?

Lisa Michaels

All rather going answer but right. you I'm I'll jump else. more give than clinicians to in, scientific the than the anything

be with at vision. the data to or LCAXX XX% acuity. less -- visual lowest that at some fact, main the only modeling retinal best in we be in expect to dose which to more level result. to start that function limit, required was identified least XX% And restore program, the at able would It's lowest a or For at of have threshold threshold able be suggests the have the would least of restore to normal order to

more suggests levels able than better to And dose is higher less the editing we're mid editing. that in provide the we're predicted actually of clinical XX% that nonclinical or actually that deficiencies in are Our cohort the or study in to data current fact, much that. achieve using

really to dose So finding of response. level of the is what the optimum actually of study appropriate determine to is get part editing the required

And actually In to nice therapy right. the the RPX both the is well editing, one knock consequence to real a through a way it doing approach. for is the account gene causing edit, vision. some effect, LCAXX, RPX particular be as that's able the you're type the actually too able replace autosomal the which an gene, replace gene benefits is normal gene the to large mutation the gene a needs to of out as efficiency This the to of to As CEPXXX case in to RPX corrections. take we're make of absolutely being program, able of because gene like is dominant dominant as editing into ability able knock to actually

the may So the be to is expected that threshold have at actually clinical treating here what projected the be we XX%, but the higher. our to to get ability moment response, able setting

Joon Lee

Okay.

has vaccinations have LCAXX since And second dosed the remind data. of inclusion enrollment many to have been forward the the enrollment? And advancing, clinical us for you that Looking following, many this changed so and And criteria an you you -- patients -- accommodate you're cohort so how are for sort how out? the can how to the rolled you've far?

Lisa Michaels

much really concerns patients patients, actually potential enrollment primary Because to dosed dose finding some written order allow some first acuity is So, were that easier through able related only complete currently of not to for perception, The two enrolled. level identification we completing well no the least study continue as being be actually cohort of patient strict so mid year. basis patients. was of far protocol we the are cohort way not who most patients. quite itself four measurable dosed of editing. and actually is the lowest Probably the [ph] allowed first and this made purpose the the at able protocol made to to to of the change The And visual protocol provide -- much safety more important being as tracking by start at in date to forward is very be and the saw light is moving we if well the dose total that the in end those two so of enrolling safety we selected the that population is more thing change first were modifications protocol just is of actually have patients on the small have dose and restricting predictive by the before largely to concerns. year. actually expected beginning in And the

been benefit us and for to hospitals get sites and As restrictions of the COVID and really loosening to somewhat travel honestly, to it's loosening allowed within the have also patients the that vaccines, real of their treatment themselves of travel has the for I therapy. restrictions, mean,

And so but easier things changed sure I'm moment, at get made centers. vaccination the able that to COVID the it has anything, to the patients not being has treatment for

Joon Lee

Got it.

how us doing [technical many of capable the sites difficulty]? are remind trained Just this,

Lisa Michaels

in day have really we're moment, So at at people really expand with the And study, injection. already history -- are non-interventional study. part to comfortable to sites at center end those centers and of set looking we to the -- our we're and natural patients continuing sites have -- we is And have been that treatment as our study. the beginning really fact, going sites as retinal that our now are into the where we clinical of with up roll have

they nice But very trying So into where are different back to to we nice on observations injections in these sites we that very baseline us a begin patients consistent eye. know somewhat we move the surgeons comfortable and to study. it's and of have centers the of that number to as reproducible the able retinal limit the and it are give

Joon Lee

Thank it. Got you.

Operator

Thank much. you so

Your J.P. Kasimov Morgan. next from question Cory from

is Your line open.

Cory Kasimov

guys. morning, good my for you. them questions. of taking for Hey, Two Thanks

have then IDMC the Thank more If need? you. factor and active to package in more meeting what to trial adult think investigators sites do prior any what's the your many are you'll First how happen dosing to LCAXX to so, program safety dosing pediatric RUBY dose since sufficient patients trained, one to with to patients? needs you additional the are prior regards summer, you have do the there? patients And to is gating

Lisa Michaels

question. the Okay. Hi, Cory. Thanks for

-- sites us told centers take actually We that in transplant had a what to patients in terms the does dose what the treatment. selection identify had already be patients to of I is they the patient guess and had trial. site willing question who for asking two pre-identified would first and the take, RUBY undergo So it

one -- relatively two centers least those and now running at of and up second those So shortly. one centers we're expecting is the

approach patient protocol this the it the that to details at the At directly site the So consistent have fairly process. And ability space. across actually actually we point, through disease signed. center, is point, the the process that's opportunity relatively step patients laborious entire to consent and gives go a the freeze that wise and complicated to start the with sickle cell of is

So they've start allows they the regimen a already to which chronic basically the all once down need transfusion the because the undergone of quite sickle mobilizing on freezes can transfusion regimen, is or screening patient procedure cell procedures, hemoglobin either to and identified F be process these and the a severe. precipitate actually dilute them putting they can crisis, and cells patients through be

signals them procedures. all till those then So for and to months, to is different, multiple days a since in get we at patient the the the to the have a come the But and able patient transfusion start dose can protocol, of that sites once we have the the we point, adults or X time as and actually At primarily to where screening, observations is order from the to to return patients collect the end then or demonstrated pediatrics. done. not we've we're can centers made walk and will adequate who this on But in patients cells treatment. of by to from back time already and it The enough of have a patient sign we and we've least days question, on it the procedures actually forward. do safety bit actually we're IDMC start Editas to is be to to that decision. order those the of It's to coming patients up a the in also used that treated. signs To in upon been the our and cohort able you're the months door have the adult the that to door to not to benefit second to will do takes schedule year. communicating upon the will number process cohort. the And a open and in really would know in it's come the safety at we so probably for the two order to be we pretty enrollment Patients the confident cells And procedure. moving recess schedule deep back into that complete Once the And dive that's whether walk why looking the for that second calculated decision to tree out been potential data, have patient part and process. the of editing able plan first advance. of to has in first decision come of second is actually the based consent editing in that the back the there is then editing consecutive about back of one-time decision whether be the interest looking for not the the efficacy in them based -- X give regimen be

whether at scheduled that. additional or that of or not would the few we're go directly so meeting have or treated and adults a do before like we us is will DMC time that determine children for hoping to And that not that point can June we for whether at

Cory Kasimov

you, Lisa. Appreciate Thank color. the all Okay.

Operator

much. so you Thank

Cowen Phil from from question Company. Nadeau Next &

line Your open. is

Phil Nadeau

Good my morning. questions. taking Thanks for

First one follow-up to Cory. a is just

the hope what and what you terms do look give of efficacy see enough? meeting, pediatric really Is good would at you what to in signals stabilization hope promising then patients of terms acuity? more adult meaningful the to achieve visual in the And and sense consider patients broadly, of you you IDMC can In efficacy? some measures us in do improvements? can you achieve to hope you Or that

Lisa Michaels

Okay.

follow-up decline late what the is general, of these patient not a vision this observed of with lot in of we've population, patients. So in there's

of the place childhood. loss majority vision takes actually during the So

and get one in them neuroplasticity is the of to to of that our amount well benefit. as intact, level vision course, less the to that hope the patients retina than particular better of later is when the actually try the about of the real experience will a between not sooner -- kids we goals What's some [indiscernible]. benefit So there's degeneration potentially this is significant of of end the or a give outer unique day, at disease But more layer. as

able we're comes to the normal photoreceptor, normal once that So interpret restore those have of in to hoping function to we're we'll results. actually function the ability ciliary really the photoreceptor. down It brain's

looking, So in clinical twofold. terms we're really of the at that outcomes I'm looking

actually of following is is differently, been One anatomy but there's effect similar And measured measure done as having that of of any changes to measurements them and acuity the just LUXTURNA. that patients. going to what itself. having be will And could in of edit if patient's also in functional eye back the was the of of physiologic part be see doing related to little meaningful evidence solely terms an But of very for well the bit will the not actually that which slow that responses, itself as to visual both the observations we're our signal that an of changes validation long or ultimate pupillary of has through something have light, me of different all continued responses a patients since of in in manner. for resulted tend goal maze as the editing responses, of a the to by we're a seeing light procedure a responses these pupillary not that's the measured as of or course, types whether change of whether objective colors could in I give be can terms to

Phil Nadeau

question second Then in fair or following just type sickle of amount cell different a program. therapies program helpful. on patients the XXX you Do [indiscernible] from of I'm MDS controversy generally gene the there's being currently treating on sickle treatments of genomic about gene where the the editing, have therapy of light preconditioning sure in very cases the That's as and vectors and conditioning, that with AML used? any a thoughts difficulty cell are of

Lisa Michaels

the moment about seem -- concerns what transfusion as who something because originally and At of methods. editing gene of host much population, the favor intensive and the the is products graft ex of just the the really being is quite in other therapy said but more of it's think to one said I have big happened next the to know think well intensity suggests [technical in to toxicities of have I I'm philosophical therapy rejection. was kind not be I'm still one interesting are The chemotherapy. difficulty], is to as the be of that Plus to sickle quite frankly, which big important in cell we associated cells. a the gene with space, it, good an patients study. malignancy not with sure attributing transplants acceptance myelodysplasia old the that that stop, does still chemotherapy. now or others point. to of news I'm bone at in patients space who we an related to happened terms as next also this [indiscernible], think as versus this to really of in that's getting disease not is the doing or in day, and it's I I and in almost both The biggest patient Whether have this confident, XX the the vivo end limitations lower ago this was those much toxicity. The timer benefit marrow in not step risk feel disease years And

modalities now looking we edits that right at. day, of our things the the end At for we're really of looking of one it's of the are the advantages showing So efficiency proof-of-concept and the different to conditioning targeting but and therapy. toxicity other next locus BCLXXA, steps lower of acceptance of as regimens at the beta course that be the improve even the maybe the looking opposed could will the

Phil Nadeau

That's Perfect. taking my for Thanks questions. helpful. very

Operator

so you much. Thank

Wang Next question from Barclays. from Gena

Your line is open.

Gena Wang

wondering update just product RUBY looking is is you beta-thalassemia? taking hold like technology us are also that Second that two. a a to all for for. FDA these I’ve bit as if vehicle. sickle little you questions. using in for in delivery indications. Do is a clinical my regarding One the you in partial need the consistency to I'm disease. of and trial more platform the regarding question file give Thank can clear But IND regarding order indications your AAV for eye cell disease question

is the show long the data be longest safety given wondering could [indiscernible] the what Just animal lasting? you CRISPR/CasX

Lisa Michaels

to on supported which question second that I'm first going one focus skip answer But the because to will the be program. you asked, the the non-clinical on not to uphold. I'm fully data these is you able prepared that I

the So program to is the in what variability the creature that's consistent I relevant the looking cell is in not beta-thalassemia those beta-thalassemia, reduce for program. sickle really a distinctly evidence different want kind than clear is to is of agency thing first clinical manner sickling And cells. for actually the a say This outcome. is of

probably to probably to In really is F fact in be thing. going to beta-thalassemia conversion the really, our more demonstrate ability F hemoglobin the hemoglobin is important

in beta-thalassemia, we news good concerns at don't potency as have least the place. for that any that is of the assays the So we have or of regarding today,

holding back in For not or sickle us way cell any form. disease, it's

were going main the time doesn't so of moving our for non-clinical We run interfere clinical been do to something part has with This potency studies assay to the hold have on agency to of we number the the the are in to move with moment support hope this We our plans study. how engage a forward. lifted start the with completing always going plans us at to parallel, and after we it are summer. actually forward the in on to

Gena Wang

Okay, thank you.

Operator

Thank you.

question Suisse. Tiago from from Fauth Credit Next

is line open. Your

Unidentified Analyst

Hi. on is question. Roger Tiago. Thanks This for for taking our

data some So you the program. on shared highlight Can Thanks. not vivo just these whether and in differences or they're among ARVO preclinical modular? you programs construct your briefly ocular and key

Lisa Michaels

modular? you do What mean by

Unidentified Analyst

potentially areas the or the other indications applied it that In you're that to could other within sense any future. in ocular diseases exploring be

Lisa Michaels

traditional because them be type In cannot in And one these all the are the treated too there's a a traditional order between So approach. in to hitting Therefore that RPX them that's also genes. problem and that order it produced able this inherited, therapy the editing programs definitely same programs thing with methods. rhodopsin through inherited, by the gene. really therapy is -- actually disease. what big to advantage not And about with are reason to USHXA is of the mutations the recessive means blindness. and LCAXX, also result for treated that we're I things loss gene the way two clear gene think probably targets they’re to are their extremely ocular in actually, of being case, in an be autosomal the the diseases those interesting gene that gene dominant that which vision genes RPX, two large creates partly why they're the of But is is correct the be of is

functional, of and gene again, the in clear have out allow gene to that's the ability in not for corrected to a portion very a the to to eye. function knock So we here normal knock able be

very they're So different.

advantages able why to and some in the genes to of gene is editing would the correct these be is -- these not of the right editing whereas construct really therapy energy diseases. So approach approach, putting shows

In sensitive very cut it's paste, gene. we terms of This not and insert or just medicine, actually can directive. guess, talking cut you're sort I modular paste precision very of whether and and [ph] is high like so

of of one the So these really the targeted each specifics to is disease.

to with moving growing program able because findings being platforms for experience the upon demonstrate forward, the However, one similar experience efficacy subretinal in we our are the with translate building injection, to really but to other levels leading as help to also stepwise of higher higher of efficiency. models. editing a the build well is eye It proof-of-concepts, as levels additional progression, to us the

Unidentified Analyst

All Thank you. right.

Operator

you. Thank

Next question from Jay Olson from Oppenheimer.

line Mr. is your Your line open. is open. Olson,

You may ask your question.

question Zhu The next Securities. proceed. Yanan let's Wells Okay, Fargo from from is

Your open. line is

Yanan Zhu

questions. Thanks Hi. for my taking

starting first that the what the in if you enrolling studies? XA not, program, IND Usher you. are have So [ph] IND timeline Thank Or for Syndrome enabling on is study? you started the --

Lisa Michaels

the we're to more be hoping this still we'll first clinical the candidate in and able identifying timeline. process So of the communicate year. I'm later

Yanan Zhu

how knocking And many there underlies -- are that curious, knockout, in you was in a for knock in program, strategy. the you're in give in gene knocking Thanks. the the knock could of address very us disease have are idea this is because would you with mutations causing obviously, knock able to I gene. approach? many Could some big, you the whole your you When condition. pursuing mentioned mutations a you RPX be

Lisa Michaels

Yes.

perspective that out of from of So going is prepared part in, you're [indiscernible] actually reduce being able the you autosomal a be of the quite total to knock sense range we're of give the this of knocking with value to extent but follow-up the I'd the you. happy what in expression. the not to certain I'm -- to to so dominant really be right a

Yanan Zhu

strategy, of antibody Got cell broad iPSC NK the could mediated thoughts or cell it. such updated the a give NK with on strategy? you some terms of pursuing you CAR that in in Thanks. might terms And NK applications as strategy, type your of be

Jim Mullen

Yes.

yet say this responses. details very environment TGF-beta that data the ability show out evidence to helpful the that quite from actually knock the supporting at of is impressive of we TGF-beta moment quite to overcome have micro the no simply to of able tumor think and the So I CISH, also immune suppression being point. at it's perspective But the

would targeting So solid here place the really TGF-beta. helpful that of an be high entry really from and production key committed think tumors tumors perspective into are have I that's is that that we to going those thing easy

later. outs As that be for talking about, we're able provide knock additional more on knock information double the ins to that and we'll

Yanan Zhu

color. Thank for Got the it. you

Operator

much. so you Thank

James. from Steve Next question from Seedhouse Raymond

line is open. Your

Steven Seedhouse

of patients. patient. taking safety could the two mid safety the signals Thanks you first morning. the on dose I hoping comment good question. you Hey, know mentioned in I just the was no for

was I the that's or even the to we case. study. if just it or you comment verify So fifth cleared wondering dosing as patients sounds I relatedly are currently and if you the that. could And wanted speak in maybe six on like

Lisa Michaels

of safety, that the lined additional progress in scheduled as being and terms have up at in us ability So study patients there the least have speak. We're to observations. -- the next pretty do to not been actually we next doses signals optimistic to safety for are feeling any any the required

Steven Seedhouse

still me for Okay, might I cohort need enroll dose to thought wanted if cohort want just I I And to anything if misremembering, please and maybe I dose the either obviously to pediatric high that the correct treat if the test you're or cohort. that that. I'm higher pediatrics middle third you're you clarify, with would sounds then adult dose. the that perfect. or advancing Thanks in to in guess It like looking or the before after wrong, data you intend cohort open be dose. well don't be but to contemporaneous dose so adults, completed to suggests as seeing

Lisa Michaels

that the because keep The keep any the would me be need go translation dose safety concerns, entire to -- only to to up me I to efficacy. and from response thing dose next really higher explore going would be

dose dose. that supports to we there because or dose they levels no data lower decision to a go made with particular it curve, highest ultimately even whether remember this that was non-clinical to with you the a LUXTURNA, do response, think that advantage of In can higher way safe. the and response If achieve a do And which case, appear an to we editing. the actually dose have was discern have not we didn't

level. dosing safety and that the at well patients ahead pediatric evaluation no the moment, cohort first basically adult dose to the adult at a anyway. the one I I allows dosing as So be inherited for quite patients there's protocol that's working is the concerns, of the high DMC to allow after long going of as The will the same dose

evaluated a until pediatric level at we wouldn't dose we start the patients safety in adults. after higher So

Steven Seedhouse

That's right.

clarification. questions. the Thanks the for appreciate I Okay.

Operator

you. Thank

question next Your Sachs,. Goldman from Madhu from Kumar

line is Your open.

Madhu Kumar

Oh, questions. taking our yes. for Thanks

threshold kind generalizable. XX% ARVO described of of described I up specific. XX% it the that on at specific so like mean, be question the following So kind before, at And so LCA seems the But Joon's beginning, it rhodopsin, was is to though could threshold was an

in of clinical what that So you on benefit do all think is corrective today, needed for fraction are practically, based going you be the know? LCAXX to to the have data photoreceptors

Lisa Michaels

or based we to of least looking I'll moving LCAXX the [indiscernible] editing our what we're say mid or on higher. XX% supports higher XX% at dose efficiencies higher. non-clinical upon certainly data dose for data the Well, achieve to that certainly that is at level the And actually have, at

edits predicted be going edits be least study the us result part ability translate with outcomes to meaningful the the required to actual study. the is of of define will one be or of vision. help at in that the of what number secondary to would So vision, able actual to And

Madhu Kumar

cool. Okay,

So an from then editing kind kind of rate. predictive this that, following the XX% from of for dose average mid

[indiscernible]. mean, I So I as average said, an that's

if average So the practically? say, to below is is what the rate threshold fraction threshold, of editing distribution you expect productive that so, XX% if XX% the XX% patients would be

Lisa Michaels

that’s what actually Well, we're average is talking I'm bit a the the little at dose. higher. threshold, XX% as expecting only editing to that see

Madhu Kumar

okay. then Okay, -- cool. And

pretty tumor cells tumor of I ratio about cell killing at AACR the And thinking cells, the data the killing was at IMK we the as X was kind so to cell tumor data, recall, I'm program, looked the So interesting. of the experiments INK those X. saw we in --

to effector more achieve cells? if a relevant use you So rate you maybe physiologically you with will ratio intervene. of ratio, What your how killing lower that therapeutically even are kind INK can

Lisa Michaels

Okay.

So, of studies. the refer would clinical I to probably that level detail, those -- who mean, for to you scientists scientists research I the who ran

Madhu Kumar

very Thanks cool. much Okay, everyone.

Operator

you. Thank

RBC. from next Luca Issi question Your from

open. Your line is

Luca Issi

companies and circling conversation, on appetite taking bringing so questions [indiscernible] a questions for that collaboration the developing Maybe my for earlier are novel and board. in setback regimen. the with congrats the some Bluebird sales recent preconditioning Thanks Bio, Well, Mark on your the what's exploring much all back I on fantastic. progress guess, of for

color manufacturing So on are the maybe this again, will to second pipeline, Wondering question transferring ocular may understanding bridging any study a Thanks is any you call any will my are great. at great. update you potentially AbbVie from there? seeing so the if run you CDMO. some what you So point And much. a that have And for be be the to there? the ask there that FDA

Jim Mullen

do you try Lisa, me take that to one? So, want to

Lisa Michaels

track what thought also apologize. of moment, the at Yes, was. my I'm I lost just the thinking first about one I but

Jim Mullen

Okay.

question simple at -- question trials have short the planned to answer to the planned, we have for material. and So, short the don't is make the so the we that least manufacturing need is, additional -- the we question cohorts

will be that some There You're comparability to correct ultimately questions have that. product will answer. in we

that but to the try -- maybe studies. Of through non are would attempt. going some to we in human vivo be do vivo, mostly and course, That in analytical techniques

the not require about a they with whether FDA anything. conversation would had have We

to can addition always predict. that, the et of are dependent characterize how product We always have recent And a little lot it assays this always cetera. think turns a on then bit well you the have. and FDA's good on In also just things what to experience characterization you hard we pretty with similar that's is

Luca Issi

any there Thanks. preconditioning regimen, maybe or [indiscernible], Super big And the appetite was for novel helpful. collaboration spare first question around thoughts? that picture any

Lisa Michaels

the earlier. I'm So getting platforms editing show focused I was that and I really on on guess our of a to that had And meaningful proof-of-concept ability edits the moment, basically in answer fall our patients. at similar that I'll back

reduce important, won't as the the that because is to have if we do And those reduced test clinical As appetite going think even to changed the also that treatment is forward move in we're new regimens will to but the conditioning in the order consequence, of toxicity, know able be become the be think overall I acceptance, programs, we I important. don't or opportunity a there. having to efficacy that

Luca Issi

so Thanks much.

Operator

Thank you much. so

Next question from Matthew Stanley. Harrison Morgan from

Your line open. is

Costa Sean

do -- do on much Thank need work the CRISPR/CasX you This on we using this us to this front wondering Sean somewhat we what quick Matthew. from to From it is for on to program is stage? safety IND the it's program. platform, safety Costa the One prior our but question. this taking the understand XXX the that for Thanks be question were given how you. Hi. is at

Lisa Michaels

So of IND IND related normal Because testing safety programs. question enabling about do part to? toxicology our as -- our safety we what's the and of all

not sure So different. that I'm something see I that’s

Costa Sean

data the do. because to no the data, what -- In why referring was are safety there. to planning ARVO That's there ARVO I'm

Lisa Michaels

No, no.

that that's I follow. mean, the So will work

So, yes.

Costa Sean

Okay.

Jim Mullen

was and package on required one. don't think different anticipating any package or we're one a for I tox So safety unusual

Costa Sean

Thank you. Okay.

Operator

Thank you so much.

from question from Jay Next Olson Oppenheimer.

Your line is open.

Jay Olson

do Can for IND that hey, cell taking is would thanks that the in-house? something program, the for an us you you NK your And the the through question. then for program? Oh, timeline cell walk to of file NK manufacturing

Jim Mullen

do So to you one, want me take that Lisa?

Lisa Michaels

Yes. Thank you.

Jim Mullen

-- can of take I'll it. I both sides

we a about constructs we that leaders a So for make to decision first to what have IND, forward move have of the number opinion the developed with timeline with consulting with. construct

TGF-beta. You we've CISH, have seen working on. the data sort that the no But been on of that by means construct is only

With believe the side. along strategic which clinical doing just constructs. -- we we've that partner would anticipate either and partially or manufacturing, initial the manufacturing, don't Although we would to on with have other in-house a the be Catalent manufacturing disclosed the largely I respect in-house

desire. a or sort So the there's part [indiscernible] the developing the point, processes part whether a handoff in there's process yet if on process, finalized in process be we'll a if -- in-house of you -- place to process do And you of there's of Catalent. at in and process entire actually in-house we're where the this natural do the that will,

batches down comes just we make. we're that of many need Some think to to to going how

Jay Olson

the taking Great. Thanks questions. for

Operator

at time. Thank questions Jim, any We this have further may you much. you don't proceed. so

Jim Mullen

so that to we It'll as you for everybody quarter. the but try support turn medicines next of tidy call will in Thanks. wheelhouse the because some with to and back Thanks you need we'll to want -- transformational to great call, Thanks participating next to much questions on. answers and for much and great help we talk Mark few Lisa's, Shearman maybe got have Okay. into get I next be this the up questions. than new than to to thank technology in more and real bring so We've some his probably patients. those for it for on the questions today's your a all

Operator

Thank participating. for conclude conference does for today. That our you

disconnect. You may all

Editas Medicine (EDIT) 5 May 212021 Q1 Earnings call transcript