EDIT - Earnings call transcript Q3 2017

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Operator

Good afternoon and welcome to Editas Medicine’s Third Quarter 2017 Financial Results and Update Conference Call. [Operator Instructions]. Please be advised that this call is being recorded at Editas Medicine’s request. I would now like to turn the call over to the Editas Medicine team. Please proceed.

Mark Mullikin

afternoon. Good Mullikin, Finance Mark at Investor Director Medicine. Editas Senior is and of Relations This third conference quarter our to Welcome XXXX call. which updates We issued third the this and our afternoon this covered company, call. regarding a press quarter release XXXX earlier will on be reviewing results of available the A Media today’s Investors hours X will website our of approximately call after & be replay on section its completion. we will Q&A. for open call remarks, the prepared our After

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

any forward-looking relied upon In be only any as our views today, representing subsequent not statements of of views addition, should as and represent our date. as

While statements the we views upon disclaim we statements obligation these update today. views date if any even do as should forward-looking as future, not to our so be change. to specifically representing point some These may our to any subsequent at elect in forward-looking relied of

the turn to Officer, our over will call I Bosley. Now Chief Executive Katrine

Katrine Bosley

a an incredibly advancing one, Chief cancer including am Technology joining and Corporate manner data. in and an over primary efficiency of accomplished high steady XX% important approval genes T and this Editas, Medical with today three, efficiency; multiple cells in We've immunotherapy the service CAR a in of that this same Andrew our leadership; Vice at editing proficiency cell internally, gene-editing Tim T achieve all area potential X business We've of Officer; our two, Therapeutic you like T to genes precise drive cells Vic team, of Financial a inserting it long product medicines and T we're quarter of capabilities progress everybody, and we're more been genes on for essential Thanks, time building our cancer first this of type I cells thank may strategic At our the to and human our Chief Myer, with well of we'd T in with presenting organization. continuing afternoon, cells Juno's been comparative; to also cells. cell Officer; X treated. Officer; at for sickle with Juno engineered program basis this the key quarter and along disease substantially forms the We be T engineered the that This two, Juno, and developed Scientific the editing developing Gerry clinical technology Cox, the a big President of for to collaboration as Chief pipeline moment our progress has solid of members Juno range Editas against our capability. the for enhance Charlie safety, has with outstanding hematopoietic we're three, progress shared I'd and Hunt, but our by Senior to the Albright, therapy. This a be that Affairs. these our has can taking efficacy call cancers therapy. combining it. and; beta data cell next-generation has our the This way joined Chief field, cell relief picture. thalassemia. ASH Mark. T third look Officer; consistency corporate Good the treat and us expand our of we've In for exciting quarter Hack, XXXX. several executive with update compelling on working One, in stem to field the is engineered is this as in our made cell therapy and and incremental term the for

been and a with been we've able First, XX% PD-X efficient the editing to case, out than range to of we've no including off greater knock target. genes able wide each inhibitor achieve with efficiency checkpoint detected in

engineered targets, with and to can significantly hard specifically malignancies capability knockout efficiently simultaneously cells. treat not genes. the that only Second, knockout expands range gene This address multiple we can T product we individual of

progress on potential create it and capabilities or the precisely want and be can in would cells that engineered targeted editing transduction we Together, Juno Third, with Editas and with achieved insertion. on major we this we T data genome genes The alone. to progress. meeting gene to cannot allow put us with where lentiviral we approach, at the has targeted the SITC made either gene this gene ASH believe upcoming insertion show any

of these simulation. important through is editing will T XX% survival efficiently, have that SITC edited greater can antigen at intracellular and cells CBLB more enhanced CRISPR than with Juno next-generation CBLB. have engineered which CAR-T microenvironment. the CBLB the in of cell. intracellular leading cells will together, development on potential at T in the suppressive gene-editing on meeting with to Specifically, inhibiting these studies, transmits we repress primary show with Editas and TGF-beta as data along to Juno, of capacity in gene greater cell CRISPR through II the system. function repressing phenotype since show, all CRISPR the report this in platform ASH present Juno cells receptor molecule effects its believe an the to in signalling have also December a of This particularly for induced more rate This cells T BCMA-specific knocked to protein, evade T conventional Editas We'll knockdown Taking block that difficult. production TGF-beta T gene cytokine kill prevented CBLB will the response and very tumor CBLB work TGF-beta human knockout the This using immune is in expression the demonstrating cell therapeutic knockout is cancer. result proliferative well-known down and using we modalities of and tumor interesting signals and

CRISPR to disease distinct is primary show I we important hematopoietic protein. CpfX that locus system, that globin efficient on beta Turning from it stem potential company CRISPR in with other efficient access on the a its with grow internal have the of recall the internal and and is work. Highly distinctions into high CRISPR a the in the sickle of that ago. CRISPR/CasX. up-regulation is specific will gene since a CRISPR native beta platform successfully present CpfX engineered a about CRISPR-editing efficient present to progress as opportunities from genome work highly cells Combined system Editas a treat CRISPR/CasX. we editing been now with with has make the our in gene a show our and engineered CRISPR develop At and also integration T eliminate to at is We're confidence levels the simultaneously targeted CasX, to and an at with therapies from We The reported hemoglobin our result ASH, scientists system ASH multiple work CpfX Targeted human would also excited replace we our at showing nearly we cell we'll into and cells. new only production only but CpfX to CpfX. insertion HSC show significant will thalassemia. compelling to for best-in-class beta editing ASH made with hemoglobinopathy. medicines data to our and this programs cells continues data normal believe Editas CpfX has in cells and besides DNA-editing as data hemoglobin, has we and previously year eukaryotic CRISPR/CasX. it location fetal globin to work it of capabilities integrated sickle of of

by have Last is well is the the correlated by second data and the CEPXXX editing EDIT-XXX. showed our significant dose the at were one dose to therapy, animals in and detected consistent now also LCAXX. we time-dependent filing of carrying results we with data in made was patients. is through EFGCT are for The mutated genetic in package that gene the and candidate human that to machinery lead these our product support pipeline, target editing cell preclinical rapid, is which mutation, is robust the and for for for studies and X-month study of third, then studies. oculus with We reported durable robust. targeted EDIT-XXX show is integrated progress transgenic. IND program the We're that treatment the a our This to track previously editing first mid-XXXX. and their CEPXXX first gene efficiency in now primates. preclinical with results the on Overall, in administered the the pharmacology treat month, on to remains only editing was from studies, EDIT-XXX type EDIT-XXX. nonhuman inhuman Moving CEPXXX EDIT-XXX these photoreceptors, building results of life these the In

the. person that from gene restored this in demonstrated human First, mutated expression, cells edit CEPXXX can functional we show successfully through and we we

photoreceptors that the in show to eye successfully edit we can primate price an that the is similar nonhuman target the eye. Second, anatomically we

we with of that treatment EDIT-XXX provide cell Third, And human for photoreceptors fourth, demonstrated We transgenic time-dependent for with human editing type in this results believe that in human patients predicted show the retinal the the we development we mice primary target support can edit EDIT-XXX clinical of for dose these durable and in therapeutic show mutation carrying target. We we successfully strong LCAXX. study. genetic cells.

trial filing and enroll our plan patients. EDIT-XXX In and investigate Europe tend this for approximately initiated the site product approximately developing front, designations This ATMP, and XX a Ear States Therapy is LCAXX LCAXX over multiple received will for history evaluate designation. time. in the robust we patients the history natural and above endpoints. study In quarter regulatory open ages additional granted the course a of quarter, United study Eye patients to potential and studies EDIT-XXX we're addition year. and data to to Medicinal up IND plan. On this the first Massachusetts to the from are of clinical EMA at Institute or our We least the Products, clinical we EMA to and sites at for clinical building natural disease package X recently, to Advance earlier patients, sites and follow assess medicinal X executing

development. organizational to now Turning

The these. build gene hires like of I'd Regulatory as therapy make team to of past continue just across including has joined and ophthalmology years our up and quarters, to President She therapeutic areas areas, experience number key and Affairs. We program. over a a note several Vice XX several of in of couple

added over hand the our have And Andrew the on late manufacturing scientists for We now senior CFO, quarter. like who've from I'd programs to to clinical financial worked also members team including and in retinal the ophthalmology, review diseases. stage Hack, results call to

Andrew Hack

made our and of summarized Thanks, approximately our in marketable and hour equivalents capital at financials XX sheet cash, business, press quarter we couple have believe cash uneventful quarter the XX-Q relatively securities. was through we'll an development. available about and ended position Katrine. to current with of and we we'll cash ago trends we the the quickly a fund statement, finance we least the a $XXX of months balance Starting We cash on the in from our flow Based perspective shortly. Form million and with It release file an items. run that

including milestone. Allergan Turning related Allergan previously to and million milestone In of and million we worth million licensers a Juno alliance payment related recognized noting in our development of made $X.X associated expense, that a to research our disclosed. revenue one-time Juno statement, with Therapeutics, the to we upfront the $X.X with we payment quarter it's our alliance that to income and the $X.X million $X.X

to $X.X back sheet million To Katrine. in technology have I'll a extend we turn strong, remains in noncash robust to our compensation With capital needed transformative of expense stock-based the pipeline we fund leadership Finally, balance the to and summarize our the that, medicines totaled quarter. it field. believe

Katrine Bosley

the A up Thank comments isn't over we of XXXX Q&A. couple quite you, yet. it Andrew. closing turn before

Editas. preclinical for with back, tremendous candidate, really regulatory lead a We data has robust progress Looking important across it decision. advanced product preclinical of been study species, and multiple year initiation significantly EDIT-XXX, of

additional Allergan's our and product critical Allergan us program sets progress capabilities. to expand partnership provided with capital and candidates rapidly to move EDIT-XXX substantial from In area the the benefited in a addition, that and We this candidates expertise we pipeline of diseases we've in done think established up made which the product with with well technical a eye.

Our in property Patent in we and had the favorable the both secured Trademark and issuance year substantially. decision U.S. earlier CpfX. from We first for the strength U.S. and highly Europe patent of addition, in CRISPR Office

questions answers. team exceptional. continued culture. they With to for Our had ASH and and progress we'd The sharing look and that, meeting. organization. of continued people advanced blood to we what these pride engineered diseases the cell your interest forward We for Editas at strengths and it for are all are and to of you upcoming take our Operator? therapy drives cancer matured And substantially grown and our support we and and the and up finally, like significantly open thank accomplishments

Operator

[Operator Instructions].

from Our Cory Morgan. first question comes Kasimov J.P. from

Unidentified Analyst

on some programs for Sean the the the one of Maybe is and you're expect the data might we or you a knockout, just entering of could just see collaboration clinic? one knockout cb pX for kind on lb when either ballpark presenting the This genome Cory. so the

Katrine Bosley

you, Sean. Sure. Thank

them will programs you externally we externally collaboration haven't keep -- have timelines on we As certainly Juno programs, updated with with so program these specific don't put on folks and yet. in this on work on haven't we progress these put we yet dates but just -- know, we

Unidentified Analyst

early don't appreciated the that it's infusion, the provide so you CAR of here? CAR knockout, stuff option. anticipate to with any talks T PD-X PD-X have similarly during days the we've with that cells the Do been still got some for what color can you expansion. knockout. much is Kind Okay, T companies any you. say be guys absent very will behaves PD-X could so or you But that for kind Again, are seen have very, example but here concerns because the really after the far, of thoughts of sequencing your the PD-X

Katrine Bosley

Myer, Technology speak. ask Chief He's Officer, I'll our actually Vic in alliance. involved to Sure. genome deeply

Vic Myer

will important prices localize difference and clinical important speculate between Our for over of ultimate systematic that Do sort our cells think systemically you T us not antibiotic therapeutic. note the the PD-X to so output. to that the on approach administration a clinical be will I it's think CAR an administered be of

Operator

& Our next question comes Company. from Phil Nadeau with Cowen

Philip Nadeau

First, cell and program that sickle on cell. for one you the question beta discussed

frequency the hemoglobin you're much talked the CasX integration? on You about working made CRISPR targeted into you've doing reducing integration progress beta how when the the focus. I'm a on of curious

Katrine Bosley

So our one those I take to Charlie, to do to Charlie please? want Albright, Chief will up, that speak Officer, actually you Scientific ask programs.

Charles Albright

at integration be cell Sure. We've target the rates from primary getting data a multiple and with success about so good order we're the It and types will some of of meetings. integration the XX%. in upcoming disclosing

Philip Nadeau

problem? a technical or they problematic solvable a Great. idea how they are are challenge? Can of you us some give are? still

Katrine Bosley

Charlie? head will and Vic comment well. then as go that on Charlie,

Charles Albright

Sure.

depends they therapeutic you're at going is application We're of where specific right cutting our represented on as that some to where of not. now. but details not are are or the not. they if depends those show where problematic I are We'll think that or in a problematic into exactly post it on case clearly lot

Katrine Bosley

it. on are the I key points think those

Philip Nadeau

IP the the One the date great. in of give and the Can Okay, in update question disputes? you here European also IP. of then the on any timing appeal us you an the hearing milestones U.S. on are

Katrine Bosley

issued in as this with February patent in that been other the procedural patent regard then recall year party was there ongoing office have and the. the the number you'd results by appealed like Patents CRISPR/CasX then. favorable are very Sure, we'd that for that U.S. States, to United the steps issued of since

brief a brief response filed was side had other then filed. recently a The and that

then way a date, is proceeding, more time frame. there bit summer a couple of decision QX proceedings particular in sometime hard of In the transferring and or a So it client's a which the settle some doesn't early we'd in were have, can timeframe. specific steps late procedural through general have the hearing a to other QX anticipate going court QX,

quite here has As all keep patent really office regards Federal them, patent is other anybody so which I'm a and the With that dimensions it made there's in a in different Funds, patent the when a cetera that are issued make The thoroughly. discrete mind office very then X question is profit be offices focused the this of that knows faces different issued decision Court frame the file make Europe, Circuit of of to so to can of is it's appealed. if on the during a everybody end examined etcetera. people the doesn't is that at in they period sure whether the then has months, mistake file Europe can questions there in really the then law opposition Circuit in that who and specific appeal. X-month profits can after patent so other looking and overall hash reference, number the Court decisions of responding the patent and parties office a et questions

is take some that this time. a fairly So will profits

that instead not earlier oppositions having has in a patent further recall, early gone in to as much issued on Europe process that such. as very and broad and they having may process not file you sort and jurisdiction that in and patents had other As in responsive, are issued along of being through opposition and haven't filed of issued

over appeals and in So of to time and Europe, given processes it that of and steps but nature add responses period. will there's some oppositions plenty the

Operator

Stanley. comes Matthew from Morgan from Our next question Harrison

Unidentified Analyst

cell work and Matt. this cell? Cyrus to electrification? cell sickle program. sickle This ask program how about viability from been does done CRISPR when has cell do then for beta wanted What you guys on on the And also I is therapeutics differ

Katrine Bosley

Sure.

Charlie, speak CFO, to to cell ask me our sickle Let question. Charlie? viability

Charles Albright

think viability we had establish the from to the the We over last and ability be showed that and good was thereby to post We cells that several record not reconstitute the that cells editing viability that and to some we've or could in of Sure. electrification them animals stem and the data shown of years. electroporate detrimental

Katrine Bosley

when defined for -- that also particular is we for when it, And with approach to disease by the is target our working any a by part the over working way so gene treat regards how at and what the our about, patients think about think really sickle and defined to for competition different. isn't be to we to that disease, working a Sure have celebrate. patients program about therapy say to actually of all products you this just can and technology, its is reason so noted competitive company think working cell -- of bit other the working it it's mix, all us those of landscape, at therapeutic question product this lost on going competitive profile we that help those this that not and and of fantastic gen-editing decades, we that's another are set is patient many frankly, case in successful we to if are we this then given any

product hemoglobin three there's we've and the about we what seen from Two I'm it that goal looking approach work, stage in when that's we to our about to been fetal there will can landscape targeted sure as full and talked so the have what's if regulation target but really and achieve up editing it think a that best-in-class surpass that strategy what the expression of deliver give we fully what studies. different different is we a and what goal. insertion different ways there's the think to with best for of product, Editas profile potential do of hemoglobin terms we're the be showing results achieve it really work think an a will I think we fetal we Again, patients. it's at we better, strategies, about think competitive than for potential profile

Operator

next from Barclays. Our Wang from question is Gina

Unidentified Analyst

This is Parag on for Gina.

The that question on first quick wondering the what We ESGT data over expression RNA presented. that just the one was is saw mechanism decreased for of decrease and the time, CasX detailed was? a

Katrine Bosley

speak to data? Charlie, Sure. to do you these like

Charles Albright

We now. the we're mechanism observation interesting and that Sure. well as thought was investigating

Unidentified Analyst

update have believe Great. you the wondering you I One with rights on and Duke, with for an have given you program. on Dr. D&D collaboration that's Duke? can announced with give collaboration also recently exclusive that us if

Katrine Bosley

we've programs. a some of of our think I number early with this noted discovery

more in seen the decline. a in there work tougher. call we challenge, it's at is some where tougher of that are to cell some we areas other the mass challenge that D&D, targets the are to As preference example, to where have than tissue reach. have -- work of technical for deliver is we significant is easier higher technical a are delivery other early because looking a are there that Those we the

because So challenges. from to the quickly would the ophthalmology path said our longer of marrow even more and that you D&D bone program be always see a blood is we've areas have pipeline in technical they're the is technically and much that so programs straightforward the going and the more

making and there D&D a some a in of years, did in from I technology the had of in think CRISPR building doing important We obviously of to platform. be Duke, important that we've the great collaborator medicines. the the basic capabilities this think include to portfolio intellectual same that this to for There's think early Editas create other and recent and interesting hard part I DMV point people of over advisor As biology in as number studying. opportunities medicines It's is of the working and there have is great company, know at a so Where we been confidence and really Charlie overall our days daily and part how been in our and scientist. lots has depth editing while diseases. a work of of of thing have great get and of a I not he's they field to will serious property license we

we're the and in to confident that to translate about our unique quite So so. medicine our do platform biology capabilities ability into

Unidentified Analyst

last to the Great. editing what us just trying you're recent you understand give methods just And to you one to and a if the route? in that base that And reported then go one, I license? if would can color from about little want were access abroad wondering have to if can squeeze MIT, thinking, whether you on

Katrine Bosley

and other could Sure. founders is there our well been very as It's be of important creative from no one that. scientist. I for editing and with familiar cool fantastic some that work David that and come in question that about time. very area think therapies and the as science, could excellent We've his base

context. in As Editas either we in better our think in or fit and how platform we other we what build about versus what think would include might do we

some Sometimes the will times away in, yes answer that move answer forward. from to we the be be better will bring to want it be might it

base editing. ongoing. we part overall active property base currently our have of we So is do editing work Intellectual IP don't have but some

huge fans there from actively we're science, that the come not that But science. think but huge and I exciting our incorporating think of be to into it's could right it's platform something definitely very of data fans I now. cool opportunities we're

Operator

Lawson Peter SunTrust. from comes from last Our question

Unidentified Analyst

of an on either are hemoglobin hemoglobin And I there understanding gene approach can corrected is for I confused. are and like the a data just using Peter expression? either the for deletion I ASH or so right? program Lawson. kind would can that 'XX? by quick Did The an Shamit you targeted do us get by of which at go got question, gene expecting -- This CpfX? you by insertion give Or had CasX you IND

Katrine Bosley

estimate where from. come sure not your am I IND has

we haven't that that disclosed think timeline support any yet of as you specific We program it to timelines and up returned sharing for in to yet. and timeline program haven't the projected for certainly forward look but of data as that I

of approach I CasX ways and think making can explorations about to and you're when to the which it best what couple on pointing approach different we've area you you out , clinically. very in is can -- think figure data are our in which of doing product editing is you the can best this correctly possibilities from is part there can view use been what we beta-driven, different use approaches CpfX, we our a through has

see data part that does the sure Not more of I support of You'll we therefore, answer, at kind think ASH reveal that what shall the can and see you'll works. progression say you of is obviously, that program what I going the we're publication. a you putting the and and see ASH we help But trajectory continuing work the have internally we're advanced on. the in from think will

Unidentified Analyst

be after is have while... Or History question. when over? last Or then meeting? it Is the do Study And RAC an should it? Should idea we expecting be a we like expecting you National

Katrine Bosley

advisory addition is IND meeting meeting profit. assessed RAC in States in committee the to The FDA the United

IND. closer a running time this study in it's at program and with the and really of not in to So essentially of with as not point history the RAC, to package regards the roughly as to it much There's is. parallel related let set more we per national certainly really preclinical se, when date folks that it's that the think know key get we'll

it's know, package lot the there oral that As into I process clinical we often that a investors you get of times is an it's we've an for IND published community, stage but the data that's somewhat an patient and opportunity of community limited would more but broadly package and think the also anticipate probably actually additional presentation at I window available as be well. publicly, the will available because data that the and RAC, benefits overall probably is for think public folks certainly to a the RAC

So As, be in running the Again, but a that Bs not linked They're the of one scheduled, IND. books the a will not if we'll yet not unlinked but can on Cs, Both that into explicitly. another happen point think date not know parallel and with prophecy. it's window into to folks good clinic the of they're actually they're connected, there's like before it roughly time. let so

Operator

showing Bosley, questions CEO, in call to I'm the no back this like And you. Thank turn remarks. to for closing further the time. queue Katrine at over any I'd

Katrine Bosley

this really think do as your have Thank Thank support questions if and medicine we you, in about advance advance call afternoon. We couldn't you the appreciate and work any this your please our you participation and call everybody. to you, company everybody.

Operator

you call our day. and Ladies for a conclude great Everyone, today and gentlemen, does disconnect. have may the this

Editas Medicine (EDIT) 7 Nov 172017 Q3 Earnings call transcript