SGMO - Earnings call transcript Q3 2018 | Sangamo Therapeutics, Inc.

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Operator

Good afternoon, and welcome to the Sangamo Therapeutics Teleconference to discuss Third Quarter 2018 Financial Results. This call is being recorded. I will now pass over to the coordinator of the event, McDavid Stilwell, Vice President of Corporate Communications and Investor Relations.

McDavid Stilwell

Hello, and thank you for joining us.

As we begin, I'd like to point out that we will be referring to a slide presentation today. of a on the page the Investors Media find our presentation of may + www.sangamo.com, website, Events section you link slide to on the And site. the and Presentations that information to like call the forward-looking this everyone I'd have we upon available also during the remind discussed based we projections that and statements that are conference today. change This information over will time. likely future. performance are provide the future of the not updates an discussing you we Sangamo By today, to undertaking obligation in with Actual from at and later from results substantially no one date comments today, may we discussed valid. differ what our still should a today assume are that filed statements are Exchange Commission, the our to that subject and on Securities annual These are specifically, assumptions XX-K Form XX-Q. report reports guarantees company not are quarterly detailed performance documents and with and risks, Form the our uncertainties future and certain in on of the that as no except date, under stated forward-looking this and are information, such of update undertakes made Sangamo as law. to The today applicable statements required duty With several President Officer; and management, President members Rebar, Chief Turner, Medical Technology me Ed Officer; Executive Executive Chief Boissel, General Ed and of call Strategy; Kathy Financial Officer; Officer; are this Sandy Macrae, Counsel. senior Conner, including: Chief Sangamo of Chief Vice afternoon Corporate Stéphane Vice on this Heather Yi, Senior again, And the during we refer to presentation a call. slide our the Presentation of page website. + and section found Events Those slides to of on be are the Investors Media call over to Sandy. like I'd now And to

Alexander Macrae

Thank you, everyone. McDavid. Good afternoon,

biosciences from MPS X steps the studies successfully finger taking diseases; a symptoms II development areas: And potential by expertise CAR-Treg I strategy gene with to company CNS the transcription we investing inherited in medicines MPS for lives. remarkably company, way. medicines We've how to the to new As is genomic year. we the our now on CNS development and is have TxCell, in to fortune the and have autoimmune this we year the the zinc diseases. patients' start some to to clinical portfolios our we have execute We're the Sangamo with of is of the we I've taken diligent time our to Sangamo and proprietary program the shifting through important became approach final far year. a our protein treating is different transform medicines, are of experience, regulatory acquisition diseases. of Sangamo immunological develop reflect towards the therapies and to on our pursuing technology XXXX. field clinical the clinical progress therapeutic lead We're for Genomic field our of immunology. disease from factor weeks Fabry conditions; the having transformation IND-enabling at therapies disrupt emphasis organization. programs genomic of a in a and potential moving metabolic advancing clinic. and approach At T-cell novel cell the focused XXXX to cures. of for skills Sangamo, development drug healthcare regulation the compared lead to made come

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in a pipeline II others, assets our a the in genome same using so-called for I or vector for using editing vivo you and most B. Success MPS will platform in approach other implications platform. positive five the hemophilia are is vivo on gene particular, MPS programs. clinical technical albumin have important with technologies. editing We progress same and hemophilia hemophilia success across share approach we within and have employed we're Today, for MPS same the in for I probability using locus for for The therapy while will A I lead genome AAV focus programs. new hemophilia II, from through will II A these MPS advanced Conner the MPS of to of Ed B,

Regarding studies forward path trial. MPS and to MPS II, Safety two clinical October the Monitoring released I each appropriate the Committees independent for convened discuss

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our call, Ed Officer, Conner, Later CEO the I'm introduce for us over to acquisition prior Sangamo to and pleased who development. in from Sangamo other update who the build share allows SB-XXX, was provide medicines details We Chief hear and company. the it Stéphane CFO, finance, corporate are Boissel, business XX experience him genomic of into accelerate first, of premier and joined third in across years asked And of clinical in President Stéphane patient Stéphane? you'll as will recommendation strategic vision TxCell very from Medical a realize for Kathy us this Executive the our help quarter. will with Vice to recruitment as pleased and the their of brings strategy financial progress But Strategy. programs; who has Corporate to Yi, study. I've

Stéphane Boissel

excited team. Sandy, you, very join I'm the to thank Well,

CEO landscape genomic as past TxCell, few the closely. of the years I medicines Over studied

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X Our possible. i.e. possible patients the many goal of as a in therapeutic to as leading global organization, disease, eventually technology as is elected to extend pipeline medicine also become one order to strong of therapeutic autoimmunity, arrestable build genomic potential development assets but and franchise inherited CNS each many under a to in the as our in diseases metabolic of treat

combination elected Our both outside for a of partnering, pragmatic this medicine strategic be therapeutic to to for and focus. partner both internal will the franchise going With we the of respect future approach and pursue is field program. of

will of develop our our is our maximize partnering And party to are when when of consider to our value believe only position for them come when technology plan future program third a and medicine in are so shareholder assets. we in commercialization. acquire us or will to ancillary out will technologies reach to also we we to There continue and accelerate patients. believe us potential the efficient. patient a effort, better genomic than more maximize delivery finally, doing to that to and continue we those to will We In that short, the or is

and this I'll you, for be and a to and a Ed stakeholders, a new payors One with such that, Our field to potential, serve have will for I'll genomic therapy course, including medicine new one a that, turn model. With of update? patient, clinical appropriate all commercial physicians. large is thank

Edward Conner

on going earlier in Thank hello, and our review hemophilia clinical independent the for everyone. data Sandy is progress update efficacy programs. you, update A. X on II dashboard accumulated I SB-XXX Alta with SB-XXX the In for subjects study from met pipeline program on safety SMC trials, date. program provided for our this focus begin other Stéphane. The to And MPS I'll the XX. from and therapy the I'm an to MPS Slide gene October, enrolled and to the

tolerated of courses oral use tapering continues serious steroids. adverse events be review, of As with no no treatment-related well of that SB-XXX generally to and

study continue to escalation, in Importantly, an the step. see we is The continue right agree additional recommended on the dose with strongly efficacy levels also the factor dose-dependent that we serum. SMC which next therapeutic

the For patients the product profile that to the A. continued that factor of excited of pivotal suits an with competition, we therapy. dose we the develop dose optimal immune a risk. gene data do needs allows A would trial, with is the and to low not balances have field safety hemophilia that find We're we for presented therapy hemophilia want as yet SB-XXX and want an that profile others for profile gene best the This escalation safety levels appropriate that support believe highest

the to safety the progressing We the only and and Pfizer has clinical this study potential and effect far. Alta phase. among dose novel the gene present the now We is Accordingly, I'm data therapeutic positioned with to efficacy be dose-dependent with made well month to for and is after observed lack a very plan continued in that the development. very this submit late-breaking encouraged hemophilia escalation therapies year believe safety, abstract the last the SB-XXX, to other decision to not expansion cohort of escalation, is dose a immunogenicity so trial SB-XXX A approach complete from we ASH. For

eligible of in and XX, on that Regarding And this in Monitoring the AAV locus has expect patients editing our now vg/kg dose directly third ZFN will right genome patient study. and Indeed, efficacy II, program data Committee pleased our safety as identified for to patients trials the study. available the determined B SB-FIX be and albumin SB-FIX dose, dose other into is step MPS clinical hemophilia and cohort I around Phase the forward used from team soon. progress MPS Slide announce XeXX very hemophilia turning B tremendous programs. other enrollment vivo made Safety for clinical for I'm program at reviewed the and the our with the highest the I/II this X enrolling to We've dose

I, on greater was result to which today, As move encouraging dose to us Factor dose based to allows IX of clotting profile the higher go with observed potential levels. a the to safety SB-FIX to has high MPS the that rationale in

sites in quarter with initiated updates we've the the third and the on made to patients SB-FIX hemophilia and providing for those U.K. in enrollment at Additionally, program are for further screening I'm forward clinical calls. B progress subsequent the encouraged about team pace look program the of this actively sites.

now Turning gene is company. therapy Bioverativ, cell ST-XXX a of to been program Slide collaboration Sanofi on which beta-thalassemia developed edited our with for XX,

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proceeding to Regarding and Bioverativ the activities clinical expect year. site first BIVV-XXX for startup this sickle initiate cell are disease,

in participating has solid programs disease accelerated. TXXXX strong pipeline Patients and transplant. about for track clinical for studies, therapy now gene for our clinical our Patient both, trials. on With ST-XXX, in remain all Phase in recruitment excited we established on XXXX. across recruitment our program vivo our updates brief active I/II a trial genome organ our very Fabry we've CAR-Treg are Finally, editing for and initiations of

X hemophilia TXXXX. X soon, have I And clinic sickle disease beta-thalassemia. hemophilia II, have MPS disease, and the cell We studies MPS and Fabry and A heading and including into B, we ongoing more

clinical to therapies. Sangamo. incredible addition we a details to development financial we results programs, look In are about now review. an the time for and allogeneic oncology from immune at to doing Kite cell next-generation for call turn these is forward with the Kathy? I'll also providing advance This Kathy to updates work

Kathy Yi

our third which current $XXX And press cash cash, are of investments. cash in includes good XX. the Thank The everyone. with on $XXX the we're you, quarter guidance We this detailed quarter afternoon, summarized earlier issued year-end Ed, and release million. previous we ended maintaining results equivalents XXXX, Slide afternoon of financial million for

simple the shares, to As of of TxCell. purchase ordinary stock the a shares. October remaining X, November ordinary first offer we of tender TxCell of share X, XX% completed the represented rights we on successfully And launched which acquire

Since to expect have CAR-T autoimmune the editing next to cell We stock we before tender through forward of offer therapy into year of this operations exchange. the of our TxCell the integrating French started technology. Sangamo end release from the complete for the diseases generation beginning to and and look gene TxCell the October, developing synergy ZFN

general mainly agreement, by XXXX. and was net expense The transaction is year. for increase of period expect revenue the per million $XX.X $X we expenses, million was of including $XXX quarter pipeline diluted, basic be of for and clinic. third which to XXXX increase year Kite acquisition. our of the and the previous well million from research move to $XX.X to in in of same clinical primarily trial $XX.X million, share, This $XX.X Revenue reflects acquisition. expense the share, $XX.X for or TxCell quarter the programs further from support increase $XXX an clinical the loss was $XX.X period of with driven $X.X million G&A for versus $X.XX the XXXX diluted, same associated due $X.XX the $XX.X onetime or in XXXX to of in and of as is third R&D operating basic the total increase growth is prior third of The operating inclusive million of G&A million, million, including by as million per range compared costs into we year-end, million the services. in as the cost and At TxCell XXXX collaboration of our Our reimbursement quarter cost cost mainly Research manufacturing and The $XX development million. million driven

in X final trial MPS strong progress. extensive hemophilia that, and comment, years, pivotal We runway to preparation B continued pipeline our advance investments genomic in account forward a approximately to call into very position remarks. we our cash with looking assets expect the medicines, reporting and autoimmune our And a we our pipeline for Sandy for much expansion As continue are to turn to of closing the for taking in and I'll further financial our over be readiness. to believe we're of lead

Alexander Macrae

Kathy. you, Thank

the and I we've questions, this beginning clinical over time solidify unrivaled Before exciting people, therapy an companies. investments conclude made we clinical forward believe and and as in in very an turn what early be company. the we much editing among which look is saying to of Sangamo XXXX critical efforts to in leading results call to last years, I'll the our I see we biosciences development, beyond. as into gene to and medicines we transformation in data focus from of by technology In establish at these the genomic pipeline, are a gene X our now flow

And Operator? so we'll to turn now questions. your

Operator

[Operator Instructions].

question with first comes Qian Merrill Our Bank Lynch. of America from Wang

Qian Wang

a I I So if couple, may. have

II The the program. actually first is MPS question on

So dose a the dose safety? expansion it data or what GAG of they you with just is on cohort? kind also certain biopsy the clarify decisions -- reduction, plasma, Do mostly have so like And for enzyme, do? it's Is Could they the on just curious please see efficacy? what far consideration that? these seen on committee? from And

Alexander Macrae

available and available benefit But they fair very question, based see and is efficacy could I a and go. development the safety decision for a that's they answer at I make the see should product it and whatever the they So time risk of that where then wish all the more data completely. data on

until then, parts And to We on the equation. we're will individual sometime share broader of the community the not results comment XXXX. in able with

Qian Wang

patients protocol recommend that on guidance they just Does the and prepared remarks. and appropriate the Got still I'm curious from under depending it. the what during patients? ERT means? taking mean the on said and that really your off in that that's And you physicians consent

Alexander Macrae

makes each I they what way always the a patients just they about reassurance, will guidance regularly have for added how with own patient it their everyone the the ERT. recommendation particular there very their They wasn't various stop it's protocol with and this investigators, a seen we we pleased be to across are few discussion investigators. them is But That study the way. a SMC talk and that when there's a under so each data it's the one collected. between So small talk -- far. doing then, of that give standardized very

Qian Wang

And lastly, if I may.

the I A on not lot are is see. really November say X, It's to think On a investors to of because didn't for decided which hemophilia an see submit you we clear now expecting abstract you LBA. that

given curious decision? the durability Can FDA with is normal the than range mean, or patient? six you please? rather patients what don't driving clarify, of I you about thinking level just it were Is similar that of guidance, Just Or the see you expression the a XX%? the

Alexander Macrae

deal one, please? you can with this Ed,

Edward Conner

as is, seen that of are had and with excited to in And We us any we're we where again take the drug-related earlier factor on to who about Sure, patients steroids. to levels escalate. tapering We've dose we have I that before, know safety courses very see seen no were a allows haven't continuing yes, the range. call, we're talking terms are. reminder, it as so about, earlier safety as with that of items said, we And just as SAE's. in our you therapeutic talked being

approval with trial. range. that, -- when factor given of to with the and guidance, want the partner is forward FDA are in are that at with normal we that recent data discussions do we've Pfizer, we they're our release in be the the when going that pivotal possibility our recognize levels accelerated That would levels So with we with our

Qian Wang

Is that you're that fair to aiming normal for say right range now?

Alexander Macrae

but and will to the right good us and decision data safety. III a to it time it's isn't efficacy data to possible. the most We looking Phase give make want the we're patients important as for end, III release as allow into that to in a get No, balance of because collect to and into Phase quickly get the go patient-by-patient to to mature, thing also

Operator

. Our comes next Gena Wang from Barclays with Bank question

Gena Wang

the prior think the basically question. line, similar follow I I'll

higher? X? So dose regarding And what of you II, wondering did in the makes to see SAE Cohort any MPS expansion decided you just instead dose

Alexander Macrae

us decision, -- any continue Cohort haven't at we So the we that's see you we and efficacy so expansion very asking And we of to and -- as if cohort, third next we the for SAE's hope view continue safety X available for The level. will in data. well, beginning cohorts, X the look treated data SMC of more holistic only you present allowed this the they the in protocol XeXX remember It's and seriously. is and more patients each give X, and so because at to seen the XeXX there's safety year. thank cohort a an then,

Gena Wang

Okay.

also So patients how enroll? you higher thinking be are planning for based many step is dose expansion then efficacy? you will the a there to And more on to the then, that cohort,

Alexander Macrae

will So we dose of and will -- we almost are to a higher it is the make right logistics. go said, similar needs interviews are on I dose the a SMC them And simultaneously likely -- whether regular to dose The review X basis. and we a this just a matter decision to it able or data can whether level. another we then it as patients level would

Gena Wang

And then Sorry. Okay. --

Edward Conner

there was SAE, so but an clarify to that. treatment cohort, not wanted that clarifying third one it's just related, thing, I just

Gena Wang

Okay.

sharing us another hemophilia Okay. is And regarding with the wondering, know what MPS share you the doses just for is dose? like programs. the reason just I doses? cohort wondering not And A, first the A question what's then, hemophilia

Alexander Macrae

both much that we're a product. off to most the is hemophilia the we've having balance our this product. allows And in pleased steroids, no product long-term X allows fact us more safety that so think, the position the efficacy to competitive companies themselves optimal and we and of safety to trying each tapering A why And to that's to, space competitive doses it's have the the escalate level. dose as us Because had

Gena Wang

Okay.

Just one question. last

be will and dose for what hemophilia bar escalation A for the So move phase? expansion then to you stop to

Alexander Macrae

That III would over and will agree study take who the SMC advise will that partner, and be our together. for will on, something the we Pfizer, Phase

Operator

Wells Our with next question comes Fargo. from James Birchenough

James Birchenough

clinical all momentum. the on Congrats

MPS came So not the know we II initially maybe I reductions, the when thought you blood enzyme. with saw out urinary was -- be on the any there really look data might and compartment enzyme for but impressive not GAG the that the level right place to

just And us that have And give your so could wondering interrogated with thought experience, still Or on you other updated maybe compartments? view? is additional you that? an

Alexander Macrae

it. an So consistent in any that rightly, and in changes talk an in assay give of was and able sensitivity that be last coming about being very really been and the measuring enzyme And at I taken enzyme hopefully we still there it you it tissues ways pleased year, Cohort things few in editing. similar was GAGs early am weren't will new the was and effect, and X. was there months report The we effect, next more on from an place that to have because was team a the was that. to and IDS. the we idea the them think in saw about year, more to done released there and a look very why And report holistic this, We as remarkable the with quickly enzyme, GAG impressive what when past able say liver reductions of we discussed up the that in by increasing talked must've we're

GAG yourself our talking of like And felt it we is happened. analysts as year of to we're need have other puzzle as that and having closely, followed us are possible followed story do others was understood who will the come haven't there you that many Our those Jim, next and and as will that the representative editing allow experience that have, and as closely to understand pieces effect is on hold the pieces with that effect. so the of what others you back try realize

James Birchenough

That's very helpful, Ed. a question then, for And Sandy. maybe,

to on but potential already gene for seen vivo as not is aren't therapy lack as we gene self-renewing. transducing Just durability disease are actually, ex pure in stem beta-thalassemia explanation look general, one of and we've transducing differentiated the the of any ahead cells program cells or a sickle and cell of things that effect editing,

to transduces speak the the you becoming cells and important. it better using whether vector than think I think you're can So you right Because that's others?

Edward Conner

pleased the So transduce with Rebar we our who electroporation yields other we're the are previously, using data seen, and We've that but of CDXX we're and on edited has the Ed stem to as Ed presented mRNA. very percent with cell that cells getting well.

James Birchenough

helpful. That's

Edward Conner

if to that? you to want Ed And add

Edward Rebar

question pertained delivery, a comment modification is to I additional your occurs, a bit X into little Perhaps, to types people as a bit I of occur opposed less of be cell that's have knockout, that -- but a And NHEJ of I HDR-type that think those including which play, optimally there's that too. harder the does to say. that okay, get doing, cells that add type occur processes, to think, -- type little are something will that know in that things if whole generally modification modification in seems go come to we are set which is your fine to -- more let's recognized NHEJ-mediated

James Birchenough

a get And showing will I a on you a what Maybe A. profile in when seeing is on competitive what true the well? that consideration you helpful. as asked competitive the get profile part is? ASH hemophilia think I landscape just think past competitive in waiting think on get to better question be? what that's we'll it's be you yet Do clarity? guess, of Do we've data at determined. final and to are set, to the expect others So fuller

Edward Conner

other would of Yes, their the cohort no. the data, this any data say is, area. aren't this folks or the aren't Because either presenting so competitiveness I answer reflecting short presenting high --

James Birchenough

fuller a the is? sense a feel a feel proper like the of is benchmark to there have you like profile? have data look you benchmark to set, Do As competitive do clear you good what we

Edward Conner

is from I be feel you're standpoint critical immune I not benchmark. on But having significant just said reaction. there monitoring the we overlook may call, a associated like can't that of the and safety. as take Safety last if but pretty steroids to is the having

the it's really of X. So combination the

Operator

Our of Baral Ritu next Cowen. question comes from

Ritu Baral

-- going question XeXX, there on as us differently that is XXX X the and believe deficient the differential the in far same two lead GAG PK to the in more diseases the specifically, enzymes dose, the should is doses XXX, XXX My PD in any dosed as enzymes conditions at reductions? that or be should of XXX. the two as is to work

Alexander Macrae

that's a that level of the to yet. think this hasn't clinical around I sophistication got signs

them in are it's understand able we similarly So through only and we'll that be there this experiment subtle to from the differences are whether doing treating PK/PD these.

what compelling on from to the is II we take as is and in possible. delighted call, have when the all forward in and the have SMC highest to we decision want cohort We safe and were the because we GAG showing much together MPS said story. world As the patients use and said look patients with the enzyme to to what from us the X hopefully, data a, reduction they've the as Cohort we made data the collected in I sensible to dose one I the MPS

Ritu Baral

program. your to then, XXX, moving And A hemophilia

on unveil time? until to is durability followed What of of amount good time? Just think data therapies? And give XXX? for amount you therapies, the you focus had the you a of especially of gene complete of durability of idea is set these gene you time. You mentioned a a -- What didn't meaningful that patients that your will meaningful want the right that of data AV amount do when given

Edward Conner

that of toxic and just to the even stability commenting we're you the we're perspective, to high but I a it's the again, So what at durability if up not on overall range. up, And see the they course, terms patient of long-term sometimes factor up factor looking I in not levels. mean, varying at, not stay go it want look they're think levels from

at looking really the It's how and curve of really the at day. whole are it's performing factor so month-to-month. consistency the week-to-week, the end understanding And levels

Ritu Baral

a some you And that's you your an across but seen reflection seen that, can love mean, comment response that's drug I sort that itself? a ability of of across of obviously, whether you inconsistency to on it. think other to Or think seen? have answer dose? the that a we've consistency selection? Got AV Whether function just the pattern barring of disease you've And cohorts that comment rare for within patient therapies, for you response on we then patients cohort. I'd of

Alexander Macrae

are all patients to thinking Can But are treat with each from little we other. I isn't and a points anything certain this. about screen physician, I that our knowing was it, it consistency. and a if and think patient give because a the our and about and within a meaningful, I'm have with focusing stays And so appropriate have worry time, that the able if I factor within-a-cohort I the view construct, that share long them about number. about safety, number. of It's go want be ways But knowing a if certain data the about medicine patient this factor at somewhat could the don't of think in meaningful the for and obsessing a I try have I'd And are There so a the few medicine it's the back of for to speak years. see different a predict wouldn't I to predictability those that's steroids. was It's between more it a possible a and and than -- studies what dose courses safe on consistency. patient, and that predictability study once to they we your to I because a important as then, pull knowing and of for level that period getting different was patient,

Ritu Baral

question. And it. last Got then

competitive the right you Obviously, space at now? beta-thal look in to how have bluebird. -- Just

bring other unmet address you moving to do in mechanism to some the couldn't? therapies have others the table forward. You think you can need beta-thals alternative What that

Edward Conner

going first. Yes, I was say to

we've about The so for different seen. and, patients off-targets, very I end approaches. potential, am help think view, are Why companies is approach excited exciting that many our no editing it's the robust of I I so level many mean, process, my detectable that so manufacturing a product to our least all day, I of in view, greatest lead in the the to think, the at my has of those at trying patients.

I'm look precision are -- our doing looking specificity only doing products we out the approaches long-term that in at they're of some provide we're editing excited that other precise enhancer the contrast of will, the where about as for not think, I that of to and but integration, think to others I we're also safety compared very long-term, approach there. erythroid-specific the the random currently

Operator

[Operator Instructions].

from comes question next Ijem with Whitney Guggenheim. Our

Whitney Ijem

one preclinical could product your fourth cohorts is I potential that to you're or guess, work, A. beyond is to hemophilia of with get is additional Regardless there on it profile, there on that? the of expectation in you your targeting, go to First terms what dose based cohort have

Alexander Macrae

at and will SMC. cohort the present one data the and living that safety are a We efficacy and time we to it gather

Whitney Ijem

Okay. And spoke assay. measure then enzyme about new ways developing II, MPS to for more looking of in sensitive kind for you

those in And missed the are sorry SMC has practice now data? that this, any but any if I of of seen and

Alexander Macrae

not process ready, is commenting to be well. on assay will working improving on you are of that give We they but I want assurance that, when the

Whitney Ijem

XXXX? we coming kind in can or similar last from just or look of could of one, year of whether programs as in how you materiality in disclosures timeline the think just of changes to Okay. then, about not so, as and us up additional clinical next about sort kind data other we see additional we understand And think you updates updates the help

Alexander Macrae

because MPS thought II as data. accountable share reduction. as data here think that really story was we -- we complete rather excitement to the what to groundbreaking from learned I is this, possible our than when level It's I'm useful data my the present most for excitement, the showed the GAG

everyone I will helps as, so has call this benefit story think from conference we patients. you it that to you'll see and understand a present when And find will it's the think I and complete data this

Operator

with comes Our Morgan. Joseph Eric JP from question next

Eric Joseph

get just really I one. of is liver biopsy had utility your in wanted on pieces editing type biopsy question in on willing whether the earlier filling to the MPS of some undergo, for updating taking program thoughts I you're something determining of up that's follow of data and II patients, place on and the on and year? the the whether when it of that table are the were, as program? And one, something is that is to whether, puzzle, next liver sort

Alexander Macrae

and choose So of have to not in They're particularly will difficult have kind have invasive, to to liver these biopsies them liver patients patients are a do, biopsy. patients. all so

share whatever different that and to that earlier the hope reported data bits in would people help GAGs reduction story all have it construct will a we year. underpin We the with this in we

Operator

Our comes with next question Maury Jefferies. Raycroft from

Maurice Raycroft

two quick. questions. be quick I'll I just have

For get the been yet, we you met clarify any decision MPS -- for the if been in patients? has can SMC wondering whether so and can if II, just ERT wondering withdrawn on I'm -- that comment made if has October.

Alexander Macrae

is solid have present to days be reveal withdrawn, everyone ERT sure We because data so can we want able make was said could as the that we the that. withdrawn, understand that to to we withdrawn, wouldn't earlier be that

Maurice Raycroft

Got it.

then And hemophilia. Okay. for just

that use For any next the dose dose? level, with steroid will you

Edward Conner

same not taken No, prophylactic before steroids. approach that will have be the given patients we are it where

Operator

Instructions]. [Operator

question from Our Wang America. next of Bank Qian of comes

Ritu Baral

follow-up. I quick just a have

it's these that all safety editing programs. profile guys see you gene the So with encouraging

example, you the dose. skip the MPS For of like were one I to able

wanted So I'm doses. three just to because evaluate you curious initially,

will So is that go than they the XeXX? higher possible

Alexander Macrae

the present and whether we needs else of we cohort make asked this available the us all about will data whether for SMC the us is -- have have the that to decision So or the dose them and they'll is right then make doses for the help protocol three to be the expand something done.

Qian Wang

but not I'm talking MPS MPS II? Yes, I,

Alexander Macrae

there, Safety are some Monitoring Committees -- -- same MPS so both. overlap, so there people we the on that are and It's the MPS the

the informative. because very So one aware they they're other what's are happening on and of reassuring

Qian Wang

doses? to can And decided us X remind Yes. choose these just how you do initially, you

Edward Conner

were preclinical with discussion doses the work that of the chosen was of FDA. So and course, that in done based also,

Qian Wang

Yes, FDA, the that is have there a example, and terms heard we that kind noises thinking people dose generally about some of a from high given signal? for feeling in the of are of

Edward Conner

very and very program It dependent dependent. vector is

Operator

of next comes from Our question Birchenough James Fargo. Wells

James Birchenough

a related expansion to wasn't around that, just for experience I not expansion? not to get just I I expansion quickly, II, that to you'll make for cohort reason related? the to cohort SAE event. the sure MPS Just drug cohort more want that want the confirm get adverse the was want it's Sometimes to an confirm that just

Alexander Macrae

Absolutely not.

Edward Conner

Right, it was not.

Operator

I'd I'm turn like any back the the showing to no for further in questions queue. call closing to remarks. Sandy over

Alexander Macrae

their look thank We everyone Bye-bye. for to and forward in interest the again to would like you to year. and new talking support continued

Operator

that gentlemen, does participation for and day. conference. wonderful Thank today's a conclude and Ladies you have

all may disconnect. You

Sangamo Therapeutics (SGMO) 8 Nov 182018 Q3 Earnings call transcript