- Earnings call transcript Q3 2022

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good day, and thank you for standing by. Welcome to the Sangamo Therapeutics Third Quarter Earnings Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that this call is being recorded. it Investor Louise Relations. turn Corporate Head now and Communications will over Wilkie, to of I Please ahead. go

Louise Wilkie

we to Good afternoon. joining I'm several Sandy Investor call Relations Macrae, Louise On members Wilke, Sangamo's Investors our Media and include, Corporate today. therapeutic page. leadership supplemented Thank fiscal Vice anticipated statements the Form materially regarding risks specifically the year clinical the and of advancement Communications. our differ Chief Sangamo you under statements may angamo.com, in filings and team, SEC, today's and XXXX. and our call clinical forward-looking forward-looking other initiating and for our stated quarterly required this time current of to candidates that for by update except us today. in executive operating the and non-GAAP on on this statements website. XXXX, discuss the are The are Events plans collaborators President GAAP December These XX, Sangamo's we including quarter is on of and and the patients for Cockroft, September we of Slides expenses Executive to McClung, but conducting of Presentations historical Chief XX, Chief this discussed Financial our on call, Chief Sangamo are statements on Fontenot, Jason product Nathalie can website, our commercial report operating Officer; made on this investment with product upcoming date, no expenses. such fiscal Operating Dubois-Stringfellow, call catalysts information, Actual statements advanced undertake Chief are and be the Officer. Mark release, as our ended our the screening Scientific and right duty Officer; Development Chief uncertainties as includes XXXX corporate what to measure are our presenting in & our relating presentation and as section Bettina certain results of available by -- report today preclinical and sufficiency that subject the with annual not A the XX-K Officer; On from These law. programs ended our of lines discussed focus are press Officer; clinic, the Prathyusha found candidates, Medical guidance, Officer; of which limited for our to financial reconciliation our data, be found Duraibabu, expectations. trials, resources, dosing potential our can This from statements facts. XX-Q not to, Form

our I'd to to turn Sandy Macrae. Now, the like CEO, over call

Sandy Macrae

innovation the to science medicines of for turning groundbreaking scientific am Thank potentially continuing Sangamo the you, data our on afternoon and the genomic programs across call. our the demonstrate our to clinical transformative momentum pleased we into achieving with strength third are to pipeline. with Louis, continue we everyone patients. I as our development progressed pursue excellence quarter in good and mission of

Regarding program, last the our conferences preliminary to were wholly-owned I/II our Phase call. additional excited study medical since X Fabry we present disease separate at data STAAR from

last patients, date, favorable dose the rather lifetime begun data alternative study. safety in of to a we've promising our for showing by female onetime These expansion moved provide a first ERT dosing care that was July. demonstrate cut continue share generated Alpha this with profile I'm of and the replace to study current than also of infusions. has to potential the the and patients therapy pleased dosing standard along this the data the including phase, data into Gale We encouraged to potential the and be activity in sustained the elevated of

the We improved make I/II manufactured PRECISION in to dosed patient sixth This owned program quarter, in is the sickle methods. cell patient continue study. candidate the our dosing Phase using product our to second progress wholly this be with

the or successfully behind In transplantation. RMAT advanced study patient FDA medicine our HLA-AX post-infusion. sickle Momentum reached mismatched candidate. TXXXX the late I/II cell the granted Phase product to has in September, patient that second therapy our first has to pleased of in designation regenerative continues am I while steadfast kidney addition, was announce X-month dosed

XX us of important while to APINE resumed transformative staying closer step September proud I'd significant do A it potential our of medicines value for team like sites III with who strategic to advancements clinical discuss trial incremental we hemophilia and our announced data brings of Natalie? of continue Natalie, that our in treatment dose the the the to area over true the we our in shortly. closer goal unmet creating The to the in an our medical expect from Each in to turn our milestone for resume programs for recruitment. research Trial with study partner, of call resumption jointly update Lastly, and patients to as detail. make, Pfizer development capabilities. will shareholders. on creating Development, had long-term I'm is on Head more reopened focus enrollment Phase patients to getting this one us need. takes this an Pfizer, to September

Nathalie Dubois-Stringfellow

note, also additional Israel currently the Society candidate have from Congress study dose We phase, data disease. ERT final gene dose the of MIM the inborn more. above patients. STAR exhibited first X in X experiencing alpha-gal Thank this nearly Momentum safety dosing, were more after on Friberg, for across marked changes Phase from I/II escalation in after at are higher screening of elevated at treated first been presenting ranging call, At in report And X patients. we XXX follow-up presenting provided All afternoon Phase our Annual preliminary study. the the the the receiving the in D.C. in Fabry level, the to and follow-up organization SSIEM of for our required see license the pretreatment in of dose the we monitoring remain as this of Fabry of as not activity In including was across and not of fifth in has sustained patient nor subjects the reduction to presented evaluating presented patients Meeting metabolism, for you, February, with we XXth Edinburgh, X In patients levels good importantly, including patient being with effect normal station from progress Xth progress pleased post Also cohort Since the substantially BX cutoff call. and therapy clinical to twofold All ERT a activity everyone in elevation baseline the patients both This years have these time. month, plasma updates for to event, level, the have ERP. updated We alpha-gal had phase committee up Society continue several of and and further X owned as of treated patients lyso-GbX disorders phase. The X at the weeks who dose X phase, for July ERT. the alpha-gal all expansion lines present data the XX a in of CEVA XX-fold replacement fifth in time study, alpha in symptoms of data program. Study in enzyme or be expansion they medical normal All on clinical and dose ST-XXX, as year product XXXX, patients elevated proud almost dosing. study XX, to withdrawal to withdrawal. weeks activity durability Germany, the rare Washington, undergone longest from patients quarter continue we last conference August, Scotland errors assumption February X Fabry data of made update to ST-XXX escalation elevated show Annual ESGCT female long-term awaiting national Cell female withdrawn dosing. at conferences. for treatment from respectively, to and by are and with Gene the been of as XX% Last on date also the dosed exhibited only After maintain withdrawn European the data cut. endorsement subjects Sandy, the the patients encouraged X started in data of Parvovec, XX% third maintain well III XX Therapy, in or promising therapy to X

than higher subjects X been -- treated well were serious operated generally one Importantly, adverse with ST-XXX adverse treated for continued or additional additional been Of in treatment-related have with X, to no per patients treatment-related event the no sites. last X have Since in note, the first reported. grade except Asia and Axia no requiring site our events global have be call, in opened, and new steroids, treatment. Pacific, prophylactically actively no this been liver either including enzyme there reactively study we're preparing elevation grade

clinical first half first the XXXX. including study, the of data STAR expansion further the update from the You cohort can in expect from

cell shown disease, a the Phase second therapy a with We in improved of candidate dosed are method have of now advocacy continue I/II zinc Sangamo, patient This III X experiment wholly number sickle to authority, the product. group in potential cell manufactured long-term been for and is In increase to candidate patients. patient for nuclease the a progenitor health final owned study the plan internal cells product who we gene the of with the dosed finger engaging Phase using by BIVVXXX, edited study actively investigators. treatment PRECISION

to look continue providing progress forward time. to to pleased and in appropriate update are making study an Phase at We I/II additional be the

expositions, Sangamo's Medicine The Therapy, designation clinical therapy clinical earlier, for our Holaniana, therapy poster with Regenerative a were and of has participate medical place reverse intended look MI potential presentation medicine the August XXXX. granted is to cure designation Annual to granted retinal programs, RMAT a to including preliminary need. includes then. in medicine New designation condition at BIVV or to all updated request an the breakthrough upcoming data track fast We interaction As address FDA and indicates Sandy American benefit evidence RMAT forward X taking for December XX, ASH for accepted Hematology Furthermore, or designation unmet which to in or modify, the -- Meeting of presenting mentioned that FDA. the on the XX, treat, serious XX, Society we Advanced this early

a from in cell CAR-Treg rejection therapy study, OE-ownTXXXX donor. Finally, in candidate Phase Phase the HLA-AX manufacturing our mismatch the kidney in enabling and transplantation CETP of Progress progress. continued are for study living immune-mediated activities design, readiness III prevention I/II

late the The second in product to continues have pleased in been both to candidate To patients and study. be in patient was demonstrating report dosed terated that and September, transplanted enrolled ongoing this control interest patients. well I'm generally

We will date. dosing scheduled, on for provide further of confirmed have when guidance third the the potential patient and transplant we has a been dosing timing

the gene resume trial, of the Phase we expected A trial fitelparvovec is the jointly hemophilia is with half that's Pfizer, regarding for and resume pivotal to AFFINE Trial sites Finally, first September III readout partner, in has A, shortly. XXXX. expected investigational therapy giroctocogene enrollment evaluating in our dosing an announced recruitment. reopened

Phase accepted a the on providing I/II at XX turn look call for we've to our In forward now Pfizer, study. presentation XXXX, Officer will sharing research December. Jason? ASH, program. updates to We our updated to in our I partner, updated over XX, preclinical Scientific been poster with ALTA the participate data clinical in December on data to Chief addition

Jason Fontenot

report platforms partner genomic everyone. to and pleased the Natalie, Sangamo's cell clinic. leverage towards cutting-edge to afternoon, advance wholly good continue programs and owned Thank that therapy you, both I'm to we engineering and

programs to progress Sangamo expanding preclinical remarkable well these genomic as team proprietary regulatory zinc developing as enable making also tools. relevant therapeutically and more effective Society proprietary and Conference, donor work European T the Cell our and nuclease use Therapy are platform both engineering We of presentations cells had year's The paces. allogeneic, capsids in healthy engineer toolkit to finger novel AAV of of this our describing Annual our multiple autologous delivery at Gene including

their International expressed specifically Additionally, at were Germany the science performed our September. data to opinion future. to ability of extend mouse factors look this at finger studies feedback in Institute model We in the hear thrilled excitement prion now and We to overview prion in our an turn the Conference in brain were in protein zinc updates the preclinical survival XXXX Officer, at We for of providing disease. key potential engineered for of financial from need. I Prathyusha, the Sangamo's Broad programs other significantly work the leaders strong This Chief patients conference. demonstrated reduce and and protein results. over Institute on transcription forward this to collaborators expression sushi? the a with with from call presented the the Building in Technologies will its Financial Prion of the Massachusetts the

Prathyusha Duraibabu

and Thank everyone. you, afternoon, good Jason,

in million results decline Our this from are release a in of which quarter speaks markets found both company. detailed quarter capital in and net of aftermarket for cash interest the the approach proactive since available beginning year. cash, which quarter. press this balanced This raised $XX on proceeds ended in the million investor securities, equivalents We with reflects the under offering our be prior program $XX approximately and afternoon, We financial prudent uncertain raises can and net approximately management our $XXX website. issued continued to capital these in marketable to the the represents our million our

X cell of TXXXX, and our continue of cotriand progression We in-house preclinical of focus our in CNS key our and to advancement Fabry, manufacturing pipeline clinical including capabilities. areas: optimization programs, sickle our investments

Turning year our to guidance. XXXX full

This approximately year full stock-based than our land $XX between $XXX $XXX be to expenses expect previously range million. We and compensation non-GAAP million. to operating of million expense noncash estimated excludes lower guided

I/II programs, to in T sickle We planning portion for activity loves indications operating continued Sandy genome our be expenses cell Phase TXXXX I of remarks. call Phase advancement including for closing back and the the of engineering preclinical CAR central Fabry disease III invested regs expect in now a our activities, and and turn significant lead to work system. in the will

Sandy Macrae

Thank you, Prathyusha.

clinical are we quarter our during achievements preclinical across pipeline. We have progress and pleased with the made this our and

to genomic our therapies mission. of a are our worked transformative in Singen clinical step out company, our I'm so here value need us advance shareholders. science, carry patients for long-term team clinical-stage proud programs in-house and novel delivering and with takes manufacturing. one momentum progress medicine to closer quarter We tirelessly The building at execution this creating while who potentially for our

with cell Phase updated updating in I/II ASH the several delighted and presentation are We to presentation term momentum as updated at the Phase in A. data positive with in forward the you milestones, expected including near hemophilia sickle disease, and data look of of I/II

phase first Phase in this of questions. please patient open the we up for half like including data third Fabry dosing XXXX, I/II first ERT of data Additional Phase in withdrawal the to would questions. line the the open additional data for At I/II and and from Operator, the Steadfast study. the expansion time,

Operator

Barclays. from will Thank question-and-answer first a you. At with Instructions] conduct this session. comes question we [Operator Our Gena Wang time,

Gena Wang

I have X questions.

first cell The all sickle today. ASH the we one the earlier is released and the at abstract saw

a a off like seems cut it So placeholder, May data X.

And is the patients program. my we then the presentation, more second wondering see Fabry ASH? at at full X Just data question

be half data will move sharing? be type your current patient like will X-patient next patients? what Or of X license, kind hear will of to biomarker expansion on you more Would clinical the inroad the data the you that to we see step. data data profile what Alphagan goal be the mainly data? also, regarding first beyond the those the So in 'XX, cohort And

Sandy Macrae

for your you thank Gena, question.

with question So asking as and a a I understand, Fabry. about question you're sickle

February going then I'm to Natalie? So Natalie to over cover and will Bettina sickle pass question. the

Nathalie Dubois-Stringfellow

patient product was improved So the the to the process. manufacturing was candidate receive trial dosed it XXXX, patient X May and a in in first using

this an of preliminary update patient first poster via expect We presentation, share in dose. from with ASH patient at the along December early data to

Jason Fontenot

you Fabry. Gena, question for the thank So around

cohort. as seen, treated So we an care. you we of we're to the which it potential have to means now in alternative CXXX to current X at study, dose X expansion patients math, patients highest Fabry actually That in believe of level. has are sharing -- very the We patients brings the of total to dosed you have a we the have do XX standard the us do excited be provide if dosed that the

will the biomarker out half, the XXXX data additional of will and clinical patients, early exact the XXXX. We data which we cohort as first from in the of nature so first data early as dose as from the will in pointed include sharing be in both as escalation half expansion the this from well well additional as data data, share we

Operator

comes from with [ph] RBC. question Our next Luca

Unidentified Analyst

This Perfect. on Two today. Lisa is on par Ziluca. you for

on is data. First the

baby presented follow to data wanted month. Just on ESGCT at last the up

is at least plasma withdrawn had there subsequently the after from patient in I one alpha-activity was increase but the noticed who withdrawal. think an we ERT,

just expect ERT drop withdrawal? I And given we how wondering that after is would on this III the So secondly the response was to you should Phase levels the alpha-gal rationalize planning.

you reiterated this planning rate base is You said -- underway. today a at

Just the what primary would given a gene approach. what wondering need had you've III what be would like, FDA terms with a look and in if could of is you Phase show to endpoint therapy here this the durability discussions on

Sandy Macrae

thank you such our questions, thank presentation. for to your ESGCT for So you and attention paying

a Phase agency, but and that -- of the going help had and neither I'll help time little lot with III to part. over we'll of her -- time. pass a we've study. this to details second the study think spoken We've had Betina, I'm We've share about So the to this to plan I'll the

in the know Bettina, the me details of answer part? forgive can everyone is because that So study. first how I interested you

Bettina Cockroft

so that question. you for Thank much Absolutely.

to is expression in patients time, seeing, ERT -- about that's sustained who be have activity naive our standard were fact, ERT are on we of ERT. or objective from Alphaga, not ERT. who provide And patients the to that care you of withdrawn alternative AlphaGale That asked has expect from We also what but that ERT. patients on ST-XXX therapy. to on and current are an the believe we naive, do only pseudo of withdrawn the So in expression. who over potential

well. plasma your patients happy patients lyso-GbX to and the were I lyso-GbX range you patients addresses ERT. hope are the doing specific The If ERT. the with off data within investigators levels, that referring in are all The on question. plasma remain remains the seen

Sandy Macrae

on the have And to data early the withdrawal is that that we remember are science. the is we've first ERT we seen the We dose cutting-edge you high data with this of Phase dose I clinical be lowest show would this you've hope that patients encourage forward to and gathered treatment, a to all These the with with and withdrawing response. that. into III plan dose, from go ERT withdrawn patients chosen

for been thing think showed therapy, ERT other benefit. added their of long were gene you for the they is who we gene speaking And power in ERT function, cardiac there on that was this of time, sweating a got really stabilization additional better to feeling our even think therapy The even overlooked. who or sometimes I is of to and have medicine. about improvement when then would Patients they reported ask patients on the I this evidence

Operator

next with Truist. Nicole comes Our from Germino question

Unidentified Analyst

on for Alex is This Nicole.

you Sangamo. on does pharma? change? might that For regs this some types are pharma so, or does the thoughts dropping CAR would that you seen in rent And space? the autoimmunity. on Or T partnerships engaging And in color what pave we've IL-X in the this consider the way big consider work related, for platform, could the in other And would collaborations programs news for outlining your CAR-T best with you of provide that some if

Sandy Macrae

group. want our business that? to Would talk Mark, you you run development to

Mark McClung

your for Thanks question. Sure.

company patient making obviously, with we're have with believe excited the we've where Tregs, the dosed first to progress a as earlier. So we we're communicated we're that

resources continue we patients therapy always be study might any they of the is conversations in proof will commitment we've can, if move it and having we're approach that commenting that companies accelerate than Our concept. to that. faster past, forward to what If we accelerate and that current know-how the not the do -- But funding similar with can drive to in actually to to a towards regard. bring us evaluate we patients on done

Unidentified Analyst

-- Have do seen in big And IL-X we've dropping also you on seen on the And you pharmas platform. that? have comments in any this? news some

Sandy Macrae

Jason, -- range amongst you to Greg the would able Scientific of on where IL-X Officer; Jason comment programs? be we in to and our fit

Jason Fontenot

thanks Sandy, question. Thank for and Yes. the you,

in on in the T-reg the T cells IL-X mutein news to think I'm point the and approach systemic a latest that because are the is entire IL-X The the do and case was But is receptor autoimmune forma for regulatory the cells It's of of regulatory can IL-X than oncology, important used of expand that fundamentally biology enhanced offer using the IL-X we're cells disease. program regulatory aware dropping which the a a to patient. it's the to it being on target different cells T and out body approach relevant the whichever to related some IL-X tissue. chimeric that I different of potential to mutein benefit approach an delivery biology. is, T to that relies of taking therapeutic increasing regulatory use with explored while be in car antigen

the is tissue and effector a cells new the directed with despite do the in way very regulation much a that's teams different afford Moreover, kind towards T T regulatory that reactivity where of immune cells. the the cells NK efforts, have pathologies and some potent so of IL-X autoimmune T still specific where And occur. proposition kind best

actually of interpretations by so effectiveness of confounding one any risk the because disease. autoimmune T And runs also is cells, effector hitting those exacerbating this of the

Unidentified Analyst

you, the Jason, Thank for color.

Operator

by for next Please question. stand the

Fargo. next with Our Yanan comes from Zhu question Wells

Yanan Zhu

on cell question a and program. Fabry's sickle on question A

the mentioned has since who cutoff But data nobody early discontinued earlier. 'XX patients think And So this resumed increased to data the off. alluded that you that been it's I It XXXX, fabless rather and since like think months looks is you levels presentation, dry few ERT. cut a lyso-GbX I ERT. GCT for in quite

could those also that could levels. just patients was lab take you what's the ERT? I criteria So we stabilized somehow meaning us have resuming And for remind as their wondering,

Sandy Macrae

your some Thank on can that, Betina, you please? clarification always. you questions for give us

Bettina Cockroft

Thank you that question. sure. for For

So very EOT I now happy off we investigators resumed X who latest them have The patients present with are at the ESGCT. have confirm We pleased therapy. we and data plasma can that seeing, were they of of patient. to data clinical seeing. Fabry series the are resumption ERT are -- And that is for really ultimately, on ERT, criteria of with terms including in investigator dependent biomarker data data a lyso-GbX the the of a that and and they are decision the of none

And well. are so far, doing those patients

Sandy Macrae

I And GBX. there is therefore, patients well, investigators most other think call. you've for the keep The levels to it's list the what comforted that's the off. thing ERT important would seen to similar on doing patients an have are investigator are at them their and

Yanan Zhu

it. Got That's very to Indeed. good hear.

progress sickle-cell informed find you has how the product? do program? patient readiness And are saw manufacturing of been #X, talked -- by III you For the how what Or is program partner in program on I about patient those plan for was much the by to to it wondering design yourself? underway. this Phase you also, this and manufacturing urgent a new pursue

Sandy Macrae

one? that answer you can Natalie,

Nathalie Dubois-Stringfellow

forward, Phase really enabling completing to currently data Phase continue program. our So your you thank assuming study to currently III have in Phase we're excited this on question. program this III to planning and hand, the the -- a progressing program we're support activities. We're take I/II focused We're and for

patient -- poster III. not very that we to sorry. will and to continue wait to next second Phase that a a we're body bit see it's see manufacturing of a planning have more be ASH the cohort, have those to improved for informative have you'll ahead data. evidence to if little But The So have patient cohort, next

Yanan Zhu

And it. Got partnership incentive initiatives?

Sandy Macrae

our for -- the intention program. best is the always We do to

do so really new half intention next until first and gather the full process at year. won't first quarter, to the improved set from of Our as is or see decision. partner it's a data we the And data, the whether it it, look at whether That's we'll thing. successful a point, the does that patients. we mention make We to getting important necessary

Operator

Harrison our next comes America. for question -- next of And Bank with question from our Greg

Unidentified Analyst

This Fabry what guess you Fabry, the gene as is physician comment on ERT freedom the the and for is this population? trial, in approach efficacy Mary interest patient [ph] clinically in the regarding Greg. meaningful could Maybe I impact and of therapy you here? for

Sandy Macrae

to Could proxy I follow. repeat is line ask just to question, you the the please. hard Your

Unidentified Analyst

Yes, absolutely.

you Phase III could and on also should in So the impact and trial, therapy ERT you as approach poles... the gene the in advocacy position comment of you the interest freedom the

Sandy Macrae

Okay. this you. And think for is maybe one Mark, I

Mark McClung

question. the for Thanks

rule, input our as of a with very engage work So all, and to first I mean, better to just also general X we but protocol actually them provide understand Phase the actually with prior efficacy early, designs to into patients.

can patient so We and researchers scientists to also the what and requirements bring that disease actually their understand share developers really patients in speak to us our to are.

proud based that And of. something experience, well. Sagawa we're on that's think So my incredibly I does particularly

we're think with But terms making steps, appropriate the the I of next next we'll at program. I time. that In steps provide great think progress

then can you we provide later. to that it's and that, details think important the guidance appropriate the I of finalize we

Operator

[ph] question from next Stifel. Kelly Our with is

Unidentified Analyst

had [ph] your Burnett. the Kelly could Huntington's. into these phase. quick talk is when This regarding one maturing more I be disorders if disease, wondering maybe in such on bit preclinical a the Ben these you'll just tau clinical the some for question about detail I prions, actually little you of as and seeing programs brain was just therapies, in

Sandy Macrae

here I committees committees, them. love the around on to We But the sit share these with question. partners, moving table to communication partnerships, partners. research the about the committees, people want reassure would in development your for they're you those more forward. you Thank is would by Unfortunately, what and is on that steering controlled

hands. Prion towards prior? of excited science ask passionate early They're that Can and Sangamo, And results. hidden about say also know I remarkable to through our the a hope almost -- we the how of love about on think partners' as that science. milestones so we the Jason, they we able -- did be them. you is have portfolio soon a it is They piece clinic to you get I in about touch because the and you are more

Jason Fontenot

of disease was to disease to compared extend and dramatically Sandy. several tried to as compared our Thanks, potently field, scientists, impressed year, approaches at Massachusetts the model in Sangamo in where comments Technology, Institute remarkable. of the similar mice Yes, collaboration very Institute studying we from the the that this data repress with were at were data. other opinion key And ASOs, expression prevent in Bryan prion of the repressors made ability and this leaders repress of the really to disease Zeitler of international of there data Prion that have generated the the thus expression life experts one a mouse our the Broad protein Yes. disease. how models Prion the finger has the mouse in Sangamo XXXX such with to zinc prion finger presented of earlier transcriptional been preowned transcription pre-one-protein mice of delivered we've was spread and that and known were zinc control and impressive they about the of brain in ability model generated pre-owned and be repressors of -- the the

comment the one of was I the data most think even there was it that exciting conference.

soon internal into we it moving we're as program, this a So can. as exploring a very excited humans program that we and wholly are It's about. is forward owned

now an was together us next dealing humans the Of in to data. aces complications. IND put that to testing allow course, steps And This possible. would forward with into move it we're for there are additional if mouse

Operator

comes question next Ritu from with Cowen. Our Baral

Ritu Baral

guys their of I've aspects there to that cardiac cohort, further, inform potential ask enrolled. about of profile their this the the And a And you're to I the enroll of patients follow-up. Fabry involvement they full dose? in ERT you And kidney to disease. that Are wondering or expansion I'm looking patients got versus is III? the female Phase a target on wanted about female number then

Sandy Macrae

you can color Thank Bettina, patients. Yes, give it's we've them? moved more female you. question. Thank into you for exciting on any your

Bettina Cockroft

Yes.

characteristics, patient detail in what slides. you done that can see we mean, terms for a our the So ESGCT data. sure, have There's of lot in I of

allows cohort. We of we includes be the the a It dosed expansion both and do and part patients cardiac to includes a phase, renal that to males characteristics. up also various have plan and XX cohort, So patients with females. as protocol

we're have So how this guiding patients have to update but as be early of specific in those making to an at moment we many cohorts, details the not around we'll that. XXXX plan or in

Sandy Macrae

cohort I'm most there cohorts. here, X hope I potentially are X Bettina, but not different different speaking

Bettina Cockroft

presented And That's I that think ESGCT. right. we at

antibody-negative cohort patients We patients. with have patients, cohort is with another another one female one antibody-positive

of Another one another latitude is course, cohort a female. cohort. renal one cardiac these is can both a And and and include male cohorts

Sandy Macrae

forms the females the females Does other disease. help? could that plus So with cohort of that be

Ritu Baral

thing. on It does. that have for CMC things sort for And launch pivotal that comment of guys assays, of you stand especially potency where on all tripped That's commercial helpful. other of that sponsors, product you then up assays, can the assays potential on Fabry, validation

Sandy Macrae

that CMC piece airtime do attention because the take And the the medicine as gets we asked to of clinical clinical I'm we you've under manufacturing. control. part way So most and very -- piece as that glad to know But is the the piece the I only and paying the we're excitement. clinical development these of develop seriously

go set the We commercial... for Phase have and everything both up to III

Ritu Baral

cohort? last I patients bit in. you elaborate, Can Sandy, the on can a Got one it. squeeze question little for in the schedule biopsy And expansion

Sandy Macrae

Absolutely.

and this for to We're come to volunteering back patient reminds cohort patients grateful to gratitude their to for possible. X cohort are patients from are -- in we are or kidney progresses. biopsies kidney to for study one, collected and naive -- well, data not biopsies. minor patients later our have have that kidney the read to who So those and we biopsies And the data. expansion data. out another the XXXX, patients for the have biopsy naive of patients are patients whom in first as the expansion drug a have share X what for us we that also all, cohort, will then do plan collecting in very do they We who and earliest have with both biopsy early are patients thing entry development make and to to of soon information we biopsy the have

Ritu Baral

classes XX-month XX-month these or Are is...

Sandy Macrae

These are biopsies. months X

Operator

by Please stand for our next question.

with question & next comes from Co. [ph] HC Wainwright Our Patrick

Unidentified Analyst

of follow-ups. couple a Just

one seen we've if about from that could specifically first future is the is it what some more tolerability And program. adverse editing you've more in or to these some and talk And the in Fabry on profile platform? to has be detail events of programs enabled current the with I'm in and you seen wondering therapy program. the of development that the in learnings discuss Just effects these safety to other or space? side can programs it the gene you what extent so you far applied the that avoid if can

Sandy Macrae

Thanks question. for your

have about safety profile... don't to You really the much say

Jason Fontenot

Which is good.

So thank you, Yes. Sandy.

are and extremely side, So on seen, we The extremely patients well. the safety doing doing tolerability you've as are well.

any needed not steroids not with with are have We treated -- of prophylactically. steroids the patients treat to reactively. patients

We X at those continue cohort. dose now the earlier, to monitor patients. And as highest have we mentioned I patients,

of patients it's schedule. patients. really So the the details what next to around trial lot paying infusion clinical the a a And doing education attention has well around the team of question monitoring think I these been in is

with on and pleased the And we're so very tolerability side. the safety progress

Sandy Macrae

MPS fabric. now And the AAVX hemophilia and we've used the across now trials, trials

yet the understand experience is is must why enough seen it seen and to events we quickly any cargo difference tolerated followed And The so seems is have that and And What's don't in and that broad patient that easily we kind why some is why occasional or the trial we the steroids. And thus is hemophilia these XXX. that? be quite with experience, the LFT have Fabry that far we've trial. the thing got of are that this. saw in question is that a at even enough remarkably truth patients is we it only make well haven't of resolved to interesting in

Unidentified Analyst

of therapies and on for preclinical cell programs some and CAR-Treg Yes. up the following both autoimmune pipeline, well with many internal on a in disorder many indications, for just partners. That's just editing preclinical externally gene the then as disease neurological helpful. And the areas discussion of with earlier. in There's

programs? for wondering some clinical not some program frame of of think So for these are or expect programs. programs if perhaps as those us which And some do of could more appreciated on could advanced you we when these the I'm as you well next underappreciated updates

Sandy Macrae

of excited the highlight? want would you're which which the Jason, say very particularly to ones programs CAR-Treg you CNS. by Can you your and

Jason Fontenot

for the lifetime T reg disease. both kind the I'm T the are of moving long-term forward the those. in inflammatory believe regulation treated by for benefit of and a great to disease the in we've that constantly of excited the than use that those targeting opportunities advancement in the medication for -- Tregs. to for We therapeutic of with transform discussed immune cells space sclerosis. CNS Andy. programs multiple potential of In offer CAR thanks, really, disease. offer being Well, both the rather a example, patients the a specific to and the treatment to space, we're sclerosis autoimmune ability And durable tissue, Regulatory multiple preclinical the about vital areas of of

we diversity potential what's view pipeline. that those excited -- So treatment of the a exciting of autoimmune the way In space, platform programs CNS disease, it's the is the very the is CAR-Treg transform the something most has forward. moving about to and really we're the say I'd

which, used a being can with our progress just and and collaboration have our Biogen about excited those made We either down-regulate being finger zinc very reminder, We're neurodevelopmental in diseases Novartis genes that's are as and with -- upregulate our in the collaboration respectively. neurodegenerative or diseases, there. transcriptional regulators,

are partners Our really excited.

that, the looking have. advancing is that those Prion we we've was advances our forward that data fact the that and of top programs most internal amazing and with nondisclosed highlighted, that AAV also toolkit companies and those came probably the year we're share partnerships the to in I'm appropriate We're own last on in several time. work. we at think And our engineering excited that other I area acknowledges capsid have an the programs to made about Sangamo

our zinc capsids So we healthy effort across deliver the there. tools And new the by to advances either or via very can that through bread barrier. injection remarkable have blood made we've the finger AAV in some -- blood CNS, a developing direct

effective, that those evolution that work, are data ASGCT generate And continuing was those been to the identify capsids are Some directed exciting year. earlier new presented studies of that more have teams doing this at to even -- results. that

more up zinc going space, at open diseases the captives, it's amazingly really, working indication that getting matter that's that's developing to well fingers go right will And the after it's space and body those are to the of cells tools more coverage even with the to we're because what really so and more and a afford. AAV the the getting those moment, AV necessary. And in new capsids

So we're well. as super excited about work that

Unidentified Analyst

helpful. very That's Thank you much.

Operator

it closing like Wilkie to this I to time, Louise for would remarks. turn now back At

Louise Wilkie

once Thanks questions. and today us for you. for again Thank joining everyone's

the keeping to As forward Thank Investor earnings updated you. developments. on Sangamo a reminder, access you Relations look and future on release section We can presentation you the website. our of the

Operator

for does you program. the conclude in This today's your participation Thank conference.

may You disconnect. now

Sangamo Therapeutics (SGMO) 4 Nov 222022 Q3 Earnings call transcript