Earnings call transcript Q2 2023

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Operator

Good day, and thank you for standing by. Welcome to the Sangamo Second Quarter 2023 Teleconference 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 today’s conference is being recorded.

to Wilkie, conference Relations Vice Communications. like speaker to and now Louise today, President would hand over the your Corporate I of Investor go Please ahead.

Louise Wilkie

Investor Fontenot, Chief Medical Officer; Officer; Chief Wilkie, our Prathyusha Thank including the Officer; Jason joining for Media Sandy on and at leadership Officer; Chief Duraibabu, members Chief Investors Dubois-Stringfellow, the this Rojkjaer, corporate found Executive Vice section I’m of Officer; under the Officer. Good Scientific On Development on website, Corporate be Financial Mark Louise Macrae, Nathalie today. Operating are from sangamo.com Events you. President our Thank & Chief afternoon. page. us of presentation McClung, and Lisa team, call Relations can Slides Presentations call you the and Communications. executive Sangamo’s Chief several Sangamo

Sangamo’s resources, not upcoming financial our with includes XXXX candidates, product of regulatory lines the catalysts are and statements runway, for feedback reprioritization capital, the statements our submissions, limited XXXX forward-looking data, clinic, to, and statements The and expenses, of and thereof. call our additional trials, regulatory our plans for conducting and dosing seek facts. estimated the clinical and of potential initiating anticipated cash that This and from our other preclinical and interactions our of are screening, expectations. commercial advancements operating sufficiency strategic advancements to regarding agencies, but and include, programs to guidance historical anticipated candidates, patients and milestones clinical estimates collaborators benefits These time not anticipated plans and presenting of to of statements relating Sangamo current and therapeutic the product

specifically certain XX, the quarter ended differ materially we law. Form with SEC Reconciliation to supplemented that discuss and undertake ended June XXXX uncertainties filings press our today by is stated to our our this Form XX-K fiscal statements except discussed risks for from today. on by year operating our annual GAAP December be required call, quarterly made expenses to can date, duty Actual the XX, and this non-GAAP subject for our the update we XXXX with on results of this in which filed what the our on forward-looking as report report statements such as are SEC. discussed On of on available found are in These we may as XX-Q information, The no release, our are measure operating website. expenses.

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

Sandy Macrae

Thank you, Louise call. everyone and to afternoon the joining good

The signs has for of medicines a second Sangamo lives pleased genomic exciting to one preclinical afflicted translate patients and groundbreaking and into share progress been busy the mission serious some our to quarter as I’m with transform that diseases. advance we clinical genetic

Our innovative I a been future. has alongside towards forwards our will genomic driver key believe in to-date, continue drive visionary the both which approach Sangamo culture to to medicines, success our

study STAR make programs, continue patients. dosed our XX total X/X a having progress of clinical our with to Fabry for in Starting Phase we disease, good now

Importantly, trial strategy. we feedback from recently received the our FDA productive proposed Phase on written X

forward it’s we it we regulatory X stands some a have potential as clarifications, seeking we’re today, path Phase While specific feel a trial. to

their of to to enormously work get I’m team us for the point. proud this

with expect of studies to the XXXX. the Phase X of to as driving and a logistics forward team our say, strategy as Needless early protocol trial our the these operations submit is FDA parallel. refining in We’re end proposed to

encouraging next rejection. candidate safety from Monitoring progress to on We the CAR-Treg received the the Based for renal we have good our to-date, also Safety our progress asset, made to clinical data advancing endorsement Committee therapy TXXXX, transplant wholly-owned dose cell cohort.

patient we of European enrollment protocol rate pleased zinc that encouraged to is this of are for study approvals to regulators the initial genomic us seeing we from updated which accelerate and our the in value believe be an enable we are platform, We phase. are through more receiving study will of finger at quickly The cutting the edge dose believe medicine. is by Sangamo escalation underlying

role platform. exciting advancements on will helping of and the neurology important we this of signed gene shortly, at we of business three the Jason two lead Mark with industry on and the share on our plays underscore context of and heart along recently more which all present programs, our progress AAV the science capsids some forward. quarter and development importance in neurology therapy to our that pipeline are These advancements deals our scientific elaborate the our shortly, cell Sangamo the that will scientific broader

being to and that in delivery are proud happy of the be field to important serve patients are necessary is recognized We broader the our best by is the part collaboration industry. order genome editing payload within technology and that

and as a patients. moment team welcome Lisa as to joins our as continue advancing and we confident Officer. our I’m leadership I’m Chief of experience physician take strong programs preclinical to like member to focused progress medicines developer. bring value drug our a she’ll great clinical closer on as to to I’d delighted to and Lastly, potential Dr. a strategic her the Sangamo with a new Lisa Medical Rojkjaer, us have

at funding strong and each the progress We as and further operating recognize of proud our of me day. makings This we priority expenses. a the additional is in the reducing importance have every of for of forefront made leadership rest this quarter. as we and well the top decision team bringing are remains Nevertheless, the

prioritized and the forward to at our we updates While these in committed areas execution and look providing to time. clinical preclinical we stay appropriate activities,

call Chief share more I’d now programs. over turn on Officer, Nathalie? like the to who Nathalie, will our Development our clinical to

Nathalie Dubois-Stringfellow

quarter, you, beginning Track Fast our provide the we last the FDA the with It’s candidate for call. designation an to gene May, disease. the on update for that pleasure U.S. program disease. ST-XXX, of treatment good and afternoon clinical wholly-owned our In Fabry therapy product Sandy granted Thank over Fabry my on everyone announced to

facilitate X/X total review our study intended to-date the reflect the important data dosed patients and STAR for options. need dosing and development with the therapeutics of the is demonstrate to Fast Track study Phase unmet overall. patient designation treatment we substantial XX this with the regulator to from have presented Fabry medical XX Receiving desire patient STAR provide the advance cohort patient from reinforces now expedite disease. Phase expansion a for of improved X/X new encouraging to The continued and Fabry with designation

We X/X data in additional from We’re Phase making early anticipate presenting good progress XXXX. in demand internationally. enrollment with strong site

something regulatory is we Fabry strategy describing We submitted increasingly a feel encouraged agency. patients. to quarter briefing have feedback with pleased and the FD Phase recently a the Phase X common which This and A we proposed the our for with clarity the that potential book a X received path the were disease written response, of for forward trial and trial

potential detail with studies. While can trial we that the enzyme population do strategy naïve patient currently of cannot Based maximize share trial intended I disclose that for received, study. this we on time, comparison expect feedback FDA head-to-head and this the a not is ST-XXX at in be the patient pseudo-naïve to required full are designing replacement across addressable therapy to two we a

next FDA point, some to in the to We’re protocol specific the end the step information on to submit expect of on protocol on early Phase additional from point specific to further FDA as clarity seeking XXXX. provide We able and quarter. as hope a coming the X be

seen sites. candidate therapy mismatched of Moving progress we A study studies CAR-Treg to strong immune-mediated six country the kidney total of across in interest now of a across the X/X Phase wholly-owned four cell sites rejection for from living STEADFAST donor, this study with our patients TXXXX are actively open prevention enrolling transplantation and some significant quarter. our made HLA-AX being

The May three two met pleased to on dose based data review cohort the from in the actively next data the and Cohort encouraging patients. to higher control the from the moving level dose for X I’m dose Safety the patients and that Committee to Preparation safety endorsed progressed. next share Monitoring

patient received We has first have in this the dosing expected of is for and the product cohort candidate second quarter transplant, the four, year. kidney patient successfully third the in their the manufactured patient

to we believe more X. In to-date, pursue safety dose accelerate as used for it times continue possible highest opportunity XX than the the one to with can parallel, we the Cohort be to is dose data encouraging acceleration accelerate through the greater best dose patient and regulators where on in quickly based cohort than to safely as

Europe. advice have amendment We regulatory protocol authority study an submitted in to received scientific and

share to We to approval. by have the end received country intend thrilled the full our of year. are first initial data already We

for Pfizer with developing are to Phase hemophilia to progress. we therapy gene with investigational continues our currently fitelparvovec, A severe giroctocogene in Finally, moderately X patient severe

work As readout we dosing to and primary required for mid outlined towards the continued BLA quarter, last in second a anticipate MMA Pfizer incomplete half potential Pfizer pivotal expected XXXX. XXXX. of submission analysis in and

from Mark will I Officer, Mark? will turn development Operating over discuss to McClung, our quarter. who call Chief the business now the update

Mark McClung

Thank everyone. simultaneously advancing pipeline drive business value additional value. strategy wholly-owned to core our afternoon you, by is of A Nathalie, strategically partnerships while and leveraging good component create that Sangamo the

or added focus We technology can our access and or partners value areas when deliberate company. strategic our advances provide towards biotech with a more working patients programs have priority, is non-dilutive to but their accelerates they in own can own made and brings and our to This medicines important of they’re to decision bring expertise to pharma our the outside apply learnings quickly technology. it technology we that proprietary this also funding and when

have development seen work in the the up the is world only been value of last we as our that has related fields. months, an a partnership result two The our excited strategic also the technology, with In but incredible partnerships business not highlight biotech to interest. uptick in potential opening on going back three and announce team

payload reflect patient betterment progress by part feel of in both technology, be and transactions something our and continued encouraged importantly feel editing for We delivery offer. honored interest Sangamo genome being are lives. innovation such perceived to this can and of as These the

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pipeline. Officer, Jason an overview preclinical now call Scientific the over for I’ll the of Chief our Jason? to turn

Jason Fontenot

Mark highlight the I pipeline. the opportunity past the engineering great from Research focus will good focused pipeline advancing our and specifically made therapeutics our It’s afternoon have to Sangamo progress neurology preclinical you, in Thank we’ve in genome quarter. everyone. vivo on work and this to in Today exciting

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via primates. delivered performance data In AAV transit exhibiting identification mediate generation multiple AAVX improvement blood capsid administered circulation non-human this an in non-human of showing describing when barrier blood-brain in delivery last cerebral tenfold capsids to variants primates. second presented characteristics increase enhanced data of identified describing Also and with consistent additional STAC-XXX we novel the that quarter, optimization presented that in quarter five our new spinal over we this substantial fluid CNS experiments

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combined our our We technology as potential we be feel The the forward about responsibility is of own to enabling companies many the from continue receiving that and editing. to epigenetic as reflected zinc mission. is internal deploying our the our by to partners are are a we a to capabilities our partners, apply other our of this our with and commitment to to we unique earlier. great announcements medicines taking technology patients finger spoke how possible, recognition From to Mark advance to testament programs field

for Financial now our I Chief overview Prathyusha? over call an Officer, to will the of the Prathyusha financials. turn

Prathyusha Duraibabu

operations believe with the a quarter. the months. in combination We XXXX equivalents June ended other reductions, our afternoon. as you available million million of for at XX, represents approximately prior and the least sufficient securities, from be potential will XX cash, marketable fund which Jason, decline $XX to next of We marketable $XXX equivalents cash, cash Thank cost and and net planned securities in cash quarter good with

efforts We as to range have development explore progress outlined, in great continue options capital. raise made proactively this to of some and Mark business our quarter additional we a

primarily compensation to [ph] as $XX charges approximately and XXXX the restructuring the second for period $XX clinical was which exclude XXXX, stock the compared trial. same impairment costs expenses, advance operating million post and for quarter in to June reduction to expense due announced operating ended million based our in quarter related the expense this higher Non-GAAP XX, we

quickly current three safety be as available to Phase directed for of investments financial Phase found through being As A results our possible prioritized a are in website. Advancing reminder, our our X program focus. in and our towards are issued press the pipeline. potential TXXXX development the trial the X/X Fabry CNS XX-Q our afternoon, for prioritized progressing this areas and of release can detailed on as quarter which

full year to expense full $XXX to our XXXX impairment year exclude of unchanged and to we non-cash operating to a $XXX turning million expect in million. non-GAAP compensation remain the and guidance, stock-based Turning expenses, – to which a certain range approximately be

As announced million expected a approximately is result reminder, forward. QX we at annualized an earnings going the savings, $XX to restructuring

decisions We priorities. these expect our XXXX continue in key reflect strategic line months. programs drive and to the be savings in operating further focus with to expense our to coming we as investments resources The our readout significantly lower and will from

further provide earnings regular our XXXX our into quarter will in per We guidance as fourth schedule. a insight

will I for back call closing turn remarks. to now Sandy the

Sandy Macrae

Thank you, Prathyusha.

today, signed partners. The programs clinical payload this of potential business and quarter programs of technology and the Sangamo focused focus determination. continues genome show capabilities delivery our editing wholly-owned to both and driving broader development importance As and manner, forward recognition heard our with our deal demonstrate and execute in to strategic preclinical to our a the you’ve

regulatory important had faith interactions in we’ve further clinical our on Fabry productive The programs. to these TXXXX with strengthen the authorities continue in

to able remain more important this we we’re in Fabry our preclinical pipeline, area. through wholly-owned data that our and share to very clinic, pleased I’m regulation promising advancing While we’ve on focused the dedicated and neurology epigenetic of TXXXX advancing been

prioritization fiscal and funding potential seek a ways bring channels. additional on in of continue operate with recognize discipline of we Importantly, continue importance the a to to strong through to focus and variety

to make we as inflection are in reach decisions. continue will a focus strategic position internal Our points

it open the like for Operator, to open would please we up lines. time, this At questions.

Operator

Maury question Jefferies. first Raycroft with from The Instructions] comes [Operator

line open. is Your

Maury Raycroft

my congrats questions. thanks taking Hi for and progress the on

follow some For entail Fabry, proposed in for the the what Phase there details would plan terms and populations, studies feedback, the of two the are elaborate Fabry any can of other in X you on patient head-to-head for is can versus on FDA of ERT And timing what as you the scope, think up, studies? you and necessary, an given share two don’t use would a size, a that comparator? as it

Sandy Macrae

afternoon, Maury. Good

So for your thank you questions.

going aren’t pleased to from had here, the some I’m we, interaction agency, really with So get help Nathalie? the Nathalie we’re we but with

Nathalie Dubois-Stringfellow

Yes, absolutely.

had we start lot of and very optimistic X think about a I clarity the on year. protocol our proposed clinical the optimistic of a by Phase end design submitting

So In very studying progress. we population. patient of entirety of term happy the question, with your are the the

both the As heterogeneous population to patient treatment address in and is Fabry and we our you strategies in entirety. symptom are the know, disease

two address and patient and the pseudo-naïve on had both the They studies. So ERT. naïve we proposed population

do we We of patient do on ERT need additional share X seeking Phase at not that the on clarification comparator protocol of pseudo-naïve strategy, naïve received date. the to and are we some that with additional the will detail but feedback just finalize X share our detail can Phase later the we a to a we

Sandy Macrae

with and to have I Maury, is these but whole getting, And put the you clarification. in know document, their opportunity a the you they agency having response you you know of ask process for this, they, interactions give

is believe said. we So very back what then they’ve to it understood normal go sure and us is we it for make fully

therefore response that in the of recently are process. came So very, agency middle the very and still the only we from this,

Maury Raycroft

Got it. Makes sense.

start for so be studies time And same that? staggered, anything they about two at those the say would additional can you or

Sandy Macrae

quickly details that We are very and time. get you them possible, give the going more at right we’ll our doing to on best as as both

Maury Raycroft

Thank good. sounds Okay, much. very you

Operator

question. the RBC by next comes with Capital. The Issi Luca Please next question stand for from

is open. line Your

Luca Issi

naïve Oh, sounds great. is on Thanks questions. great my for so follow news. on with much Maybe questions you like It and pseudo-naïve up there alignment great Fabry. which the have for patients, more taking a FDA

there? any the much go However, is on patients what’s asking that FDA are color ERT? Again, the be What plan to would actually appreciated. after

and less here that me different love time have their marstacimab much much. monoclonal maybe maybe can just maybe a about of any then [ph] for stream? would give looks wondering and on and energy focused behind excited therapy, Thanks subcu monetize a you hemophilia on view is And putting have but you to how A, and if gene therapy if It bit sense royalty plans a they’re related really so to gene Pfizer hemophilia? antibody you that

Sandy Macrae

Thank you. There’s answer a to that in lot single question.

going it suggest reverse I’m So to in order. do we

are on Mark, hemophilia, So, about the talk Fabry maybe and epidemiology on can patients on you are reflect you can that and on ERT Nathalie, you patients the about the then patients. can of the and that comment not ERT then perhaps

hem So Mark, A? if you start with

Mark McClung

second having interactions guiding For the with consistent program them have has BLA forward. fact top-line changed anticipate their still not The XXXX. half that they are move the hemophilia on they’re with to in submitting a They data continuing are guidance. A, in Pfizer half of the sometime the first we that that of XXXX, and

progress the about continued excited very on we’re So that.

and now if what milestones talked monetization right runway. provide and in are about, past, guided is an we something as for the of our update. royalties We’re not going to extend of comment cash the the it we’ll on need We’ve But in to an royalties. option we’ve changes, us terms any should

Sandy Macrae

Mark? And

Mark McClung

Do you want Fabry?

Sandy Macrae

Yes.

Mark McClung

Yes.

in at over I at a I But three only patients course look particular on have at a So database. sometimes got Luca, measured the that’s least the healthcare What low there’s as year scenario, database talked it’s about time year. diagnosis low of we’ve that this we case per take that X,XXX. the striking about X,XXX as of by patients is visits given that at Fabry engaging any X,XXX at with providers. patients, there And that if think two find Fabry are the to are end, about with best a there’s you the before, had as

I So right there’s been X,XXX is the any diagnosis sort way of triangulate not had patients have prescribed have clearly that that a roughly therapy around now. X,XXX

being the just probably they’re by physician. monitored So

on at compliance going place. kind put enzyme that replacement they look the were There take all But is have are probably about therapy. X,XXX you when regularly rates, a been ones over the the that in. are Those of

as given that And you’ve got low got, as in month, patients on so X,XXX basis. you’ve in any a are regular going

regular substantial And and of population, The patients the the small diagnosed number that into naïve with compliant is about category. enzyme that think are of replacement fairly number patients so, fall totally particular when the you population. actually of a therapy their a number pseudo-naïve

Sandy Macrae

that news. study, we is a great the how – And going said ERT? naïve, which on will need so you’ve to, are for comparator those are And Nathalie, the pseudo-naïve not

Nathalie Dubois-Stringfellow

seeking we’re So exciting in some the patient our have an we from potential X population needless really both naïve, to well on patient. FDA ERT. – specific finalize the that pseudo-naïve forward ERT proposed as as ERT path But for for on question to Phase protocol trial order say clarification

Sandy Macrae

And that we because important some them. ERT. that They’re their of up, life weeks. are having they gene will It be year ERT off a staying wear where for off a better. quality goes the these patients therapy two of look proposition done of studies ERT, believe an much go In are come at the the on every ERT and patients we’ve that feel them patient over better that patients avoids to

to questions look like. as study can that obligation more feel let the and And know you need what the some design to so an we’ll we we as will We soon them. address of agency ask of

Luca Issi

Thanks Very so helpful. much.

Operator

next standby Please Greg with next for question of from Bank Harrison The America. comes question. the

open. is line Your

Greg Harrison

taking Hey, question. Thanks good for the afternoon.

would to For does the be look enrollment you for the the how after on demand Fabry Phase FDA patient like? your turnaround what you protocol expect submit the timing and initial X trial, to

Sandy Macrae

groups. patient patients The medicine. very the to coming clinical in get with this engagement trial the eagerly are great support have to current us We

submit of if we So Nathalie, at year, are to? what end we the the guiding

Nathalie Dubois-Stringfellow

FDA with X. So the will go say, to the soon as on having team and the or for days have preparing as we protocol review start comment agreement our final as after the give we are protocol. for have ahead possible us CFD And operation some needless XX then then a to Phase

Greg Harrison

into fund baked in. you [ph]? could I your whole guidance consider cash partnering development more And runway latest you. What to sneak your are next And one any stage over Got if the of programs would the assumptions months? XX then

Sandy Macrae

Prathyusha, one? can that you take

Prathyusha Duraibabu

Sure.

funds end fund operations $XXX for we and that least are would XX. at quarter of cash was at our the the our million, it So projecting

I assumptions, in restructuring least in have XXXX, we what savings. guidance – the is that that we’ve mean, million, at we as announced expected you to of million – result the executed the QX, for QX, in and keep to mind in and to $XXX the far As million it’s did $XX for $XXX

guidance several but we’re with market right, the And XXXX, be lower. up. partnership our looking expect to further the at at looking spend encouraged funding shared do for Sandy we’re we yet both we opening for as in savings, significantly a along and as options alluded So to from also by OpEx. these haven’t we’re looking really XXXX the for how call, we perspective we’re streamline at And our

Greg Harrison

question. taking the for Great. Thanks again

Operator

next The with question question. standby the for Barclays. comes next from Gena Please Wang

open. is line Your

Gena Wang

Thank you for questions. my taking

clarify specific trial Just – the design. from the only X FDA. additional want ERT That’s clarification regarding Phase question to FDA

Sandy Macrae

question. the an for and it’s it’s – have take agency to this clarify of piece us, important the we because opportunity such opportunity tells everything Thanks information. Thanks. It’s important to the

So things commented we across whole go and the back ask to able range were on. they of

Gena Wang

clarification from there the Phase before or clinical initiation? data any requests X the Is FDA Okay. trial or data like preclinical

Sandy Macrae

Nathalie, I can one. that think we answer

Nathalie Dubois-Stringfellow

Yes.

data X. is So for the starting clear be no data added we on the there have path to and that no, Phase a preclinical needs forward

I both those. think clarity So we of on have

Gena Wang

can the administered then internal capsid? CNS own Voyager’s target CNS Okay, And your target elaborate contracts you regarding great. like bit compare a AAV capsid, AAV AAV a IV little very to quickly

Sandy Macrae

pass I are me Voyager to that clear we that, a working but do and let careful capsid to Jason, keep over just before very Sangamo either capsid. So, on a the teams they’re

very We team to to their capsid. giving Allen, Voyager access are for grateful the us

make. come be And Jason? the important point to very a so have them very together. to to It’s careful not to partnership information from

Jason Fontenot

data disease. we the for our collaboration and that Broad We’ve animal really through Yes, the models, about that in to get that MIT capsid in develop progress with saw been Voyager we’ve the partnership patients And believe really Institute generated prion received drug Voyager way so extremely the on we’ve potential capsid, the when that that to disease. a excited from prion in feedback therapy the regarding possible. administered their we it community And the the IV to quickest in there. positive made uniformly was way the best

And quickly. if you extraordinarily I’m you’ll so devastating understand die disease, felt the need important. can is a disease where sure was in prion appreciate why people it This we

to also as patients partnership we our Voyager for around there, there getting really quickly possible. so their was as that. recognize And motivation that thank as they And

capsids. effort programs. we’re our of to there. have year or are over lots next from we optimistic lot make our some Nonetheless, we’ve that see also course of we seen the that mentioned developments we’ve a developing own to of we shown and at very major the improvements progress in the continue ASGCT, work I two, a major own And going

so commit capsid, to combine the at manner in a on And we with and time. we make what and a to strategic maximize advance a we we case unique forward pipeline, but to amount we our to potential a reflect we that look as also to available, our each to reasonable pipeline, things when our have probability and in move need of maximize are recognizing capsids choices be could cargo of success, need capsids

it’s believe but our more capsids. decision, So own a we multifactorial we’ve that got

that. at emphasizes recognizing to a strength Prevail that program. I there’s the community development we’re the the something think always time, capsid open with to are in partnership doing quickly, the need same others our more if Lilly But that of own move forward and

Gena Wang

very you Thank much.

Operator

the question. for standby next Please

question with comes from Anvita Cowen. Gupta Our next

open. is line Your

Anvita Gupta

Hi questions. guys. for Thanks taking our

to dose many followup. Firstly, from have Phase expansion FDA do signing see planning data program, off any you patients X/X cohort? the on does Phase in want Phase Fabry And the designs X, the two the before possibly additional how the a I and X to more are patients on these

Sandy Macrae

that? can Nathalie, do you with

Nathalie Dubois-Stringfellow

Yes.

phase to something patient is So Phase do study. we Is start we for Phase in we the protocol. we trials starting those X/X data data and enroll sharing to are any point. X that plan and need continuing this But at a additional not a patient are not X this there depend the on

later at will a point. clarification provide We

Sandy Macrae

patients important sure study X/X a very see. showing the the when we I incredibly it’s will that will up from Phase longevity file BLA benefit And agency, that to patients this and for think pleased will be Phase years. to we it be the those that give data realize XX supportive believe to X will could I’m that be of data, because

Nathalie Dubois-Stringfellow

part will durability BLA of and the safety database for be they Yes, filing.

Anvita Gupta

be end? Got are on then it. of guided you. what your secondly, could what so, give there the to us for? from And you data and any TXXXX, we for expect biopsy data looking if Will Thank some sense initial

Sandy Macrae

this take you can Lisa, one?

Lisa Rojkjaer

Sure. Thanks question. the for

as it the we safety and a at year as of is tolerability data some we’re into what a that So data about going details be study, present not primarily and end of may additional would presenting we well. the

Operator

the question for Truist. question. comes with Germino standby next Please Nicole The from next

open. line is Your

Nicole Germino

congrats Hi, and afternoon good progress. thanks and the question my on taking for

on ones quick platform. the CAR-Treg two just So

TXXXX production. immunosuppressants given expressed help the have some study suppress of investors of immunosuppressive patients couple a you use So to may in Can understand ILX us the after infusion? the agents that concern

in from to a progress are for several question CAR-Treg with pharma timing their programs IND? my pharma. the big lot big partnering us space on, is And with off the you your IBD platforms second of IBD and remind Can IBD in interest CAR-Treg potential there’s program and companies just

Sandy Macrae

going suggest So interest takes question, I’m want in how there the technical you do Tregs goes is if to in clinical about Lisa talk general, Mark, but Lisa Lisa, the first? to

Lisa Rojkjaer

Yes, sure. Thanks question. for the

are standard following regimens therapy study adaptation immunosuppressive we the those – time. over you’re So in the, and using in regimens

the related it’s concerns practice. using standard of care, heard we’re that there that haven’t actually we because of So regimen standard have been to any

following, in clinical patterns outcomes, therapy immunosuppressive looking as the as terms the we’re the used evidence investigators. rejection by of of at of So well

Sandy Macrae

Mark?

Mark McClung

Yes.

invest of leadership a in them that. And – looking And the investors we as because interest to of create CAR-Treg inbound a partners had in Tregs, the of opportunities terms so of mechanism before we’ve into we’ve in a potential in Tregs. that our interest communicated for of from think for have potential lot directly are terms to the obviously lot for and platform I

so looking are And of we both those. at

having look conversations taking and update at that We’re partners, allow provide direct to we’re with for at investment appropriate time. an we’ll mechanisms a but also

said, the to of that on, provide still we’re in is or able we along show commented what end terms there interest of in the progress lot at be to of the what an furthest dosing will we’ll of But Nathalie a point, making. hope in year. Lisa by as actually patients. update of And yes, and the And we’re this data that the terms the data CAR-Tregs we feel potentially

Sandy Macrae

furthest And talked talk us that’s what hear the we that and along, about that about one it, we’re from people the in only clinic. that’s come we the the to are being

control allows We’re process. that’s, only the in GMP manufacturing [indiscernible] with that us the one to

cell only capability, that’s INDs doing editing and of experience an the the we one the are We’re and and with one gene got therapy.

data. Tregs. So the sharing that’s why forward the we Sangamo they’re And look they do like seeing at in what to

is it study remarkably This have first a CAR-Tregs the is time foremost. As first is and this been Lisa safety well says, tolerated. and given,

Nicole Germino

you Thank so much. Great.

Operator

Yanan comes Securities. next Wells The Instructions] from Zhu question Fargo [Operator with

open. is line Your

Unidentified Analyst

taking Yanan, questions us. for [indiscernible] thanks Hi two for so the This on is question. from

The first on is one therapy.

So for withdrawal Phase and on do off the second TXXXX. the then ERT remain patients one X/X ERT start the all is

forward? need what you. safety patients be the Thank decision? move besides would how of for the to So make data the required does that to company many company see program And to

Sandy Macrae

Nathalie, as you your easy Thank an one. Fabry always for question,

Nathalie Dubois-Stringfellow

the all in the that ERT. have Phase in X/X remain ERT yes, ERT withdrawn, on patient So off

second question was question. the that think And the first was… I So,

Sandy Macrae

TXXXX.

Lisa Rojkjaer

Okay. Yes.

the what there clean about we’re And no data forward? And more this very a that far. that to move second open data So in level, different with expecting to from Well, And within allowed advance within safety thus we’ve thus also that to think has forward And and your so patient us by dosed I had first quickly first were dose dose quarter dose the next we potentially that need three patients to to question been opinion program see the sorry, Nathalie protocol SMC was already DLTs. now we’ve to do level. this the move levels. we’ve far I dose seen patients can safety we mentioned, the were as the the – encouraged amendment the the through quarter. say

long-term, are terms So we’ll on to study. have of how encouraging in evolve see all the things these

Unidentified Analyst

so much. you Thank it. Got

Operator

[Operator Instructions] The with next Wainwright. Trucchio question comes from H.C. Patrick

Your line open. is

Patrick Trucchio

Good afternoon. Thanks.

Just up follow questions few a me. from

what manufacturing can The discuss X.X of completed IND first submission NAV on in of program, just side the one remains you to the in anticipated if particularly XXXX. is ahead the be application an

wondering might then interest earlier potential regarding increased on and development receiving you’re future secondly programs platform I’m can the earlier of look comments that call collaboration partners. just, are discuss the what there you most potential interest aspects you if in from if the specific could And partner. on form collaborations to stage take. And what

Sandy Macrae

the Nathalie, Jason what’s about can will you in X.X, and So for then platform? left about talk to do his passion NAV talk

Nathalie Dubois-Stringfellow

Yes.

able planned IND for XXXX. be to a primate. terms of main for in on manufacturing an GLP the and time and next tox trial. to the really we’re non-human component the first lot in year. things underway our are, are, underway well is remain And this to in IND is are we also be really be in we the completed to the patient manufacture The And X.X clinical So completed two the to excited about to NAV two thing that gearing the be that

the working we foresee delays. to point, those team So stick at this is fiercely no to timeline. But

Sandy Macrae

And we GLP do dose the still the although Nathalie, range. already we’ve to done have tox,

Nathalie Dubois-Stringfellow

ranging we Yes, done study. have the dose

path development this study. to So where normal this is, need we the have

Sandy Macrae

was issues. the there was, no tox And

Nathalie Dubois-Stringfellow

beautiful. was It Absolutely.

Sandy Macrae

thank Okay, Jason? you, Nathalie.

Jason Fontenot

so that different to way there area. then inbound capabilities Treg many seen wide talked all from, the the advances from already our a partners in terms of running cars We’ve interest, lots about around, there. go covers cells interest potential And in our Yes, platform. that partner patients. we’ve and particularly designing manufacturing We’ve built it of the trials in

of them lots Tregs. large. there can’t some very run we variety recognize indications And a of that are ourselves. we all around So of those of studies There interest

on that discussions So in front. we’re always

is the a lot next platform. the discussion say quite been next I’d area where zinc around our finger of there’s

compact be about that as ASGCT was think a known always the value there specificity, kind things that for of this the year seemed developing zinc of And we One Sangamo the at renewed based therapeutics is the kind the the I finger proteins. nature on, something true. of of of that discovery real platform have to of the interesting tunability,

their renewed technologies so with different different pair platforms that And we’ve lots programs. really had of with a zinc companies interest want from to our finger

how One our way strategy. those of that, fit evaluate kind collaborations is we are into they programs, those

vivo, we’ve about we have in on as focus strong So a genome talked space. lot in a neurology today engineering moving the forward,

cargo of enable finger the very one – zinc with always our outside their providing the and that space cargo, are for that partner So, is are open first the them companies we programs. of working very places to to to neurology platform always open

would through When get soonest we really to way, for where the that are always then ultimately open there collaborations. we space, to we that quickly to if on? can and company? And do in want drive what smart those a things execute forward and focus both the patients value areas think the things Where other move that benefit into there to we’re can we neurology have of are companies feel areas types Sangamo,

over is team with highlighted and things on capsid been about platform. two very been very would progressing are fast an years by area seeing data the has evolution the And work excited we’ve we’re the then area from This last and is been the say it. really that great Prevail maybe doing deal I lots that last also that our the we’ve of

need area of we’re to going not an if to lots it it’s And happy discussions. to that, earlier, that’s then because have of there is interested someone in we the ourselves, else capsid if but the a, platform. capsids for looking of these mentioned there’s types is delivery we field there’s we’re that are able as doing and that, And an a whole indication have those pursue diseases facilitate that right

in teams and the zinc in on our of to move So preclinical Treg those Lots all think of work are, tremendously going interest I inbound platform the our proud forward, platform really, evolution of I’m finger forward, and all getting that’s platform. the advances research areas. we’re capsid we

Sandy Macrae

spend your Jason, said, lot I talking and partners how about a prospective the science always by time you to excited Well talking know they are are see. of they

Patrick Trucchio

Great. Thank you so much.

Operator

call Louise at to back closing now to turn no like time. questions remarks. would Wilkie show I further this I for the

Louise Wilkie

questions. today you once for and again us Thank for your joining

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

Operator

you This concludes for today’s conference call. participating. Thank

disconnect. You may now

Sangamo Therapeutics (SGMO) 8 Aug 232023 Q2 Earnings call transcript