Sangamo Therapeutics (SGMO) 25 Feb 212020 Q4 Earnings call transcript

Company Profile

Participants

Aron Feingold	Head of Corporate Communications
Sandy Macrae	Chief Executive Officer
Bettina Cockroft	Senior Vice President and Chief Medical Officer
Jason Fontenot	Senior Vice President, Chief Scientific Officer
Mark McClung	Executive Vice President and Chief Business Officer
Rob Schott	Senior Vice President and Head of Development.
Maurice Raycroft	Jefferies
Geoff Meacham	Bank of America
Gena Wang	Barclays
Ritu Baral	Cowen
Ben Burnett	Stifel
Andreas Argyrides	Wedbush Securities

Call transcript

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to Sangamo Fourth Quarter and Full Year 2020 Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Aron Feingold, Head of Corporate Communications, please go ahead.

Aron Feingold

Good afternoon. And thank you for joining us today. on leadership are Chief team, afternoon the members Executive this executive Sangamo including me this several call of Officer. With Sandy Macrae, Scientific of , Rob Chief McClung Chief Officer; Fontenot, Mark Business Medical Officer. Schott, and Development Chief Head Bettina Cockroft, Jason Officer, Investors corporate Presentations the page. Media from sangamo.com, our at website, the can presentation Slides section found in be our under and and Events statements includes forward-looking regarding call expectations. Sangamo's This current and these and our are not limited statements highlights These lines trials for house additional plans present to the Sangamo demonstrate for to statements data but include, in time benefit manufacturing and collaborators relating to, plans clinical data conduct and to on and facilities clinical bringing patients; clinical potential to for launch.

and not that other differ performance resources results discuss may Our and historical from Actual expectations are what we substantially today. our statements fact. regarding financial

required in to future and the potential this believe discussed is comparable on helpful considered which and measure comparable our The this of update The guarantees information, these in GAAP be Securities our call discussed today's to for we XX-K uncertainties risks GAAP isolation XXXX. December statements and for are is addition, This annual release, we In call, undertake this XXst, with date, We are statements future meant or on the past website. that non-GAAP duty not available subject discuss substitute a of measures are stated specifically performance year as we today no in to certain financial ended our measure. Form Exchange the press our measures Commission, documents On file GAAP are in is and that to performance report of financial in are included and measure. the non-GAAP made as a understanding and law. fiscal reconciliations except as not results. on under forward-looking applicable this such

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

Sandy Macrae

Thank we saying call. everyone afternoon and bolstered manufacturing XXXX. with product on cash execute start to by pipeline. how executed you, challenges just we of in strong executed good are the in position forward Aron, the all progress our to and pleased Despite I'd like on infrastructure on We on pandemic, the strengthening our our of to candidates the moving therapeutic clinic. Sangamo's

transformational online Phase disorder. headquarters. Biogen disease, on we initiated achievement under candidate house disease, A. pandemic owned line our product treating diseases, hemophilia of to and impressive with COVID-XX our X, with disease, Alzheimer's This testament of treating into AVV any our together in Phase our studies to bringing and the California collaborations X spectrum product manufacturing tackle our as team. Parkinson's an is Novartis a Specifically, X our capabilities candidate in Fabry neuroscience particularly during our partner wholly dedicated and challenging trial brought and entered was We Pfizer, autism a facility tremendous We condition Brisbane, manufacturing such

programs. And highly year candidate owned marketable payments partner through strong million in up with primarily we collaborations, all advancing then cash CAR-Treg TXXXX also XXXX wholly was cash, equivalents $XXX cell engine the of finished grew product research Our and our built and position, and productive, cash our from our upfront securities.

to execution. further ahead through on Looking we're clinical XXXX, value Sangamo unlocking focused

enroll end Fabry the patients present Study owned interface initial wholly by of one expect and data to to programs, XXXX. our For we to continue

continued momentum can competitive in control. capabilities flexibility And half we continue advance the phase initiating house partner XXXX, rejection and expect of We manufacturing cell X/X our end the proprietary Also advantage the in to with to the in manufacturing bring quality year. we programs. also by lastly, our manufacturing capabilities online of believe anticipate study facilities expect kidney We transplant our greater provide a and in XXXX. our second therapy

our For Pfizer expected presentation with AFFINE A XXXX. Haemophilia of an expects pivotal trial in Phase X continued enrollment example, data in

Additionally, ultra the within Pfizer year. from ongoing expects study the next Phase update X/X another

of by expects of clinical Car CDXX initiation our partner of Our study T end cell the a anti Kite the XXXX. therapy

several the end and Lastly, anticipate Development, initial our then our by developed of of by to disease Rob team of data the development I'd Phase member Schott, conclude our thalassemia our update late we end our we of of efforts highest leadership beta Sanofi. X/X on development the cell product an our on investigational like candidate will success remarks Sangamo by on today. team, advanced anticipate And programs our science. newest prioritize Therapy, anticipate as XXXX a experience likelihood study, stage interim with evaluating program robust Head also opening in presentation his our my the partnership as with He differentiated introducing from impact. sickle the milestones. XXXX. development Rob our Chief and capabilities and drug Rob's us Head pipeline significantly call and heritage role as the executive of who help joins and year, I'm to collaborator, Jason which the also very instrumental to genomic our be role Effective enables and Jason acting former Jason during contributed team, in will advance is Research. continue and this year of of his of beginning to rule between Officer. significant excited new in This promoted Fontenot designed the latest clinical pipeline. the to Head technology an building Sangamo the and to Scientific which research we has cellular clinical by in support we Jason leader Cell

Officer, work keep who details the to clinical our Bettina? came who These were am progressed achievements. of who our I'd over running, turn circumstances, Medical Bettina and dedication will home. provide Sangamo proud call I so the the Chief to team's additional I and business engine With from team, employees, I like to the all accomplishments. acknowledge of working over challenging those our our that, call research the into will labs the Before turn hard our to on

Bettina Cockroft

afternoon. Good

date, with XXXX, propels Pfizer in At week from range us results for one levels through X/X the XX our updated cohort gene which into Sandy patients, factor vector follow longest alpha fitelparvovec in we remained year A As clinical and dose announced giroctocogene activities phase and momentum eight XXXX, high that the very was The clinical median tolerated. longest dose treated was study the patient. nine achieved of A. results five for we're state fitelparvovec weeks XXXX XX genomes as factor multiple mean and Ash the for XeXX the Steady week activity treating mentioned, all of through well factor cohort generally showing by activity cutoff showed through kilogram available a pleased potential the Haemophilia in therapy, catalysts. therapeutic eight up sustained with giroctocogene following per high

XX is five AFFINE is to during median larger assay. study patient infusion. nine enroll. patients events activities was week activity year, also see years. XX.X% to mean from the vector over following of understand in XX.X% geometric followed Phase the no Specifically, the therapy for to Pfizer in via X target group these two There It X first its bleeding potential chromogenic results of less sample and important note second group X the were time and factor patients leads are joint year one that a was It factor which these to important and watch when continues reflective we continue than we only

our Regarding Fabry program.

in evaluating patients ST-XXX We Phase X/X Fabry to disease. start three have dosed study the in the first

we enzyme Following classical of Key and last or year ERT-pseudo-naïve an includes patient first assessment Fabry the who ERT include dosing prior have in efficacy patients and our years secondary activity this six the cohort. in trial ERT-naïve this in second GBX evaluating patient The replacement and received older measures tolerability two safety XX or old fidelity months. month START the and of GBX the patients comers, establishing and of the treatment the analyzer levels. dose Earlier not cohort. study therapy dose ST-XXX is first first

XXXX. by present We end the After cohort of we expect have Fabry data expansion. adults identified to initial for clinical

expect look will now collected I not to levels. this data continuing and later Mark as turn kidney We be over initial readout include will to to well readouts keep forward enzyme biochemical at to as results. do initial as date. We for which financial substrate you safety the program. the of the and to progress in data, measures, tolerability expect the a call overview biopsy our We include informed such on an Mark?

Mark McClung

the quarter $X.X Bettina, achievements. for same $XX.X and with the in fourth quarter good same our compared share, our revenues pleased full Thank cell period XXXX. XX these Haemophilia A. the achieved expected collaboration primarily disease quarter clinical to in milestones and $X.X sickle first to of $X.XX with $XX.X Beginning a study, afternoon, million net Phase We're million results, XXXX, milestone the due Sanofi from you, XXXX. loss our Total the is to financial for or per patient in million for under net transfer we reported million million, completion of we or for million share dosing for $X.XX share XXXX. included the and the were for which X/X our agreements, year results fourth income decrease $XX.X compared fourth per IND in revenues of in period everyone. In

in expenses, manufacturing also the to the Non-GAAP we gene due Phase genome to reminder, was the CXORFXX-linked quarter the AFFINE in the in trials and in compared milestones cash regulation earn million our study, expense, the of The increase $XX.X expected third program activities research non period a to million primarily our support XXXX. our X of of advancement in XXXX. participants to Pfizer, for to in of in expenses based quarter capabilities. Haemophilia million clinical A stock fourth excluded dosing second ALS. which therapies develop firstly treatment as for headcount completing and collaboration operating expansion from facilities same and the As was in growth, our Related operating $XX therapy $XX.X compensation total

licensing in agreements of $XXX.X partially headcount increase to A due expenses our net expansion. total Biogen reported upfront entered in primarily the results; The or to was collaboration collaboration million $X.XX Turning under XXXX, to transfer of in Pfizer operating of million loss $X.XX growth, was share $XXX in facilities fees to revenues year XXXX. $XX.X due compared per and to to IND $XXX.X full $XXX.X The XXXX. per revenues XXXX. the recognition million operating million with offset in following in XXXX. Non-GAAP agreement The were decrease into by a Novartis in XXXX. from expenses December in Haemophilia increase million and XXXX increase compared net XXXX primarily million a the were for in compared the is or $XXX.X share, we Revenues loss

mentioned COVID-XX the capabilities. our As advancement arising expense reflect travel, of these corporate offset partially the a in quarter in clinical operating pandemic. in XXXX, increases due was and to fourth trials discussion, costs decrease by increase manufacturing This

Moving ended securities. approximately to the balance sheet, the cash, $XXX million cash with in equivalents and marketable we quarter

our its sheet believe potentially approval. will review of in A, allow proceeds sale offering through We our market strong In program through XXXX from our BLA net that potential we addition at filing including balance XX, XXXX. milestones about our reach received to us balance important $XX.X February and million possible for established the XXXX, several stock remains to this Haemophilia of and R&D have common the under of in we and

stock expenses, $XX Sandy, which cash million. Turning be million remarks. of for to XXXX operating approximately in turn back estimated excluded guidance, range $XXX full of non-GAAP to compensation the about it closing I'll non year $XXX to expect now million based to we expense

Sandy Macrae

saying company we we're and Thank R&D advance house you, strong manufacturing our clinical Mark. like our and capabilities. our as our progress XXXX in where research by XXXX. building with And ourselves momentum engine made enables pleased execution, conclude a that pipeline. balance sheet We're very in to I'd to us with in find

are a updating the year. please question. compelling you. We for to milestones we open position in And this Operator, look important forward and line capital to several achieve

Operator

Raycroft Maurice from [Operator Jefferies. first Instructions] Our is with question

MauriceRaycroft

the And questions. for progress. everyone, my congrats thank Hi, on taking you

and is on ST-XXX Fabry. First one

the about guess perspective treated, the on and the patient patients you could for key So they pseudo any So I you've can AR you Anything experience else status enrolled say and naive, provide their that patient. are additional too? naive? or backgrounds

SandyMacrae

all can the well. end often So, give results study the study. for product. Maurice, is state the patients going Because to of just that to our we're or Bettina we you be we very until the easier being to that's there that think the say the better wouldn't is characteristics know careful anything of I

BettinaCockroft

thank you, -- We Sandy.

the three three mentioned, have enrolled, So patients are well, as those we I patients. patients,

enrollment the of Sandy And XXXX will is subsequent shared with completed toward part after So the as ongoing be we mentioned, we've patients. data dose Two expect Cohort end escalation

MauriceRaycroft

And second XXXand you. Thank Okay. is question it, then therapy cell Got Kite on programs. the TX-XXX

and if both the there is between standpoint? wondering can can -- these the whether both to of any factors from insight programs you two Just you for And provide into there comment for gating synergies programs? manufacturing a or studies prior are one additional starting

SandyMacrae

Well, that's question. an interesting

Gilead So both are program. will to the soon be for forward TREG have progressing, IND that and and with Kite guided the clinic moving into they the CAR-T very

are there. We a patient that year we said have great half this forward in dose second of and to will momentum the moving with

across comment Jason, do. CAR-TREG learnings interesting. question is can and the what about about in more CAR-T what between if are we're background the trying learnings do Your learn? there wonder I to you we difference It's it. And

JasonFontenot

both of and in We're these platform working excited in our our site also with are tremendously understanding T-cells very around use our TREG and pipe engineering both certainly of the partners exciting. finger in programs engineer TREG about There And program local be are synergies to the Kite. for at excited Sure, we're our program. program.

that's where would I say are. So synergies the we're biggest

heavily T And As it. where think for And work our nuance. manufacturing off own for we TREG development and is T part manufacturing about of oncology. which for I lots in critical and to differences really the Kite of probably but cell their different a our we've compared from be regulatory we're affect process and it's CAR-T know doing manufacturing And that's and building their so paying cells, program can manufacturing product invested has and it excited the why our quite the CAR-T program. there for real TREG some manufacturing are between you is

SandyMacrae

on cellular the manufacturing? can the Mark, what we're and you strategy touch manufacturing for out building

MarkMcClung

manufacturing our so a we advantage. competitive believe Yes, provide that capability can

So a our through and we're developing to as investing manufacturing access in-house capacity partnerships for contract process well with and supply analytics necessary balanced lay a chain. and manufacturing, beginning securing the building achieve foundation to We're strong as manufacturers. in our

Operator

Bank Our of next comes question from with Geoff America. Meacham

GeoffMeacham

Hi, guys. Thanks For taking Geoff. -- questions. for our

technology, into What get AV -- getting some these you delivery that steps moving Just quick into to allogeneic couple for steps a A. are some more studied Thank then allogeneic? could of the on autologous love the ones maybe non about gating little us you an of a factors have from but first therapy maybe the talk for from been And mechanisms would for you. the from bit hemophilia high to side? thoughts on more to what do your the to get TREG level, approach I gene based are

SandyMacrae

Jason, part So, versus large Yes. talking yesterday autologous we a science. spent edge about It's of cutting allogeneic.

JasonFontenot

Yes. Sandy. Thanks Yes.

the from that and product, is engineering needs to to product donor's a of in we One aspects the So cells. done to autologous move are between and avoid rejection are an the those differences allogeneic healthy and an to autologous main definition is be allogeneic which it. cell by two there and need of a many patient biology

So there's engineering there. complex a of lot involved

allow still silent, So our understand themselves engineering making that sure have requires that mean that, then and that which once effective finger medicine using and we biology potent dual to have platform also make their how affecting delivering and that you cells, engineering it patients. product. scale, to engineering complex is more we a engineering And you to then a on more of top strategy the for have strategy bring the the to can

effort. areas are places. Yes, key those those are investing a of where we're lot the both of places So

SandyMacrae

larger TREG of treatment diseases, and such arthritis, different allows And treatment want for us as the where multiple point rheumatoid into to even allogeneic for is importance inflammatory an oncology. move disease, more that population sclerosis, the is price also bowel urgently, normal to patients the

other other delivery about to was really and question is or delivery us. second Your treatment important modalities

based a moment to ways state. the of also It's look area. ways a particularly deliver, to the is L&PS find complicated horizon the always other to view pattern AVV encouraging both complicated complicated -- but deliver to deliver. companies constantly We on beyond is to are a who find It non are deliver interconnection a on are other scanning lookout

Operator

Securities. from Our Germino next question with comes Nicole

UnidentifiedAnalyst

for our Hi, Nicole. everyone. is Thanks taking questions. Nim This for

the on update the Just a tonight share conditioning quick forward question on Can discussion, other been program want the can intensity you. source, and Sanofi awkward that concern reduced [Indiscernible] that occur the risk there share you sickle to I guess all beat Thank or of time consider? ontogenesis with step, face changes and thoughts the have as shed resurfacing option or from as your of using any your may to it thoughts contributes competitors cells. reconditioning with you on there an is you

SandyMacrae

listening best we're our And something and and similar patients. our a that anything Thanks on Sanofi for the it they great comment the kind feel making our data difficult that about doing does to different. the have be are discussion our they're what we that at Bluebird. we of people appropriate also happened sure know for from your what right that but in understanding question. are Bluebird, on company And that know We seen not it's there know the doing very and several very to medicine, goes is learning we'll patient understand we safety confident we the be with We with friends quite from coming watching, have they'll We're relationship. journey. confident thing; that

that path optimize a conditioning You I'm understand we're to conditioning what first best will now question to about all the are regimes. us I asked generation. out technologies do continue the rate efficacy, of about And using is. once and we'll to sure to learn sure am try thing the work another we And how regimes.

Operator

Barclays. Our next question with comes from Gena Wang

GenaWang

I of my questions. sets two you have Thank for taking questions.

is Fabry regarding program. First

wondering three, Just so based on dose to dose vector you of often substrate question TX-XXX. Galle based say one the the do a cohort choose measure set the choose second as dose how alpha the you to to you've regarding Cohort mean from the second have well seen far data. And you my do certain AVXX on the is when freedom up? and data I is can shedding? question And cohort as and you

additional since the transplant? as acquisition, Just regarding collecting completion TX-XXX TX for be a improvements your half for kidney data the the second of also what the initial this construct wondering will will the cell be sets you and any year of

SandyMacrae

you of cell. so with why first back thank a around we'll to is Gina, with Oh, Bettina. then questions we come lot part and Fabry start don't TX-XXX a TX And

BettinaCockroft

thank Yes, And the Sandy. thank you, you, for Gina question.

cohorts So escalation. on low strategy at disclosed and we're what that a have are at dose for looking high dose. a three for looking now, medium we that's we And the a dosing our and how

we levels out, we're sharing the doing And point of that And towards you alpha the regular throughout now and study. data frequency. end are, collecting at Galle expect as that XXXX. we As be substrate of those to are

are that escalation be I going earlier, to mentioned waiting for data. As dose we to share

SandyMacrae

shedding. vector mentioned Gina And

BettinaCockroft

right That's shedding well. vector as

vector we at look So time. we're the you also appropriate And well sharing forward to that data shedding. with as collecting

SandyMacrae

ahead. go Please

GenaWang

have that vector some you dose, the to then is so interesting. also low collect question, high, dose adjust data will collecting Do those? actually I the market up And very like the and yes, the kind wanted biopsy. that's fixed Sorry mentioned ball but regarding you assume freedom sharing, you the to accordingly? follow just mid of that

collecting or you at terms Galle substrate and can point once frequency also two talking how do little we that? more every about alpha and give you shedding of weeks if Sorry So, just weekly bi-weekly? the also how data. bit for Are will frequently monthly in collect or a a do A often the color vector what

SandyMacrae

questions. always detailed have such Well, Gena, you

together you monitor Galle not of no -- committee we and So second all from been to acquired the to that we and found we share vector dosed gotten our really they haven't, we about we cell, discussion TxCell in give we it ask when often choose a question understand good dose be that will shedding. would the effect process continue of package comprehensive then meds. And understand we reveal protocol to we once that done was have we on the TX we have and were the how that TREG, the them them we believed that and we safety cohorts but since a believe allows because you nothing We they can developing the And acquisition. were we in fundamental flexibility TREG. you have alpha chance so why measure at there's we going and good put have escalate, with three with that something fully acquired work

GenaWang

existing recall, clinical I it's to actually if XXXX so XXXX, they planning in was Okay, enter TX-XXX. their if with

what to what Just share additional additional with Constra on little the more order clinic, time, enter can take be data us. of color in data and type takes wondering of what any you And what or additional hasn't also bit you'll a you collecting you since once prep if sharing clinic. kind move steps changed, to

SandyMacrae

get tech longer. was don't transfer in just process time we of I clinic in hope the in XXXX. we think there COVID takes be I ever that little in a And to the a XXXX, think expected the

first their feel year. the share that as clinic the good about We are as in and own now of we we we place. into we And forward and patient getting can When a the be renal later to -- dosing biomarkers sharing data, the we looking function. will this a patient biochemistry much

clearly later. year. to with showing the pleased with moving will Our the I next their data very that but program's ahead that able more on be But and forward its progress. is ultimate are hope And that you to reduce immunosuppression, patients data look

Operator

Fargo Our with [Jonathan Wells Securities. next from question Joo] is

UnidentifiedAnalyst

Great. Thanks progress. for on my taking questions and the congrats

program. maybe couple questions on the few a So Fabry first disease just a questions

cohorts the first end are are second patients from patient. cohorts. said total any a many you. has And cohort? patients at be treated year we in expansions see And to as data, you dose treated these those would escalation think when by present to Thank you How in well dose cohort dose the cohort and end. escalating that dose how year the I see in all chance many second patients as

SandyMacrae

Bettina?

BettinaCockroft

right patients will you dose escalation well. expansion imagine, commitment many from the to after to the looking then course, year, And Yes, patients. from two, to dose on patient had up cohort the that we move now. two two dependent two COVID cohort things that be end cohort expecting of by dependent, as we data year patients but the those of the two recruiting cohorts have the the going will of will as patients. dose far three. and be four at cohort three in able are three as we at Data possibility in will cohort, enrolled really first as once end have to will of first least we've per data at Obviously, patients cohort objective cohorts are from and to in accumulated two of will can to cohort concerned, sufficient patients two whether are also have COVID be-- share confident we three beyond need are presenting the how time the and we do two as We cohort the also to be patient

will see. We

UnidentifiedAnalyst

to few clinic. program closer That's the a closer the see great. and then Great is it. progressing to questions TXXXX. And on Got

are perhaps of curious by be TREG? how of of you level okay contamination about you markers? process. Just cells with measure comfortable Is do your surface kind first be T it regulator. the that lastly, And the about TREG achieved? kind seen could you. with with with function T kind tolerated? way a cell of since talk purify it through be manufacturing purity Thank you comfortable assay are therapy, what are the potency this can its as that process, effector you what And could How you And if can

SandyMacrae

can Important that's of to their purity understanding secret questions the TxCell, we and anything that the there and say Jason, sauce the TREG? knowing purify acquired is you TREG. about hope

JasonFontenot

critical of mean, a cells. engineering I and of as we cells is is pure part a the process very this -- the go population the we obtaining Yes, regulatory expansion of into T

in we very terms standard that use, developed, are variety we're so have we a that the kind both that confident of of ourselves. dish and And models, assays achieve purity. of in extremely in function high we're T into benchmark in regulatory excited all a we're the to culture interrogate vitro very what assays in be assays for about to this of that the obviously, animal in so why assays field of with using those we're that's on cell process And clinic. we're cell the And, moving happy function, the wide the we've And forward we seeing. measuring

Operator

Eric from comes question with Joseph JPmorgan. next Our

UnidentifiedAnalyst

on just us. for questions, this isn't a couple Eric. Hannah from Hi, afternoon, our it's Since good

recent safety mediated Fabry with have potential compelling notable, like lentiviral patients, First, they concerns. accompanied been the pretty some approaches by regarding from in showing Fabry data efficacy

on and a And received one gene a be that. I patient gene to lentiviral for after therapy going therapy efficacy? you view market a anticipate it AVV your with we And have with you So there in from meet then what in a therapy to prevent vein, that treated AVV an a setting? commercial with that set that do think expect another dynamic up therapies would Is relates bar ex lentiviral as anything would to have also lower that AVV Fabry? in both might might who's

SandyMacrae

-- a area. you. well, it's Okay, at It's do your like easier thank time. them to just one seems Mark, this

MarkMcClung

difficulty your some hearing I questions. yes, So had

SandyMacrae

and there could the of versus pros therapy AVV. Fabry the is question And approved in with a the them of -- both place cons the So for cell is market? you

MarkMcClung

better any gene the require we at that. patients things one go a be think recovery IV do regimens liver and like the option that, delivered so than system, as to obviously, other Yes, time preconditioning doesn't and therapy infusion, directed having would preconditioning through

whether you the that there would therapy in of getting believe candidate would important But of patients kind I'm of the of make not where a for therapy. antibodies of would rationale cell that terms things sure obviously distribution terms that a one a also would the market. that can their them the We someone or be have into that be have the are the candidate. non is make the not they expert agent a broad whether exhibit treatments, neutralizing in is to

and I answer, we of We more the have whether So won't clinic clinical -- in our phases through work. bio evidence therapeutic, the would candidates. obviously, initial of that would to happen need see terms cell post patients ongoing think in what trial they be

So to they've need once the that reached something be market. be it'll done will

UnidentifiedAnalyst

Okay, thinking Finger thank thinking Technology, level CNS helpful. Do about have we indications, of then ST-XXX. meaningful terms the benefit? just be of strategy how in -- of for and advancement about specifically clinically wondering across you. That's here XXX, clinical any should And what you Zinc idea

SandyMacrae

I'm sorry; Can latter part please? repeat we you question. of just your lost the again, it

UnidentifiedAnalyst

in Oh, for would or ST-XXX meaningful tau be just any protein reduction level wondering clinically if of humans. of sense what you mRNA have

SandyMacrae

friends Biogen. that's our So with at

much that's Alzheimer's, So experts the understand Biogen why we more with lead and they do. And will the partnered in biology that are than we conversation. because they

indication. would now a of And takes code right platform. that you it's capability editors, enhancers, zip offer of zinc it then is So the things a delighted zinc to repressors, whether us the through And matter we of the to that the of collection do. we are a itself base capabilities the the with genome. matching technology set a whole in it can allows crisper of a range to with finger We, finger to attachments, companies of series unique require that attach transcription description, nucleases,

Operator

from Baral comes with Ritu Cowen. Our next question

RituBaral

end Hi, remind dose for again, are was to a a guys, thanks as you critical taking could question. an treat of before enrollment might or could and its pre for expansion the to do I me the of and amount order what of by have on your I'm follow treatment. treatment such pool needed then each you that you protocols could the dating patients Fabry quickly screened when Back pace. patient could that could you gating you for end increase the dose mass get study; wondering that such believe escalation year? that wondering stuff. And do data pick dose certain in patients up there of screened far a I'm that as

SandyMacrae

try Rob, afterwards want have? the could you Bettina, comment to we and do you plans that about

BettinaCockroft

Yes.

in will move yes, of data have look of amount terms mentioned the weeks pre the committee patients have from screened and agency and of we two And we've monitoring I an And at do that move think data So in lined place before, patients safety cohort. the need two up. to to to of we data we next assess data.

cohort. earlier why at to escalation our feel on, that's stage was end COVID confident, I So year the to of permitting, from data the reach mentioning sharing the be able we pretty to that obviously be

RobSchott

the data that. urgency Rob move us less my driven, four and we to so journey quickly patients here, into as data feel getting the can as We'll level. as fast allows, comments do now, than to my weeks higher in about as as I'll completely we as data at we're allows keep Sangamo, treatments a the can,

at to looking and forward the end So we've what carefully sharing look learned the very this at of we're year.

SandyMacrae

And medicine us into Rob And of X. drive is phase get We're one expansion that we're company forward phase works into says, of success. X ensure as for planning the cohorts, the that ready when to we're planning hopefully patients. focuses main for to this

RituBaral

dose a enter that Two be that expansion product? to and you to the quick when the ups you what that. say your when you'll across One, dose, CMC going your treatment final have good is commercial cohort follow you duration are wise shape, in that think to to you expansion pick

SandyMacrae

hear muted. the can' Bettina? I think we treat first we do you? you're Bettina, patient, when So

BettinaCockroft

the patient QX did create in year. around cohort We one last first

And so of of we cohort. worth will end have year for out report we a the by year's first the up the follow towards this time

SandyMacrae

that dose processes, we're is as it's working X as beyond. something into moving regards And one continually the this improve not phase that do And to and we gating see manufacturing in when this field.

Operator

Our comes from with Ben next question Stifel. Burnett

BenBurnett

ask do thank trying TXXXX about am in I efficacy how TXXXX program? study, low about also the CAR-TREG get you. And the wanted Solid thank dosing really out do what's another Organ for expand? Hey, just to program. you expectation cells I you in I Transplant to cohorts much. expect the dose very you like to guess question the think in

SandyMacrae

first piece when prudence TREG guided TREG Jason, never it's that we or patient balance that but don't the given? happens study will to any is what between a of expansion So until to hoping you study, of -- dose always benefit and some get do know. you the

JasonFontenot

TREG CAR encounter for the to the cognate the Sandy. the engineering Yes, We that in patient definitely ligands expand upon with. thanks, TREG with we are the receptor expect

and the to will antigen receptor. tissue, acquire that suppressive expressed, will and And TREG design, to be regulatory immuno get anticipate certainly that by we to properties. the where their them proliferate activated and trigger the their core that through So, when they is

data and what part public. partner we -- shown we've data that's loving that's the see. preclinical And what our the that So to academic seen your expect in and are of we've preclinical the made that loving to

definitely we expansion. So expect

BettinaCockroft

may what I Jason if something to summarized. And has add

to will study trial we TxXXX intend expect or approaches following HLA patient year. year. clinical the study. the preserved. several dosing year undergo genetically of a patients receive his initiating the cells year this but therapy initiating other procedure isolated of will such And And engineered period be we will initiate But terms personalized may result a as as this transplantation detailed after will In and and TREG to And a their which patients the the similar occur certainly next to that recovery enrollment. dosing and year, do will not drug this subsequently from negative so then their of committing cell engineered months this We're to surgery. dosing undergo candidate process, be reset to

BenBurnett

candidates I just you if the provide Novartis also Okay. missed timeline could additional the And then apologies Very but helpful. any on color I if under if wanted for see to it, collaboration. selecting and

SandyMacrae

instead of coming list a with three, laundry shared targets. they we with up to choice on and made decide they They would It's know of them -- their the us. XX potential that targets

and are their know we closely working So them. with targets

BenBurnett

of there? Okay, but in expectation asked should that have disclosing target, differently I any this terms

SandyMacrae

is it. choose choice they disclose to Novartis when That's

Operator

next Wedbush with Securities. Argyrides question Our from is Andreas

AndreasArgyrides

in I and evening. be Andres from know, for and Leon. milestone good Thank then, done just for them we an past, those progressing? asked are indications on are updates guys excellent how throughout what any And those you But discussions Fabry. Thank on how thoughts who've taking et daily our Thank expect cetera. would partnerships, question. you the Two additional is on regularly based getting updates everyone's cetera? we about just How you This can thinking with selection questions. on speaking, and broadly the you. about job And et And year?

SandyMacrae

said safety thank dose won't either any indication you We've we've Fabry that profile give three questions. of doses trial, we cohorts. So, or or all for the until your more efficacy the

will So Mark, we it's end do talk -- to see significant. anything of it be before the partnerships? the want you year about

MarkMcClung

Thanks, Sure. Andy.

$XXX potential bringing a at their they or had really in science more our and take about we recently of many and at in say, expansion partnerships XXXX. or expertise, about can of as way we've medicines should billion that our see commercial would as and ways, upfront therapeutic well addition resources million pharma patients, $X promise the the partnerships And we R&D at with look portfolio. rapidly is in milestones, benefits where companies strategic look in we've an to in the look a across then we and Most chip the a in to So, payments. to got our clinical collaborations way this have I earn Novartis to the for as obviously, bring experience those collaborations royalty to these we these payments, Biogen platforms. reinforce important us. completed provided blue They've deals opportunity milestone multiple financial with These believe

they're So thought technology applying in and they're has that areas things coming our identified us even cases, many have with not disease team targets to within that in experts about to. our

that create forward, So and an believe more we for opportunity value importantly, going the there's patients. to industry to

our creating we a to But really we look we address at And these so a and to can products, take allow that through have the Sangamo expertise of on all take want to which partners, now to where way and areas. real we'll patients. underpin in those wholly owned, portfolio continue will a leveraging resources are markets, focus

AndreasArgyrides

we question update year. program stands where the the the to until to cetera, correctly, expect no clarify, just the then provided phrase and to data Sandy, And on that. you whatsoever on maybe have Appreciate didn't of I end are updates cohorts, the until et also dosing, different

cetera. So the we calls questions any in et ask won't future

-- know to Just

SandyMacrae

Let me try and navigate on.

we understandable kind our see some or will But dose we experienced three or calls of progress the and we results those things data. story can is the are the the study. know is of you at won't simply biochemistry quarterly more So tell you of tell if period you because and cohorts

as to to as want you get we possible. this quickly So

to forward speaking year. look end of the at So the

Operator

last comes Securities. our And Debjit Chattopadhyay question Guggenheim with from

UnidentifiedAnalyst

Thanks for taking on Hi, this questions. Aaron digits. is four guys, my

So there on to poor They are the them or as, to expect not opposed treatment responsive going perhaps the Fabry they push would to enroll have the are program, or can ERT other towards gene haven't you you perhaps using renal in therapy, a sorts remaining talk may patients ERT? that such of on types ERT. symptoms --- we if as And function of to these about

SandyMacrae

any you patient. given the thinking that from guarding of about and about haven't a the because choose one with very carefully it's there you studies great -- are We categories of these each of type input outside what -- detail is the is It's several experts. something patient. And companies for Fabry

UnidentifiedAnalyst

any regimen learning if at comment trial Haemophilia from about then Okay. all can incorporate you being program. Fabry into And steroids if the using in the you the

SandyMacrae

Fabry, we said learning to we're We've learned the liver and haven't applied we Fabry. lot in we hemophilia but have and from a in about function of steroids do tests use what what doing

Operator

would to our back for session. remarks. I the to And call like turn Aron the Q&A Feingold final this concludes

Aron Feingold

great us today Thank your developments. have to again and you forward joining for you We you future once our on for all a keeping questions. updated night. look Hope

Operator

for in ladies we program. you with And thank today's participating gentlemen, that, and

night. wonderful a Have disconnect. now may You

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