SGMO - Earnings call transcript Q3 2021 | Sangamo Therapeutics, Inc.

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Operator

Thank you all for standing by and welcome to the Sangamo Third Quarter of 2021 Teleconference Call. [Operator Instructions] Please also note that today's call is being recorded. I would now turn the call over to your host Aron Feingold, Head of Corporate Communications. Ma’am, you may now begin.

Aron Feingold

Good morning and thank you for joining us today. Officer; on leadership are Chief Scientific team, Mark Chief morning the members Executive Officer, this Financial Chief McClung, executive Chief Medical of Sangamo including Operating Bettina Rob me Schott, this Fontenot, Development; several call of Officer; Officer; Head With Jason Duraibabu, and Chief Cockroft, Prathyusha Sandy Macrae, Officer. can Media website, sangamo.com, Presentations Investors and section Slides the our page. from under on our be found corporate and under presentation Events the regarding statements forward-looking This current expectations. includes call Sangamo's These candidates, statements limited of our other facts. revised not statements plans of include, data, for but are to, guidance clinical of statements financial potential that XXXX timelines are presenting collaborators execution relating not advancement commercial our and our conducting product of to and strategy, historical and clinical Sangamo and the candidate, therapeutic and our our trials product anticipated corporate Actual we discuss results today. materially what may from differ risks September with annual discussed to supplemented are fiscal and year by XXXX, subject that are as Form on our ended report SEC, statements the fiscal December XX-K for XX, ended report for Form certain the These XXXX. XX-Q year uncertainties quarter on the filings XX, specifically quarterly our in our are today forward-looking statements this such we date and no as The stated duty except required made by of as undertake information, update to law. On this call we operating non-GAAP discuss our expenses. on website. operating be in found our release, can is our expenses of this measure available GAAP to Reconciliation today's press which

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

Sandy Macrae

several and in towards to Sangamo across programs we have stage is or an for such good updates as that assets Thank development. This late important we you, data important clinical Aron, everyone progressing on demonstrating morning call. the three and share moment business

is advancing efficacy accumulating Hemophilia Our programs. A data in gene and our Fabry and therapy safety portfolio with

point and study require finger sixth that through abstract The nucleus post XXXX companies with the beta in sickle The announced preliminary year. cohort expect for Sanofi. from data engraftment sickle the study, Data obtained we from The a vg/kg the percent disease the this Grade dose X/X proof-of-concept cutoff data the was ASH. or F to were Further no results X/X we results patients. which patient. zinc to two civaparvovec to were events data us and four focus treated date the to allowing And hemoglobin, one two and a XXth. XeXX preliminary program. Interestingly, from the this cease symptom advance further last proof-of-concept Based evaluating the follow-up ERT elevations, the activity the showed preliminary patient patient announced initiated from Sanofi gene These all the or of X patient tolerance measurement ST-XXX, cells. throughout recently planned ability with in and who in cutoff first XX total provide hemoglobin at the study events the as patient as patients ranged these SARXXXXXX, cohort. date. patient treated the limits or program. thalassemia friends Those the recently above There for in maintained decision weeks patients alpha-Gal patient. ERT first of for finger XX, our adverse is now for present serious stability events. in clinical patients to at in be alpha-Gal also first on Sangamo adverse we encouraging is increases following screening Separately, serious first treated patients one technology indication patient business required disease September at as we of currently a the Phase our This patient generally for is than enzyme patients date their cohorts. in of four poster weeks morning, of STAAR patients and treated for an gene engineering, adverse to in three that steroid related meeting. four XX clinical above higher treatment based delighted of in Phase recently data, potential cell edited treatment. of investigation XX and none were that third first important demonstrated experienced withdrawal guidance four the are the results XXXX Sanofi levels events therapy date. improvements and fifth program fetal adverse that disease candidate. other disease blood provided study, data the to to reported treatment data the disease. and also are with This Fabry requirements development PRECIZN and product from partnered up December for disease made Phase the treatment. experienced common No dose A as of tolerated. we've [indiscernible] on the continued A the these at on studies. exercise well transfusions no FDA of on reported normal the complete primary sweat presented filing. most four we This cutoff updated is is our ASH will third Fabry cell will dosed. year activity, fold cell cell XX in for our were genome June the exhibited through zinc liver treated X, longest treatment cell related All in The the Additionally, resources cutoff sickle patients morning, and of from normal potential evaluated the for preparation Levels at ST-XXX The presentation for isaralgagene related was on No levels dose XeXX therapy at sickle manufacturing clinical medical the showed

partnered program morning, A Pfizer, at we from with the Hemophilia study this our updated announced onto of ASH. Alta presented X/X be now results will follow-up Moving Phase fitelparvovec giroctocogene the

For followed the VIII XXX XX.X% year was throughout the assay. this measured for at in the four and cohort May after have activity Factor follow-up. dose of was for total cohort, who zero week treatment as least as XXXX XXX patients bleeding In Mean duration first the been X.X of rate weeks by chromogenic highest XX, cutoff. annualized at

FVIII experienced patient Pfizer as Pfizer On greater clinical with has will protocol announced treatment. Out A. therapy than management Pfizer none guidelines of direct some December patients FVIII caution, provide this put have in resuming the dosing screening, possible. some at of risk. paused patients clinical following of Phase events anticoagulants tolerated levels. abundance study, in amendment, voluntary patients the have gene generally patients and to oral Further for well these to XXX% in poster of the the presentation that and soon on provide XXth To an reduce been which trial today committed a XXXX. that hold. X also in data to AFFINE FDA was of November thrombotic on We in date, demonstrated the the was the the ASH As dosing severe Pfizer with hemophilia and as implement experienced Xrd, treated a treated levels trial thrombotic informed are will trial Sangamo elevated order of in and

with with trial. for for gene patients We share which hold represent an hemophilia timing important authorities A. health the treatment to this continue after to protocol option provide respond will will be to step amendment the to the is proposed companies and therapy believe able The next that updated clinical the

to program. kidney our now transplant Turning

their patient believe a have Phase evaluating first now STEADFAST will period this receive isolated, kidney TXXXX, cells to autologous cryopreserved. and procedure their autoimmune therapy negative engineered Treg CAR this similar TXXXX a therapy personalized our this patients Treg recovery that be promising challenging will and and first candidate. the conditions. inhuman genetically X/X In The Treg approaches, with therapy. in leukapheresis undergo approach owned subsequently as for engineered, excitement We will is study study, enrolled study, We from following field is undergo other patient the will which that CAR transplant cell growing HLA-AX much cell wholly our then

patients first to this by middle XXXX, two of dose following their in the expect study We the transplants. kidney

will We advanced the process important CAR renal We as represent development continue patients. portfolio therapies believe study understand in patients transplant to us this that sites Treg CAR undergoing owned knowledge. help indications. screen this open Treg foundation of for may We and as a and treatment hope establishes well autoimmune for study humans, proof-of-concept wholly for an biology

our genomic December. marketplace. well Prathyusha expanded Sangamo, Mark a updated important working to Operating been that Sangamo this turn bring effective the medicines XXXX, the We update. share for present we over to patients presenting I'll to ASH and the thrilled with the is as momentum. role forward Officer McClung Prathyusha? we an data owned as at through the with COO And continued call support results to continue collaboration which in X. will with and capabilities to November Mark's wholly build I'm Finally, as look Fabry the for as to step Everyone clinical leadership to that, investigators Sangamo’s forward And programs. delighted and partners look to advancing partnered to Mark's multiple appointed as Chief has organizational financial

Prathyusha Duraibabu

and good Thank you, morning. Sandy,

pre-clinical in this cash to the including at exciting we we can approximately and sheet for our balance want in found in the us quarter as $XXX this company end quarter, and financial remains and strong. TXXXX, million quarter, Our a capabilities. believe for results this Fabry are be equivalents to that issued this that also release morning, With clinical invest research of continue website. and press our is which on advancement an I our of our moment cash, our the pipeline in-house reiterate the marketable manufacturing programs, securities available

year Turning guidance. to full XXXX

the expect million guidance to This to back have of the compensation for previously is that million to closing expense $XXX $XX provided. operating call the we be of between Sandy We cash excludes now the turn expenses stock-based for which estimated range year, I $XXX non-GAAP non will within million. remarks. range approximately

Sandy Macrae

you, Prathyusha. Thank

have delighted the data, four the quarter. manufacturing France facility in in Phase Phase and study of of to ASH generation CAR including the preclinical headquarters several first patients in X/X medicine advancement positive additionally, our readouts. gene protocol focus of in STEADFAST forward updating therapy, for California respectively. with a proposed momentum completion therapy. for and a data. We once manufacturing are presentations and in-house and Phase potential the allergenic generation XXXX clinical Phase dosing therapy X/X the hemophilia genome the and in-house hold autologous authorities, Brisbane, term traditional is show neurological near we'll completion And about this very cell XXXX, the where a two-year presentation you next patients Fabry in of paved our sickle with throughout shared autoimmune data of regulation excited the and pipeline our Valbonne, the provide past X/X pipeline and at following potentially first cell update with dosing These genomic patient updated therapy X at meeting, to Phase about trial health middle programs data on Treg these way six of robust amendment location cell the our X/X We of medical the response ASH look including We're diseases a catalysts of two presentation disease we our facility by timing cell at

to questions. So open the operator line we'll to for now turn over the

Operator

we Participants, America. line [Operator is first speakers. will Instructions] the of the Aspen of session. you, Bank Thank question-and-answer from Speakers, Mori begin now question of

Your is now open. line

Aspen Mori

Hey for question. guys. Thanks the

to I than in replacement getting one is think that from Fabry, I that appears some to a patient data from maybe a to expected expected. any that end you're it in also enzyme dose see on follow that a removal milestone kind hadn't guess, bit of with of got up. seems timeframe timeframe that the ST-XXX withdrawal than year therapy So patient in therapy? share But And faster longer the good four the patterns you takes had who seeing the there this to little release. It's mind, it maybe of previously, to quicker

to wanted there there's see if you. explain you're seeing anything to Just Thank that?

Sandy Macrae

question. your for you thank So

I'm what ERT is think about I hearing about asking you're withdrawal.

of treated important appropriate it effect. a been And to So patients, we year understand that treatment. thought see the this been consistency there's time. the will so for withdrawn And only and and appropriate the the they them the recently. have course to that longest effect very discussions is truly withdraw was second and we've very of data planned we share patient their will the at pleased was year, on with one a over And And duration. first investigators then to and withdrawn patient, the point be for we

Aspen Mori

you're if with there's there? three? have thanks. one would alpha-Gal might maybe among on seeing there and patients Okay, that on more what's then that point two And driving you three response but a A than can lower two think you're I Are I patients had out anything a four any seeing trends baseline signal and GbX, – the to have better one and help Fabry.

Sandy Macrae

so only Well, four patients. there's

not. ERT notable that some additional think two speculation ERT are patients that to to only on and understand though leading that the more to is effect it's it But say I from may is and So are difficult very patients is specific. It and three it. dose four patients the effect. And and be need that is we there one to anything

Aspen Mori

Thanks, it. I Okay. Sandy. appreciate

Operator

of Yanan Zhu Fargo. question Wells is Next from

is now open. Your line

Yanan Zhu

maybe be a in Hi, I comment two, substrate thanks for Fabry, for enzyme Fabry the questions. but and I other that also updates. also think the response to on levels my dose additional there seems questions Sandy question on here normal, increase and – But was on taking above on patient flat. levels you could have

light some shed you that could Thanks. finding? So on

Sandy Macrae

on the the has been dose seen that's consistently been good don't a they If levels into the of change treatment And your something this But period. response question. research across thoughts disease. all we've I that are this think the substrate it's in low, Fabry seen? on So Bettina,

Bettina Cockroft

Thank you, Sandy.

dosed needs off but regarding two cohort lyso-GbX patients the low. So patients This have in lyso-GbX And And Sandy sure, the started to a as cohort levels be as you maintained this been remain they lyso-GbX it small that with lyso-GbX, time, at levels XX of reduced emphasized to in mentioned size. two we of reduction those This three, one by about are XX of three seen patient's in weeks. this Sandy after is across still And mentioned, levels now point patients this patients, sample XX% two. for look at – and four levels stable, were these low was start through start high. programs, the has dosing. if within if weeks in off

relationships. So is time at there out this a medical such so point more to in difficult to more with about will that really anticipate and data small it those data sample and be granularity meeting, that we a throughout come. also talk say data be share shared would I XXXX, will size, to

Sandy Macrae

four been four are the served. excited you to because want level. away effects we we already the they've as physiological very is shown we'll I higher patient about are normal And this the are encouraging of that how response patients the All next says, four But drug. of Three this all year. patients about And cohort. take are next by are in patients time of the effect into talk the above consistency have reporting Bettina already

Yanan Zhu

XXX% the thanks. on hemophilia how And we program, had from hemophilia A, is seen clinical Great, for like I studies. the hold wondering level? A VIII thought I the was Factor high collaborated other

the for patients? shed we And – question VOC lastly, could for the Thank is ASH, BIVVXXX, that a very some see So that light you. at one the on rationale a hold? you treated on quick data for clinical would

Sandy Macrae

Rob, talk the to this? question So hemophilia to I'm you pass on can Rob. going to

Rob Schott

patients. was this. a with elevations hold resume clinical the we're submit levels trial trial and very an precaution. abundance with X were with to the This an It's followed Phase plan seen events pause is associated voluntarily appropriate of thromboembolic with we discussion do venous the the than FDA XXX% getting of and to Pfizer through so. amendment around have our have not VIII data notified protocol decision elevated greater And to the in so specific with ongoing that the trial, when agency we a were was late that is the in to of some as it yesterday. Factor Yes, anticipated, no these There out

Sandy Macrae

– sickle in And ASH data at regarding set we'll have December. show we cell full disease, the

Yanan Zhu

Great. Thanks for that updates.

Sandy Macrae

Thank you.

Operator

question Next of RBC Luca is from Issi Capital.

Your line is now open.

Luca Issi

comment Oh, maybe much proof-of-concept. On how early are X the for Congrats study color wondering maybe progress. bit protocol risk so design? great. Fabry can disease on the if you much. beta so May just A, my thinking Thanks you on be hemophilia then about if behind rationale the you And a about thinking additional any exciting taking question. little all you're you the on on on Thanks can I forward? thal, program? expand minimize to that can a terminate amendment going under provide decision to Phase a

Sandy Macrae

Goodness.

to exciting So which cohort and see we to X with programs. the about third behind be the of are planning already the because results. we But study. energy we can started design reassure to the how forward you Phase I has on it's and to dose understand able these pleased results taking so the very Phase full we're designed need But that be X talking really the

beta the me Let then hemophilia one question the to Rob thalassemia pass to Mark. and

Rob Schott

the clinical… disclosing agency considered promptly this Yes. discussion submitted is to at With will the amendment, time. amendment specifics the be amendment any But to regard the of of the being protocol in response in protocol and we're protocol not the and with

Sandy Macrae

And a find management guide plan that best to has the level. the any of is way to higher current patient

Rob Schott

are with patients the XXX%. levels of management greater and VIII monitoring of than be that there'll Yes, Factor

Sandy Macrae

has adverse be abundance conversation events to And is a it a Mark? And this. comes up, restate I every is this to no related harm. therapy of we need best gene that caution. feel abundance important for and time seriously to do no come patients. of the because the to There'd patient caution correct this very think that we But an take

Mark McClung

Yes.

sickle we decision of timing felt competitive set relative the thal it it where focus in their lies the our beta resources development. really on business partners cell terms Sanofi and decision, that of to that took the really and at a disease. to in So to due We terms effort important our solely was is just our alongside

Sandy Macrae

together. forward drive And

Luca Issi

it. Got Thanks much so guys.

Operator

Next of question Jefferies. from is Raycroft Maury

now is line Your open.

Maury Raycroft

and update everyone. Hi, the today. morning on congrats Good

wondering can could than guess what's you about data six way competitors your data fold best the kidney competitor to to higher XX substrate and For the if this fold I at data higher. Fabry, more how talk just month seems alpha-Gal to two five being translate reduction? than to XX about showed higher versus normal, that contextualize

Sandy Macrae

want Bettina, about Maury. you to you, Thank Fabry? do talk

Bettina Cockroft

Sure. Thank you, Sandy.

alpha-Gal So with expression in to super the excited we alpha-Gal all the obviously terms that we're physiologic four very are of patients. data, see seeing

place look more in data early it XXXX. compare granularity to comparisons this meeting I on sharing forward an think at at across do point, not probably programs. to and this upcoming is to We the right

to additional data that to also any related So goes kidney substrates.

Sandy Macrae

medicine You is, enormous data. for data and that it find early though, is we it this the compelling compelling and into to efficacy a gives It patients can Fabry. is initial as going it energy us cohorts third seen higher imagine really with useful moving be having will at that be that two cohort

Mark McClung

A on drive to we expressions And I tissues. clearance would super priority, and gal from targeted of had alpha plasma that add physiologic

this what we're a seeing. So desired is affect of

Maury Raycroft

gene maybe and observations talk contrast that. can more all expression program. Maybe Got just hemophilia compare your can you gene it. helpful. follow-up, about with one if dose and the and you Fabry therapy And if That's

Sandy Macrae

That's a good question.

we've the across that programs understand programs, of we've very used several of now in fortunate events. it. safety and And all we are AAVX adverse had We few the

program. there's In the of even than safety hemophilia, Other gene addition, which EXX in the in now at disease it's pleased the look one and it's that earlier that, Fabry if seen three an to and we've you therapy about efficacy program. our two efficacy, with at point very we're inflection than the in

Maury Raycroft

Got it.

for Okay. again. questions. Congrats my Thanks taking

Operator

Securities. Truist question is Next Nicole from Germino of

Your is now open. line

Nicole Germino

questions the Good morning, congrats on taking and thanks for my progress.

bit So little dialed some missed I in and I a remarks. might late the have of opening

can baseline A, happen about to have But these if long So how are questions for cardiac already I how assessed from on over going time excursions addressed. my talk any and Fabry, from were how the Fabry did improved for forward and you color infusion function? primary Heme symptoms levels often apologize being And monitor?

Sandy Macrae

little a unclear. So your line is

So the let and patient I back read is we to about your Fabry, understand patients me in see correct? it's question hemophilia. how the symptoms that about – Is manage in see, that

Nicole Germino

Yes. And the from long happen? how for excursions these how did hemophilia, long infusion

Sandy Macrae

in what we So know reported patient Fabry symptoms about Bettina, say the can you please?

Bettina Cockroft

course. of Yes,

as three that hidrosis emphasize sharing, to we're reminder, again, had the patients reported preliminary patients, data, So, a early data is two, four at one, the I'd of like this baseline. an early

these patients, reported have patients for over these have in of three improvement some study duration seen the which patients we the course that far. And in outcome favorable post of an so the is or time sweating period

seen the had of on of imaging. on structure patients serial also cardiac have One assessments stabilization

we of So information of preliminary more reports And mentioned before, year over are we are details. of be these gathering. will next sharing course some I as the these that

Sandy Macrae

the observations the the pointing of expression, add any all totality the look including if in at to adverse like events, the patients’ that all investigators, that are that the direction. data the data, I'd alpha you of to gal relayed were favorable

totality, you component. look at not its So just each we like would data in to individual

Nicole Germino

Great. excursions how the And infusion the long Heme then A, did for from happen?

Sandy Macrae

Phase We This potential trial, specifics we it's to want X that data. keep ongoing aren't is disclosing trial registration Phase X a the protected so trial. around because the an

Nicole Germino

thanks. Great,

Operator

Barclays. of Wang Next question is from Gena

open. now line Your is

Unidentified Analyst

Gena. This the taking for is question. Tom Thanks for

question patient A? of about data and plan higher how that the change your to who Fabry, enough? you Heme level for in the depending withdrawed the dosing goal level gal readout ERT level, is alpha serum and the the what one enzyme And for the the from alpha enzyme after and the physiological current just super on dose gal automated with So do is

Sandy Macrae

So your alpha never be the disease. Fabry must do we make we really therapy in that is We've sure Can Fabry this it questions. cover people Great first? done have because time questions. set gal we all first this gene for

it a patients eat And we're there on to levels. We've and the level. than EET always And And had so are to pleased at level. isn't both be physiological definitive ERT thought better is what off normal we doing now the are by and where levels until understand the out. that and ERT gene seeing the And won't level this we do provided fully. withdrawing liver therapy the experiment, that we really

dosing into compelling XEXX, to which is so important to the dose outcomes we are we also to current patient because as And like Rob exciting that that gal, about three safety, totality, with investigator the take broader them, are first that compelling felt the they X the to than that once be times in things swear from share ability were felt patient had decide related that We can with to alpha with see and the data wanted two we that came dose. at forward we'll patients to with says, data was the and been, but it now its spontaneously Phase the cohorts which higher exercise, talking community. this able

Unidentified Analyst

the medical that be you set up very meeting. And the will including inclusion set data or will help be on quick that? That's that just follow include the an will EGFR, helpful. data, to GbX share filtration that us XXXX function in great. Okay, expectation additional Fabry for it in comment clinic – the kidney measurement. you Can you maybe And function

Sandy Macrae

It's then months patient and an undergo one. biopsy we'll unlikely biopsy in a renal the later second there's would that sharing some be initial XXXX, data

XXXX. is data that So in available be to unlikely

Unidentified Analyst

for steps there be require would other that be Okay. Heme any the for the that And the the amendment. order do anticoagulant to oral than expect will of that. hold one the will in be, remove that required? or so know implementation only you we clinical be additional Thanks protocol A will And

Sandy Macrae

always colleagues be And an agency. with in will patients the able confidence our makes together to put the Pfizer then puts first enormous it move and that that we'll sure trial. they safe. to hopefully patients have I that this discuss present that and amendment And with forward remain

said, had a As into the patients Pfizer to have to and them been the pre-study ready of now have to an trial. have enrolled XX% patients, the over they've collect go

sure will Pfizer's that So doing thing. right the we think are and this move forward

Unidentified Analyst

that. Okay. Thanks for

Operator

from Next Baral Ritu of Cowen. question is

line now Your is open.

Ritu Baral

for Thanks guys. the Hi question. taking

four your expression of wise? X/X, this asked life there that up you confirm anything the seeing you're and of clinical can capturing patient of clinical like symptomatology, you You reported proactively that Phase curve sweating. of sort in sort fold then are you a part for other way about that continuing As the like week other that say guess, what being Can at are patient Are one about the I improvement things that patient can XX? that that's on you're for talk all XX top of or mentioned expression curve? And quality clinical you've seen Sandy? symptomatology you and at

Sandy Macrae

Okay.

to to the This important. was things, tell spontaneously it So changes their spontaneous very their formal patients clear. we measure whole they’ve known. us We And was compelled them of a outcomes. the to because investigator – to related – patient felt talking tell want including investigator be slew that of will of

capture it’s about it’s And the about their the they the like neuropathic It’s to because all things. sweating, the We important that pain just important about will not we energy these quality the want this X. in have. their and is on things Phase like it’s life the patient and what’s of what’s to measure so going enzyme and

has at study the the of fact. and patients it its to because the long I question, after as pleasing think of to very that patients level continued in continue this And we – to that remained the want duration of as the it we will – those duration the duration patients. solution us the possible plate these gets asking level, for that’s Second once you’re report throughout hopefully about is all follow is a for and

Ritu Baral

regulatory Are or Got just to in going – months biopsy the is there are next you in I I Fabry – study? to for mean? And this the to the last kidney it. a the XX over study that given any plans so. guess change Or landscape add the be biopsy reserved potentially

Sandy Macrae

biopsy this We’ve that be the we not initial we of for would part in phase. was biopsies now the always the study. will will plans medicine saw have the be we results that in effective XXXX biopsy said just available effective of do study. the It’s within patients and not the medicines routine that until the see We the

Ritu Baral

Got it.

Okay.

maybe biopsy the with and plan? but the Is highest these the that six patient patients, So maybe dose. not these from fifth

Sandy Macrae

four first renal and the have did So future biopsy will. not patients patients

Ritu Baral

Okay.

Sandy Macrae

the study of part As protocol.

Ritu Baral

it. Thanks taking questions. the for Got

Sandy Macrae

Our pleasure.

Operator

from Ben of Burnett is Stifel. question Next

Your is open. line now

Ben Burnett

Hey, you congratulations the will a data. echo on my I to I Fabry did this. disease want much. very ask couple around thank questions

lyso-GbX in agree data would on that lyso-GbX, guess just of what’s two is specific of one, the I good impact I of one, more just the is first happening the plasma I activity inside interpretation seems guess wasn’t what is the It how on But – how and The patients enzyme profound some competitors. four. representative curious to those with to and others the like also call. your on least quite cells? relative at

Sandy Macrae

for of that in of an that lyso-GbX low, make to a indications from what’s all that that’s are effective benefit kidney. that’s hard And having in producing already don’t and And trials. Good showed a reduction GbX at in example, is we that effect effective GbX that the very go have period AVROBIO They question. and who high producing us across it a the They lyso-GbX have had is want. in again, the very trials a show be we’re won’t of one-time it’s to the we also reduction lower. the already Until it all and And alpha-Gal done this that seen. low safe, even notice that what we that of have the lower. plasma. I’ll in We go yet, in over even each patients want injection who is competitors say trial, a at think system it’s showed to It’s a If patients that here. correlation. producing is Fabry our biopsy, time. they different trials direct long

need that benefit then decide full patients soon go more X we together hopefully as the pull to of possible. design hire as this data and show even dose all Phase and the this get medicine Now to on and and for to next study

efficacy, so benefit We we’ve pleased patient are with the consistency, the seen the and safety, far. the very

Ben Burnett

Understood.

therapy? activity if That’s XX, just show but at that clarifying made that? good, assessment looks, weaned think after was Okay. of helpful. one you this week prior four on question patient replacement could an were was I off sort Or ask enzyme alpha-Gal that And I of they assessment really to

Sandy Macrae

Bettina, to that? can clarity you just

Bettina Cockroft

Absolutely.

withdrawn from four number has have cutoff And that on been ERT recently. you’re only our we patient therapy what these corporate So posted slides very data for seeing after the website. on

trust all clarify, And on ERT. is So the at the you’re with the ERT just patients data level. to still points data are seeing

to is So the ERT dosing infusion. was prior

So withdrawal data will after been the this cut. have ERT

Ben Burnett

Okay, okay.

Sandy Macrae

think the to a is And is had and of the all declines part the the level, think be make trough about ERT dose offers we next there before rapid patient the from their is point ERT I we is about enzyme ERT, but different all reason to of particularly to something that careful. last companies need residual trough making wanted that’s in solution all studies careful than the at day sure the therapy no in trough. the very was we sure this different plasma. ERT has be better very the quickly, it’s after Cmax it And XX therefore measured I the And a then usually that –

Ben Burnett

sense. That Understood. makes

very much Okay. All you right. the for Thank color.

Sandy Macrae

Thank you,

Operator

Speakers we for one Andreas have time Argyrides from more of Wedbush Securities. question

Your line open. is now

Andreas Argyrides

you questions. morning, Hi, thank our and good taking for

Yes. Let me this on encouraging our reiterate early congrats also data.

A, for does just And amendment protocol may data? preexisting restart around quick have. ABR on the a you. the potential the So the your the for any hemophilia one thoughts Fabry, then time impact antibodies, on Thank that

Sandy Macrae

then had these Why be gene therapy. to one Bettina. answer pass are treated let the seriously patients this me the So second to antibodies in was

even the stays the trial and their is get when they So in meds paused. if it body,

is by collect being of There’s from collected and continue paused. about we the Pfizer medicine into will so prolongation still data patients, recruitment even Pfizer. to the though and And the effect safety information of

medicine. to trial compare So it’s still taking where patient continuing there pause the the and stops is unusual to we for the the a medicine patient The have benefit antibodies? hope. Bettina,

Bettina Cockroft

Yes.

I as time. And of are patients on to whether data of to are is emphasize need point antibodies, a is, it that effect an patients. at and make these We antibodies on this so what inferences to mind think what this to early patients. not. having four important to in data in degree lot in are is antibodies There the bear is is For these on early ERT to two variability the

So better being that data forward manner. cohort, next in able we coming to seeing to assess X, more from and our out impact a look Cohort

Sandy Macrae

but And haven’t correlation any optimal. we we anything, seen –

Bettina Cockroft

say. would be At this difficult point in to that time,

Andreas Argyrides

Sure.

you encouraging Thanks. Understandable. congrats and Thank Okay. for taking our on again questions that data.

Operator

back I’ll call participants. over to Feingold turn now you, Aron remarks. for a Thank closing

Aron Feingold

you questions. today joining up to – for on our for and again updated once look developments. forward future keep your you Thank you We keeping us

Operator

call. today’s all joining. concludes That conference Thank for you

now may You disconnect.

Sangamo Therapeutics (SGMO) 4 Nov 212021 Q3 Earnings call transcript