SGMO - Earnings call transcript Q4 2021 | Sangamo Therapeutics, Inc.

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Operator

Good day and thank you for standing by. Welcome to the Sangamo Therapeutics Fourth Quarter and Full Year 2021 Conference Call. At this time all participants are in listen only mode. After the speaker’s presentation there will be a question and answer session. [Operator Instructions] I’d now like to hand the conference over to your host today. Corporate go of Head Feingold, Communications, ahead. please Aron

Aron Feingold

including for today. candidates, corporate call statements Mark to strategy, our relating the product trials and conducting limited not and With to of therapeutic our our and with afternoon clinical commercial Head what timelines Development; are events leadership page. Medical several and of Financial are from expectations. this McClung, and not us risks Jason you that in from of our Actual to that other but thank team These the collaborators are regarding data. materially include, candidate, corporate executive our Chief website facts. Prathyusha differ Sangamo section, and Officer. of filings this on Good Sangamo’s and found subject under Chief me Cockroft, Officer; are our our and Operating clinical Executive advancement presenting guidance Duraibabu, plans joining presentation potential afternoon Bettina Fontenot, product of uncertainties Chief statements of may our certain results anticipated and and discussed the historical Sangamo initiating Macrae, the Officer; the statements today. Sandy Chief members Chief we SEC. Officer; for and statements Execution financial be XXXX are discussed Rob initial Officer; on This investors forward Schott, media call current Sangamo.com can looking Slides presentations These includes Scientific statements

release this On law. press GAAP to our found is of website. expenses measure today and required undertake update our available as Reconciliation in information our this XXXX. can December the for of no duty fiscal this The date, call, today’s annual ended we our Specifically, be which on XX-K as operating Form operating except forward-looking stated statements made on discuss expenses. XX, to such are non-GAAP report we’ll year by

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

Sandy Macrae

I’d Thanks using Aron technologies. significant start and continue areas good saying by afternoon for year call. therapeutic XXXX innovative genomic to a as advance of to that on multiple across Sangamo our patients we for like to the the development medicines everybody was

We’re challenges our the pandemic. year progress despite with very the second pleased as the of

We’re indications advancing potentially of genomic clinic, facilities. These to and our AV efforts cell therapy capabilities transformative are pursue in-house, medicines need. using our by the unmet in supported infrastructure, R&D including and manufacturing strategically

we promise progress as for also upon generate drive executed our medicine. genomic Our And that several with help we of on important collaboration achievements. us believe positions towards this XXXX, shareholders. In the long-term partners to strategy us our value mission deliver our to well

partners our Phase our clinical Fabry presented month. earlier for program, owned this advanced we data updated three presenting disease our Starting wholly and we compelling lead First, with the data. programs at X/X symposium World

sweat by encouraged some the challenging to in Investigators to-date. three have the the safety importantly, report We’re better. study and of we that feeling observing data symptoms, including patients patients. in And seen improvement, was treated they’re the them first most ability efficacy

Fabry we year, date. competitive of half updated Phase plan we’re to changes additional the In recent X/X landscape, the position. With second believe the leading a this in present in we

cell long-term candidate dosed study internal in been increase improved a by across shown patients no the reduction importantly, at treated ASH showing with several December, biomarkers, improvement have and clinically the We’re most X/X product cells. groups, manufactured patients and actively painful planning experiments crisis. the X Were will next sickle We sickling and patient anticipate health the investigators. methods for Phase discussions four treated significant to using that of adverse disease advocacy the a emerging four presented authorities, data study, delighted events including Phase with treatment be also that in in in progenitor

to expect third as of energized the partner. Transition this quarter Sangamo potential to of in move program hands for the asset planning in program from assess we well, our that a back our have way year. these on of is this own, we the dosing soon We are forward with going best patients patients and be the complete Sanofi to to or

sustained by partnered last ASH presented Haemophilia with the gene program Finally, Pfizer. at following results we’re in encouraged Updated highest A two X/X years dose the therapy. from cohort follow show our data bleeding year control Phase to up

treatment. authorities experienced to XXXX. X and Regarding Factor Phase first patients the The of process their have health AFFINE in conducted, been than enrolled was patients VIII some with trial. Pfizer following authorities paused Phase the obtain of submitting the trial X been study, the protocol XX% announced health in the to previously where responses greater is the a preparing this that hopes activity in trial of resume amendment the countries agreements Over XXX% it the FDA clinical the in hold. of of to trial has AFFINE Pfizer currently has when half to

we’re progressing technologies, second Zinc neurological autoimmune preclinical and for generation Finger Transcription our Treg Second, for disease disorders. our candidates CAR factors based on

expertise transplant medicines. technology be elite operational mediated CAR offer can first and the that in synergies We facilities position by a to a believe comprise where genomic we our prevention internal are the with engineering Treg capabilities, with mismatch are rejection that supported genomic for manufacturing cell for and that robust program strategic platform first immune in and evaluating CAR of CAR enrolled kidney have multiple patients into dosed companies donor. differentiated infrastructure, following to a Treg HLA-AX living therapy in this leading dose and We we this from advanced Treg soon expect will ever area. and promising several a patient platforms very our believe which TXXXX us We across potentially patient the therapy we be our

our And factor study, technology inflammatory bowel sclerosis year. allogeneic Biogen, study with of data we’re we last multiple preclinical renal and concept proof IL a zinc as CNS to transplant our as of progressing IBD programs, the regard including are addition in and transcription preclinical first to well TXXXX, from in candidate disorder disorders, presenting addition advancing in Novartis, In rejection our finally, with internal multiple programs protein Treg programs. Takeda CAR partnered in treating Pfizer, finger our to XXR allogeneic

our Third, we platform, precision, genome and hone differentiated specificity, zinc proteins. efficiency engineering continue improving finger to core our including the of

these for a to designed therapeutically. to resources and to research nucleases partnerships advance that of development progressing mechanistic preclinical our the our a completed including activators, more development strong our facilities high us develop diversification, our partnered assets, our manufacturing strategy we have applied take and flexibility therapy and progress. continue quality and catalysts. potentially Fourth, chain capabilities. we position to many domain expertise supporting we approach driving providing of from believe from pipeline to excited for transformative and We editors These programs while see resilience, through validates range representing of programs our we and reimbursement enables our modalities, a of and by control, and our quickly. internal intellectual also stop shop capacity needs, be partner’s with diligently needed facilities buy We about They’re our cell from portfolio. for R&D supply clinic, advancement geographic a our capital with We We’re capabilities to to therapeutics financial across are receive advance for supports cell in in believe as And value work believe partnered these engineering bass even one while partners. us Fifth, drive patients. which genomic therapy advanced from capabilities operational strategic partnerships and that the have continue provide Sangamo’s helps in collaborators brought and process our repressors, the now of overseeing deep a the a AV substantially component therapies have of incoming the property range provided efforts The advantages, for to cycle. programs Brisbane key facilities a a been online can pharma benefit support we in-house. Six, that Valbonne. expertise,

of mission, are colleagues team programs for in hard detail. and turn Development, our we our a our setting up what pandemic. will strong for like Head dedication Our my the employees Schott, accomplishments team enabled us Rob diverse work over our multiple data year more XXXX. salable and their leadership in about second all talented for be XXXX to and and of to who I’d the accomplished expect leadership a have clinical and discuss And grateful our I the to passionate call my very that, am challenging to with

Rob Schott

afternoon good everyone to and call. Sandy, the on Thanks,

late this updated symposium concept of star earlier the At month, the and in presented execution X/X disease. believe Fabry from the important clinical last our stage candidate development treatment our clinical evaluating We Fabry for presentation of Fabry gene ST-XXX, civaparvovec isaralgagene are delighted World therapy or supports study Phase of preliminary we program. proof cell by We the for results XXXX. year data sickle

XX-fold dose one tolerated activity X, above for the first dose patients threefold the normal XXXX Elevated therapy from generally that adverse related of treated across maintained As candidate last grade five in patients measurement. cohorts alpha-Gal date, in as November cohorts the three events treated cutoff treatment four the with two were assessed has to be continued been or well to at the mean mild. ranging gene

ERT patients For normal in at activity the patient begun Cohort the at fold week folds week long-term two XX above normal therapy on the planned naive the activity X week XX mean in Cohort Cohort second study following up and completed the A above a enzyme and at mean have patient enzyme expression Withdrawal patients three alpha-Gal alpha-Gal above ERT follow mark, XX and for X. for at on the in of fold based stability trough X for the Cohort at remains four replacement mean activity one alpha-Gal of XX in the replacement ERT patient week X alpha-Gal measured on has for now their patient normal patient robust. at patients XX-fold cohort XX is one one above year and the patients been mean treatment. the from two for normal The therapy two in pseudo through was with

alpha-Gal the Cohort into X. has in normal patient increased X, first the mean week A range For at activity

SPXXX a cardiac confirm parameters three sweat. benefits This patient is with dosing, noted, tolerance treatment The magnetic exercise be a of from with resonance reported in reduction significant levels clinical two recently of to patient of Week in the patients. with through baseline Patients during stabilization plasma pretreatment the lyso-GbX the important Sandy individuals. maintained of significant biomarker cardiac XX The elevation XX. the of showed date. active the cutoff a ability MRI weeks to lower study, Cohort steady intervals approximately greater and who within X in after cutoff one suggests for that after baseline was in the patient improvement The will date. in patients followed As maintained This with data dosed therapy. allows second XX% greater this levels sixth imaging lyso-GbX in and

in expect ASH of study one for announced planning second preliminary SAR clinical cell Phase to study of the star We from concept At precision X are treatment X/X XXXX, provide the trial. of disease. currently and of a proof sickle the half for XXXXXX XXXX updated data from Phase results we updated the

recently no product XX, September had study patients. for no and there longest the on hemoglobin to of been reported. serious single SARXXXXXX, cell four have XX one investigational most therapy had patient post events F in fetal nine there XX% XX XX, and months with followed the events four the increases In crisis after treated weeks, transfusions BCL gene hemoglobin, As were related total in XXXX a treatment, to sickle percent crisis, were a adverse SARXXXXXX XX drug or between all adverse vaso had blood treated been cutoff for all additional has modification target been patients treated There cells. date, occlusive the patients required in followed engraftment None patient of the weeks.

Additional patients XXXX. data be presented to from a study be third expected this this to medical quarter expected the meeting by at is the study And completed dosing in in of are XXXX.

of with collaborating for planning back under forward a June. regarding this of these look responsibilities With for keeping I’ll to future this program, studies. clinical Sanofi are to Sangoma We that, We apprised to you updates its a transfer turn over financial it Prathyusha. currently update. on Prathyusha exciting

Prathyusha Duraibabu

you, Thank and afternoon. Rob good

press research the significant execution quarter issued be fourth with release in-house at for we the programs. and end the are we available found I cash advancement for believe that preclinical our a want approximately our our platform and reiterate year cash, across fronts our year, XXXX as progress of in results With on financial was our to remains year for and to lead million pipeline, the the programs, manufacturing marketable $XXX full many of on that Our and equivalents in capabilities. Sangamo can continue also our website. securities balance execution continued and sheet the

Turning million. initial XXXX approximately the between to be we excludes stock guidance, estimated operating to $XX to million our year. expense range of year non-GAAP This expect based full for non-cash $XXX compensation $XXX million expenses

Treg for activity CAR in CNS and We progress of expect pre-planning a and significant be indications. of work invested portion preclinical continued programs, operating Fabry and including PhaseX/X expenses elite activity, TXXXX to our

investment in [Indiscernible] remarks. now cells I in back grow We also year, for call half expect following sickle turn the will to the closing to the second our Sandy the of

Sandy Macrae

Thank you, Prathyusha.

where as we by company and excited we believe have stand a bright We’re future. a

the XXXX, Cell it the Data and We look dosing with transition in now our data, X/X fully line the updates for forward thoughts, progress X turn selection study trial. patients in expected operator Sanofi over open are to a Treg medicine manufacturing. and Sangamo questions. and expansion We’ll Sickle on X/X In momentum Dosing we CAR the from to X dose to Phase the Fabry building of integrated Phase clinical and Phase planning. noble Cohort the Haemophilia company the program a Phase precision Pfizer’s trial of of execution of A steadfast to patients in-house providing and for one pivotal for genomic

Operator

comes first question [Operator Nicole Securities. Germino Instructions] with Our from Truist

Nicole Germino

and the ask everyone. afternoon, thank could Good on progress. all If question. taking part my I two And you congrats for a questions

integration other it ST-XXX experiencing vector confidence The your is Treg the your A CAR don’t switch concerns put for need? Fabry. for their second, another and holes is, Heme And for Or safety clinical therapy first rest one of give backdrop will a necessary way, agencies? you in with gene regulatory the programs. kill address that companies for how you the Can for you maybe platform, around for

Sandy Macrae

you thank So your for questions.

Rob. I could if on and pass address then first So the one one, to I’ll the second

the now the as the increasing of this that MPS an programs, that’s factor. endpoint safety look throughout disease, really why AVVX on done safe So with all by gathering really safety of studies that with the the thoughtfully convinces of have serve. with have us. that important community we’ve as Fabry far. the benefit the so that The is we’re with to doses sharing spend because And choice We’ve with of of progresses. been data programs, sharing protected. haemophilia remains starting delivery our balance delighted maximally then A, And is safety regulators, risk. primary and to us our body we pleased and for we concerns us safety the and to all diseases any of form progress always long I’m we we a is that time trial sure the important preclinical allow patient patients data that And be we gathering

with I and whole lot because having worked effect I’d we’ve it think a into the of advantage have it’s field. say difficulties to safety. others that and does have unfortunate of gathered us run close

Rob Schott

Is clarification on the and -- that that you’re to call off that cell programmed that Yes. can into I’d turn we’ve like suggesting we the so some therapy kill switch the the Tregs.

Nicole Germino

couple like, any it? this? is regulatory have, And will agencies, one? had a agencies Do give need confidence is you of or input don’t regulatory necessary switch kill not, competitors your switch, you kill it if you A have on Yes. a for need

Rob Schott

cells Yes, we’ve here, that these to through Again, regulatory programs. not engineering in these switch. been kill asked were are T

So responsible of immunologic they and inhibiting transplant. response in case for are rejection the the renal

cells, Jason, CDA to killer other are of too. comment I’d types like us? So help T that can to cells actually you on have cells. Jason engineering it’s different proposition T But a T that,

Jason Fontenot

in cells think and using and program. efficacy say evaluated that TX what thanks, Rob. one the is dosing the that first And companies clinical the of a consulted And moving very patients. that I be with what we’re for confident a very we’ve what are We’ve they’re happy the to other be can patient experts, going as obviously approach is seen, X is a XXX our benefit is safety is forward. safety had focus a about own this quarter. we’ve regulatory models. in regulators take keeping And conversations safety, we’re we’re to that’s that our of approaches, and Phase Sandy been in eye preclinical we’ve the Obviously, with study I we’ve internal we’re paramount different patients but is this we’ll And confident variety approach our Yes, where Yes, on. close treated. our offer on and and the with excited

Sandy Macrae

Jason. you, Thank

Operator

comes question Wells Yanan from next of Zhu Our Fargo.

Yanan Zhu

questions. Thanks taking for my

program, First all data of additional going second so that wondering you you’re will this on include to half is year Fabry the And up? presumably in have patients. follow you the six what might the mentioned be

any of length is ERT the enzyme level think is the can important that’s studied? the would also question To stage? going be I we at that the be endpoint, informative additional up But substrate. assume to clinical endpoints? there follow be reported endpoints and So

Sandy Macrae

for you Thank your question.

be closely. longer able Fabry return we’ll chart Prathyusha can term So very to you and on about show, this the what this is find

Prathyusha Duraibabu

question. thank for the much so you Yes, and

So accumulated later especially earlier more the will have year, from patients. on data, we this dose

enrolled years Alpha been have have which follow expression we out be well And as T other up we some long-term on in follow over endpoints, study, of biopsy data. data sweep the you point presented. clinical patients the four of have available the patients A treated naive at that parents is study will will later of these we that into this terms to not will now know be able of studies, At this biomarkers we additional be Gal been be collecting in in beginning, as collecting like patients to forward, your expect prevent withdrawal have time, year. those rolled update, patients may and type data an total. GBX, be of You clinical the one will the year these up one And to moving this accumulation over kidney data can

Next outcome. an reported on year, we will also presenting patient be update

And so those really, in intending we and more have baseline. in screening we to also move along, we are and patients patients currently

anticipate collecting patients be So wealth that from be data to, exactly to dosed. well. of nice some be we and that a as are will we And going profile will this

Sandy Macrae

remaining talk of investigators the data no so far like complete it’s talk remains seen until flat, the getting like understand. stabilization, that about mark have up that four data year talk be been the that about, patient’s their still sense is from, been claim And able sweating, for only the the treated to decline have symptoms We from GbX benefits, is Alpha elevated, all about data remains those time we’ve of are is lyso there’s encouraging longest gal the that a we’ll patients in we through and really one MRI. that

current data are from the look for and to that Phase on matures. X as absolutely the full study the planning we want I to study forward However, our in be clear, initiating

Yanan Zhu

Thank you. the about looking design. Phase And Yes. to forward X Right. hearing

maybe think I question. So to early that ask too

ace on Obviously, based you I’m might, about going fingers. So you’re on. programs be I is may, about zinc working if what I this editing mentioned that curious to

it the double in degradation terms the part asking editor very efficiency, Or this editors, is is, stranded retain does So the nucleus? nucleus editing because the forego the nucleus actually i.e., actually the capability think because ability I’m strand. based reason cut prevents question part to I case thinker the of edited of useful of of strand is one of in the my that in improving the does big it base your DNA

editor basically. so light, Thanks architecture in that for interested I’m whether your under your So base

Sandy Macrae

interesting question. Very

DNA are So comment gives range be allows some time these. us on right allows to editors technologies our a comment we’ve for and the or these important, same choose enhancement, crisper, added breaks working Jason, to patient. this to specific thing, been get on a of platform the to that or Jason for But you minute. and whether a repression What please? localizing when zinc is question, can the double in great like the I’ll us finger pleasure things of right stranded for company and things are the there’s Compared nuclease to each

Jason Fontenot

at the genome. Sure, approach. it to the and to think the the base base and scientific discussing editor made, I’m thank you, don’t double to conference. change I Sandy. not into really of create excited architecture deploying look excited This designed, we breaks a very And base but in we’re it is the of the stranded sharing going it’s will about very go therapeutically. a appropriate we details we’ve that novel a them soon, we’re advancements that And editor that about forward

Yanan Zhu

Great. the Thanks for color.

Sandy Macrae

benefits us particularly and won’t. some that’s of crisper to choice, almost we and in of a the a the are like in a increase is way architecture what base It experience how reason be XXth it like, zinc the the uses fingers, to therefore and And that and allow years of from partners size it packaged understanding zinc it what of allows size finger about we until editor the we’ve to our it how small AV which architectures specificity. with and that’s standard tune had like. The

Yanan Zhu

mind, my you much the well. that Thanks. Got a for very Yes, Thanks color. Thank all that’s in highlighting it? Yes. as for advantage. question

Operator

question with Our comes Maury Jefferies. from Raycroft next

Unidentified Analyst

for Hi, taking thank [Indiscernible]. you my question,

have questions. I actually, two So

all you question. this talk what can is my been Phase And the X? That’s The to first get a at designing Fabry’s started. more you study’s are first where about are the one factors gating

the you Treg are say Second CAR question. enrolled and also already this patient been program? the first transplanted one And the what my talk you about in processing patients Treg? can has CAR currently if can That’s is, else

Sandy Macrae

just take me anymore. is talk let with please. general, up where are you about we So the Can text easier in which one,

we I’m the know, a therefore And as about Phase the a Phase haven’t takes planning been sure we’ve of So shared but at design this and X time. looking for talking anything you X study, long to experts for months, And because are talk at more progress right share time. excited can at we’ll about making. and we’re Mark the we’re least about the six broadly you the very risks with pleased that?

Mark McClung

about with Yes, first just to XXX candidate. so we’re the patient our TX

And so as expecting about very patients that. And dose half excited of by we’ve the talked two end we’ll XXXX. to be we’re about, the second

to We’re is And going a be opportunity because that XXX on. this major indications. is establishes because the effect agents, and goal of portfolio foundation really understand first this immune Tregs establish forward, us CAR the what’s take delighted TX these it’ll of critical auto for for really biologic the our to for

the inform In allogeneic as disclosed, advancing the disease. will candidates as XXR preclinical inflammatory meantime, for using autologous. we’ve we’ve this we’re And so as sclerosis, IL bowel us, approaches. multiple well got And against

be have learning those this advance we’ll the we out that the candidates. as also particular so platform, trial applying of we but And coming

Unidentified Analyst

Thank much. very you. Great. you Thank

Operator

next Capital. with Our comes Luca from question Issi RBC

Luca Issi

biopsy level, X Fabry, Phase to you a process that you key provide that can what maybe then you’re yet? quick better confidence patients? start data second first the the here kidney talk so drive of are the about without for on of Thanks you just taking manufacturing you any will disease, not the new sickle on confidence the some much on outcome Great, the the can here. design thanks you Two will What And can one, at the much. maybe ask high on parameters seen you Congrats actually additional having and quite new here Phase just gives progress. process X, optimizing so but color I give maybe manufacturing question. cell for

Sandy Macrae

the I other please? was can sickle, absence can this And the let question, me we design see biopsy we the about in So you data, the these. of how Phase of the said on expect split the XX, correctly. think inflation, decide talk and which how you I will question answered in Rob, And in kidney absence see we decision to of go X.

showed said, with changes we’ve they that itself Gal been the gives in in talking should Bio, very regulators confidence small for when believe one benefit in our that some And data from to and competitors, ICG, stage. As renal for the are we that alpha the biopsy get data, we the this of we I designing get that tissues VX, us changes perhaps Afro time. gal seeing Alpha where we

data. plan to look dose we have we we and forward accrue So And one curve, decide the in different as doses response we advance. and have to at take the which

Mark McClung

internal experiments, shown the And about some with but manufacturing, in cell specifically we’ve the haven’t talked this respect it increases progenitor process changes to sickle in program in, cells. number we have made of process changes, we

optimistic into So we’re through with the will carry this yield. clinic that better

Sandy Macrae

until will of direct have hope very we can’t we correlation. experiment, being believe we clinical results And But clinical I say transparent. we’re a realistic And that these to the do benefit.

very say too patients transition during period. Sanofi, study are data seeing forward we coming months. interested these are why three the next will that who from friends dosed that’s drive continuing thank or at I And four the the to our over the So you in want be to to

Luca Issi

it? so Thanks much. Got

Operator

question comes with next Our Burnett from Stifel. Ben

Unidentified Analyst

Brees Hi, had our one, quick Fabry. just this about Ben, thanks I for on for one questions. Kelly taking is

and Or I And if from was do. the is GBX second feedback what you one life then just you. program, Fabry yet the about biomarkers have the holds? Thank remove the something talk at biomarkers And already they the all treatment? regarding expect to about you under specifically So situations FDA this the if is to would received hemophilia. wondering has can necessary of our move to Pfizer steps clinical ST-XXX with

Sandy Macrae

can do Prathyusha, for Rob So Fabry hemophilia? you and

Prathyusha Duraibabu

for thank questions. the Yes, you

start So like San Diego. seen world’s level are in weeks for GBX. we different of different a the to And on GBX, you off patients just symposium with lyso at exhibiting will couple ago have presented data baseline

patients are movements patient lyso cohort this, that time And point we’ve so is to infusion, number whom GBX first post other higher I’d had XX first based up. than the and And differ the than whose within expect that that maintained until the latest on started X, to the like reduction been to for follow over the three, XX% patients significant and in more who weeks can we going has reduction. patient,

depending likely their baseline. And next dosing, is to they’re be seeing the we time, to going so, to how forward seeing on look be patient over phase three

Sandy Macrae

has adult Prathyusha been programs, to hard in it’s seen to phenomenon got this And other do. be

Prathyusha Duraibabu

the It’s you important because be GBX seen with in that high does have approach a we significantly therapy out, to and across seen other other been the us need really be lyso has it to that able programs. point gene programs that to for same levels thank Sandy, impact to at

data going And seeing dose so dose. be as at we also patient’s encouraging is next we’re look that to we’re confident higher our at that the

Sandy Macrae

And Rob hemophilia.

Rob Schott

Hemophilia.

than of remind to markets the that the First partnership time Pfizer trial more guided trough will Pfizer of that first this in at us planning I’d class. acknowledge resume resume and or program was has XXXX. on terrific XX% like half to with the the this in everyone

that where we are first guidance half into to specifics year. trial, the So without responding authorities, with toward point resumption regulatory this I can the of getting of in

Sandy Macrae

well, pretty all they’re going and their efforts. putting It’s

Rob Schott

enthusiastically pursuing this They trial. are aggressively and

Operator

comes question of Mori Bank next with from Our Aspen America.

Aspen Mori

that if on approved the that all Thank your And already in partnering that in any just for is where through FDA presence niche a that talk FDA? be thinking of out how available. think indications kind question. the someone you have Sanofi question, of your or Do if gene your ticking there’s you the for updated maybe it Maybe at you Or transition that the then guys. prior alone some for therapies it of you to as US on you kind that out. see thanks seen over of you. may within more stricter priority partnering, Maybe fair with second therapies terms implied been, interactions assessment? was update partner? peers thinking therapy your preference stuck six, that’s you or an as the with progressing play has that dynamic guys, have of there’s can space, is Hi, And

Sandy Macrae

sickle. ask about to to around touch I’ll And the Mark then general comment. on going strategy talk the I’m

Mark McClung

had transition XX, they us. that cell to strategic sickle we decision I to heard mean, obviously, back December made

Sandy we the very progress been has mentioned, the culminate team Sanofi, this XX with around of transition on with very pleased to as hard working As the engagement transition, have which will year. we plan, June we’re the

proceed in will utilizing that even manufacturing way that now to Phase feedback as process, right which on hope X/X to and priority better through just both the meantime Totality preliminary we the highest kind the the can those that the around manufacturing, results the complete as precision data of terms Phase in come we new authorities, well X. want discussions mentioned, of Our is forward, demonstrate have four inform will approach of we teams trial, Rob engaged to as ensure in patients.

update an provide we’ll time, appropriate the for at that. So

that severe. clinical disease commitment you if to the we’re get those sure It’s In we and as are perspective data make which to on patients. a it’s right early cell data. US, commitment that. is issue. remind do can not is And it’s to the our access more a on differentiated going medicine. to the our subjects of partnerships, it devastating sickle we we’ll in the whatever ensure terms that if particular affects United And But that medicine, comment we forward a to this of patients know the through And that get to I patients XX,XXX take there’s XXX,XXX would geographies about that Outside about XXX,XXX States of This provide a of too is patients, now. population.

Sandy Macrae

And one I talk to could general the the more about if agency.

ahead We of have FDA. in Because people emerging data I the the to they and enormous with this train have exponential understand a agency stay great with relationship to one and understanding. I and and the has respect think field for to deal to have and that growth Peter an

more years the IND I new think too to what medicines at there will And be have understanding preclinical first. the had ensure conversation would be have which we of watching the which we are agency, it’s and patient what many the of new a the is seeing. at our to having first bit takes any to effect of about any approach, safety I that’s toxicology some the check But concerned data clinic, the challenges about now, Sangoma and safety and agency’s are more everything the the the team patient of found one back to about me the and in or that in and have that as reality are development difference in about said in the model, to that if a with necessary we’re medicines. factors to having many haven’t being glad field, questions partners our felt moment, earlier is inherent in unemployed them the are we’re inevitable them same tested, of testing of is ever indications in

Aspen Mori

Sandy. Thanks,

Operator

Cowen. from comes with Ritu question next Our Baral

Ritu Baral

important Thanks the Well, one. Good program of what preferred you’re you’ll will cell looking more to or program percent do since to on you question high is the more metrics more cells one such which the the love drill a in in to F, going And had certain know guess, patients by I that’s level on think more down especially in levels for I’d at a afternoon, guys. you general, of tell we’re show for taking question. ultimate and F of And say sickle on be a also which that, is gauge landscape level, I sort I I sort of proportion the levels, benefit? reaching important that percent precision for to F getting cells this where guess clinical the need will you of treated do Hbf do a to therapeutic. need be you high to XXs that crowded. percent to but the think fit

Sandy Macrae

a ask you’re you, what remind more is would to used comers data, getting into that are clinical Rob to landscape strategic what to technical pipeline or comment the of the talking many, can But bit. about on competitive modalities, the going other talk successful? I’m you there new Rob, So, from with and editing we’re in largely clinic the

Rob Schott

reported to were what What’s we episodes. very the enormous frequent had frequency important whereas expensive they we’ve that parameter the the patients patient to a seen prior in we’ve which have painful, have the is treatment, of is occlusive size, having been effect occlusive most single crisis. an VOC, sickle is releases four to important most That cell of is they crisis. And

all what look I magnitude has effect hemoglobin factors F All keep protection, profound. fetal if of are the important time that most and relationship understand most the So the durability the you percent what’s and patients. is lives. at has, mentioned, other and of to that percent the on cells, but is patients that impact that hemoglobin it’s between important. eyes But And we’ll you really on therapy with the effect, to that need our we think important fetal F cells

Ritu Baral

analysis couple now some or years well be VOCs relationship you potential be endpoint? other it will or some think Just Hbf could enough right clinical understood Or enough of Do a quick Hbf come that. that snapshot or time that the really is pivotal or approval, aspects? a accelerated next of understood will approvable follow up to to down in in

Rob Schott

VOCs are to measure. easy

of I remain unaffected, You subtle particularly effective that the disease. the patients got in assessing this laboratory just with think that a will report, patients based therapies provides cornerstone is finding it’s

Ritu Baral

Thank picture. you. Bigger it. Got

Sandy Macrae

the that, registration shown of Yes, results, clarify they’ve not to which study the be know, year, any would vertex terms updates, much towards this far don’t of dataset guiding for this, further the me, at of but least I directed of so answer crisper as sometime file yet, I please end as in that obviously, expected. as they’re the terms in in going particular,

And agency. terms discussions we’ve seen with the of tell the in so will go also they meantime, bluebird how the once file time In withdraw.

emerged, as in really data alluded increases F clinical any today, competitive manufacturing but I additional to see percent better really for the that four the for increased has so, levels, it benefit think how and it’s change importantly, more terms Hbf cell looks as understand the dictate as whether process if program the we with becomes forward. presented us Rob the the that that’s to patients. take in well patients that will the And we’ve of outcomes enough profile And important to

Ritu Baral

Thanks. it. Got

Operator

Our next Wang comes from question Barclays. Gena with

Gena Wang

Two taking for very you Thank my questions. ones. quick

would regarding the kind base to Or is keep be editors. clinical the question cell what program, second possible you crisper follow The thinking, like Would be the The sickle first of profile? be better? profile there will in-house? one regarding that also

So a use about Do right proper what IP I to MNAs, part? assume you use that? the the have you

Sandy Macrae

Maybe And why base don’t you then sickle? talk speak Jason about editors. can the to

Rob Schott

Yes.

of well, an a patients. cell of a the to profile At it’ll you’re terms sense, be us day, manufactured alluded important see patient. bit differentiated the which crisper personalized comparable end really little got the a that hope And I clinical update give be the of are the the think or four need mean, in whether product, more of they means we’ll therapies, hopefully, roughly that we’ve from it’s product. So time I in we’ve vertex, to, whether data, I these sort got for around mean, a to they I to just see going

the As XX,XXX I severe. of them described, US, of there’s XXX,XXX are in the patients

so long to one more time service cell a And in sickle the the importantly States, worldwide. communities, of if even not market, only competitor going it’s the needs there’s United to the in take but

or I either a earlier, what differentiated get this attractive ways as so, to an do, vertex, we look for therapy And we will the find met same got crisper these mentioned that or novel to to can partner through need we’ve profile, we patients. that’s this do

Sandy Macrae

base can Jason, question? on you editor help the

Jason Fontenot

is regarding the a this excited program I’m incredibly about. Sure. Thanks, editor, base Yes, Sandy. that

of But functionality where that on of molecular they’re innovating target doing accuracy of that team side, biologists finger And the we’re platform. are they’re molecules structural side also biologists, on the zinc great right. refining the the continually to a both finger have We molecule, of around it portion the and doing the specificity specific our sequences.

we already in toolkit nucleases, that functionalities regulators. of group transcriptional have transcriptional, core the beyond our and activators moving so, And

we’ve exploring we had if moving we program think now We’re epigenetic wouldn’t And, but something base didn’t that our we’re excited including new about, freedom other Editor also base be we and forward re-combinations got really editing, functionalities, operate. in to editors.

we’re zinc we’ve developed. think pointed advantage real that in in being unique viral of accuracy advantages a specificity vector, the deployed as as out, offers finger taking it So the of Sandy And pretty single with be aspects architecture really platform. the some great to well comfortable and we as able

Gena Wang

Thank you.

Operator

Our next question comes Wainwright. from Patrick Trucchio HC with

Patrick Trucchio

the good on for progress quarter. Hi, afternoon, and everyone this congrats

just in Fabry what lyso start first off kind have guess components the patient’s patients result elevated can these actually the how to level do that some of have GBX increased diseases the GBX? I are two is of I one having lyso on, So questions for and readily

Sandy Macrae

patients, see those are our are rare the and Patrick, are few Prathyusha those can naive to screening asking? it’s that lyso and have lyso GBX. a of what think those what that can I you proceeded talked benefit that I your question we elevated medicine many we asked for I patient by levels patients is make already where understand repressed on earlier ones you sure the have GBX. And ERT And will the

Patrick Trucchio

You be high GBX patients. screening lyso will for

Sandy Macrae

patients, the those patients delighted take, ERT, six take Fabry patients, We’re a we GBX what take the on all to supplies categories, [Indiscernible] rejecting and find level. are take we’re we lyso and low we’re they least a patients even on with we have take we been patients high That’s up And months. and we when for that three naive not take if at delighted have we patient. for

Patrick Trucchio

like of kind a just then And up question. follow Great.

patients, that and in like, something see so increases that do is on. also see later lyso GBX? six, lyso the baseline you we’ll five Or And these patients for of

Sandy Macrae

the patients on five six. GBX lyso commented We and

Prathyusha Duraibabu

Thank the for you question.

that year in that of not is clinical commented This We update. both this can is part time. point something later we that provide have we the at as update our in on provide levels

Patrick Trucchio

Okay, additional great. for Thank color. you

Operator

turn no to Feingold time, questions this queue Aron for to in at closing the back further remarks. showing like I’d call We’re

Aron Feingold

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

Operator

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

You may disconnect. now

Sangamo Therapeutics (SGMO) 25 Feb 222021 Q4 Earnings call transcript