Earnings call transcript Q1 2023

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Operator

Good day and thank you for standing by. Welcome to the Sangamo First Quarter 2023 Teleconference Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions]. today’s being that advised be conference recorded. is Please hand over to would now speaker conference to like Wilkie. I your the today Louise ahead. go Please

Louise Wilkie

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

product current trials, This These potential guidance strategic statements to of results for historical and quarter-ended anticipated our that regarding dosing initiating the preclinical expectations. resources, therapeutic targets our are facts. candidates, limited estimated other and statements operating to plans clinical XXXX our advancements capital, and and collaborators and Sangamo product statements preliminary patients milestones, financial reprioritization for upcoming the for and catalysts and include, not our the advancements cash to The March sufficiency and call statements our of XXXX, programs restructuring the commercial runway, to, thereof. and and clinic, includes and clinical are not benefits of seek time beyond, estimated data, additional XX, lines presenting relating candidates, our conducting forward-looking and and of screening, anticipated plans Sangamo's but of

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

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

Alexander D. Macrae

good of where morning firsts. you rich the call. Thank I Louise and At to our am today, is proud innovation scientific and is of everyone history joining industry Sangamo target

committed of value and in which our to shareholders. we so developing bring promising transform been doing have results. potential are In genomic to have pipeline We are patients the lives the forward medicines to driving for important programs producing

chosen the an want effort believe sit why focus. spend I to our significant of announced I and these in time spend achieve the company today reorganization value that vision. some the and and then Today strategic resources create potential vision reiterating is we Sangamo back for to a -- will outline prioritize in being areas to has to

creating challenging to believe program portfolio ideally capsid newly for and genes field years. well portfolio are neurology as assets epigenetic regulation delivery potential repression treatment X.X underpinned our owned by the on capabilities. Prion to disorders the Zinc unmet disease, believe we of or suited Creutzfeldt–Jakob wholly the large, which our treatment power year, chronic the strong of XXX a in neuropathy Finger a market opportunities. technology is leverage addressable coming over our our pain submission advance ability existing program today these neurological will the the medical of NAV to has for with promising and We repress of address as technology, preclinical We both and enhance often Anchored present targets of that result the devastating needs by

same leading continue we to At provide advance the time, patients have of programs. Fabry the Both clinical TXXXX to potential shareholders. and industry our to benefits to significant which value CAR-Treg and

with progress address of programs. promising to safety We important treat therapy are and We to trial patient program support this design good have proposed we termination ideally in have pausing summer and population. of data in these preparations this believe presented gene continuing of the potential to are consult to the leading Sangamo on in we competitors once needed the conversations. or data reinforces some development to making needs the Fabry the FDA X today announce is The the efficacy Phase disease placed we

us suited to to TXXXX a demonstrated, being also be great space, an therapy in CAR-Treg Sangamo the a the with We STEADFAST transplant treatment manufacturing is ideal new capabilities of that believe is could R&D we modality. trial. a only leader for adequately in CAR-Treg concept continue safety CAR-Tregs clinical in a a treat range and to CAR-Treg leader field. believe our efficacy therapy ideal the and broad our be and proof to advanced cell coupled those the our in study, promises model well holding position know how Through company cell autoimmune Treg are of indications, renal places patients known if

be inform what company requires industry of disciplined forward in using data have a considering the in. date, each in to to approach is we for signs very asset. and of which take can success potential commercial to alone, To the than more a focus also progress while this position assets This company rare seen to a

shareholders. and our for it generating we long focus While carefully fund must goal progressing by to our investing resources with we the the manage meaningful business and must patients, signs the on capital delivering creating for term for of also returns

of critically evaluate programs a to us for Sangamo, general steps necessary decisions make have market but investments and difficult number burn. reduce next months, prioritize recent capital cash in factors the and to requiring access to broader including In challenged

our neurology X the term for value newly being ones today and investments resources the others and prudent seek program through TXXXX Prion believe X.X a prioritized with with renal a the Phase Phase the programs business. industry owned trial; and only in our wholly to three, not We our one, maximize unveiled NAV as CAR-Treg key prioritized transplant drivers we're the Fabry strategic potential two, in Therefore, and focus program value core X-X. our spend. clinical are these are carefully announcing being portfolio; We R&D to rationalize sharp near areas; their to three as advance

which return Novartis programs programs had the wider forward set the economic while how from we With upcoming the of we reevaluating carefully Biogen select backdrop, priorities. to to take neurology also resource and our within strategic

be capsids expensive these California. We as to epigenetic led our from and and necessary and manufacturing the that away especially in as IND. through asset. has an as footprints we have assets but of California can research to programs manufacturing difficult allergenic internal the This believe decisions delivered prioritized recognize value resource existing intensive well program today targets internal regulation for are our future importance capsid We in step evolution underlying some company reducing significantly to preclinical potential advancing neurological specific to drive

value and term. activities preclinical our to focused on in time which this to drive believe clinical should the we be spend -- expect at we So value near

with to result in $XX initiatives savings We reduction believe will cost fund in of us restructuring at roles U.S., initiatives are our months. other Sangamon of the cost XX% XX XXX plus combination plans expected current are planned U.S. least at We the The million. announcing today of therefore these annualized allow elimination plans in approximately other changes approximately operations the representing workforce. to for reduction that

to and company. consistent the continue will ways We reduce annual to the operating our the objectives progress assess priorities, with expenses of

leadership He and has the forward I Ramsey, Andy Head President appointed I of capabilities these as us dedication all and his really development, be of of brought been as Andy organization Ramelmeier, with our current May with expertise share Technical industry Vice sadness, manufacturing Alongside is real support Operations head look and is legacy the passion to technical Sangamo Operations, Sangamo. that great a at programs excellence Phillip company. technical Executive serve four leaves Phillip to with leaving priorities. advance to of a development. and will XXth. building strategic clinical to has here. us known working technical continue throughout that advancement our manufacturing allowed personal to Technical into has the align by the recognized effective we His our of announcements

and the by as our They to their individuals many will Chief have like this who us these the over we great have will are, neurology decisions been vision been are our innovations call to them. Jason, the very decisions decisions difficult. share believe difficult and the I'd to to no to more are our acting instrumental leadership right that they pipeline. thankful on committed projects. a future mission their most years, to during have promising and we level, helping matter I've Sangamo for company. truly brilliant Scientific the in Jason. turn how Officer, our and now upon our been I'm a are talented achieve and committed redeploying On personal capital been has for Sangamo, service for but team, these focusing preserve and for many so responsible dedication extremely who

Jason D. Fontenot

everyone and good on the Thank to you, morning Sandy call.

the patients. Sangamo, of and we Here strongly capabilities, transform our our help potential the of scientific believe in lives platform, our to programs at

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we of neurological ready important by unlocked in into to will programs preclinical these our to be outlining significant to treat value I disorders deeper assets. dive start portfolio why see

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our disease. focusing a are with on diseases and AAV that on developing in we strategy -- on hand value AAV Alzheimer's We're the owned capabilities We believe designed long-term will build addressable the high our we of few years. existing have broaden to set a probability neurodegenerative the in capsules. potential focuses novel capsids such treated To have success concurrently believe greatly including developing indications year AAV in we the over that with be delivery future, has can maximize that result of novel currently focusing the as wholly value portfolio this one submission we neurology next diseases technology IND

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our Biogen terminate this to us. we to partner Novartis that have collaborations both and regards notice with received programs, With neurology decided quarter

cases, these from broader same early from seeking week. resulted prioritize the While pharma stage is reflective two and to In stage large This an these coincidental late unfortunate of completely unrelated, and reviews in growth, strategic portfolio away investment risk value, had step in balance both decisions de-risked commercial in coming timing decisions to and risk. trends product.

for decisions, these In create savings. cost out Sangamo’s companies bold is medicines. While discovery we understand focus of is such pharma to work transformational favor as look

the of progress from data are to and pleased We near programs preclinical future. look the forward with these sharing them in

comprehensive million capabilities exciting Most the necessary three programs Novartis efforts programs for these upon cash work opportunities and in are we demonstrating future look platform significantly and After owned on month targets to driving these focus as to pause indications. termination will and focused our identifies have time. portfolio, epigenetic now XXX Finger to these the first revisiting to our the previously potentially be pipeline. pipeline in These decision activator notably, capsids Zinc thereby advanced, of new Sangamo us recently a strategic generated returned for our SIFTER wholly Biogen the completion review partnered and significant instead programs. of Sangamo forward activities. our We Notably, the neurology to facilitate our delivery indications AAV value and from more have programs success for us returned for made

now assets. our will the Chief pipeline Operating over will key and who contextualize broader Mark. to in turn I Officer the clinical call progress Mark,

D. Mark McClung

continue Fabry, leading announcing another with this therapy yet and to This with Fabry termination we available Starting this medicine us double everyone. gene the space underscores those tirelessly with in for morning to need good and disease. recently make working development. Jason, you Thank living remain the of the to down competitor

to the diagnosed the many struggle the the on for to remind X more disease. diagnosed market States helpful the that adherence a like diagnosed not know with those XXXX for that We although well are around with prepare trial, are replacement opportunity in even approximately of actively many it's asset. patients regimen. actively XXXX as incidence potential to Of we enzyme are there of Fabry treatment familiar this X,XXX Phase therapy everyone As as United

on as recruited alternative already and patients desire options over which into patients above to treatment successfully study, of patients naive demonstrates and of well as our have We X-X standard improved care. ERT, Phase have pseudo naïve the the

to continue clinical chances and commercial of program therefore success. maximize appropriately in will the We invest this to

to to favorable study. this the X-X dosed study XX last we total The have quarter medicine, with to profile in QX those patients is sufficient of study on X proposed believe Since benefit meet in data we've urgency potential half enroll a and dosing discussions. clinical to from anticipated further the health depending a a of second summer compelling patients and this we first ST-XXX FDA patient for We general planning so happen as of data shared with accrued regulatory of the XXXX, is the Phase data safety scores. we're trial date, The earnings, on demonstrated interactions. we SF-XX kidney progressing to first the a and could and strongly design achieve evidence commence continue three once STAR the biopsies to Phase study of part plan strong biomarker as from improved the in early the XXXX and

X, for X-X to As is factor for complete the be not a additional regulatory dosing of data, expect package. to we expansion expected the gating in but a commencement the reminder, support will XXXX the which Phase provide to Phase

progressing once our T-cell we detailed TXL biology regulatory programs into research and acquired we're expertise in and understanding to the leader XXXX, in Now married a of and and experience of we our portfolio, When manufacturing field turning development. CAR-Treg developing in again in where with clinic. that clinical this INDs deep our gained

for a indications we are new range treat modality. treatment to maximize combined, pioneering well placed of type a and factors CAR-Tregs these to we of opportunity believe are the With autoimmune

first programs, the move to The the mismatch clinic. through continues HLA-AX kidney TXXXX of transplantation and

to This quarter the Cohort for progressing, pre-screening second and dosed higher dose enroll. and third to continue do in well. the and One, we patients continue additional in Preparation we patients patient who actively have all is cohort

Cohort on the we the positive agencies end working two escalation. we've As enhanced in One also that accelerate We're the share that clinical outlined trial the by from European to pleased to announce will to feedback been for from initial of XXXX. design I'm dose quarterly we've received call, last track accelerate of pace data required study. enrollment on this

our of CAR-Treg portfolio, the plan to we has this the decision autologous our As and investment in allogeneic our to part United all see California sharpened to to facility. of Brisbane, activities in self-manufacturing States facility France near-term transition prioritize from focus, resulted a our research Valbonne,

While resources important the towards are this the TXXXX our it's be advancing capabilities important allogeneic development time. to of at that will long-term platform, CAR-Treg directed

other to of want two value important I Finally, the company. be story the drivers reiterate expect we to components for that

to an potential gene to developing mid-XXXX. the completed the fitelparvovec in analysis patients severe a generate X. giroctocogene we're and BLA Pfizer milestone half required out to therapy primary Dosing currently which when second Pfizer first of complete would anticipates significant continues read in severe hemophilia complete and The is for expected XXXX, all for is towards a Pfizer work investigational pivotal patients moderately payment. MMA the submissions A with Phase with in

XX% As reminder, a to royalties. agreement potential to of in and this allows XXX up potential to million a milestone partnership for XX% up

innovative for as I'll progress about serves of as and both Prathyusha those partners, revenue, competitive to capsid hopeful over this updates driver for results. significant program outlined to inaccessible of are resource delivery is own as our of it potential to of value like the areas. recognize system. potential that a also exciting Officer, source an to engineered addressing our issue potential Prathyusha. important in unleashing medicines, a a the financial genomic the enablers share now and the the advantage turn portfolio previously Financial which programs Chief see the address and capsids nervous with over asset of capsids We We to overview range of making coming potential earlier, other and delivery of are months. new of and our some new places importance our central The Jason

Prathyusha Duraibabu

as of review Mark Thank quarter towards financial customary worked impairment good completing results evaluation to including close the assets. and Sangamo non-cash charges and you, preliminary morning quarter procedures first of yesterday related we everyone. end long-lived announced

for year-over-year. compared as quarter are Our March follows, are for to million XXst, reflecting same estimated XXXX be million upfront to to $XXX assets of related approximately preliminary from the first million fees. by driven results Biogen This XXX of return in increase non-cash period primarily from ended revenue the the $XX $XXX million was revenue XXXX, accelerate us requiring of approximately an the

goodwill the estimated $XX $XXX period an for including noted expenses the to year-over-year. were to by to of million. of range same increase space of other $XXX several the in certain impairment agreements million primarily quarter to partner similar first $XX our operating charges lower in observed. impairments non-cash assets be million are million and This of up $XX GAAP estimated including $XX to have These of to million in Total compared termination companies what million capitalization was significant million long $XX of impairment XXXX, market factors, previously reflecting driven for and lived driven by

in represents equivalents, marketable decline fund approximately expected today to next $XX savings going result and with announced least quarter. the million approximately from expected million of headcount This million which cash, of in at reductions cash approximately the the XX in Furthermore, the is annualized with planned the combination to ended months. securities, reductions $XXX are We quarter for $XX prior cost a forward. company other

two prioritized assets ways to operating our further to and and annual as neurology a continue expenses manage TXXXX. driving to towards reduce focused assess Sangamo highly organization Fabry We our

is result announced range previously guidance $XXX to obsolete. operating our changes of XXXX a these now expense non-GAAP million of As million $XXX

value including and $XXX and Sangamo further compensation to for the of We lived expenses million in strategic expense. expect with is in reduce estimated from certain operating long assessing [indiscernible] $XXX impairment line range This ways be non-cash to of charges key in to stock to the committed priorities Sandy our raise our drive assets current call explore back estimate intensively readouts including XXXX for remarks. and parallel, months. programs proactively a closing based creating to will range million. to continue will excludes I In investments long-term to for the we partnerships. to capital, and Sandy. coming now avenues through will additional the continue physicians shareholders, Sangamo turn is and

Alexander D. Macrae

owned you, future Thank responsive, Today Hemophilia to being BLA nimble Sangamo neurology term. our capability long around development centered anticipated success. a in submission. X.X for technology can to you value position underpinned finger innovative Today closer our in a Prathyusha. XXXX, that with wholly heard ourselves NAV a responsible, lot order believe potential by the There's is capsid that portfolio and in A a and With and IND we provide submission near marching look sync announced we to forward to. leveraging

in in this begin first XXXX. leading through a technology clinic CAR-Treg of end the TXXXX and One of initial end from trial Phase being market to Fabry data assessed Our program Cohort expected by the X expected our potential at year, advances the class is towards with

colleagues and to for I'd hard thankful team by their hope the and very ahead our to I'm future lies close dedication expressing that and mission. like work to my Sangamo. for excitement leadership all our Sangamo my for

open open to this we'd up Operator, please questions. for the questions. at line like for time, So

Operator

And Instructions]. Stifel. first our of from question Ben [Operator come will Burnett

open. Your line is

Benjamin Burnett

regarding there potential so, and being color provide U.S.? would question design supply the is that needed as a any I you a was to wondering Fabry non-inferiority study had guess And a Phase X in expectation would Fabry I for I be program. whether thoughts to approval on much. any more if proposed comparator? very the you the the if FDA or thank could as not Hey,

Alexander D. Macrae

for thank question. you to on to one going I'm your Ben, that pass Nathalie.

Nathalie Dubois-Stringfellow

you Thank for question. the hi. Yeah,

on We preparing meeting FDA, we comment FDA and the with right have interaction now. a the Phase agreement are call. the hope after summer to with and this X design the will potential the We

Benjamin Burnett

interaction? that gating to Nathalie are going what

Nathalie Dubois-Stringfellow

the dose X-X top Some Phase additional we data in but from trial, our are…

Alexander D. Macrae

So of it's time of just the collection data. simply

Nathalie Dubois-Stringfellow

Absolutely.

Benjamin Burnett

just That actually makes I like ask that. disease question, Prion question a And program. sense. if Okay. maybe also I the appreciate could on biology a

it, there have Prion we think I like misfolded you supplying what continually I depicted the aggregates will unveiled slide larger there? anything Is source cleared once for these as is the proteins, material Prion is be known production understand turned of the way about guess, whether aggregates. the off, are

Alexander D. Macrae

Jason, please? with help that can you

Jason D. Fontenot

evidence aggregates There Prion the of protein off Yes. that if if the that do the Prion diminish. source is gene the gene, aggregates turned

models. we approach will data the have presented that we believe preclinical effects So, great and indeed potential, showed in XXXX at that the this have Prion therapeutic mouse

about So we were very excited this program.

Benjamin Burnett

you And mice Jason, can data the in when given? dosing lifetime of difference just and summarize that quickly the about was

Jason D. Fontenot

Sure.

allowed which mice then standard in that understanding the initiates model study protein, simultaneously gold Finger with is and inoculated disease the the the Prion with and were where So, mice disease we the And in also approach extending the has repressors But Zinc extended therapeutic the a was And took the two the Prion improved where that treated start been the considered was very used cases for inoculated to of repressors. disease. extended our we mice to to Zinc took and were on ASOs. approaches how was one used. an And, our of compared lifespan. that were develop, was really this much mice. data of repressors at Finger significantly the repressors part lifespan we And application an both then published there, that impressive this

Benjamin Burnett

Thank you. That's interesting. super

Operator

question. next our for moment one And

Harrison of next from of Greg Bank come Our America. will question

open. line's Your

Gregory Harrison

approach X.X morning to the like of could blockers how our others thanks give NAV as molecule, the target a you for for like could be development, Texas Hey, a taking choice your program? of us small good questions. little what led and Maybe focus differentiated under from and background the just on

Alexander D. Macrae

X.X again for been number similar talk Jason NAV question. of cause and with Greg, and But for going time, to of X.X, that in effects. to us small you pain, I'm that companies science. the those the to with molecules on interact for X.X tried seen pass drug X.X, NAV of have us and on period work NAV such the through as give the thank side have we've antibodies NAV clear and proteins benefit struggled And, industry to in fact any that

Jason, So why our different? is do we so approach think fundamentally

Jason D. Fontenot

clear what's of that data key X.X human is there that NAV is about molecule show so so regulator that populations pain very sensation. a from Yeah, exciting genetic this is

very of there that pointed molecule been X.X family X.X target clear with of So specificity. part been very out, small genetically receptors. it it a issues targeting Sandy a without unable has it's this similar the involving have implicated diseases. is and particular NAV in But receptors in genetic, to other problem as NAV approaches creates of closely and specifically And structurally has

or about what receptors. reduction by can't epigenetic affecting what's unique target And molecules an closely related which that is the pain receptors. that any our affecting achieve only expression the transmission regulated and us we in of are do design closely related small So is very that X.X NAV do, other this X.X without antibodies the specifically can of repressor to other targets program without NAV this processes Zinc Finger allows the

Alexander D. Macrae

other the not protein. problem this X.X DNA that is DNA do The it's that level. we protein. and we Does have to NAV is specificity therefore underline help simpler I molecules do NAVs, the all for the the different small target we from that and a the want completely and What DNA kind it to take target and Greg? target because of target we not of the just

Gregory Harrison

funding seek pipeline the now more, fund that's in could program longer-term? strategy your CAR-Treg, broader, curious, help development I Yeah, medium partners just stage like just will one for what's If that and you're to sneak helpful. earlier on programs the focusing the more other X.X and super to you Yeah.

Alexander D. Macrae

help That's question. Mark, a can you very that? with fair

D. Mark McClung

the Sure. question. for Thanks Hi, Greg.

expertise, communicated potential help about we've that the those look have And, several by As excited that interested commented at can we brought lot faster accelerate got a a technology it, really ask do, mean, partners terms as they before, earlier. get I bringing advancements patients in of in ourselves couple we're a including strong take in us of upfront. the XXX One questions. and seen when partnerships last on by about if a then program Pfizer's of serious we million history that. to years that capabilities. we a the result over our resources, partnerships and in we've I've is, of progress And approached that partners are we've

So look that. we at will

Gregory Harrison

Thanks for taking the Great. question.

Operator

for our one And moment next question.

Our next Gena Wang Barclays. from come question will of

Your line is open.

Gena Wang

X.X. Thank you. NAV also regarding have I question one

also I actually, a and olfactory quite cells as of are it's parts body. beta other pancreatic expressing sensory, there well, like different tissues the few think

what specific questions. of route is several good is is, the advantage disadvantage organ? and a of One administration what wondering, is your regarding incubation long-term selectivity the how or and So

Alexander D. Macrae

I So is there the we I other patient. And long-term of One route about addressed. three is that heard. partly questions, is about which Gena, about benefit to think specificity, one administration, is one the

Nathalie, between can we So Jason and address this.

do Jason, route about want the please talk administration? So you of the to specificity and

Jason D. Fontenot

Thank Sure. question, the for Gena. you

are think there two the I about important points specificity.

the to platform genomic engineering power this front. our where of is really Zinc First, comes Finger the

addition for NAV that We are we are for in driving are in cargo, the and of use that Zinc able related interested that, vector cell promoters in repression. this can But epigenetic the to repressors closely that design to we're that of highly specific any repression AV avoid specific that the receptors. delivers type X.X Finger also

to through ganglion. tissues expression here, of are are in selective -- achieve by very the able specifically the combination of So those that targeting, in both we interested we dorsal the root mechanisms,

both and target type and engineering capabilities Zinc is we our cell that's our exquisite targeting So gene to through key achieve the promoter the combination really can for capabilities. the the of really how specificity Finger

root delivery through dorsal of terms ganglion, delivery for In achieve can systemic we administration. the

that are is taking. we And approach so the that

Alexander D. Macrae

administration? Through systemic

Jason D. Fontenot

Sorry, administration. intrathecal excuse me,

Alexander D. Macrae

to want that? on comment you do Nathalie,

Nathalie Dubois-Stringfellow

Yes.

that population we with of are yeah, fiber targeting small neuropathy so chronic patients So have a diseases.

in a that So appropriate that intrathecal bolus injection intend us reducing for and those long to treatment to we for we the the hope patients. will the dose last time. pain single the escalation product trial one-time of dose It's a allow a will do target

Jason D. Fontenot

are first that NAV dominated we're people specific lives visit pain, these patients, show animal benefit Zinc really a ASTCT by and we by Fingers will as feel need. with by repress important, and in group unmet the is this show forward ganglion appear pain. their We are intractable we enormous a root dorsal an it an had doing to intractable that specificity only And with it significant And for run at get the benefit, company. is experiments the results a doing our about their medical will us going route gene. we've are them that single it on that there's pain, By population promoter intrathecally, ruined only to that come targeting for here what

Nathalie Dubois-Stringfellow

modality reducing I associated And be X.X to sensory to side may not and integrating think, effect NAV known not importantly neurological that I be other pain any is with add by expected affected. are

very yeah. targeting, So specific

Gena Wang

disadvantage question what my also could the like term So inhibition, yeah, long there? be

Alexander D. Macrae

this, do So, patient well, you will and take safety. you know know seriously we Gena how patient us and we benefit

But their so say and agency are at seriously pain. patients increase how I dose importantly agreed with And we appropriate will the start gradually cannot impacted by enough, the it. these

And them, them had so companies the consulted really come medicine. a And and future as for them they this is an into with we've promising advantage. see enormous we've this

Gena Wang

you. Thank

Operator

moment One question. our for next

will next from Maury question of come Raycroft Jeffries. Our

Your is line open.

Maury Raycroft

need now and Hi, factor and to will question, the NAV capsid you paused guess why Thanks and and and the delivery. to Novartis like [indiscernible] like promoter because up less programs, CMO intrathecal a for program or use penetration cell about capsids is a) reasons follow distribution for that more questions. a limitations barrier Can I brain blood because Gena’s good for partner those you are morning. delivery for Maybe I capsid the as specific mentioned for as previously advancement, you gating my talk would novel you of for targets? taking conventional Biogen one primarily those clarify of the X.X Prion

Alexander D. Macrae

Thank Maury. morning, Good you for the questions.

the we're not we're for But have that diseases So this we time. solution advancing. using for which we that believe each revealing ones are at we of a capsids which

the Biogen manner have for we ensuring are talking Alzheimer’s, is example. that Novartis programs, an most believe. that intravenous regions in for Alzheimer's by impacted that Looking the we at challenge about -- the for widespread brain particularly coverage,

so there are watching change. the there brain great interest be other capsid those Biogen, we will when has making And with evolution, crossing And evolution for with paused it's people's future, in in significantly three soon been strides captured people. with capsid a barrier program, progress, we or other assets for advanced that blood the is truly years are we've progress suddenly over and believe our I become very us attractive it

Maury Raycroft

Thanks helpful. That's taking my questions. it. for Got

Alexander D. Macrae

Maury. you Thank

Operator

question. Zhu come our next our of One next will Wells for Yanan moment Fargo. from question And

Your open. line is

Yanan Zhu

taking Hi, thanks for our questions.

there knockout you there On the of program, you opposed since And driving of is wondering transcription suppression are to Thanks. technologies? just to for NAV expecting what X.X both using as for straight repressor approach benefits? advantages approach also, percentage and -- expression are have have

Alexander D. Macrae

like sounds this technical you. for a Jason, question

Jason D. Fontenot

Yeah, thank you for the question.

start So the with second maybe question. I'll

that excited of or we're potently. our we're few believe it's regulate type of approach and the We repression reasons. we're genes really about platform. mutation, able do stranded allowed a for are we any and breaks, very able that to very particular introduction without because optimal epigenetic for really this One, And the specifically to -- double

believe term. for target be what XX% this and gene the using we the So stable essentially close we've of approach, long is will repression to it achieved quantitative

believe especially DNA permanently. using approach and So modifies any of are cells we're instance, where superior, the about than nucleases kind it's very or the for that the non-dividing, excited the in CNS, approach

two is platform think are of There's is to gene the basis, Purcell and the our needed I to repression, therapeutic Purcell and needed, can on design that repress repressed. we to things cells considered there's how that great repression number What's really expression achieve the repressors we is biological amount that about that the being effect. coverage, Regarding much the there. think the be

are And and achieve we we'll will And done what of preclinical we've necessary potent the is across doses take the at animal single level that by dosing understand cell reduction then through to that and in dosing types explore, in patients. multiple therapeutic models very want cell appropriate models, we into what basis. identified of repressors pain. to range that And explore be will so repression then to

Yanan Zhu

remains program. on on Got about a -- off this do until hear -- Could also Thanks. maybe it. to you quick data or the at whether update Thank Fabry the we off maybe one those disease moment who to have should that? we you. questions, And wait patients next comment question two were ERT

Alexander D. Macrae

Nathalie, can update you that?

Nathalie Dubois-Stringfellow

from that so been Yeah, are far, withdrawn have still ERT. all the ERT patients of

Yanan Zhu

three. higher to you cohort first and have designed it think move patients the those the this like I sometime like three And to helpful. TXXXX year, a Very lastly dose for will program, you sounds because designed is have sounds I

So decisions question. you that's dose a think, I could yeah, for yeah, escalation about talk potential there? And

Alexander D. Macrae

can Bettina, you that? with help

Bettina M. Cockroft

question. And you absolutely. Yes, for thank the

So and to we're extremely happy One. dosing concluded in enrollment have Cohort

that on by up. monitoring For TXXXX, we shortly we're can up this. the we lined And patients very excited to coming committee move have plan to And so safety then in meeting, a May we already have cohort. next

have agreed recently agencies countries dose to in regulatory trial also have conducting the who are protocol. escalation representing engaged of we're that We Europe an with where our with optimized design

plan this accelerate this we'll initial data the then means year. to end we share One dose And by to from and hope of so Cohort escalation

Yanan Zhu

Got it. updates. Thanks for all the

Operator

our for question. moment next One

will question of from next Issi Our RBC. come Luca

open. is line Your

Unidentified Analyst

Maybe our the Luca. questions. for for This first taking excellent. announced restructuring on today. Lisa Oh, is on here efforts one Thanks just

And or wondering initiatives funds? other A? Just for you either raise what thought explored potentially royalty being monetizing have stream are to about capital preserve the Hemophilia

Alexander D. Macrae

announcement the a question. was for you can on part you important Thank restructuring our The today. of comment Prathyusha, this? very

Prathyusha Duraibabu

Yes, Sandy.

organization. ways running Sangamo but leaner, strategic we'll the outlined on the restructuring in And today's call. was So assess to continue we a of a difficult it line focused was as decision with priorities

extending clinical the our the for opportunities and for As will different runway, monetization. different at avenues a levers company we evaluating as royalty far look including we're pursuing actively cash as different financing

Unidentified Analyst

Sandy. ahead Go thanks. Alright,

Alexander D. Macrae

be simply of when is. to for in Sangamo access submission to more gets about valuable The closer or It's that we revenue need return it important a And thoughtful an future. it marketing hopefully medicine, significant we and the the revenue.

for lead And so to that the value, versus looking balance shareholders. of is that's near-term all return both at to Prathyusha long-term which constantly our cash will

Prathyusha Duraibabu

That's right.

Unidentified Analyst

question. big on picture And more one a Treg the That's of maybe just helpful. Thanks.

the to going an some thinking are accelerate be next data for Treg, year data your are thinking, just be indication? autoimmune going steps later towards get this end, what we to that moving announced by will maybe positive, on You should the

Alexander D. Macrae

about Tregs think the please? help you can Mark, us

D. Mark McClung

mean, I about performance the TXXXX as we Treg. Yeah, model Lisa. we is to talked assess perfect of a Thanks, biologic think

they're we've really progress. we're we about, ILXX bowel move be as the providing year. towards have disease. of multiple them programs, continuing for can to and update focusing we'll autologous as MOG we're ensure an we those IND. the for preclinical towards of about on And We forward so talked sclerosis do to that Both inflammatory excited primarily programs, the end to looking our forward and mentioned

a and And to of assets so lot this. focus against the work we TXXXX trial. that to those excited as the very committed continue We've got execute both continue to execute we're of to preclinical on on

Unidentified Analyst

questions. our Thanks for taking

Operator

And one from our our And Patrick comes H.C. question for moment Trucchio last question. Wainwright. next of

open. Your line is

Patrick Trucchio

few I that a had Thanks, had up asked questions just been a morning. from earlier. follow good few

for to if are some to in prioritize of AV neuro actually what more I'm in intention for and how engineered this will this and you a these wondering the the the the CNS CNS selection bit capsids accelerate portfolio, was HIV enhanced perhaps platform details just The delivery? then first decision, enables was just just just tropic particular, with with autologous additional it And near identification, I you programs, the the like about separately, indications IDD? please for with just looking talk capsules, of little can you selection and how provide INDs SIFTER can the around the term CAR-Treg, wondering broader MS regard

Alexander D. Macrae

for answer, let's you So, thank question. your Patrick,

ways. in Treg, please? can you talk and And Mark, capsids, two take about please. this you try answer can Let's Jason,

Jason D. Fontenot

Sure. question, Patrick. the Thanks for

I about develop capsids. also that to But capsids. in question And what guess, in that we're two the I we're group. different the the using there's actual and decide take really evaluate in taking the evolution capsid for work So excited to There's ways. we're can we're our approach technology looking

in that development as think And research component, specifically collaboration that neurologists our the Zinc have, to is a and Finger optimal are and emphasize identify addressed development work between for to the we groups want our our scientists we our being so epigenetic second done group platform. on and I technologies that with that indications

where So think and targeted. or we system can brain really think we the in we about in move identify the once biology needle, we be anatomy the the and needs the where to diseases nervous

diseases, nervous so different the those And the different system. for areas brain, of are areas are peripheral of sometimes they

a And think, identify to we so do target, once to want both where targeted. that the that. capsid we team then capsid is best have we And we libraries is what okay, that developing are

facilitate are transduction, the high that efficient in we're pathways cells that So blood a both targeting frequency evaluate specific transport in have that randomly known interested that novel capsids indication. libraries to for and for in more can specific are over produce we we evaluate And brain transduction the diversified. capsid areas barrier. highly of animal to those we And models look

so And novel approach use capsules. we to for that's look that the

have that delivered of move can one and be And us be therapeutic have So need. in to other injection. to think approaches delivered indications, whole get use those And can that direct the that we excited forward. in and areas capsids libraries are the those that set we that we have by designed using systemically. And delivered, we are obviously these we we idea designed libraries And of both libraries some nervous for we're hand, of to to that we're the an look of once effect of we be have system targeting. interested of to a we're

Alexander D. Macrae

means we a and the couple all with And have that, as days rate of to I focus make is frame that had this ways. these burn know, of have prioritize, in on we to as to capital want we access Patrick, we've had, And to when mean, prioritizing. on a really choices. I One, the result we've So a challenge. it Tregs, to

auto to that advanced, proof of and programs, not make the little And forward. case the TXXXX to had we execution choose sometimes are do. focus in of a a moving them choice keeping concept And preclinical of on organized the and would are more really we XX there Tregs, things to trial to which

to the make the [ph] a also Tregs LL interested future. that But in work had that know, us Tregs. to investors until they're we've they want to to deprioritize I So choice say invest love and the the you difficult in very would to say our

But And coming assessing want around a that If purely and those with also to so ourselves bring do we're choice we'd partnerships. that. we how partnership do to set interested ended accelerate can make can them is we unfortunately. meantime, one that had we're looking thing conversations expertise ended programs enough money in a the of and and having best restart up up But again. that parties it's having we the in, we've to and them we've up

Patrick Trucchio

very That's you helpful. much. Thank

Operator

And questions. to would I'm back Louise I call for Wilkie turn now further showing closing over like no to the remarks.

Louise Wilkie

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

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

Operator

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

You may now disconnect.

Sangamo Therapeutics (SGMO) 27 Apr 232023 Q1 Earnings call transcript