SGMO - Earnings call transcript Q3 2017 | Sangamo Therapeutics, Inc.

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Operator

Good afternoon and welcome to the Sangamo Therapeutics Teleconference to discuss Third Quarter 2017 Financial Results. This call is being recorded. I will now pass you over to the coordinator of this event, McDavid Stilwell, Vice President of Corporate Communications and Investor Relations.

McDavid Stilwell

Good afternoon, and thank you for joining Sangamo’s management team on our conference call to discuss the company’s third quarter 2017 financial results.

As we begin, I’d like to point out that today we will be referring to a slide presentation.

the link the slide of Events and section a website, on the find Media on www.sangamo.com, and of may to presentation the our Investors You site. Presentations page everyone I’d also that discuss currently the this during have the upon and call based that forward-looking we are that conference information like statements projections available. remind to we undertaking performance This an we the market of our current information perception today, updates provide will likely to you the obligation change of future in over are with time not by discussing future. Sangamo and the substantially Actual results later what may from and today no our that should differ today a from at we still valid. discuss assume are comments one date files you the Quarterly Exchange aware of and to Report detailed Commission, risks specifically alert our in Form Form on be our the the on with XX-K and Annual Securities are We company XX-Q. on Reports that documents that actual company’s our materially These cause factors that important could the forward-looking or projections those the results from statements. documents of differ to contained include operations in call President Herberts, on Chief Executive senior Chief Sangamo’s including With Chief management, and me Research; Holmes, of members Officer. Macrae, are Kathy Michael Vice Officer; Officer; several Ed Sandy Medical Yi, Financial Chief Conner, Business of Curt Officer; today this slide will we refer during again, And to presentation call. a this the are site. and Those Investors be Presentations section on slides our of and Events found to page Media of the to like I’d turn the call now, Sandy. And to over

Sandy Macrae

review conference quarter to our Thank XXXX. to business financial you, highlights McDavid, welcome and the call and from third everyone

and continued biotech last our of months, renewing in in we moving capabilities, in the made evolution Sangamo, Sangamo, of develop sheet balance heart our partnerships, We’re We’ve taking our the California the preclinical discuss a programs, establishing and and steps through clinical process. are Francisco. And and sometime screening as programs South and and pleased In treated we’re the great Bay second clinical to with of strengthening from patients the all, share for take Brisbane, trials programs new San Area advance improving few our plans manufacturing the need development the to you community, product core to the including headquarters progress we the will and revitalizing patient progress program, technologies, the clinical we through into advancing in towards other SB-XXX commercialization. the clinic. growth

managerial, location Brisbane, now importantly than workspace Directors provided more in Board Our late as open encouraged future. and will our planning achieve to competitive expect beginning we to talent, successful has more will company’s bold XXXX, in us need recruit and of square footage. manufacturing goals. for just which a be us long-term The new the we It headquarters we the clinical, to operational provide

Bay. in East Our in our we’re and Point roots are scientists talking the to Richmond

retain here facility improvements our will We research product be a Today, our manufacturing center to the and for also Sangamo. development announced to update presence capabilities. and we

technical very and Technical manufacturing and leadership valuable, the him our and to served We years development to have of at Vice development. to Andy of in Operations Operations over happy and delighted XX new control and team. extremely comes as President, Pharmaceuticals. cost J&J, welcome a welcome experience resources Group most who And and Ramelmeier, we the be manufacturing Merck, BioMarin, experience manufacturing, to timelines operations pharmaceutical a at will Technical Sangamo Senior develop Portola continue quality, capabilities Biological product recently of our to his Head with process I’m to

both in years, of CMO Brammer organization, and a over and clinical manufacturing for genomic has later supply therapies. been for discuss we and in development have therapeutic XX Curt and to Bio Brammer contracted As vivo capacity the ex for our commercial call, with establish leading manufacturing providing our is will dedicated clinical vivo batches [ph] programs.

addition, on our Phase build initially development own new GMP state-of-the-art X/X us and manufacturing Brisbane focused facility, to enable product In the clinical will headquarters trial materials.

Our approach to goal manufacturing. establish for is a balanced

meet demand pipeline of as Bio and on our capacity own their We’re to needed development our will expand our commercial continue we’re the development expertise for greater with manufacturing. production to augmenting with process, facility approach ongoing in focus with discussions clinical to This vector us towards a and ensure we ownership Brammer product to scale growing in-house take future. allow address complementing to and a the our our flexibility current business manufacturing

lentiviral-based clinical a I’ll focused the in beta-thalassemia, Sangamo’s therapies outline other Kathy integrating we In for start addition over announced to delivery Bioverativ. Ed our application the a candidate be other therapy an call trials. for our results activities ST-XXX, finger the IND cell to now Conner, on advances approaches genome programs. gene-edited Chief will pass Curt research. review the efficiency collaboration capabilities developed of of financial clinical being up operational then accepted update recent of the our our you headquarters, approaches recent in for as Officer for today. novel specificity precision, and will the We randomly summarize third gene lease long-term and an zinc of [ph] and part In will headquarters, and will At target believe editing Mike a moment, Ed provide Sangamo’s FDA editing the the our significant over highlights nuclease advantages development. will new and with progress that on slightly on our advancement update Medical it the October, quarter. call discussing our new our of T-cell

Edward Conner

share good five progress the quarter on to the Thanks. call. third excited on afternoon, and clinical our you, we’ve everyone, the very now in made I’m Sandy, programs. Thank

campaigns head yielding A. into development As and starting the the expected, programs for of severe with go outreach I’ll the our year. through we scientific of activation sites, patients awareness end as results treatment SB-XXX hemophilia of are each

two screening to more of us slots allow by the activated we’ve fill plan and more Today, continued we study, first year. X/X Phase execute patients several the dosed to open become SB-XXX and available. end as on to three in sites opening have the with the six they For

success Our the produce no and is study of bleeds levels in factor resulting definition need spontaneous no to products. factor on replacement sufficient of of

have by a anticipating from treatment and to track be first-half in hemophilia for this program one the B continue of on severe data screening to additional be of for actively the We XXXX. we’re continues Enrolment site year-end. to patients challenge, activating but SBF-X

have by I, we SB-XXX, activates end three MPS studies. on sites these working the the SB-XXX sites and patient working II, by SB-XXX with principal and centers initiation four more currency the two with year the end year. on around for our MPS more are For the of academic closely investigators enrollment and opening activated and of five programs We SB-XXX

several advancing very through patient winning soon. first are We process. this And patients our confident, treat I’m will we

visibility patient. We studies. enrollment of movement across formation a of programs for also pipeline is important in greater An of the development new have patients the toward pediatric these the MPS

could to number normal is key and range off ERT of where GAG pediatric is the file are lowering activity scientific the of them would this from Health I treatment levels children, level percentage benefit? tested SB-XXX that patients Europe normal, withdraw benefit while collected consistent asked of programs development what truthfully as next a current our and approach in Success X/X our appropriate integrate strategy therapy. only and studies they SB-XXX plan greatest the could to to to will threshold genome mean to for treat what in into a safety We for approached the levels lead what Authorities, clinically and excited information disability MPS to in with recent ours, X% and sufficient me, this, has for in our means about a this, include children GAG. discussion enzyme of care a Current evaluate never respectively, replacement of maybe irreversible X% suffer unknown because European achieve a they levels very and lead is patient. it you in the they is study been MPS this therapies is to In GAGs. normal and effects year. adolescent above urine of Replacement both to enzyme activity advice encouraged Our have is ERTs drives study lifelong permanently believe potential the to meaningful ear for Phase within have study to adults. patients no open Phase enzyme one before provide like maintaining we consistent of X/X and levels I’m a benefit. activity CTA exactly our and on after a of studies standard gene-editing permanent is about enzyme to enzyme lowering A II, still

most received IND recent is program for We and gene edited clearance from The month ST-XXX, autologous the FDA. beta-thalassemia. three last stem last successful IND treatment was reasons. an for Our cell

to have time and First, taken we develop gene-edited the efficient specific most the product. precise,

with have product, manufacturing experience and our preclinical the FDA and demonstrated we of a of wealth Second, robust a toxicology. characterization

a Bioverativ expertise submission. collaboration technical close leverage our Third. organizations years to both with successful between ensure in of experience

XX, editing advantage believe body. lentiviral-based with strategic through therapies treatment to We potentially a for Slide that development, non-viral single As natural mechanism more approach contrast a integrating allows the protective is lifelong harnesses permanent therapeutic delivery genome a beta-thalassemia superior be the controlled involves differentiated to long-term has a with profile. a administration. gene this and precise on ST-XXX, strategy shown randomly highly a in offense, the XX potential safety that our therapy In mRNA of of for

advance in of With call Research. Turning to phase turn the to Michael continuing to to first I’ll first-half work next have Slide our to into Holmes sickle in of also our we’re Head Mike? XXXX. patient now the XX, cell Bioverativ, year. enroll over we initiated program expect disease the

Michael Holmes

T-cell work on regulation afternoon philosophy. Today, I’ll advancing good on programs: engineering in rapidly approach of genomic of for and using gene nanoparticle, Ed, three delivery Thanks, our touch immuno-oncology, and research to, therapy our recent the non-viral everyone, lipid for call.

the at with level to attributes: involved to XX. in target all therapeutic is efficiency gene single-based precision, As genome and development of therapy. Sangamo, attributes contribute ask to all Sangamo specific editing with editing clinical of continue at to everyone’s and the for specificity modification in interest efficiency. to in must three. editing system of on a genome term evaluate context together field and we the the I to the Specificity Everyone some hypodermic editing editing site. the gene Efficiency three these used target and precision therapeutic down degree tools Slide pushing to authorized the of specificity the work in ability apply Precision With desired sequence described must do we efficiency. other shown come success considered nucleotide of achieved encourage and others or can about be [ph] precision field. a when increase clinical three expense the at in the context therapeutic

has gene-edited XX, demonstrated recent comes beta-thalassemia for cell efficiency of of finger you’ll produce cell from example, and to our allogenic already relevant find example T-cell Slide Turning where therapies. even program to disease. programs oncology and nucleases one the universal specificity more sickle in application recent the Sangamo zinc therapy precision, our And our cell or

assay. editing Turning achieving and and highly the a very capture of achieve locus compounding CAR-T data TCR and TRAC XX, of of greater Gary and the endogenous XX% detectable production of allogenic inefficiency even analyzed of standard his with a [indiscernible] the cell than allogenic for Thanks therapy cell gene XX. the cell recently response. and activity TCR Earlier BXM of gene high than off-period no into the for knock-out This genome, hard is gold work product, the multiple because has effect Gary with construct Slide into double BXM of important More the an and shown only knock-out of to also sensitive on widely modifications. of but the we to immune team, oligo our the CAR greater the Lee this not double gene widely target levels Gary than year presented integration XX% to requires begun Slide on demonstrating manufacturing host greater XX% were efficiency. genes eliminate integration

HIV required of significantly gene all As Even you each Slide efficiency can expand see the contained and modification cells of of making not producing risk select cells therapeutic is the why of step more for compounding effect the well drive above throughout in in three efficiency, years XX improvement the and T-cell increased from to oncologist the can XX% only is XX%, allogenic our to clinical difficult XX, program. is ZFN that believe actually enough attained immuno-oncology. expensive T-cells expertise development at we’ve decreases. process editing best-in-class into it editing our to platform are modified having three the not editing levels gene legacy This The operational more genome of of we cell efficiency of manufacturing and for results the number inefficiency all Sangamo if XX% T-cell. in of and That modification population. generating runs result well the therapies

to LNP on platform, our like represented Annual of our delivery Moving I’d share advancement, weeks ESGCT Congress. to ago couple which the now a at latest XXXX

dose as elevation X.XX editing believe Conway Based these Turning that efficient in close to toxicity we XXX% the at gene XX% liver data, scientist Slide LNP well doses with within to This over of demonstrating presented is new load protein efficiency Sangamo in can in encouraging long Anthony dosing as knock-down LNP mice data window XX, relevant with mg/kg. genome Sangamo company at mice low other highly hepatocytes no delivery achieve as extremely approaches. a we the platform. therapeutically it our than is demonstrates non-viral on with

tauopathies already of non-viral Shifting ZFN primary to and AV amount series development on test of LNP delivery work the program, effectively non-human in of initiated studies We’re barrier. the to a share brain larger forward and our moving recent some take Sangamo species. blood of efficiency now I’d proprietary to the Vector our in our to more potency have moment to designed cross our like

regions the thing the brown transduction superior right to images comparison target mouse proprietary neuronal the AAVX, type Slide results. of our transit no XX, on in three demonstrates by range Vector AVX of seen in in in As [ph]

that collaborators a data for finger in reduction administration tau and presenting XX providing I of further to for protein In new Herberts, Brianstem XX% be Vector Meeting to next will Sangamo in Hippocampus scientists after week transcription results mice. AV And factors Cortex, meeting. Officer using now Massachusetts targeting Neuroscience. single zinc our the Annual tau General addition Hospital the from our update. to over demonstrates proprietary of I’ll of our Slide for Motor up Sangamo’s protein turn Curt? a call the forward Curt XXXX Chief the program Business Society of at business update this look the and

Curt Herberts

I’m think programs and on innovation, about Business and long-term Thanks, the progress and our and we afternoon, excited Sangamo’s Mike, strategy as good Building business and Chief everyone. corporate technology clinical planning. operations Officer, to discuss how

core As are and and that we’ve strong discussed before, is business we programs others. have a to very strategy retain platform for Sangamo product pipeline, right-size externalize and our to

from that look fully business sustainable biopharmaceutical a location, people, to then clinical recruit appropriate to commercial capability. talent into company. those we company forward For a integrate requires we capabilities This and too and the the organization as retain, early-stage we strategy progress an integrated pre-commercial full-fledged into infrastructure to

cluster step learning is towards the the securing Biotech Francisco of of heart updated Bay San on in important [ph]. earlier an this new mentioned realization Sandy As call the Area South headquarters our first

with new these XXXX, growing expect number a to in open to capacity facilities We employees. accommodate of late

also headquarter to positioned functional areas, we facility Richmond Once operational new clinical many company’s a capacity. up San the our Brisbane with offices dual is including top business, innovative research in our talent running, to center. be and to will and improvements model technical Our Sangamo announced transition our laboratories Point and and capabilities will attract strategically in remain across Today, manufacturing as play current some Francisco operations.

as highlighted, with Head timelines Ramelmeier, to have GMP, and working experienced through Brammer and Manufacturing. this that, It AAV to There with is pipeline leading product Technical Two, relationship cell large-scale into continue been gene development Bio, organization, contract Operations expanded will Sandy note and it manufacturing. of we improvement. a and the on for acquired portfolio improve strategy important focused new One, we’ve better three we commercialization. President control costs, who business advance years. to XX talented and particular we parts with a on clinical leader, us focus our So is Senior to past Brammer manufacturing and Andy are our our help as quality, our Vice therapy

genomic our Our new planning Sangamo several our own next to entire as the dedicated over GMP manage our evolution years. planning ground facility, facility facility GMP with operation enable and the new to be as to from in build in-house support will the capacity, greater our and a for products This the us AAVs GMP portfolio provides process The in headquarters are Three, company business take agreement we which we advance state-of-the-art operations floor Brisbane. build programs of perspective. control strategic the a over is will technical to in corporate large-scale key pipeline manufacturing component strategy. to decision an the housed

we XX months companies have restructuring and on such made the past the and focusing establishing in company, the tremendous balance A key Over strides with SB-XXX pipelines, sheet. the strengthening hemophilia as Pfizer partnership

laying technical this we strategy on integrate area to Sangamo, in investment best-in-class and in true ownership editing product the gene for we a foundation our and in advantage competitive greater the our of with therapy manufacturing and have take are we forward pipeline. where Now, operations, based future genome believe to

for therapeutic ready the as transition plans and integrate headquarters. updates Now smooth providing when is on products. of necessary for forward and growth to our we to establishing expansion make are growth Sangamo opening the own a new I to this we support, our further infrastructure look progress commercialize

of vision are new the committed capabilities with application evaluating for are oncology adopted cell the of right T-cell the genome to our editing opportunities partner business gene to who we ahead editing future development, therapy. Looking combine to

for to are gene best-in-call partner regulation also several planning indications. technology our CNS We

to are We providing of our study we program HP call the forward for program non-human and to this our calls. the I and I’ll strongly third Yi, value in now the believe over on CFO, in trial will future initiating update. updates currently additional investment the this look quarter studies maximize financial primate For turn asset. Kathy

Kathy Yi

in or We you are was results net or detailed share period quarter financial $XX a We share compared of position and the on investment. milestone $XXX.X earlier potential XXXX per $X.XX and capital clinic any or the same middle advance for cash novel press lead and Total cash months included for Thank afternoon good loss $X.XX to our excluding million delivery Based technology on XXXX. programs business quarter in pivotal believe full our third Curt with XX. third asset release our continue studies, issued our which pipeline we cash, to today least summarized ended equivalents have current clinical towards payments, Slide XX optimize the to and with that the into million everyone. quarter new the million $XX.X collaborations for transition and at of current XXXX, we platforms. per

and completed we With our our recently More out for CMO, pipeline. importantly, this late Brisbane, position are at we Phase ensure investment XXXX. supply to by manufacturing build of believe secured expected in strong I/II includes in clinical to cash our better and capacity our facility be manufacturing position capability future a therapeutic

maintaining to financial a least our comment, we cash. with end at final and million guidance $XXX As in XXXX expect are prior

Sandy. my the in solid today. our I’ll out concludes a new This We facility. operational and over turn to the financial the remarks infrastructure for call for product necessary now pipeline position are fund Brisbane build

Sandy Macrae

Kathy. you Thank

report half at data Clinical is clinical trials are pleased continuing in with closing, XXXX. to in make in enrollment XXXX. sites and our out progress first lead we of to commencing laid activated the beginning goals we’re of expected in that the the I’m So

of and setting the finger the critical Our across for of standards efficiency is zinc the editing TRAC specificity. optimized precision, nuclease genome dimensions technology

the LNPs. genomic We headway novel development of of are forms including and in new therapies, of making delivery AAVs

progress expect the progress collaborations with occupation, hemophilia successfully a program are for believe future. laying future announced to partnerships forge are the we foundation Operator, programs this our into A Our questions. our for are XXXX. in along ready manufacturing and and plants hemoglobin to in we Sangamo With and headquarters, we continue advancing now these new selected accelerate look of newly we to strong forward for

Operator

our first Birchenough with And Wells from Instructions] [Operator line question Jim Fargo. comes the

open. is line Your

Jim Birchenough

the on guys, progress. congratulations for all and questions taking Hi thanks the

questions, gene X think the the you So, efficiency the in and if a similar I’ve about transfection transfection maybe couple really B for for clinic programs before, beta-thalassemia the wondering got around and terms editing the efficiency in the program for helpful. detail that hemophilia through could you but now then go I’ve of right a and how asked follow-up? are is just X MPS

Sandy Macrae

Okay, thanks take to do Jim. want that one Mike, you on?

Michael Holmes

of transfection third Certainly, sort three was vector the and vectors what all so different you’ve of DFNs and components, delivery PRPs, [ph]. well component, as efficiency allow expression. studies terms as two maximize efficient I dose then gene into drive achieve the therapeutic of up TRAC target appropriate in of as we the two what appropriate our the donor levels ratio that as vectors of to of each both levels was are it as evaluated donor deliver to comes delivering And when the would to IV to delivers integration brought locus us well where optimal the think the or the one, vector of in we to three DFNs number each pre-clinical things,

how very so of locus. about high a pre-clinical that’s obtain efficient with integration least optimize at came able of to we work we and the transfection that at to the ensure TRAC to efficiency sort And least target levels trying from were

Curt Herberts

Curt, it’s is couple obviously of therapy very and a vivo situation the in than vivo different genome Jim, Hey XX a editing. ex add setting just comments, to

optimization about only as need of very submitted seeing we as levels the good in knockdown the the the laid target this the to XX% really we technology, well work to the and manufacturing as now beta-thalassemia we [indiscernible] of we vivo So X% both in gene was done, setting XX% therapeutic are you than for modification, Mike that expression. beta-thal our of protein T-cell order the have improvement and strategy, excited vivo the earlier are foundation In as that are alluding drive for the greater integration well achieving we as gene IND was think as for terms process regularly in ex year. ex

Jim Birchenough

on just then … the And

Sandy Macrae

I’m go sorry, ahead.

Curt Herberts

follow-up Jim? You question have a

Jim Birchenough

X maybe Yes, some going challenges that terms and pipe screened you screened the in dosing trial, the patients X of patients? are the so could of and being of just that in from do to some the and B as discuss in have confidence into MPS of patient at patients going you clinical the actually dose trials to kind look hemophilia that execution, converting are being actually patients screened you be

Curt Herberts

solution. the clinical to are clear that And do it’s you that? them in I talk and work need to want dose confident as Point recently And at I’m want the into wants these some had patients Richmond trial. visiting a group. MPS waiting that to we and the really should this of say patients We we to patients us like the get because Ed, you here

Edward Conner

and we studies really a is there the best just in forward. way are awareness I/II number screening first possible in to the so I community to and your are broad is Sandy’s mean letters gotten all evaluate them there to the that about we study moving are screening a patients respond I safety but are I the to the who enroll recently activities, the study received Yes, point, patients Phase these think committed MPS the from of who to think and and enrolment just in can, comment, this in study looking to want letters I’ve

Jim Birchenough

taking for the thanks and detail. the well, questions all Okay, for

Sandy Macrae

Thank you.

Operator

Charles comes the of Jaffray. with Thank you from question line and our Piper next Duncan

is line Your open.

Sandy Macrae

afternoon. good Charles,

Charles Duncan

the for sorry background guys, noise. Hey

questions like perspective those that the I patients on my I First I the of that in that’s in Also – of dose, the anymore of first anticipate you have and relative to pediatric work for ask a II, all progress congrats adolescent me, on really trial. in taking really the something safety. did couple I When to along really patient But quarter. can’t in about think question, being to you it on intriguing terms I congratulations able and MPS announce XXX lines? thanks would and that,

Sandy Macrae

that want you to do take Ed, one?

Edward Conner

Yes, sure.

So of our integrate and as authorities met benefit these potential we all European the with factor children. of studies, very realize in the therapies and into we which were health is that enzymes provide primarily greatest lifelong about taking the can the enthusiastic who we or treatment mentioned genome for for

In terms announce activity of forward announce not would regarding when working I’m at of we when would we’re give Europe. to anything, we with sort sites, and to any actively time moving this but identifying timeframe prepared in

Sandy Macrae

to But CTA is those much you simply that by tougher mentioned we have – a go through process and of and this timelines?

Edward Conner

Right.

Charles Duncan

question forward if Okay, And then of not into anyone well Curt, I’ll perhaps appropriate couple look update. else. a for that sure this is for or him

terms different with will you, a company, to headquarters that in are on certainly be difference kind visit you consider of in Francisco. local price gained? advantages San you Well, in understand see the or there were help is we’re other a change if with that that perhaps justify terms per really taking square Can us other of wondering regard incentives great evolutionary possible the or to in in foot, the of gained competitive hiring, to location. Regarding you advantage there I’m distribution people easier

Sandy Macrae

Kathy stay individual Hey and with Point that speak Richmond building the to for North, the is We you comfortable abilities they’re of can up here. that. aspect in of with question. were the Yes, so overall and financial quite kind actually cost Charles the the thanks obviously surprised

Kathy Yi

So, we’re how versus there that in we at what’s negotiated look financial read, and out this if Charles, we today X-K building. with the pleased available the commitment our you very of the and our that see you’ll is issued – length for facility the that laid marketplace,

Charles Duncan

guys helpful you CNS traction, activities, that biz to potential regarding oncology is if for technology in either any very timelines that’s the last of activity? partnering or see regard and applications for to with progress lay Okay, certainly I’m and excited making question to the want dev wondering out making you

Sandy Macrae

to be continue very active So this of them, area. we for both in

oncology, discussed and is think with I constantly you launches of rapidly environment. I changing world For as oncology acquisitions the moving and world prices, have before, and the

be and T-cell that we are And optimistic interested a to the editing are so we best-in-class have about really in. continuing very this quite of number people

because have think we to package, perhaps and the would into sure you appreciate that the why to some we activity is did that’s benefit that primates the in CNS, very asset. of agree we moment recent of that of maximize that encouraging. recent our forward feel the looked nonhuman seen allows I raise as and we we’ve us For will the I’m what more compelling so value data to this some deal at extremely data, And take mice can the be

Charles Duncan

Yes, launches, add in for acquisitions I of absolutely may in Sandy. questions oncology and making so taking quickly, also my thanks world change term the also

Sandy Macrae

Thank you.

Operator

comes Thank you. from line Maury question the And Jefferies. next our with of Raycroft

open. is line Your

Sandy Macrae

Maury. Good afternoon

Maury Raycroft

and Good afternoon Sandy team.

question, dose quick different same one the patient patient a is I’m side? at a side hemophilia or on new First wondering first the just if A, the as

Sandy Macrae

Ed.

Edward Conner

patient same new side. at The the is dose

Maury Raycroft

it. Got

Sandy Macrae

just remind of to want – us all. Ed, to have, underline sites the we number just We just

Edward Conner

sure. Yes,

has the the So more intense been three year. six end in interest have physician we activating sites. by of There community. Currently, we’re the

we mentioned, who I as and are – of patients number screening. have actively we’re So a

to So the as we’re things, yes. up in able open study and slots open those fill

Sandy Macrae

And sties? those patients are different in

Edward Conner

one not It’s across patients sites all these are that are nationally. yes. Correct, center, but

Maury Raycroft

in if provide electroporation into any on on efficiency the knockout the for And them? can process time vivo just and me you’re provide what using can T-cells ex any how insertion, in And you if comments could that? you. that you’re the efficiency for can XX% than cost greater and and Got that’s double you you translate savings remind targeted the it. seeing then if Thank gene-editing specific production

Sandy Macrae

Mike?

Michael Holmes

specifics So, I’ll the on the provide to try delivery.

for the of as donor and electroporation terms the then track messenger far the into insertion deliver using the logos. promote using ZFN In efficient to to as RNA, AVX were

Sandy Macrae

Curt?

Curt Herberts

Yes.

the So compounded multiply slide we by compounded presentation that the other. when at interest to the deficiencies we similar deficiencies talk look about where you in each

get that really were means modification all all when a what’s the therapeutic And point, with modifications. for batch. looking products out the more we integration? my that that we are a of level we’re product goods of going drives get and cells have knockout, dental number But to single significantly. target greater So more to is a XX%-plus in what the levels, efficacious have making for think, our significant product, three doing and to We which synergy we of of – double then down cost three efficiency importantly,

Maury Raycroft

considering LNP Got LNPs on data it. the that into are to LNPs last And your is are SCCT, you clinic? thoughts closer you incorporating on remind Thank delivery it you. just could strategy presented is exploratory, question just me what advancing are? you at or And

Sandy Macrae

don’t why and follow-up. this start then I’ll Mike, you

Michael Holmes

All right.

significantly this could we So with technologies, levels by I we mice think, of competing achieve where we at start at levels at RNA. of a SCCT, significant a XXX% achieve modification in I’ll demonstrated lower low-dose the in nearly RNAs hepatocytes greater long to could editing that messenger compared of messenger than

Sandy Macrae

this – but is strategy. of And part a key our

of we it success delivery. efforts delivery patients. to place. very novel why AVs good research available and successful genome to we is technology will done make dedicated the forward So and both that to at our to AVs LNPs, look the in within drive will group because what our be But have And that’s is feel a editing

Maury Raycroft

Very it. Got good.

Thank very much. Okay. you

Operator

our with the Thank of you. line next question comes from Ritu And Baral Cowen.

open. is line Your

Unidentified Analyst

This Hi. for Ritu. Alex on is

facility. to be will product? manufacturing expect of data product? programs in-house that capacity sort on to Just the Can be a there? what the expecting should couple tell the manufacturing should with that you we we first product eventually see clinical us And transition think what in-house expect first you of should the Do the questions we to

Sandy Macrae

Kathy, try can to that? answer you

Kathy Yi

Yes.

So, building. Alex, with the now the currently design right phase are we in

information what to as more out So clean clear we can – going square our But the our and understanding building capacity out XX,XXX is it release feet. about we’re be. entire

building possibility significant size product. a definitely it’s future So commercialization for has that

Sandy Macrae

in-house And also one Mark to the And people to at with capability. very future blend the of supportive with pleased – gives have in our us been Y, the dedicated dose own capabilities. and flexibility. driving Board being – without both, think, we’re work this to other do Brammer ahead they’re with dose we’re very rigid of I we it resources and one the Brammer And delighted team the X and be having giving able us

Unidentified Analyst

the asking questions. Perfect. Thanks for

Operator

question follow-up Fargo. a with have we – Jim And you. you. follow-up Birchenough Thank Thank Wells Instructions] [Operator from have a And we

Your line open. is

Sandy Macrae

Hey, Jim.

James Birchenough

more going expectations see patients Hi. we Thanks. this would be kind And expect a Hey, whether timing see little if them? liver trying two of to guys. of at bit have the you’d the to threshold the efficacy just willing if crossed to current I’ what was would enzyme we sense dose level? wondering on give detail we with to where of we’re SB-XXX. of get a elevation

Sandy Macrae

So, you one. could this perhaps Ed, sorry, take

Edward Conner

sure. Yes,

of that we’re we’re all very be So terms patient them study earlier getting moving the out timing deliverables on going track study. of yet. say, pleased I’ve we But discussing in, set the as progress track we’re and process been I’d for to enrolled. the getting with with far just this patients thus And through year. in keeping the on on not the swiftly that them the

Sandy Macrae

very is safety to patient And us. important

constantly we’re function patients. So liver in these measuring tests

Edward Conner

Yes.

Sandy Macrae

any We have you ideas look. have don’t when of preconceived to

so to continue look will these we after patients. And

Edward Conner

Correct.

James Birchenough

Great. for Thanks the call. taking

Sandy Macrae

Dr. Thank you. this at to showing call I’m Sandy And the turn time. further closing questions back like would Thank remarks. Macrae now no you. to for I

Sandy Macrae

you your for afternoon. and you. good Thank we your support a all on thank just for wish And today listening and you like your very to continued questions

Operator

does great program, conclude a you all for the Everyone, disconnect. This have conference. may Ladies today’s in day. and thank you participating and gentlemen,

Sangamo Therapeutics (SGMO) 10 Nov 172017 Q3 Earnings call transcript