ORTX Orchard Therapeutics

Renee Leck Director-Investor Relations
Bobby Gaspar Chief Executive Officer
Frank Thomas Chief Operating Officer
Whitney Ijem Guggenheim
Esther Rajavelu Oppenheimer
Yaron Werber Cowen
Peter Kim Barclays
Call transcript
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Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics First Quarter 2020 Investor Conference Call. [Operator Instructions] I would now like to hand off the conference over to your speaker today, Renee Leck, Director of Investor Relations. Please go ahead, ma’am.

Renee Leck

Thanks, Sonia. Good morning, everyone, and welcome to Orchard’s first quarter 2020 investor call.

slides of call our the the going access section Investors website, can for by today’s to You

make. forward-looking this Before to we from will those on implied differ remind like events include call everyone as with that expressed results of those may get various statements we statements in our the forth filings set and materially annual factors started, other could statements. and forward-looking Actual any I’d filed risk by a result XX-K SEC and or make any including uncertainties, and we

addition, this today views revise any In obligation any relied views represent forward-looking not date. statements. should forward-looking any on statements upon of any call We as of update representing disclaim our made as to only or be as our and subsequent specifically

that, with And to over turn call Bobby I’ll our Gaspar. CEO, the

Bobby Gaspar

like field everyone, the health our everyone. Hello, care welcome. Thank tremendous and care to you, patients during this of receive Renee. partners our efforts acknowledging difficult Thanks, start and time. to need in organization I’d by continue first the ensure in to

our last strategic of leadership, The together we company, benefit can for vision can to and been to it portfolio gene make really weeks what about can the a for and what need platform, cell our the based important When Orchard very even that become, the patient ensure us with populations can the do as thought team, potential diseases. carefully Frank therapy executive about have happen. I, take of true on stem beyond many provide hematopoietic current Orchard and that on think we ultra-rare potential taking all it’s new it few the Orchard. Since where we its period can have the an how fulfill

built is supported We forms value our will new focus by and Orchard vision taken of on creation. have that achieve around remarks the is we sustainable company four decisive believe a actions we morning. developed strategic allow pillars. plan bold that in this long-term This some and Each which chapter have to growth a and these

First, operating efficiencies.

made focus to efficiently important strategy, detail of more our execute enable our which series sharpen have changes in operations that and us I our will moment. We a a will to

is Second commercial build. our

We are focused gene model the HSC the of undergoing therapy, ultra-rare true on for value and of establishing see and approach this the diagnosis right of a patients series products. over treatment

potential manufacturing the near-term areas now our therapy have that one the be most economies space, this of leaders CDMO the scale. want We technologies network. exciting gene experienced in to right place taking are covered that is by invest our Third, knowing deliver in of innovation needs in and capacity to

beyond of pipeline central Finally, the our portfolio ultra-rare is strategy this to expansion to to Orchard’s less-rare indications. enable prioritizing

us to new programs been believe genetic already validation of time two a indications disclosing dementia or expand We first the frontotemporal clinical these disease. our research genetic for larger allow the subset ultra-rare confidence that and in these of are Crohn’s today, and subset and shown indications. has We are biological to that with a FTD,

first the chapter to new strategic Turning in our plan.

in strategic objective the patients that for and identifying here analysis need are on metrics left-hand both in was high with criteria X. started over weeks those programs side operational Slide This evaluation the an high-value considerations the on shareholders. the where several extensive and each past involved of are of shown throughout We financial that focused efficiency an organization. program for six portfolio creation there We improving our undertook using

horizons. and Each and different of many that different the intend over given in of these these ways you has value, imagine, can realize potentially As were we time nature programs. decisions to difficult transformative

Wiskott-Aldrich Leukodystrophy, believe and investment early programs the the This beta-thalassemia programs OTL-XXX mid balanced we however, late, MPS will Metachromatic a such and program, also with means and forward. stage as haven’t syndrome, focused OTL-XXX, on The a we and transfusion-dependent, are portfolio that moving Today, reduced our have have research programs. resources programs. ADA-SCID our I best mentioned

for realize through programs, We alternative value will ways look those to with partnerships. including

resources So summary its brings enable business. changes the core we’ve mission highest necessary set imperatives a at We objectives important together for and to and announced were to believe level changes matching operational of attention of by Slide execute today. X our the to that these the and a Orchard

prioritization we here, the summarized realize $XX our to portfolio. expect savings of cash million from As of approximately

the investment in from results teams and savings manufacturing facility. R&D to our in one million site $XX the decision to consolidate the Another defer

existing more each the our staged and XX% in to another will savings. headcount future workforce reduction build-out planning approach Finally, to yield $XX and commercial million the

All of cash savings expected million realized savings to be of and expected result $XXX XXXX and that these XXXX, are in total period. over over

near no With to the we finance. have revised runway term into cash plan, XXXX need and now

savings commercial, and innovations with drive help this treatment. following support million in after manufacturing, mentioning is and and other time including Slide briefly testing diagnostic no-charge to strategy, conditions In worth $XXX the X. initiatives, In technology, efficiencies identify new in MLD investments areas neurodegenerative on scalability. It’s to that the patients shown for process screening key our making to programs

exercise that deployed decision-making potential in substantial to is This future building growth in disciplined fuel while diseases of way. capital pipeline a all expenses, that ensure reduce leverage a in success our to initiatives an just a wasn’t the but manner, our In the to business. can phases R&D, less-rare across have company’s important our

elements to discuss over plan. call Now of let me turn additional key Frank the new the to

Frank Thomas

morning, good everyone. and Bobby, Thanks,

examined aspect tell this carefully release, we morning’s press of have you each our can from business. As

ago creating commercial sections evidence Bobby, manufacturing. our moment operational latest the we as thinking efficiencies, you I two with summarize will a and are around that and think heard we in from way You next deployment additional the see

infrastructure successfully. and will build to leveraged to also these Starting with market ability will future of We a that launches. product that the therapies for model we execute commercial. bring be developing the importance understand and model any our the commercial demonstrate This

will certain from the speed you’ll note launch impact Slide trajectory which the market. that disease X, can each As bottom the and we rare of penetrate dynamics with has

anticipate we illustrative having WAS can two but launch potential the distinct as fact, and see complementary from our launches you MLD In curves, diagrams. first in

established, start with with EU, an later point the on revenue me inflection the countries Let launch the think other around U.S. then where left, will to We by MLD followed the important come in we on MLD curve the expect it and world. first second to will affect diagnose penetration. patients market important newborn in so acceleration an an progression growth Disease MLD opportunity and advances be dynamic them early rapidly, treated. that screening when important a will is for is get providing rate. Because

an curve different, the syndrome, this right. slower Wiskott-Aldrich are For MLD, dynamics progressing treat filings the and and from prevalent of WAS readily more additional very Unlike opportunity outset and to are for program, also of disease us XXXX. on number the the This that it’s will long-term the patients shape a is in revenue stream. diagnosed. track MAA believe BLA reflected provide on We give and the

MLD time for line. an back Turning on the update regulatory to

the U.S., engaged from the FDA In recently if Agency approved, EU to track the of are The on later first submission a in in has history FDA our endpoint, the on the We planned decision package, with the sufficiency comparator including this data launch BLA of the provided the OTL-XXX CMC written treatment the clinical on we and the and Medicines company’s of of MLD. for data year, European feedback natural half package. get XXXX. the

facilitate this BLA. IND we this of data fully to feedback, result of later open more a to both designation, more RMAT an a believe dialogue the intend a will discuss year and we file submitting resolve comprehensive before also seek As and matters the which

to we’ll guidance provide closely filing new lines regulatory and agency, with interaction. We further the updated the on for working are the BLA after time committed

the the where future keep have often referred up can centers can franchise. the expertise. to able be transplant EU same On tracking commercial with patients that especially prelaunch also first XXXX, Slide momentum Germany expect qualifications. Many progress pandemic first half to site you we the for activities XX, and of the team country to a in for patients. launch of programs set work underway, up and with launches, we’re centers the where the MLD approved, been treat has see the We in if intend OTL-XXX are in leveraged of and being who These network are centers treatment neurometabolic to during key with of in nicely

of taking stages importance patients I early MLD steps are the long-term to we to previously achieve disease, at mentioned diagnosis and the of success. in identify necessary

efforts, Success for identification. MLD support Italy and newborn designed are disease looking awareness provide are State the initiatives no-charge such with in looking as we as these York testing partners upcoming will facilitate Beyond key such diagnostic validate at for initiatives data pilots screening also of in Invitae, implementation. funding and and we with patient the to MLD New are to that assay wider early typical

neurometabolic even progress challenging two Coming during disorders, with MLD this behind up and recent our proof-of-concept we programs made in where franchise, have quickly MPS period the COVID-XX.

our year, past next are one shown to year, promising registrational preliminary and bringing encouraging commercialization. begin plan closer the data engraftment, positive over we with program outcomes, announce biomarker and we’ve MPS-I, For excited clinical step trial early to proof-of-concept correction to this a

year a we Hospital, Royal late with planned announced last at in first continue proof-of-concept this to month enrollment Children’s in and released interim data be XXXX. was the MPS-IIIA, to treated that For patient Manchester trial

You markets these to can lead sustainable how see revenue the commercial growth. graphically aggregation on Slide of XX

of incidence designed we and you figures WAS it commercial In addition, and realize prevalence the help for the figures potential incidence included to On portfolio. each for infrastructure we’ve today. as build provide opportunity understand necessary slide, capabilities this the MLD the to see the is to the

ranges those at that tied to range awareness, and population, who has X, countries in often over a Turkey, there time, Middle the diseases on play Also, reimbursed. taken also which patient could in this the identified and may should patients East year be prevalent of believe from incident wide dynamics be We’ve differing countries literature patients previously as to where we described improved where the Given opportunity XXX I market we the MLD figures. are from conservative the view than largely on in therapy. disease with addressable believe per rare such opportunity benefit incident XXX a more for Slide patient

to platform not but product, be and has and strategy the products potential based been HSC continues gene only infrastructure. commercial multiple off rather one X on aggregation launching Our of always therapy

in Turning you’ll capital to to key our made manufacturing and short changes XX, manufacturing strategy. manufacturing. approach we allocate the of Slide On see some how tenets main mid-term. and new also the our We’ve in

on plan First, to innovative in technologies term, scalability. we the to commercial near focus enable

allow organization our those way. in a appropriate a to site to are we’ve all and transfer located on to technologies, London, to our brings the to efforts party focus quickly processes single improve easily made R&D of decision and be to currently a commercially in our ensure together whom Europe. manufacturing to efficient made scaled manufacturers, in shared will more then third Second, This which consolidate

the As of California spend. associated part of site, including close Fremont will termination we project the and consolidation, our this capital

Third, for identify the a new of with requirements. future and we product sized facility have bring and appropriately with in-house relationships near-term will we eventually is that And operations. and longer site ensure satisfy that fitted in clinical to supply to CDMOs to U.S. term, manufacturing strong techniques capabilities a commercial intend

technologies vector stable and potentially consistent. the planned the the will shows closed in the it our needed, enhancers, making In more launches manufactured, we are such to are labor-intensive, and of we’ll processing manufacturing change the way manufacturing: for invest, near reduce partners approach rely and our early Slide of build. mid-term, WAS. Today, to partner COGS product. MLD and line amount XX less as and automated of drive transduction This drug less cell continue we continue down and producer will potentially expensive investing, in in the and three invest products phases on

reliable has partner been For beginning, programs these Strimvelis. with a been they’ve example, commercial with and MolMed since the

and reducing to complete and existing additional finally, of for in build that thereby to and over us in-house drug network, scheduling for partner our tech to in with explore begun to terms have This the options when transfer the our challenges CDMO capacity. In some need market, time, more U.S. serve redundancy. enables a we aligned capabilities to U.S. product search to manufacturing creating are we And closer scale timing. there plan addition a business a is

turn I’ll to that, with Bobby. And back over the call

Bobby Gaspar

disease cell to In in of correction new Frank. specific briefly blood types Thanks, through going HSC I’m to research correct allow enable of cells, may this but portfolio mechanisms, and indications. gene specific highlight potential the section, natural expansion not therapy organ also of how lineage into our only systems

are and gene to and these as of insert including cell of the lead immune therapy, into you can Slide platelets. copy types HSC multiple in a HSCs, gene cells, XX, of through genome to modified many we working bloodstream, permanently shown on the know, As red and cells genetically blood cells corrected able the

In naturally directly to of organ addition, into an macrophage us organs, cells deliver HSCs genes to those differentiate gives systems, monocyte and and that tract. the and brain the opportunity can various GI into proteins migrate lineage including the thus

preclinical Within neurodegeneration. we therapy MLD, clinical particular, proteins and MPS-I how programs can the and deliver understood neurometabolic in genes MPS-IIIA in CNS HSC to and correct to space, through our have gene the

this an example at mechanism of is work Here Slide in natural XX.

barrier, of as blood-brain distribute express that neurons. enzyme population that a Data is naturally taken the and gene-modified microglia the HSCs shows there cross up engraft by throughout can brain, that are

seen we for approach clinical and for benefits and the using are results MLD, this MPS-IIIA. patients have MPS-I same also in with We approach

we the that this, high approach Beyond unmet HSC be other which see genes used and for conditions deliver need. proteins to neurodegenerative have could larger specific therapy gene

underlying program the therapy neurodegenerative University Dr. involves the further Italy, specific HSC parallels a with This and conditions and addressing. conditions. today, of broad the the potential already disclosing Hospital we Boston subset genetic of strategic on are Alessandra pathogenesis conditions gene to and shown Children’s of neurometabolic neurometabolic a vivo dementia, alliance One of is XX, explore that ex number frontotemporal in Slide Biffi, in with has are a we where Padua

abnormal systems, are the required organ XX. the certain in X-linked at Slide XCGD, from invasion gut or bacterial such as respond mechanisms In tract, are response chronic similar as granulomatous gut function other to prevent such which colitis. illustrated are resident macrophage GI there on to infection. an and work disease results in the severe immune lumen macrophages disorders, and wall defects In Tissue in

Slide to on XX. Moving

We mutations XCGD have through Crohn’s the gut disease also in reconstitution to can seen of the immune of and to that affect Orchard be therapy observations Crohn’s the program are subsets the This response. and gene-modified the this genetic clinical response our infection, of associated is in macrophages ongoing Certain disease. laboratories. migration already gene that into that same to genes XCGD our preclinical for presumed applicable colitis of so with of modification cells lead suggest HSCs research HSC of may our approach subset of work resolution in

are the begun more in we and show our exciting gene work we and and proof-of-concept, FTD programs As these and therapy diseases to become either we in have in us just explore advance this Crohn’s populations, truly partnership. of disease, as and share believe preclinical others, year. larger or for about patient potential these such to for development the excited alone will assuming expand We address opportunities later these HSC preclinical to

path our our in OTL-XXX wave additional XX to Slide important FTD research neurometabolic further MLD, for therapies. MPS-I, company next readouts progress and anticipate stage many evolution and in our XX Wiskott-Aldrich clinical syndrome milestones disease. in of of continue detail offers our MLD from months launch commercial filings new as data forward summarize programs registrational advance to on stage regulatory and our on next we clinical year a So the Crohn’s approval next to and and in a EU, XX, franchise the We multiple study and

larger of and gene today hematopoietic the from serve our growth, on achieve new path To number we that an could who set will operational even remarks, sustainable long-term strategic wrap therapy. our announced benefit to patients value right stem confident decisions and cell build up plan prepared are us

we’ll to very you time use questions. answer much. Thank your rest And now the of the

line. So have up let’s the the operator open


Thank you. question comes Guggenheim. from Whitney from [Operator Instructions] our And Ijem first

Your Please line now open. ahead. is go

Whitney Ijem

Thanks question. guys. Hi, for the

get Or you’re maybe I of just they wondering, more kind looking on for? color guess what IND the having first, need FDA you work discussions some additional the tool – do? So think you you’ll is of plan sort in are of with to MLD? the is RMAT? you us clinical a give there just Kind And to can

Bobby Gaspar

In here. details, and CMC release of the in discussions we endpoints, obviously, Thanks fact the commented general, of the go in talked they’ve package, that FDA. cetera. the that. into the Bobby history, et for can’t think the Whitney, But we’ve all natural the certain script, with the about I on

the already to have extensive have and like think is this – ultra-rare a very a we set, population, I’d and I disease, with filed we just data Now EMA. obviously, say very a complex

Now for so FDA. had that U.S., we IND data historical reasons, discuss opportunity the and haven’t with to the an the full been there hasn’t in in

say are a that full can looking is we the explain I able able to that want FDA for data be We we What to be and be we comprehensive be We to talk that that fully. set. body that able endpoints to asking order to have they But have data to an in feel of they them are with data. extensive the to – that confident explain do to need do to have and conversation for. have dialogue that the

filing, then dialogue. we we the So so submission can that BLA. we we’re ahead the filing with those an And that’s filing have why issues, RMAT, can can once of IND go clarify

Whitney Ijem

are one in Europe Okay. Can screening, in us you with follow-up then newborn Got remind both it. you guess, on just then And MLD. and quick I where U.S.? the

Bobby Gaspar

sure. Yes,

patients more be access important is I be screening a and important, earlier to issue, we’ll that an for able because, means that able obviously, have think, therapy. very So newborn diagnosis MLD, get to

this of our a disease. it’s very of part diagnostic kind in important initiatives So

scientist, also have a assay and the funding activity that as well. other enzyme dry in we will the increase to assay is an now decrease been are that’s has pilot that going on State, into in blood also start and and far published show assay to York is in a understand in a that year. worked been have states pilots we’ve the to be an key sulfur-type pilots, we later that developed, we’re is there looking so with at levels. we put And where done that What the this And spot New

a we’re funding in well. and in be also out. and and states opportunities that in for looking also Italy where And that other transferring We’re EU rolled to that assay Tuscany we’re – will program Italy as

So I’d say, to going start, to pilots already are are fund other there well. are that two we as looking

And screening, data states newborn also together, in onto that the for wider U.S. validate will us in but and the nomination, allow panel allow to also for for the of implementation implementation assay example, WAS

there’s I going sure order to that lot say on of So a work in make happens.

Whitney Ijem

Great. Thanks.


Thank your Oppenheimer. Rajavelu Esther And comes you. from question next from

ahead. go Your is open. Please line now

Esther Rajavelu

Hey, EU know that to on or drawn to was a it my scale, screening. all trying MLD math guys. newborn looks first the four-year the understand duration time I’m lag on but changes. the Congrats approval like help from approval almost guess, if that line first there? is NBS. if graph question and it’s us I don’t understand to Can you I again between

Frank Thomas

screening or between EU mean both? You newborn and around U.S. or

Esther Rajavelu

and between Around newborn screening. EU approval screening, newborn generally,

Frank Thomas

sure. Yes,

of be patients then Ross the in requirements to have of will As children I can terms for will patients to the order active come in positive a think time apply Bobby Panel. and certain be on a number Panel, program newborn or there of you identifying mentioned, even there’s are number the to apply expect around we need Ross the screening that planned pretty over screened, met that that

the process get on And basis that a least, from added. to then state-by-state a there’s go in at it through you U.S., there,

number steps So along I way. are a think there of the

largest we’ll on We out sort have obviously, Ross – specifically we populations. the of but years. time other that initially that the haven’t there this reimbursement, apply the but focus full once to for are take line, could get Panel guided screen there suggest after the on I think approval precedents that states we that Once

Esther Rajavelu

my Yes, ahead. go And –

Bobby Gaspar

for I EU, for later approval the Esther for year. MLD looking we’re to was – obviously, just say for this going

are But pipeline example, EU for countries also screening As people screening sometimes SCID, the in worked it’s far as on in a now on for in a And concerned, number newborn even certain screening in pilots SCID the basis, that’s is the country-by-country well. I’ve EU states. as with the there numerous of EU. and are for that

of and really And so that has as EU and in already we Frank would and mentioned. U.S., in facilitate experience, kind be that uptake in with kind of – as looking the the to

Esther Rajavelu

of to some then screening? the given approvals and is some cost Understood. the of kind related EU defer CapEx, that you decision U.S. newborn lines already the really to for into And what that Or that time have into versus went delay, facility?

Frank Thomas

and that start, can on can I add Bobby again. Yes.

with the capability I relative important sort have programs have. we believe some really It the that Working for over for to to want think, continue launches manufacturing we’re and for we a the to to have of launch. down today, in-house various that and and when capability that know have we comes beyond MLD an capacity capacity is the WAS obviously, period time. going is of that to period we need CDMOs we the

secure to We’ll continue that CDMOs capacity it deferring to makes there’s think the those launches. on imminent need work an and we today, most So sense. with not the

We look market. product more bringing be at able on a supplier for to drug U.S. the to U.S. will service easily

manufacturing And term, is site a at we to and that capacity look will longer fitted in-house the potentially, be. location what think at, needs then more

out roll So demand that the appropriate we it’s and make because grow time was needs think, WAS be will any say will as our to launch and But sort for certainly, the MLD. lines capacity always plan those wouldn’t to the tied and I investment. utilize CDMOs grows, of to time, launches

Esther Rajavelu

much. very you Thank Understood.


Thank from from Rama your you. question comes JPMorgan. next And Anupam

Unidentified Analyst

Tess is guys. This this morning, Anupam. Good the on call for morning

week. maybe expected programs size what be of here see from and us the any next of trial And scope for the should data can at data you we that clarify OTL-XXX and ASGCT out proof-of-concept at at Can clinical pointed you updated MPS-I we will interim data is the updated remind that other You conference? if other upcoming ASGCT? about thinking newly there will is just be for

Bobby Gaspar

decrease here, this that’s patients, has enrolled question. fully X activity, study the therapy. frame so into. so data of and heparan I’ll biochemical you, we’ve Bobby some is fine. the There MPS-I, in was clinical of and patients the shared time and take on all data with previously the who gene-modified Okay, IDUA early been that to proof-of-concept only just recruited cells sulfate, engraftment X the gene On remind dermatan beyond What patient time who have had showing overexpression X-year previously got reached the after you point.

So longer enzymatic further as of on patients. to activity, consistent data the longer-term who got longer on GAG more X those engraftment clinical cells, to well. follow-up follow-up, decrease be gene-modified able endpoints patients We’ll show and you in of have also overexpression there’s been more levels

on, show follow-up. to as clinical this assay outcome, issue able be we on again patients example, but will their big both be longer cognitive growth data we’ll have will on So in And which after MPS-I. parameters data a well, for data is also on

for is that MPS-I. So

longer data transfusion-dependent treatment OTL-XXX who patients as follow-up ADA-SCID. will on for well who on so on have so beta-thalassemia, also treated been be We for the undergone have There further sharing be will follow-up far. patients

there’s So other programs. as quite, really as well

how of as underlying neurometabolic and week. it on the gene of different more deficiencies abstracts immune deficiencies, showcases diseases of HSC a extensive well. detail and opportunities those body in defects therapy data, number And across can an platform quite different we’ll just So correct Orchard’s you obviously, give next hemoglobin and the there’s really potential

Unidentified Analyst

Thank you. Great.


from Cowen. comes Thank you. next And question from Werber your Yaron

open. is now Please Your ahead. go line

Yaron Werber

Thanks this for an I easy Great. And decision. – we’re sure the taking questions. was not

it move is MLD CMC. I was I albeit of FDA couple two accomplish endpoints, sort the think sort mean, on there interactions, easy then elements. – of to the to direction, like One definitely I first not relating can. Bobby, internally. this data if it’s I a right definitely of have What questions sounds in and clinical is a

So has that you’ll need Or endpoints, a to do And want of to on you have a different the you well. another the it as clinical conduct is on it is they quick philosophy FDA I CMC think and study? that clarification? side what just see? And to then kind data follow-up as need the

Bobby Gaspar

Yaron. Yes. the go mean, I the in whether get to set filing endpoints we kind set all is some data extensive show now but follow-up of X data ago we dialogue, have as to at. to ability that difficult I’d even really I us at a FDA into those And as say, of to of to from have other general, have Thanks, those might what appropriate look endpoints the full program an time certain and patients, asked details. sure. The about have we again, to we our data it’s started that – Yes, with talk to guided have, show we the plans. years-plus this you other that the quite we look want endpoints to, on that get and are is And have individuals. say think can know, about the And of clarification in front as and what we XX-plus them, want be able do, do and them to endpoints.

us IND interaction. and RMAT allow to thing and that greater I the for designation to submitting us most get to have say, would I’d have think that trying the important is So dialogue

Yaron Werber

vector request than any are drug seen what essentially maybe looking to characterize and as what for have on that requested. Okay. sense and different side, FDA potency We vastly But – expression? well. recently is CMC And the product assays new validation they the cell then Bluebird with the and the the EMA

Bobby Gaspar

not Yes. all. No, about that at it’s

that’s looking one have MLD we assay. and activity, that CMC general So is the the an And the say I for focused potency assays. think that’s on are issues can at enzymatic assay potency thing than we more ARSA rather

Yaron Werber


Or is the will as MLD to you would about to an So that have you the before chance of impact WAS very WAS? side not specific very you central? much of file next care take on This think year? sort and essentially a is this have as BLA

Bobby Gaspar

about So talking are or we Wiskott-Aldrich MLD syndrome?

Yaron Werber

was Yes. CMC on for relating CMC to MLD issues the CMC year cross-correlation to the the about WAS a – and the talking from then perspective. BLA I your file next to ability

Bobby Gaspar


more CMC are from of for MLD kind and we that. about say, for a the talking of, comparison between cryopreserved the formulations to, that So perspective, have and issues that they’re body fresh around data example, I’d related

I’m but I’m assay. follow-on – syndrome. are not not we what Wiskott-Aldrich for specifically say so – I’d And doing not kind related And the to of are potency with

Yaron Werber

so Great. Thanks Okay. much.


Barclays. comes from Your Gena question next from Wang

Your line go now open. ahead. is Please

Peter Kim

Hi. This apologize for for I about this. I maybe on bookends taking But already guess, us have is Wang. our you’re Gena question. you different the you I talked guess could Kim about and question if give Peter of look terms another be scenarios with MLD, thinking And like case FDA? much. the like Thanks what like, like? good would very you with of What best what dialogues of scenario a time in case and be line, time so Thank the line interest would be not would scenario? in how

Bobby Gaspar

FDA. getting us the for give information without it’s Yes, the very to difficult from think that really I – of kind it’s clarification

a just us to about we As We data. on they’ve We – this asked really dialogue – – natural have in us I’d a them about we They’ve at be can of that. present the in then endpoints, have, we of other feel to and that questions, guided data have in we said before them best way in looking to that need for, really to able endpoints, have certain forward. But entirety information. body we the meet certain say, its about I need obviously, them. – terms we think talk dialogue the we last significant talk think to the to and history with talked detail, to

could think really that, those So do lines we’ve able kind those don’t you we accurately. until more been once can guide conversations. And I we time then had we’ve to guide to of obviously,

Frank Thomas

indication study context, that we I think was this can mean, a of but is a started data I mean, put XX things. ago, so add a significant XX just almost clinical years. maybe just in Well, years it this in I the to over period was have I few obvious,

strength to the to for an go have IND. without file data, clinical the with intended, directly We of need the a

it’s I to some that recently think them going to with to that familiarize based need to written the the clear data. on us spend FDA we’re we time the got, feedback with

So a per se I’m data as and them. not sure to we time need I much the that I spent much having they’ve questions, actually think, dialogue have as, with think with as

is hope, provide the the of dialogue with closely now front to in and to purpose the the seeking IND process. a sure package And that. our RMAT have I with in many of but that parallels earlier, as feel requirements. a course, to the of meet helped we’re with ability and and similar EMA. right with there a the discussion. get think, FDA said make data and do Bobby And FDA, I now, we be place good to that have we’re designation, to that them, work want like the the And existing that So we think I in that had think on we data we’ll hope questions strength had the the able with with with Whereas we to we need dialogue path EMA, and the agency more answer EMA we we of clinical are

Bobby Gaspar


EMA. Just involved was to because discussions follow-up on that in I those with

were of number raised about the they the questions, – we the EMA one, a So package. came day there points clinical XX had and

to And function. motor and the some where, entire put forward that effect on function a we data to again, have we and able are the having the presenting them set, with And magnitude clarification cognitive of meeting were – us.

got a gives to answer think we questions very a and good I place us to effectively. path forward those

So I disease do I where as complex we of a body think dialogue large that important have data. very say, in such is and a

Peter Kim

Thank you.


remarks. and the that I call Thank Q&A session. you, for Bobby closing back now concludes Gaspar would today’s to like to turn

Bobby Gaspar

follow-up. our abstracts of very where next a that, Thank provide a everyone eight call. I accepted will the at to you for Thank have number I much. a light available that as reminder week’s also be in Orchard programs. will significant ASGCT wanted you. We’ll presence earlier day virtual for said today’s to on shine meeting, on, Thank you participating any throughout

So stay you much, very Thank tuned. everybody.


Ladies That and today’s you concludes today’s conference. gentlemen, call. for thank

disconnect. now may You