ORTX Orchard Therapeutics

Renee Leck Director of Investor Relations
Mark Rothera President and Chief Executive Officer
Frank Thomas Chief Financial Officer
Bobby Gaspar Chief Scientific Officer
Anvita Gupta Guggenheim
Gena Wang Barclays
Tessa Romero JPMorgan
Esther Rajavelu Oppenheimer
David Nierengarten Wedbush
Yaron Werber Cowen
Graig Suvannavejh Goldman Sachs
Call transcript
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Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics Third Quarter 2019 Investor Update Conference Call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that today’s conference is being recorded. [Operator Instructions]

to Leck, conference Director like speaker to now Renee today, of would hand over the your I Investor Relations. you. Thank ahead, go madam. Please

Renee Leck

Thanks, and XXXX operator. everyone, Orchard’s Good Quarter Third welcome Investor to Update. afternoon,

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

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

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

turn the our that, President With Rothera. Mark call I’ll CEO, and to over

Mark Rothera

Good Renee. Orchard’s annual hope the you’re of release morning, welcome. afternoon, abstracts I the this the with portfolio. therapy included ASH several excitement gene which all from enjoying and Thanks,

today’s purpose is The twofold. call for

have to progress priorities registrational highlights. programs like up we’d data on accepted our from set ADA-SCID ASH the our been releases some And at while the recent in you Wiskott-Aldrich we’ll our Firstly, date secondly, executing to reviewing in bring that Syndrome corporate against business December. stage and for

close prepare financial commercial reviewing a second Orchard’s quarter filings, beyond. our results XXXX product strategy will my remarks. Thomas, launches opening approvals cover and third multiple CSO, CFO, return in outlook. of and as Our I’ll discussion after Bobby time some Then will topic we potential to and in detail for the with more Gaspar, spend Frank the

are step and the take Before remind like we I the tremendous progress newer story back to who this are to at year. datasets a coming ASH, you the to who we’ve highlight made of to up I’d recap those

approach Orchard is therapies mission a from pure-play deliver to gene to focused and the life-threatening curative children, rare around therapy transformative with often suffering patients, and singular a genetic diseases world. company potentially

lies expertise proof or gene stem different concept platform diseases in approach HSC demonstrated a therapies, autologous vivo has cell developing hematopoietic that Our of so portfolio in ex in clinical five far. our

to As temporary label see robust follow-up the believe eight a and XXX of "patient" across including a we for be potential over clinical we of the portfolio treated the datasets With one company, field our opportunity showing to gene lifelong view the years of going we out therapy. or response our for have patients as term more, most the therapies benefit, curative. durability and and comprehensive in

for Now standout turning has we’ve been a made. to year XXXX Orchard. progress the

program; lead our in on program. focus first OTL-XXX the Let’s the metachromatic three OTL-XXX WAS, leukodystrophy, ADA-SCID, program; and programs: OTL-XXX the

lead for gene-corrected clinical three we fresh all formulation. abstract with generated or of registrational published, have measures presented an efficacy safety programs, a cell These include the data stem For and complete datasets. had accepted

while to from and beneficial two from cryopreservation for that to our regulatory believe filings. patients upcoming versus globe evaluate cells our performance be will their more cryo them of facilities. provide and a MLD the travel a studies part of cryopreserved manufacturing have much the nearer and work results fresh We by cells bridging conducted ADA-SCID, treated formulation presented as we convenient enabling around experience homes the for were For also to their the

about this and grown worldwide. we delivering dedicated in delivering therapies gene our more to organization way on our Europe two the its capabilities Accordingly, build on regulatory patients as with mission well later. for and U.S. upcoming to towards MLD. patients are has filings HSC ADA-SCID We for Orchard commercial I’ll say an

are franchise neurometabolic these for patients therapeutic by wealth HSC approach. programs. where has particular is of This a Most effective for we MLD. potential our have Not not in news a excited The our a this as options, this area creating flow disorders momentum additional do neurometabolic conditions advancing. OTL-XXX progress, the especially of also program promising had of advanced case the year, earlier-stage treatment most is only tremendous is made these franchise application assets pipeline in with but has

first seen begin In encouraging enrolling for new ongoing the announced the proof-of-concept at clinical SR-Tiget syndrome program This we in pipeline. SSIEM in of from partners the Sanfilippo will and year-end. MPS-I May, for licensed Orchard the by our in Type Milan clinical acceptance our study preliminary morning, Furthermore, an gene exciting presented application we in we seventh therapy A, data September. this the HSC program and clinical-stage patients study or MPS-IIIA in in to trial expect be have

trial effect. Manchester The enzyme Royal Hospital, under of clinical well the study MPS-IIIA being by is do taking biomarker earlier indicating at a to continues The year. treatment a with six outside this the Children’s conducted patient a data which special license favorable is and treated patient months,

The turning of and for the milestones clinical abstracts programs. in Now corporate and ADA-SCID news. morning’s important ASH data this two our to WAS achievement marked the

using for ADA-SCID, cell formulation For OTL-XXX. are the data the first from cryopreserved we gene-modified presenting XX time of treated the patients stem

that datasets, in weeks and marrow few are ago formulations cell will MLD like performing fresh a in Bobby formulations. was the now at that have ADA-SCID we ESGCT supportive bone two demonstrate one the and presented engrafting that review, cryopreserved in one As

per time this life median of around condition patient with disease three-year X,XXX there data registrational that in point typically WAS, at of morning’s is years with treatment. trial around eight-patient pool world patients as For abstract has the living today a XX and the The so orphan WAS substantial. primary expectancy in met protocol. prevalent the with X,XXX that its quite patients supportive to approximately the this are is And countries drugs, the reimburse confirm endpoints the the follow-up secondary

for the to regulatory filings expect this Europe submit in We U.S. XXXX. and in indication

and each SR-Tiget, morning’s we achieved milestones Raffaele, collaborators, others has of within the cross-functional among our Manchester this Ospedale like results achievements these UCL, of San have Orchard made University the to abstracts, Children’s tremendous that data and clinical the out the Boston Hospital, including the Orchard possible. congratulate laid UCLA, XXXX. I’d for efforts With from

Bobby hand me to into ASH the over it now more Let to dig abstracts Bobby? in detail.

Bobby Gaspar

ASH are on to ADA-SCID Thanks, therapy of pipeline compelling Mark. data all diverse potential clinical showcase we of I’m in hematopoietic abstracts. stem morning’s highlighted life-threatening of the where the this delighted breadth, WAS and the of longstanding pioneering I believe presentations Orchard’s review the these cell establish With The care. depth durability a patients, featuring dozens data clinical diseases, and in new in in in the gene to three of will have culmination data therapy. for gene work standard data

platelet by that start OTL-XXX life-threatening severe presents abstract with Syndrome abnormalities. a reviewing immune for is detail. Wiskott-Aldrich hematological I’ll patients condition the system WAS with in and greater

two severe the mortality So clinical of episodes infections. disease are highest bleeding of the major the and aspects severe and, consequently, cause

in is followed includes who results an dataset, dataset WAS with as for considered lentiviral that integrated for published patients an no including XX and making years expanded Patients gene be events of vector access therapy oncogenesis. of transduction. were from the to HSC long-term abstract This and durability treated the of using follow-up to testament program. years date nine There also patients were effect additional integrated reports analysis an ago, a the patient safety no adverse longest received and the a median related durability the from OTL-XXX three The study part patients, to of to possible eight first registrational both of this the the approach. this the insertional is complete OTL-XXX eight using of

to One of in died the the an were as expanded access that alive unrelated be OTL-XXX. was to result existing XX considered analysis. neurodegenerative of of patient XX, the XX% a the patients by condition of at time cohort investigator

successfully within the in of maintained that been eight expression WAS different months, cell number for vector to copy up three lineages years. patients in has in leading an protein increase All to and engrafted surviving

infection transfusions, immunoglobulin time there experiencing all to no function improved post months post bleeding patients over able receiving supplementation immune rates, of recorded. the summary, More in major of were poster a data severe two and episodes platelet also life-threatening and a all the ASH correction with be were aspects In immune treatment, at reconstitution. show of disease. severe December. these discontinuing Patients’ in suggestive treatment Nine in stop patients reduction effective presented complete all analysis will

ADA-SCID. to let’s Now the turn abstract for

the with twofold. the are the patients fresh cryopreserved formulation patients dataset XX patients, that We for this XX an treated formulation. data and from additional The registrational study from objectives supportive were from data presenting XX is

was to to show the similarly fresh. first performed cryo The formulation that the

measure engraftment; HSCs across XX XX and recovery, of patients. showed within six the granulocytes, in consistent number copy treatment, months Overall T-cell vector the a both of reconstitution, in treated in persisted relevant a and patients the of CDX of modified genetically approximately follow-up OTL-XXX a showed both through datasets. of engraftment performance measure which cryopreserved fresh period results of Analysis immune

cryopreserved allogeneic receiving or stem formulation a engraft to received cell and of hematopoietic failed the patients the HSCT. One rescue transplant subsequently

that have to it of We was this formulation received. believe OTL-XXX the looked into related no the have engraftment and reason failure patient to

are dataset across over between and This cryo We data consistently with results portfolio. the cryopreserved ESGCT XX partners the fresh treated the is formulations engraftment presented similar consistent patients have cryo at and now therapies MLD with month. and our last seeing gene

OTL-XXX, second XX second survival objective to OTL-XXX’s patients population, evaluate passed place HSCT overall XX XXX% or historical broader OTL-XXX was HSCT. HSCT ADA-SCID of remains profile Event-free XX% The ERT XX% returned versus or over survival XX% survival receiving of whereas patients in was historical a required for patients for HSCT. the patients’ versus clinical clinical overall to in receiving away. allogeneic control the rescue outcomes control compelling with

patients follow-up or with XX patients completeness or B the cryopreserved featured for a at be to had point seeing versus findings HSCT the immune analysis In at from of data XX% formulation, months was patients reconstitution OTL-XXX longest with illustrates on will with and, previously fresh ASH. stopped in disease. which formulation we receiving or comparable more five immunoglobulin therapy. function, poster graft this of is treated the XX%, the cell therapy. data available patients XX with addition, presented reaching seven are This In Again, has treated host months, patients eight chronic acute time receiving replacement that for the gene coupled

on completing program, We’ve abstract ASH. to first following presentation proof-of-concept and ongoing treated. into demonstrated as an Finally, stable enrolled metabolic since a a in of evidence preliminary was the patients, and accepted OTL-XXX treatment at evaluation SSIEM presented was study. at data the This skills A MPS-I study expected the motor trial improved XXXX. from is all where an engraftment signs patient patient oral one-year close of is patients as cognitive growth, seventh I’ll in in six the Data touch as resumed enrollment showed clinical score of well eight proof-of-concept and correction. successful

that wraps up section. Now my

forward looking at you Frank. to seeing all the many updated Frank? the turn are of to Orchard. We over keeping ASH you I’ll now on exciting call developments at and

Frank Thomas

let Thanks, me Bobby, welcome. and add my

You can third in afternoon’s find highlight for will I this which briefly quarter the results financial full here. release, the press

investments to We and up global under filings, credit cash need sheet including ended preparations will This strong third without the million our available $XX several an to approvals multiple key the quarter potential us and for the facility. markets, with allow launches. commercial execute to additional regulatory on capital million balance in $XXX access milestones and

any expect programs. that vouchers guidance, facility credit with runway for we second fund operating cash expenditures anticipated This investments available the our upon into under Consistent the does U.S. our of we capital existing priority include proceeds capital XXXX. the or half will our and and monetizing review our past receive may from not approval

the For expenses for XXXX, $XX R&D slightly the period approximately up third which the in were are same XXXX. from only quarter of R&D million, expenditures

R&D expect may future periods though expenses generally development, quarters. We advance in our be particular to increase there programs some through as in lumpiness

to due prepare to as million period third compared $X public as The our in investments of was for year. higher company for the quarter SG&A expenses programs the XXXX support late-stage approximately were same higher increase million of last the cost operations. commercialization to well to primarily $XX

revenue related of During the third Strimvelis. in sales quarter, $X.X we million to recognized

that minute, of is approved As ahead ex our learning gene product vivo more talk source And Strimvelis the organization. the our how the of a we important European in for specifically build next Agency, leveraging first therapy Medicines about of will commercial an Mark to launches. wave are by capabilities

licensing million million in We fund in of used which operations to our the May. quarter cash about about $XX cash $XX related of of MPS-I included the payments XXXX, third in to program

and multiple payment, the quarters own to build-out expect for next rate grow potential launches Excluding manufacturing this subsequent Fremont, the we in few prepare onetime years in facility quarterly run product our California. our in to of begin

our are our to manufacturing in addition in strategies platform In efficient in make also investment more to commercial setting. processes the and we investing facility, a multiple

focused are the patient. a investments transduction enhancers or cell could reducing those per of both amount new on line, such Some usage vector technologies a which of long-term benefit of have stable as

our investment own Our facility source supply, overall provide in capacity enhance manufacturing along partners. with additional will a manufacturing of our and

manage which several In addition rate us our always burn portfolio or to through looking on our investments capabilities depending environment operate. to macro in with licensing are enhance in This leaves focused research the manufacturing, we for to we and deals. P&L in opportunities the levers

pleased and hand a way. of with continue I’ll over back fundamentals call deploy the to XXXX close. we’re coming the to we’ll capital a rapidly in close, business, to Mark now With our to focused

Mark Rothera

global Preparing commercialize of launches the on Thanks, in and ADA-SCID gene and is therapies. Frank. our success to to a crucial future our MLD scale

journey, required their health individual and identification, care the for securing and scheduled understanding patients the said, for European commercial families cross-border Medicines patients treatment to and has delivery This the Frank support for in along Agency, therapy real-world us marketing number supply a release ex and approved and activities, setting. tailored process the EU vivo a treat of Strimvelis, manufacturing first the patient involves logistics patient. HSC and by As each completing within reimbursement including gene produce enabled

We treated be available our This multiple will these preparing at over and therapies approved, multiple at around ability scale centers deliver one when our the for is past the demonstrating therapies, quarter, to if made the center. patients us time world. investigational

France, America; global in years. operations and and information Turkey, to North affairs, plans increased and medical Germany; knowledge as end, this diagnostics, in global East key Italy a the Asia access next now therapies. to such functions commercial Europe, in our with Middle Latin To over America, few U.K., growing to establish including have presence hired footprint access, facilitate advocacy leaders and medical established we in We’ve and our the the patient market of medical and Orchard

have place expect program. This qualified we putting patient Finally, a well-controlled begun we in supply chain. launch centers the first a countries are includes establishing in to treatment support high-touch where and

potential diseases are is effective care driving gene our of This they diagnostic pathways in patient treatments impetus place. are the Some communities therapies diseases these targeting. represent with first new for the and health in ensure professionals for to

is For newborn and the pilots example, multiple with Europe. stakeholders to Orchard expedite U.S. working in screening both in MLD

multiple commitment across the At company bring Orchard and like our always gene this we potential medicines as we different With horizon, to core, patient a therapy the the year. putting by our these entire on world. approvals maintain company look could time to next innovative be novel will one-of-a-kind to first the

questions. may now Operator, attention. and time open your you the for you Thank line for


from sir. Please Our comes first ahead. Instructions] Ijem go Thank from question Whitney Guggenheim. you, [Operator

Anvita Gupta

is Whitney. This on guys. Anvita Hi, for

actually. questions two So

the U.S. cryo just And any hand, the MLD the what left there other then now can steps to for other in data on are us is the filing please and do data us With filing? you for remind – rate-limiting than getting application? the EU remind to in

Mark Rothera

for Yes. that Thank much very question. you

MLD got you integrated the data the as earlier a think come cryo great compelling saw through well from that’s recent in data. the with file. that year place I we’re We’ve as this obviously analysis

are and posted. an and us. we’re we’ll speed And condition, to that both we quality. diligently expedite waiting so the does day that, do our clearly patients is such appreciate really, to is now And count. obviously, every a keep and This the devastating for in you And job submission, important working balancing program

Anvita Gupta

Thank you.


you. Thank

go Our ahead. comes from Gena question next Wang Please Barclays. from

Gena Wang

Thank congrats on you update. for my taking the and questions, data

two So I have questions.

is in first regarding one The XXX the ADA-SCID.

not engraft, that and mention So did data related the cryo product. did drug one not to was you patient from

Just VCN? did reason was And the the the engraftment – product wondering, not what what happen? patient the was drug for

Mark Rothera

Maybe, to Yes. that like answer Bobby, you one? would

Bobby Gaspar


for So that thank question. you

of of set context this. kind the Just to

– drug in and package study is between is this accompany vitro comparability fresh and of cryo the So product. cryopreserved supportive will a includes OTL-XXX that data the formulations here

So to is it supportive that.

failures, of to even be patient patient-specific Having drug do engraftment, engraftment we there context reasons be can’t for fresh in allogeneic we said and failure the transplantation. for characteristics, at find see we will this have formulations and process it regards detail and individual. related this looked in that, in the to may in factors. product, example, With the And particular patient,

And treated we’ve a a XX formulation. of patients, for just a just small failures, OTL-XXX. with three – those now in patients very was cell failures we’ve those over again, patient fresh number, and And just XX receiving had across just one context,

these And So we at or happening reasons don’t say, minority in a formulations. and of occur can know fresh either very non-engraftments only patients. in cryopreserved as are, I small what it’s in the those moment,

Gena Wang

is regarding Great. the Okay. next my WAS. question XXX in And

cohort, you to disease. So there in neurodegenerative neuro was one and it was – death EAP due degeneration said

So formulation can WAS give related just for on wondering little a And another bit When that question you more if happen? color. trial. cryo the is did

give the enrollment. can Just of wondering if status the you us

Mark Rothera

Bobby, like handle would to you those?

Bobby Gaspar

sure. Yes,

regard he therapy post he died to patient was procedure in died gene treated And study underlying early an condition, was into unfortunately, was procedure. which, access who – who succumbed death came the an neurodegenerative that with with sorry, therapy and the patient. unfortunately, the X.X patient program an just gene in So expanded it after months to. fact, And the

So patient. regard that to that’s with

and yes. – the patients The cryo started – your second of study part has cryo sorry, was enroll started has to And the has study successfully. question

Gena Wang

if wondering additional give color. can Okay. you I’m just

first additional give enrollment can just enrolled? patients half guidance wondering And you the many is if color. already that think a XXXX. I Like how in complete

Mark Rothera

had... we’ve So

Bobby Gaspar

I mean patients...

Mark Rothera

ahead. Go

Bobby Gaspar


of So and that’s that we’ve so had ongoing. pleased far, with we’re the and four patients progress treated the recruitment been have that

Mark Rothera

happen just that we’ve maybe they’re think about put functioning ADA-SCID you data to MPS-I with had that initiated way. because the if already just in It’s there’s using data, that and think showing cryo is And for just in And same don’t we working Yes. both are and to MLD three programs, initiated first or on cryo of different. program, anything that’s transfusion-dependent cryos way recall cryo the the context, which that these be proving that, all beta-thalassemia both a the fresh. top now we’re just fresh, in the similar that these to they’re

a cryo body of I evidence there’s So that in treated just with, think, using so portfolio have now large our far. XX around patients been

Gena Wang

Great. helpful. you. That’s Thank very


you. Thank

Our from Anupam question next comes ahead. Rama go from Please JPMorgan.

Tessa Romero

on SCID, Are to submit? lines complete for gating factors remind be And a the for, specifically Hi. tonight us congratulations month, new call question looking XXX in first think of on taking tracking Number rolling presentations program, we us was BLA morning. I we and on to still if XXX but Thank one, the time the related the WAS presentations half progress. of before as this next you question out you datasets? for thanks and us, in should there are year? summaries for analyses for the any each all XXX that you think But may. the of complete can This there you the are these any And ASH the there towards the specific this, within key submission. about I the two abstracts then is Tessa from from Perhaps on of Anupam. for next

Mark Rothera

then your ask I’ll take part. Bobby part the address first So of second the to question, I’ll

Bobby Gaspar


studies. there as far What on concerned. detail just these see ASH the is two data as more is So at you’ll

patients. Wiskott patients, studies you data further functional enrollment, But further detail study. other won’t will drug So follow-up have in but ADA remember, terms patient number copy any you assays so there on these see and see characteristics, the more be won’t the for the vector and the of both on finished the any product,

Mark Rothera

the regard to your key the second second which regard – of to And with steps we’re With was presenting cryo yes. one at work, WAS. your question,

ASH. at – So

process that the think using the as rolling as which an per the material, said, working next patient have runs first year. we step are another in product the of validation whole of preparation. drug BLA the requirement BLA, half important to, one, I FDA before So we the that’s other on that’s mentioned before, which we initiate FDA we’ve But from guiding we’re and already for guidance,

Tessa Romero

questions. Okay, great. taking you Thank for our


Thank you.

Oppenheimer. from next Our ahead. question Rajavelu comes Please from Esther go

Esther Rajavelu

just have in quarter ones changes many you there thank this And taking or couple been I to versus the treat you How did specific a for question. quarter? patients any Hi, on quick this of earlier year? Strimvelis. my Strimvelis pricing reimbursement had

Mark Rothera

I you the question. Perhaps Frank for Thank one. take can to ask that

Frank Thomas


a quarter launch, there’s of number respect treated the has there. second the the of no to Strimvelis price since think question, And I and changes this of was patients with remained its in same your total any three. for plans

Esther Rajavelu

help markets Got Okay. in looking the you a of and us preparation. bit a some little for to the mentioned the – position you’re may XXXX? over some be several two quarters you in maybe that then completing next – the around Can you And understand in future. into and timing spend as filings the launch details you. Can commercial taking

Mark Rothera

of a prepare as doing. we so I’m that – for number commercialization, are there things Yes. we’re

footprint. establishing commercial The the first is

So geographically, in here U.S. we’re the

key in just I years. we mentioned, in and this Europe markets, our the Germany, are launch to also next like not year progressively, couple the advance will U.K., in of of as continue Italy, footprint over but France, We expand

that undertaking before, said patients launch. we’re have prepare footprint a diseases these commercial for need of world. all comes because to a the footprint over are treatments number With I As rare to our these is and activities our global goal

dealing aspect as think, well for already MLD, screening I rare as with we’re of as newborn for the is, some which work important importance that types pilots disease of such call generally, to doing, in earlier, And expanding the the identification exists another key. expedite patient alluded also diseases. work I of screening is of I ADA-SCID awareness think, helping So the newborn

is to of treatment are the on other area focusing centers we’re The for centers that we would become excellence there like of patients.

transplants identified we’ve good centers that good we are that launching so specifically also And disease but the at into. at areas are

know we centers of to those working launch. which in qualify and we’re So advance ones those are,

is And access. there’s think range then be stakeholders great out, area, work I of because the have call in single the And I here, a of extraordinary done market think to intervention. about final are in advance medicines whole deal just ability one of a to important launch that to we talking with transform a life

And clearly so and the we’ll we’re to of system our really and proposition value represents patients of that that care society, and each be looking these articulate at the programs what very health to before able to launch. to and

So looking according flexible in to paid needs. models payment for payers’ these at of different how terms can be we’re also

different think we’re lot areas prepare launch our I a for working programs. we’re I So there’s that successful in, of we a think and very confident can

Esther Rajavelu

trend of over several course of this sort next the quarters? would spend the how And

Frank Thomas


$XXX ended think I with of runway million, gives XXXX. second into the which us we the ended half quarter – the

I going growth course, to think there’s related in manufacturing to build-out. of expenditures in and, SG&A capital R&D, be the

So potential launches. all into upwards X, will and filings think, trend the leading I generally

I SG&A will launches. think as answer trend to closer your upward So we to question specifically, get

Esther Rajavelu

you very Thank much.


Thank you.

go question ahead. Our Wedbush. Nierengarten Please comes from next from David

David Nierengarten

the long-term the of how relate look some would payers we and with follow-up minds? on launches, pricing be as to five-year seen years? study, studies that you think talked is getting that you your a this other typically and or Hi, with you’ve you with installment-based that is had talked follow-ups just payers’ durability potential longer question Or have your commercialization we’ve set and But instituted for folks on Have plans. the do for so obviously, your other model that in to five planned in upside? at

Mark Rothera

cell argue Well, do underlining lower the can out durability you, compared fact firstly, very modalities. stem of is And important and to just such we have the therapy maybe the vivo hematopoietic that that data. with duration around David, therefore, effect ex long for the uncertainty thank other gene pointing you

approaching I So is way it real gene we’re benefit of think a therapy. the that

a one-size-fits-all just burdensome be to that isn’t place one requirement a payers, to about put some sort But as size there your feel patients don’t they feel that of think I for that It unnecessary they I data period. well think of fits over that good a question. is may in all. an it is extended I very said, follow-up answer

of depend. going it’s it think to will that with. question types And are we’re then variety we’re it us really And different for operate the of on. are I point I align that’s models. different key that flexible many the how to important value paid thing to for. think There a I a It’s and payers we of deal the do is best think get willing

David Nierengarten

All you. right. Thank


Thank you.

Werber Our comes next question from go Yaron from Cowen. Please ahead.

Yaron Werber

Great. Thanks for taking my question.

us file visibility think is need now a on we’ve bit to noticed a your the to what ADA-SCID. do the first the coming give I – then market But Strimvelis we sense good patients one, filing in the the to you Europe. secondly, So maybe things one U.S. of into to new And I on have certainly, is on just a questions maybe couple therapy a and program? then And with in do EMA able of for maybe is lumpy. be have

So I small And centers? so have question patient’s much so the it’s different how in to onboard? bioavailability numbers visibility are because do you coming is when guess the selective

Mark Rothera

Thank for Yes. that you question.

around world work, where really there therapy I’ve comes countries prioritizing I on initially for it is the previously, think when ADA-SCID. we’re – regulatory to as focused gene that our no mentioned

a geographies effort parts as and our regulatory medicine of has parallel MLD for priority. WAS the think focusing a But bring that, So world to clinical so priority been on obviously, it’s important a have the to and we of in other United then on. And other amount and where also gene we’re patients therapy huge States. we ADA-SCID in first behind

do I we priority in bring top visibility, And list our well, will to as this not the OTL-XXX to that? European want Frank, at think, time, speak market time. you the it’s but to So at of to look to the forward

Frank Thomas


a ongoing that available. in that Just been the during of it Strimvelis been candidly, some have Milan, are Strimvelis – been has lumpy. only is there’s ex mean I available some has commercially of OTL-XXX reimbursement that And you’re for one right patients, bit vivo for therapy remember, It there the But limitations and terms studies because gene represent with sometimes in Strimvelis to challenges. to ADA-SCID. you center some does go time

option And the it put to great think support behind we we and so for a continuing is are patients product, need. in

think, we’ve to commercially I and seen securing importantly, the it this those So deliver that products learnings product. get for results ability recently, past about and I that successfully. and releasing think our about And manufacturing identifying that patients we it multiple times patients, then to speak did quarter because from reimbursement about that is the is we the

we well in those here serve us to learnings think I So OTL-XXX that as into we going from the get and MLD move launches Strimvelis for are U.S.

Mark Rothera

I’m can we hoping. online That – guys. still we’re


Yes, are we sir.

Suvannavejh Sachs. go comes question from next ahead. Graig from Goldman Our Please

Graig Suvannavejh

on that. – looking first perhaps Chief of you new any so activities. Good just hope just Officer. on think is evening. four business I’ve additional appetite questions look in quick. and about taken curious tick as I if XXX know talk the can recently with you the Mark, could programs My either to was there. them is you’ve good some I And I don’t question somewhat perhaps do to you just has to partnering I the got to do that partner programs. wondering in-licensed update on of quarter. Thank what the has hiring just Congrats there so what a with afternoon, and to you. I Commercialization is bring what My Great. program, to you’re actually. responsibilities, was or know part do, second happening development off

The follow-up to do programs. the that your to capacity has your of with handle all – to company’s

You’ve well. programs, preclinical got clinical got of you’ve a ones lot as some and

preparations. cell the maybe So with scalability the cryopreserved just your handle then looks was you wondering if the kind can of comparability all formulation lastly, compelling talk quite around I of data about the could in terms you fresh And of this. of how

I’m cryopreserved just scenes data us the could convince in if And necessary right that perhaps wondering generate the terms between to to what’s if bioequivalence, color of preparation. term, the fresh behind so some the FDA that’s and give like happening, the you cell on

Mark Rothera

questions. Great

to do all So to I’m remember best going four. my

officer, first and even lot So actually my me rather nice drugs. commercial of seven the moment commercial having get side a the of clearly, commercial orphan a about having the in the launched it’s spent to business, the chief personally, for to career closer side

the as well regard our delighted closely here European really team advance as therapy a launches. with team can But role to by gene that with launches. you search, the in imagine, I’m in companies of three probably and years aren’t working many place. have I’m the getting in X.X filings these lot because there as U.S. the we’re So the with of in team interest next in we

So the search is well. going

pond. on sides in both some to great on the of is maintain launch But the preparations that meantime, candidates. my focus have We momentum we sure make

BD to to address you want those regard and With points? Frank, perhaps, capacity, do

Frank Thomas

sure. Yes,

development, a now have that today. these So franchises lot we’ve three the bringing of into last of on in we couple success years programs over business had

So we at embarrassment have an level. of some riches

seven We soon-to-be have now the programs clinic. in

be the the make there we’ve the especially milestones of all past to opportunities. lead of based opportunistic we future about so this we are respect in-licensing any today, to sure excited out focused on leverage that achievement programs. continue those the our where can we on But on executing our lot diseases near are with year. programs but to franchises remain and our term, in the some delivering, there and bar that on will get We’ve we’re about plate, that. And higher been if a got continues well And

the to obviously, the we’ve point respect to built organization manage the programs capacity, about they’re we have, various at development. various stages of With that and

on So the development, depending imagine, in as development. stage to can resources pulled programs get through different move you of the

advancing team the program, We have program. assigned and program, great have we program they’re a to job a each doing for lead each a

that need already the from good when we resource and built. new again, additional leverage ourselves like will resource the we how a much we’re look platform that’s set at capacity and that programs, for it it feel I one will perspective, And in we’ve of place. is a bars So

Mark Rothera

this also regulatory you take like to our to Bobby, formulation and then, And in meet sort the question data cryo domain? about would the needs regulator steps the

Bobby Gaspar


thanks. So

regulators number have fresh the elements with like the of at what and package, comparability cryo the as two comparability to fresh different the engaged really, So would parameters between a two. formulation, is far significant that. understand that, to between and looking we One in there at vitro some quite the detail is CMC products, they then And as looking an in and which on concerned. the is two are

number those copy those parameters the transduced cells, those so the the of viability And of the of in the cells number, activity purity, the CDXX+ within case ADA OTL-XXX. include total cells, vector

they are parameters, those between the equivalent X? So

then way that data formulation. in in in this that suggest it’s that some And have we’ve supported the formulation. that cryopreserved be cryopreserved shown that shown working by we’ve would the you And data the same you data OTL-XXX, as received the data patients fresh formulation is And cell the from MLD that’s already. the

combination a it’s with comparability. patient CMC vitro the So data supportive of in

Graig Suvannavejh

That’s for my you Thank great. answering much so question.


you. President I’d closing CEO, to I the this time, Mark to At remarks. call and queue. questions show turn over Thank for in Rothera, ahead. the Please back further no go like

Mark Rothera

you for joining the thank Well, today. call all

preclinical team clinical year. demonstrated, we a regulatory and we’ve hope we’re advancing of of what I’m I proud really whilst As continues the filings build momentum to accomplished has portfolio rich as our first our two approach also this programs.

approved, to towards therapies world potential a the benefit progress remarkable mission single is bringing have our of administration, provide great in the curative a a therapies the patients these patients which we in believe And to of world. medicine. lifetime making We’re thing potentially if to around

to month Thanks at again, many next looking seeing and you are of you. we forward Thank ASH.


Ladies and today’s gentlemen, call. for participating. conference concludes this you Thank

now You day. disconnect. Good may