RGNX - Earnings call transcript Q1 2022

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Operator

Good day and thank you for standing by. Welcome to the Q1 2022 REGENXBIO Inc. Earnings Conference Call. At this time, all participants are in listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. now today hand Mr. would to conference I speaker our Instructions] like the to over [Operator Patrick Chief Christmas, Legal Officer. Please go ahead.

Patrick Christmas

Risk Today’s Good our statements regulatory be outlook ended afternoon, actual financial The XXXX. press us www.regenxbio.com. conference anticipate, today. results REGENXBIO’s reporting call, discussion today’s our Securities These is this account the you These this annual call Any we meaning. REGENXBIO identified is future no Officer; and statements available of any and risks to and for REGENXBIO’s call may are will, new operating obligation that our call uncertainties. and provide on being Chief expect, Please our otherwise. make should, such statements Earlier this information, in Pakola, believe, development we date the future May may risks XX, for Any product report thank to and management’s to Executive provided words financial Officer. Factors may, Mills, be and and plan, the conference uncertainties reports recorded Chief as forward-looking Vasista, we will are for forward-looking XXXX, with Steve as such Chief to regarding involve President and afternoon differ on words are Ken SEC’s undertake cause call year and from Form of and can advised forward-looking and only Medical which Exchange addition and the are subject webcast. Dr. March ended on on results include website. analysis sections our described is risks of Vit With and website REGENXBIO’s on certain and in events performance available guarantees of plans. on statements not full or us Factors of X, Officer; information Commission the of results and by this Financial released the other the intend file on of sections quarterly on at first similar Comparable quarter Form release XX-K financial December XX, and forward-looking Risk XXXX, those are XX-Q that forecasted joining and update

positions pro addition, forma may financial Actual the that not provided Mills. any results company. of like now materially. does be turn would may and over or results financial the unaudited to call is I or information In purport to to Ken preliminary operating project differ Ken?

Ken Mills

Good joining on Thank a programs provide the XX, At Steve well as update an you, pleased will provide financial I’m of overview of up and first recent for our open the with recap an results an our for will afternoon, and for ended the our call corporate the to March XXXX. business Vit Patrick. we clinical highlights of everyone, then will call, questions. begin line end on today’s goals. as thanks update us. quarter

industry. I gene optimistic be help recent detail, provide the like in progress the would some of for pipeline the into gets gene future context cannot but Despite of Steve performance on therapy I sector, Before therapy. to the market the

as and by or the pipeline therapy advance from novel goal quarter as right these and encouraged this recent the to This patients leaders, a companies, gene by modalities that key our REGENXBIO with treat history find our seen possible. XX the in industry therapy our to XXXX possible. XXXX. like gene if to not the I’m commercial collaboration industry. internal gene efficiently involvement into we’ve deeper pipeline past path strategy has lay programs getting AAV developed with been have announce of Well, years the greater I to how uncommon time soon to including stronger. had was monitoring, believe have pivotal deeper field for The this potential and to of has this products XX for therapeutics key opportunities therapy decision The created as use commercialize therapeutics we our Challenges and AAV I a led Over out the To can people, us the new towards to difficult progress NAV by areas our that challenges the I’m and as to technology high have and incredibly definable science a unmet believe a better as is that safety gene the end where across of In we in across advancing like beyond. experienced plan regulatory felt X potentially stage proud therapy. fundamentals strategy was led clear of the level and investment to recent of end, will and number a our of programs in high need. and gene guide the quality therapies company the never agencies tells been have licensed understanding develop unmet relatively year needs past during X to very X our why partners challenges field a platform are times

our treatment One by of and the XX retinopathy. AMD is leading diabetic candidates of RGX-XXX the wet strategy to for X meet

to leading this broad in to AbbVie but as are RGX-XXX validates we market milestone. know, receive REGENXBIO with a payment to $XXX in also to only have validates not partnership player opportunity the and you which gene bring As for RGX-XXX we global X.X and a believe final a ophthalmology. its We space, upfront up received have therapy eligible deal million billion in addition

strategy and advantage high of launches, treatment endurance setbacks developed X While the wet data presented to changed RGX-XXX also together we internal new in profile also few field, and our the Hunter based eye from leadership for treatment by being the believe to And some opportunity in wet and for retinopathy we both and its include full has past macular landscape over the AMD of both RGX-XXX with the AbbVie’s diabetic for therapies in encouraging and and diabetic advance the candidates RGX-XXX, AMD XX commercial months the work expect take retinopathy. as syndrome. for our product age efficacy wet strength both plan we degeneration Overall, for in such treatment RGX-XXX diabetic the of retinopathy. related as we on and for of other safety Duchene has programs care improved to

interim Hurler at World for our highlight for and the of neurodegenerative diseases these deliver update treatment We presented the data data RGX-XXX we of syndrome improved patient alter programs treatment quarter, debilitating of outcomes. the course the program, Hunter Symposium storage the lysosomal potential the believe syndrome. RGX-XXX This of to in disorder positive for both

with to the clinical we decision isolated made step. take the final stage to patients are This elected unexpected trial supply. Moving all due Based vial of a trial. inhuman was an in contract first key in in manufacturing continues, delay update to This and for be manufacturing first trial we to this half a our was preparing Today, clinical announced RGX-XXX. situation. proactive of investigating We’ve didn’t the readying expectation and patients manufacture additional criteria. able this our to in informed Work patient Duchene of that our observation that filling difficult decision XXXX. safety. sites, dosing We consideration stakeholders on is clinical dose meet and initiation quality current to including

in-house own to this event having capabilities. this our Importantly, view a highlights success. and recent importance our as unexpected term manufacturing of I longer the key

commitment delay patients with be developing RGX-XXX our a Duchene, plan development current our element by does While this not XX which continues will for impact key our of certainly strategy. X to strong to

programs may developed programs, X technology our our by licensees. XX Beyond by NAV internal include also

driving field technology over NAV database Health Trials in vectors National AAV Institutes trials since XX therapy clinical XXXX. of of is the Clinical NAV gene forward registered with Our utilizing the platform

today. believe of by the and also a United of like States for therapeutics in range greater therapies number Our talk we to broad strategy. by therapy key NAV XX a one technology that, approved in is developed Zolgensma, of the FDA AAV gene over therapeutic meet X disease now the indications two being products these about to our basis additional also will With gene turn I’d the licensees programs. to technology Steve NAV contribute detail call With our programs internal to over areas

Steve Pakola

Wet loss VEGF. is XX, severely of unsustainable and injections. a diabetic care U.S., is more AMD AbbVie with receive to require evidence developed being patients these patients standard shows in retinopathy. of update Europe wet vision including Thanks, the with due therapeutic AMD leading the of endothelial multiple for Japan. to factor eye ocular to X growth to which weeks. current AMD than every undertreated inhibit that people the and anti-VEGF injections deliver fragment collaboration begin in wet a affecting X with patients patients treatments, over vascular I’ll the Vector are into NAV XX AAVX gene to frequent Real RGX-XXX, RGX-XXX million on Ken. treatment cause world indications the The and to or wet uses are burden which coding antibody an in treat AMD

that vision Data vision loss as has We is wet treatment AMD. durable a experienced preserve and thickness improvement represents RGX-XXX Phase treatment and As wet majority for wet of treatment loss with treatment stable significant burden. approach of the three AMD the patients to vision treatments for reduction our believe anti-VEGF in and the onetime developed for frequency AMD including subretinal the X/X RGX-XXX has for presented trial now the a to-date using retinal reduce effect demonstrated potential RGX-XXX of in a of significant for result, treatment a advancement delivery the AMD patients wet over clinical over wet being potential time. anti-VEGF mean years AMD.

two subretinal eye be under enroll trial submission with collaboration AbbVie. continue agreement delivery approach. clinical support in and to our our safety expected pivotal patients evaluating for in first a with using ASCENT in RGX-XXX AMD are patients efficacy our to and is care the trials two ASCENT These XXXX. wet PLA of We ATMOSPHERE trials to RGX-XXX initiated the

developed and RGX-XXX. receiving that the AVA collaboration for and that recent anti-VEGF an stable trial, burden our presented are Super both from advancing two AbbVie the of the wet the six with choroidal in compared prior part also the have being demonstrating data using additional treatment phase visual to most are cohorts AMD, XX% wet programs well Super we are using delivery choroidal delivery, to office RGX-XXX first month retinopathy with We as our AMD profile In during approach. cohort in and acuity clinical the X diabetic emerging and data XX injection rate of months mean in to as retinal thickness, evidence RGX-XXX X of showing reduction RGX-XXX annualized

last of was incidents serious dose topical on Looking is intraocular safety XX all cohorts no both reported of similar levels. tolerated observed at patients cohort well with drug out events. the inflammation slit cohort be as November X profile across was and to four and RGX-XXX XX year, of in related XX out across in patients lamp That patients in with quickly examination Mild X corticosteroids. of at in two resolved three adverse

expect of XXXX. do after or trial the steroids XX this half receive in not We RGX-XXX. enrollment in prophylactic to first of complete administration before in Patients

blindness is of XX for are the complication DR of and million adults diabetes RGX-XXX between XX. patients in DR. to this the An and and the macular leads proportion patients of diabetic estimated Moving is treatment DR edema vision leading ages affected in is cause a large to threatening XX to including DR DME, blindness. slowly neovascularisation disease a that debilitating worldwide. that disease lead can of by or progressing complications, of

reduce demonstrated treated from control. hurdle angiogenesis not disease the the XX conference improvement cohort developing this presented choroidal proven baseline our step their a treatment We to month anti-VEGF February, of alter these compared due RGX-XXX important complications in therapy ALTITUDE administration could a single two significantly Super to on that the recently overcome potentially in for vision this scale patients phase data to X observational burden. the However, in after many have X threatening to at and with clinically receive in patients provide treatments six from RGX-XXX DRFS X% XX% condition the risk unsustainable progression. meaningful therapy trial do the an of

from increase months. an Importantly, this was at three XX% observed

potential is administration no with time seeing to before this intraocular receive RGX-XXX. SAEs completed Shifting not by inflammation. of well are no dose We the rare RGX-XXX our in gene the Furthermore, as was stage. ALTITUDE in trial, related portfolio this and drug reported of XX, trial cohort after in the and for encouraged X what our disease steroids January now did we at treatment in to RGX-XXX Enrollment XX Duchene. patients or are therapy patients prophylactic tolerated of one be

We induced RGX-XXX preclinical dystrophin. or it muscle a dystrophin for occurring designed of been shown be the leading to have the several of product CT mice the and naturally key damage and a resistance C-terminal presence studies, the muscle dystrophic the CT functional developing differentiated proteins muscle domain domain that are transgene to has a to to highly elements membrane to in includes novel to In contraction recruit improved models. cell found deliver micro

gene and is our targeted to translational expression, vector include Additional and optimization expression gene well codon characterized which a skeletal to and promoter. delivery and reduce design heart immunogenicity. RGX-XXX reduce muscle throughout the AAVX has NAV content improve support specific using and efficiency muscle designed increased CPG features potential

we or RGX-XXX of for encouraging along indicating or Hunter developmental possible. year, of the Ken Earlier for fully initiation this profile presented trial soon Hurler demonstrating initiating evidence from trial World the committed ongoing the to trials As CNS prepare for to at clinical with discussed CSF as We as this readying neuro data activity, supply. site treatment both earlier, MPSX we manufacturing both emerging biomarker for Symposium, clinical including remain additional continue and and MPSX of treatment candidates. for trial potential severe syndrome, assessments syndrome, the an RGX-XXX

As XX were last drug with related of both SAEs. tolerated no RGX-XXX December year, RGX-XXX and well of

XXXX We have expansion providing forward both throughout we in the patients in and Overall, the to plans already continue trials. look enrolling further for arm year. of significant updates we made progress

call turn Ken. back I to Now the

Ken Mills

in for first this Thanks, the of over good part past updates the year. we’ve made the Steve, and to team this the for and progress quarter

to mentioned Before call highlight our turning that Vit over progress the to we’ve would manufacturing. I like about

we’ve our As share to scales process. Maryland excited up Rockville, XXXX platform of production manufacturing new begun I’m that facility to vectors in our this operational, meters you for yield utilizing culture our suspension cGMP houses new headquarters using NAV now allowing know, that is high and facility. cell

With infrastructure. the basic headquarters GMP commercial to from we in and new facility, expand are to our continuing scale research manufacturing capabilities development

X,XXX remains GLP analytics corporate art both advanced over to our have of our of for multiple strategy. XX,XXX of flexible headquarters liter support GMP programs. key square our suites bioreactor differentiator for feet We drugs key element and manufacturing dedicated Plant, REGENXBIO fill to of and state This in-house Pilot a lab, multiple capability the finished suite a

production therapeutics. cutting facility to move to of which Our of selection material supports edge clinical accelerating from of early AAV the our quickly allows us the candidate development grade

team enable the readiness. formulated need has early Additionally, a we control on development innovative product with and manufacturing that device to. focuses pioneering Steve clinical choroidal use from believe during process quality the our techniques approach to distinguishing of competency be REGENXBIO. to to a device lessens innovative our AAV alluded in a the intracisternal of used which therapeutics and therapeutics efficient the clinical commercial technique Furthermore, trials leader and for is changes interventions for clinical super transition including delivery AAV delivery believe We number being of solution

over call to Vit to of stage, the at our turn going So this review financial guidance. I’m for

Vit Vasista

decrease million capital on the to reimbursable $XXX.X XXXX. trials quarter the through lead quarter Beginning collaboration while result in It compared with the expenditures costs expenses accordance of and for XXXX, offset increase development cash for to March attributable marketable REGENXBIO we during operating eye ended trials XXXX. marketable collaboration. other funding development activities AbbVie. March agreement with was temporary thoughts. The March compared RGX-XXX for a activities related ended on equivalents of costs as XX, $XXX.X development to partially March certain XX, for million March were debt our unrealized continue ongoing $XXX.X AbbVie of cash, that, call equivalents cash operations securities Ken and quarter you for and of XX, $XX.X our the RGX-XXX provide $XX.X Ken. R&D as through of are by used December under the Thank to current the million fund In majority quarter ended REGENXBIO was and well all for securities Based was ended XXXX. manufacturing back losses REGENXBIO XXXX, with I as of to as clinical in clinical end personal balance XXXX the candidates. marketable primarily with care costs securities be XXXX expenses. will cost to million primarily cash, fund associated and AbbVie million fund will XXX will on XX, shared cash to totaling The by XXXX. product responsible plan, the our XX, into With expect turn XXXX to final the operating and XX,

Ken Mills

of Duchene reiterate RGX-XXX, support unexpected to this RGX-XXX the I want GMP Vit. plan strong first you, I this Thank for RGX-XXX at our continues communities for therapeutics. their facility. to the delay and take thank our the We patient moment would a in-house to to Taking just our perform our Overall, clinical to facility commitment in on and the community. REGENXBIO back level to one of REGENXBIO circle manufacturing innovative our team, runs This to for commitment AAV high importance our program. development and like time very to supply reinforces investigators

the heard from expected on be what therapeutics. per to far are my overall used also s vector today, to our we NAV summarize be approximately improvements us There of over in leader to processes. the our of in produce, field five status to So that new to improvements progress for we plan to of RGX-XXX platform. begin with dosed install that process yield will in AAV than previously year, technology so example, you’ve We’re case our derived batch times continue AAV views facility in able from the therapeutics patients this in a X,XXX use

the and programs continues Steve choroidal eye in track our on delivery advance and with by in unlock mentioned trial first is additional global progress AbbVie clinical filing enrollment collaboration data excellent for PLA XXXX. care expanded supports to trial Emerging Our super to value.

have We look AAV behind advantage to accelerated we high disease our where unmet clinical and to take needs. strong of science rare of therapeutics, pipeline data pathways address

GMP is of internal the capability across support a AAV diverse Our scales necessary and clinical programs the operational, quality yield manufacturer to supporting development commercialization. and high at range therapeutics of

We the NAV the AAV generate scientists platform the application and engineers differentiated the applying innovative expanding amazing technology an therapeutics. have also and team vectors, understanding of of to capabilities potential and dedicated the to exploring AAV new of

remain five that execute stage it benefits, We capabilities. expression, that in on NAV research cell commercialization proof XXXX. and and step believe systems mission on to conference and more late fund development clinically announced use including optimizations pipeline advance this ASGCT of on strategy by we foundation And identify week, XX type our of and which therapeutic expertise the on to reflects strategy, the summary delivery process the therapeutics device combination, in and capsid, track next for fact, of at the and areas XX AAV our capital specificity innovative to and vectors point, and X AAV next development characterize that concept to operations novel by XXXX. tissue stands as In the designing relevant with AAV such our of and manufacturing features evaluating we our week, into analytical presentations engineering therapeutics, enhancing all in of around informs to promoter leadership enhancers

that We’re and lives. to more potential confident people our ever in diseases our patients to significantly developing than capabilities our support therapeutics impact science, the for AAV have

portion call will this turn is the operator it over of I So over. for questions. with to that, the

Operator

comes first Our from of Stranahan question Certainly. Alec Bank [Operator America. of Instructions]

open. Your line is

Unidentified Analyst

questions. our Hey, for you everyone, for taking Thank this is John Alec. on

from Just a couple us.

question. in little think XXX delay to kind What the bit more ran will exactly first So on think issue the delay And was this decision competitiveness could elaborate you you do question you of of that the DMD? how the I the field a that’s the overall XXX affect the the of just program. into? first

Second on DMD. was also question

and obviously That’s you So the in kind you news how concerns issues and other question. approach issues study? into the of how your those learn do run second the companies recent wish kind of do all from on with by that to eliminate of safety of

manufacturing, what that? But one Third in-house forward obviously, the with is going you question, just short very do to you. manufacturing regards a think on Thank are promising. challenges

Ken Mills

also I’ll back clinical John, for comparisons the questions. will thanks manufacturing. in-house Okay, on Steve circle start. let I on comments and trial

of XXXX ours reported, observation dose have And So we quality a delay. patient manufacturer process late to the we of that XX delay nature first isolated reported at report and this half first which was was month to stages third This X caused that us final I to for our this contract have party think of criteria. a a meet and something didn’t telegraphing that stage very is as re-guide unexpected of the this manufacturing supply. unexpected the had of RGX-XXX the in I nature clinical in this to

and So been of online supply the of facility the commercial that we’ve a to bring house. GMP our facility our look communicating been And with we landscape today how that at investment, We we’ve facility. and supply availability with headquarter is of operational. the that out the relates in in is pipeline pre-COVID, for think clinical GMP started announced looking build to intent to which bring your you third the the for is all we quality important question, about it while, John,

approach, able strategy to respect those XXXX in in the team pivotal other to or turn by XX commercialized to comparisons So in to it key a elements be support. GMP with With bring the house for to us, of capabilities our of I’ll five to to and company our Steve. a Rockville trials us enable level here imperative for phase over XXX that X like programs by for be manufacturing

Steve Pakola

Hey, John, thanks. Thanks for the good questions.

we at data historical of collective our can to sets of in that AAV of field later, understanding licensees of all benefit but and collaborative of the And think other the of the On been do advantage in both the we field our look I all take in have all this. think in got Duchene also of I programs. appropriately. field And it’s very the we

get what’s So take all been I learned? of think one really where we can’t advantage you program each program to of aspects. to carry another is forward the specific and from though, much too different because

other very AAVX clinically and well from for of learnings out for dose versus at of our abundance been as a of example, other complement been of So that initially we typical in that AAVX example, transgene some to advantage really activation, in high human robust activation example, way where just we’re caution for as course programs for with being with with doses. first nevertheless for immune short suppression where regimens including the complement the this AAVX, AAVX taken immunogenicity, how we’ve that an seen but inhibition, complement as potential AAVX study has of our Eculizumab so higher same aspect. seen cover And at look the the issue real hasn’t have program in finding a

we’ve good by of where very clinically at we’re dose at proof pathophysiologic So the that the way first preclinical well concept and both clinical doses we our seen excited good that looking safety our based very model. feel we very on including see package as in very as

innovative exciting to all together of this within very promoter about AAVX on. feel So an advance really us ties make program transgene our as good

Operator

from Stanley. Vikram Morgan And next of question comes our Purohit

is open. Your line

Unidentified Analyst

This is for Gospel everyone. day on Good Vikram.

So the I’m And is, patients you see aim prior to is would need of updated? do sharing were and or do generated that once to data begun much questions follow that RGX-XXX outlook program, XXX competitive dosing many safety two up for how sort for initial what how a the then initially profile? you think have data? to going has the efficacy you And we to first the have establish our set initial one

Ken Mills

color, Ken. I’ll this necessary. let Gospel, more Thanks. some is provide if start Steve and

we’ve profile for being therapy a of studied see one design, for microdystrophin levels the we’d RGX-XXX three safety such evidence dystrophin is profile at to similar parody, up So with about support treatment we dose that at RGX-XXX to the microdystrophin, will, reported the candidates as of evidence months, and of the dose it plus measures secondary of initial be comes are a as, the design XEXX. improve three three XEXX, includes at things as as be of When this that well. on cohorts moving basic improve, the that those potential to to that that two intermediate similar similarity levels to And And three the paradigm. safety for are and trial cohorts based existing stage and outcomes, micro continue expansion as design has well as treatments patients, days imaging expression you execution looking XX complete well look to of beyond. primary measuring gene six be endpoints to we’ll looking the of the to if months functional order some clinically. place like in look the AAV We’ll be for to that a there each

be of we I that others continue points that capsid. types that extent AAV demonstrated, status, to of same products because of about pre-existing using market for immunological in with who the that I key different the because a access AAVX. could to think there to microdystrophin in AAVX instance, to differentiating based profile And immune using other access we capsid, able that or place for are emphasize believe or think can types think the have the be a addition we’re are evidence its we see differentiating therapy, would with aspects gene couldn’t RGX-XXX of patients demonstrate, other

think term facilities itself importance quality as cost to that we market, our we profile. wouldn’t years, differentiating an today yields to that well at a facility term those with to contributor our the term on only potentially longer the process at those or we to and advantage supply, likely advance us control provide the year, able to early higher well supplying meaningful, a functional for also terminal And sort differentiated first not being importance and domain that on the manufacturing addition months outcome in over Things at opportunities contribution and start with our to in our but things months, emphasis having processes, show to are XX term having finally, be manufacturing over long will to look pick as control on maybe to as that to new clinical of the meaningful, the marketplace. XX addition, the for we six with plan and days continued processes, bring then longer of In patients and here the to a with that having processes in And of our improvements. or profile. Gospel two C of a lesson the major think the long side, improvements people, looks learned for are goods be is control development, would that up having being would paramount Duchene follow but up helping

Operator

Wang you. our Barclays. question of from comes next Thank Gena And

open. is line Your

Unidentified Analyst

Thank you.

We for have on this Tom is three, Gena.

questions. three have We

one about the first XXX. is The

two of level cGMP re-moment see choroidal protein the facility. thinking, is we and I completing data the expect when you the to regarding Super second as am to trials can So

is what get Just for supply? analytical are beyond to And to clinical the you skeletal the ready hire in folio go are versus the saw for you mentioned there last mediate should new in steps Duchene AAV clarify program I any of disease driven other expression normal the other to transgene that model wrongs ASGCT or through mice, a the question down the you abstract factors. muscle regulation the by

defining the also you refine a is does relevant dose how escalating in to And wondering clinical this human? Just and study. fraction dose to help strategy how strategy

Ken Mills

want grab Thanks question. you to for Do XXX? the

Steve Pakola

Sure.

other response. in of diabetic have clinical have good delivery studies, as to wet we continue ways studies, we assess the retinopathy and in the both XXX, on dose proof we So in in where non-invasive response. one both indications, you have choroidal super the office evaluate two In concept very AMD to we mentioned, clinical

new aqueous assessment. a Your done a measurement And signal we on for these on choose that aqueous the and were include studies the humor protein of question for retinopathy even that’s just did now focused with as on when super used of that for actually diabetic case data first chorodial variable have signal we’d and any and given studies guidance ongoing ever endpoint to route the signal in are of protein, this administration include humor approval. clinical we with that haven’t to meaningful clinically these even we have particularly actual delivery

focus there. heavily Our is

not clinical to where path, there. question your supply far second as we think I as bioreactor on fortunately, related critical that’s the advance how

completing that’s mentioned, the to with we on to our XXXX. be as pivotal two submission really on Ken a track driven PLA So And continue in studies. plan have

Ken Mills

and efficiency, at to bio respect safe into support as different certainly that our to selecting evaluating totality of There disease distribution. safety the proceed. as well Right, respect models We relating work including background models doses candidates is and with things to like for types a of obviously looked Duchene the and preclinical purposes evidence for the went granting IND around done FDA and RGX-XXX. and transduction for and of to with question

there may or models across other what development what think mics in I that people types are space with in disease and find might of well this differences it’s people models. animal of model, our black transaction you terms six to in see organization, and as learned you in a and as have respect research

things, of the all This those that time to reconciled evidence package of appropriate last the over been XX. question. that the build the escalation roll inform to dose the end for alluded continuing be Thanks starting different to able filed parts IND, doses to the since of out at we the we we’re IND package XXXX. into Planning for but year. went So Steve today, totality has at of the there,

Operator

Mani question next SVB from Securities. our of comes And Foroohar

Your open. lines is

Unidentified Analyst

Hey, Rick our good questions. Thanks for Mani. on afternoon. taking This is

you could dosing? the initiate just need the simply that some XX of you delay walk before of take us can So given Is month roughly a facility? to party product Or dosing have other seek you in-house would steps? that or to to protocols there’s new steps produce through of you potential third in from any it have new your batch manufacturing potentially clinical a revise a manufacturer place RGX-XXX,

Ken Mills

for the that doing primary here our X have capabilities we establish month in So the so is new XX Rockville. re clinical using the GMP the and supply delay I plan of is essence to think to

relying both of get us strategies I point last was from end and that for think at outcomes it that to safe potential then the of to And fill get be to great drug do think capabilities is IND proceed the make we essence mitigate it will sort going the have that we different done that sort be as have filed year. I wouldn’t patients always is here was part will it but on this the supply we year. of under of we that to like prepared hitting announced early re-guided of

As on this a but that to of in progress to that addition next year to and make for get is we we’ll this the update closer half milestone at us unexpected guidance, stage. and something event recent continue first that

guiding this is So, now. right we are where

Unidentified Analyst

All taking Thanks right. it That’s us. for from question. the

Operator

line comes Wedbush Certainly. from your Argyrides open. of [Operator next Instructions] Our question is Andreas Securities,

Andreas Argyrides

taking one We’ve for your afternoon, got Yes. question. thanks and on Good XXX. also

mass so talks on Thanks. domain. a an guys targeting question other have quick abstract and that of just ASGCT You as binding recruitment how competitors may from about to differentiate the that XXX [Indiscernible]

Ken Mills

of glad on to things and to I’m on including that of improvements candidate as the call, enough to in its limited closer have truncated got of effort the dystrophin safety. structural the as entirety biology a we part We multiyear domain dystrophin other circumstances we again the we domain closer expressed our that based to try have think the to, we integrity I’ll you aspects and there of function, published and complex C-terminal in Andreas. contributed differentiated the sort the scientists protein on of But well being I another the But was We’ve or but on we data to this C-terminal as that that the fill as that should RGX-XXX, in candidate the to construct protein that. and brought these. think, of in from science, abstract became that complex. raised have of R&D associated fact are are final based that stabilize at ability selected manufacturability, select based thanks, the a apparently. RGX-XXX talked about that the looking things, view was part Yes, that shows essential the to of team recruited recruit selecting to recruit elements order not that which other the were

And know so been part which construct we that instance, the the brought talked been of, we is think something biosciences them, [Indiscernible] that clinic. into we for has know among about as has that solid

candidate the a components and always making component science, us biology to that of been C-terminal improvements evaluation is key of view the and is for has has Our been a direction to selection. the domain go dystrophin of good

Andreas Argyrides

It’s live very Great, on Rockville. going congrats Thanks. cool. and at

Operator

is next Danielle And [Indiscernible] line your question from our comes open.

Unidentified Analyst

the there for manufacturing of related as taking And any news that XXX other a The XXX. could the question. well. and you if other have affected? of programs? are so, programs all the those I Is Hi really for thank be is parts one that any chance on for

Ken Mills

this for use the part that pipeline. for of is other the program a party that And any programs our of group we not REGENXBIO manufacturer third was

Unidentified Analyst

Okay, thank you.

Operator

turn Mills closing this now like showing I’m back time. further I the remark. conference to at Mr. would no to questions Ken for

Ken Mills

want reiterate. to again just operator Thanks, Thanks, everyone.

to REGENXBIO capabilities for of as progress continue patients therapy we field and the excited help we’re for For gene at as be possible. the to the making here have time the today many

So support a for you forward day. questions Have good everyone’s and we’ll thanks to seeing soon. and look all

Operator

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

You may disconnect. now

REGENXBIO (RGNX) 5 May 222022 Q1 Earnings call transcript