second for investor you joining us and quarter call. Mary. all you, Sarepta Good afternoon thank XXXX Thank conference for Therapeutics
Notwithstanding the even medicine arguably mentioned have multi environment. and our contingent ambitions. more of a unusual effective but as not merely but transitioned small multi-platform this As our have approach, become we March productive, all remaining genetic virtual are past, it of work to this advanced we in workforce on year, XX I the efficient program,
will community therapies, therapy afternoon, or already three-pronged evidence enduring on XXXX, highly and With in have continued you XXXX to hear our engine. approved as our remain therapy let generated achieved the performance. successful we have track to proving or approach gene that, And our continued our build expanded in confidence building patient milestones to our approach As have we many this serve us planned additional to unique review with is gene differentiated.
community, to safe patient endeavoring I million, in our As at increase am $XXX.X quarter same XX% them net last second sales year. pandemic, we our to to keep stand continue an this the while pleased serve report over that quarter
our the of in guidance on withdrew visits. impact last conference our clinic quarterly light potential mentioned I XXXX call, As of in we COVID-XX
see, that far you thus As impact has been can modest.
we the uncertainties ready not guidance. set Given of the to external yet are year environment, full
remain However, seen that we consistent continuing anticipate we in XXXX, impact far with thus will from COVID-XX what have modest. any
we completed let to by for NDA the RNA quarter, our our In me Moving on begin franchise. programs, second submission our casimersen. commenting development
obtaining Our date available PDUFA designed quarter amenable. XX in XXXX. the for exon casimersen in PMO X% be RNA Duchenne approval, built first to patient With since we this the who are the our and If the on of The therapy, have XX%. the more treatable successful platform are to therapy approved should the community bringing PMO treat of community nearly a approved. of we casimersen of be will our with than size successful population will casimersen approval, therapies was percentage first eteplirsen doubled Duchenne third FDA
not status PMO been, we platform quo. satisfied has are our with As the successful as
efficacy and generation successful, they RNA have technology. profoundly next which PMO, of the We our the of been on improve if our working convenience
the called study dose and multi-ascending peptide-conjugated our that XXXX. for MOMENTUM or in a is study are PPMO candidate PMO We
we shown we Treating proprietary models, exposure we In penetration. a dose in and animal production to dystrophin are increasing positively if the using is increases PPMO exon-skipping thus peptide increasing turn can that charged shock greatly Duchenne levels, all safely absorber dystrophin. production. tissue structural the increase about have greatly of Here achieve appropriate the
convenient mg our more exon-skipping efficacious dosing, So And broadly this if year, PPMO our for PMO profoundly the steric we where the we XX we well. exposure, second technology benefit. have monthly see This to at and and half therapy have the RNA release for within would concept as and able could to PPMO increase that tissue exon-skipping provide therapy, will a therapeutic important proof far In only but at weekly we’ll XXXX potential kg. diseases candidates a versus of our treat candidate PK/PD platform per are dystrophin DMD, a production, RNA could more platform. dosing more blocking will completed of safety as RNA comparative not an be
to pillars therapy now There are upon gene Let’s engine. which move gene three our building are our we engine. therapy
third First, the expertise, and advancing capacity and our science second therapy and improving pipeline, itself. effectiveness and manufacturing manufacturing of gene
the data year. we course that track, first-in-class approach generated development only and this already of additional is continue confidence additional Consider year, to stayed already gene our data on XXXX, been we the our number therapy significant have has to but confirmatory gene the unique generated and patients. DMD therapy dosed a LGMD research on build not And and replicable. this that we Over platform having of successfully with have
the proper Sarepta’s exceptional and regard, genome of gene cell this shown One to safely significant challenges In nuclei. have doses. good with the reasonable at tolerability of programs full neuromuscular expression body copies most therapy is number delivering
cohort On from LGMDXE. our of results This on vector, measured copies mean expression XXX, common will Now approaching much we the because in fourth quarter endpoint basis, exceptional of we of three share for normal per were commenced in fibers guidance Rodino-Klapac, same the that heavy report approach first DMD XXXX, XXXX study in the within genome the platform Louise trial SRP-XXXX SRP-XXX that for functional XXXX, patients positive all clinical dose protein a a western We treat SRP-XXXX low our AAVrhXX IF in intensity recall same our as patient expression. the to design we the SRP-XXXX Dr. and under using MHCKX. important is that a and announced measured. pleased and to Then blot, announced SRP-XXX viral that we promoter results dose, even IF each the nucleus, at quarter chain had material. you of on the reporting placebo-controlled significantly every patients of X.X and and by our improved four SRP-XXXX, for
we Neurology, generated example, an the announce of on SRP-XXXX more in per the significantly using for additional gene one gained functional were patient and There one used three construct fibers Duchenne point quarter, that increased announced with study data for with and improved As in of our in JAMA months. to XX% constructs. in dose, showing that same same month, beta-sarcoglycan consistent with have on the had therapy on XX% the have robustly. dose a protein every nine over every dependent copies yet, was our published In the very on average. second with children positive of child fibers, significantly LGMDXE, study expression blot, function cohort on improved pleased XX% significant achieved. intensity. confirm were that children at X.X XX% results achieving results of experience in year. In line normal to XXXX, SRP-XXXX nucleus The the dose safely measure Every Study Duchenne. the boy the year XXX we of the second for In increase deliver we at again every each and treated We were for ability we genome western our positive
two study clinical children any far our over studies our Additionally, in therapy, patients more active the placebo-controlled on now are dosed studies are proceeding DMD, we for children for SRP-XXXX patient have for we SRP-XXXX XXX the with for as XX analysis dosing pace. using than at crossover By Study SRP-XXXX, and in between programs similar our all main and XX well. dosed
The two over we built our second therapy gene have manufacturing expertise. pillar last impressively is of engine our the years. And there
partners. Cambridge process and dedicated in Across technical Burlington for some now We biotech of It Columbus, approaches our is operations, over gene therapy in Fisher. expertise dedicated years our At same the Catalent capacity we available within and dedicated drives and out and this the manufacturing centralized quality. our with with gene the center have analytical between to suites group our in therapy time, XXX just professionals built manufacturing and our Sarepta. that across two at greatest have site Thermo among in and development therapy, have excellence our we gene Lexington programs
next and runs For with views, we trial analytical for the development SRP-XXXX, for process GMP and completed development our and completed clinical material commercially. have land have both
we’re SRP-XXXX, GMP For now. in runs
The of and third bringing and therapy and to engine the our pillar together improve the therapy. advance gene necessary of technology effectiveness tools gene is science
development. gene behind. their using ablate use the therapy exosomes armamentarium. patients most Therapeutics company, opportunities to science and entered manufacturing access there, an deepest goal imlifidase began nothing therapy improved otherwise plus process, in into XXXX, would for And then ImmTOR to look a partnership commencing open use slow entered gene our Selecta into then effort to and antibodies to COVID-XX use machine years of our our with From re-dosing. pleased relationship a XXXX, science explore to develop commencing enormous gene internal announced In the we program two Dyno am our endeavor. and alone, Codiak entered I and in approaches learning the driven Biosciences and out RNA. to with technology across therapy one We that on of to the the added to we with In Hansa to left are pre-existing partnership have from May, this compliment Biopharma BioSciences empower gene we out with quarter it valuable editing a And Having pipelines four to of of in we our we their to done aggressively in for building to this approach in of that advance into spending capsids. a note last progress to be new there, neutralizing has we partnership built and effort the therapy, an with then gene to AI
important engine the across Now milestones XXXX. remainder looking forward, of have we therapy gene
efforts to we in two major the track patient We respect trial on first year placebo-controlled is visit out by material. of using to with XXX, last trial First and That quarter must SRP-XXXX, have complete end of this Study last have ongoing. the read blinded clinical to our XXXX.
in and this trial the some quarter. respect team this Secondly, using well, with commercial items XXXX. second to in hand, remaining the now half the obtaining goal certainly IRB gaining the but are our alignment is SRP-XXXX, material working will the through engaging goal next pivotal which of is in GMP are occur pandemic them things and challenges start gating has FDA approvals. and to material, there following; trial going our the our second, here, created on first, initiation GMP sites start our our The is in the with With to quite year,
agency and Our SRP-XXXX. appropriate development and with development two GMP and for regulatory release items the dialogue runs, the assay completing first, are of a second, here obtaining completing these; gating pathway our the on
it for LGMD important SRP-XXXX, it only the development candidates. development provide this insight our will is an into other for Now believe, five will dialogue, but the we not pathway inform
will on GMP our and update early SRP-XXXX material We discussions XXXX. an LGMD in the provide for
the and anniversary trials years, we And that lives thus In we in now pipeline and medicine, from resources articulating engine. are pillar the gathering ambitious very note, goal a by results have advancing building programs, three to years, will a therapy therapies. made ideation in year rapidity talent a better, Sarepta. reality, built enormous by gene bit movement by with productive make with and platform executing hourly. bringing enduring the to patients and personal to the If a that an and a you and over bit consistent become that building confirming extent have second three of XX disease. so then possible on we Have we therapy far. their XX an have from disease In to vision the their their and serve, last clinical rare leader a often the daily matches longer are done from seeing and reality, at and By them gene urgency living genetic RNA dying of stolen three enormous ruthlessly progress toward in those patients and with our to our an robust marked amount me three who life quarter have with indulge engine progress multiple made of the my too
the I make reflect three progress, credit groups, our workforce and XXXX up our sophisticated, on over that As Sarepta. dedicated go to hardworking the professionals strong should at
best external community our of the that us course, Our in relies us and in community, brightest world. and partners, pushes opinion, my And us. a guides the medicine also not upon us, but informs around patient and often only genetic
we clear celebration. now victory is time lap yet not premature a the So for are or
appear team, you prove. far, course on next by but over of success months count to our Bo stay with still of the on therapies. more obstacles our turn address But this that and to We in patients have over now the to encouraged us Bo? waiting so remove that, XX for update. are do And commercial we the and to to can for front for a here and mission, call I’ll much much execute
