FDA becoming In the medicine afternoon steps, the of in Therapeutics’ plans vision including give results have gene executing into continuing our us three clinical near-term recent very the strategic micro-dystrophin took corporate and second commitments. programs the genetic Sarepta its on one in call. this States; CNS-targeted significant our Good United in and at casimersen look move potential a direction newest second our which very you advance therapies; Churchill quarter, to observed, famously meaningful, hold global program. our all should But, perhaps and our and therapy for discuss most Ian. of with to our after meeting update strategy, partnership, fulfilling afternoon, results”. will accelerated us to you, XXXX quarter conference we while the indeed Thank thank of leaps Winston productive We and for joining the achieving beautiful responding much remain focused progress FDA our approval occasionally on and companies, the to to you “however the new
the Sales same reached over half begin $XXX achieve $XXX year. of remain So, to our do announce guidance revenue an second to first $XXX quarter today results. pleased strong we were last EXONDYS million track another We XXXX let’s our sales XX, of increase million, to very of quarter. strong $XX.X quarter with million, on of earlier With million. XXX% approximately
well If and a team development and and as of Cumbo of therapy approval, to first you affairs half commercial from company that then XXXX. take strong fully can for the breed rare very to like his that will community Sarepta with in supported conception, integrated indulge commercial both biotech a execution. medical is me, credit special fabulous strong give I’d our as organization,
for our received second Committee Human for eteplirsen or the the Medicinal Products CHMP, in the in also regarding MAA Europe. We that Use, negative for Authorization from announced a we quarter opinion Application Marketing
of have the neuromuscular experts of discuss application or Group, the opinion. will a SAG fall Advisory The CHMP to commenced in meeting, reexamination. Scientific of the We XXXX, reexamination CHMP hold our and
While to remains patients for we mutations exon the challenging. a amenable Europe, reexamination XX process case believe eteplirsen in strong with for have access we
year-end. We expect the final decision by
on PMO our candidates. Moving to next
We are our two golodirsen very we’re next casimersen. PMO the pleased with progress candidates, making accelerating and with
proposal C XX among week exon to amenable to the to with As rolling you a quarter of with patients for analysis we an submitting treat Type trial the FDA to discuss from designed positive mutations. for muscle will therapy XX NDA amenable treat mutations FDA accelerated ESSENCE exon a Duchenne PMO of our XX supporting will a in the casimersen, approval we which recall, week, for potential approval of Last XXXX. of be guidance in our amenable held complete date in conduct following at meeting golodirsen, other first process XX target with our year-end a in a are XXXX, the the our and of designed things, by exon purpose with PMO biopsies patients patients
as is Our to for pleased skipping the at so golodirsen. and report without trial preclinical that casimersen to that expression a and efficient that as expression do dystrophin and golodirsen confirmatory agreed FDA it casimersen. supportive an was is dystrophin a for compromising to models our as as suggest proposal analysis sequence ESSENCE of exon possible perform am I
larger in that and We be expression means approval should for EXONDYS position to dystrophin position will casimersen, accelerated with significant before population file conduct which analysis year, the end of we and that combined a Golodirsen in if we to by mid-XXXX. a have XX. serve be that even this casimersen
To to these the XX% this is in candidates serving successful, will put therapies near-term perspective, by if States we approved community. of significant. the have XXXX, three Duchenne the to PMO very bring into United the community So, nearly opportunity
next is amenable for mutations first study next XX. And RNA Our our for performed by the of year. XX five dose generation talks PPMO where get ascending skip quarter a with work beyond progressing will single insight next the we technology, dosing candidate IND-enabling is underway the being
to Turning progress. now gene our therapy
life The an those for second important extraordinarily has and quarter rare particular longer, dystrophy. XXXX disease, fight with a with for muscular of our dose brighter to been Duchenne one in bring
at know, Duchenne of Dr. who from therapy gene day Rodino-Klapac. has very Children’s presented on three To Nationwide early by been you Jerry designed the Hospital, Mendell first our our June our results doctors micro-dystrophin Mendell micro-dystrophin As received the patients you, R&D and XXth, remind therapy. construct elegantly
a lower as First, other rates should humanized vector, vectors, compared rhXX available monkey-derived show be meaning appears AAV to patients. AAV immunogenicity to more to it rhesus
important which promoter complications. typically its muscular for in was patients chosen models, is to in is robustly the Duchenne who for from with pulmonary preclinical In micro-dystrophin critically Second or the ability express die dystrophy, specifically expression the the heart, micro-dystrophin to been be up micro-dystrophin muscles. third, protecting the muscle recently in heart the XXX% to was and of the And levels X, transgene was reconfirmed damage. designed which observed X skeletal repeats to observed crucial spectrin has to be from maintain in cardiac
As is the pathway damage Mendell Dr. drop of the to therapies. three-month robust rapid draft that Western you, showed creatine measured our an new is its no that particularly results What ongoing for FDA aware levels, enzyme Duchenne disease muscle gene kinase associated development, market unprecedented efficient gene with therapy patients by is that in as reported are aligns doubt, on with innovative drug creating July showing blot expression biopsy all immunohistochemistry guidance an goal the the fortuitous three the issued of rare muscular hallmark that for dystrophy. on XXth, with and
Encouraged Separately, pathway plasmid on hold of goal next based issue. and with trial B third-party XX program trials early FDA designs. are a the meeting to preparing that potential discussions our as crucial, Specifically, by next the of the clinical the to we end FDA for mindful agency that our before Type to to also It commence FDA the to pivotal registration to design trial. sponsors announced we consider before trial our July that and its encouraged our placed on supply on has a study, clinical alternative micro-dystrophin hold, on first-in-patient meeting executing the guidance, but for XXXX. align studies are submit recommendation is
As previously, research the as third-party for programs early is grade, clinical material we that standard in setting. current academic noted was this the
and moving completion we FDA’s of will of respond the plasmid near to before be the supplier. forward letter review, Hospital’s to August. to certainly a and end in the completed Children critical committed the Pending already Nationwide our in the to third-party GMP-Source audit have material fully of going We hold have position future
our the anticipate the should meeting with hold, in Given nature clinical on to of pathway. advance agency our the align of be lifted it we
our next Moving to Limb-girdle.
is to which we dystrophy, the as umbrella quarter, beta-sarcoglycanopathy, the you the in for programs, so patient of known first As all Myonexus announced a August. XE new muscular therapy program, recall, current five with LGMD. is The plan transaction a in called also will under broad the gene Limb-girdle in dose second
map four in To micro-dystrophin you, of Duchenne with the out dosing We on the we’ll share opportunity later and the are that. working the Myonexus with common LGMD XX% next year of of LGMD five an represent growth. remind have this update about programs and diseases, much to our
therapy Now, consistent upon with our franchise. build to our long-term the vision, breadth of we gene continue
of stage make in University significant a we spin-out therapy partnership, therapy Sarepta the the therapy seen of therapy afternoon’s number of is led gene of Florida Therapeutics. made gene by contributions this Lacerta world gene a in and terms transaction, As $XX from this therapy equity excellence founded University the Under renowned investment into programs million as today with clinical to announcement, one Like Lacerta’s research. the have have another time Nationwide top a in have field. centers Lacerta. will gene in was entered of Lacerta Children’s, and researchers. you numerous Florida founders the yet advances of gene
also on digit and and sales. have of to gene majority Pompe additional preclinical well the program We pay development clinical rights two development, programs. net CNS-targeted, as Lacerta an will lead will commercialization. sales to single will license therapy based Sarepta and while Lacerta’s as the Sarepta Lacerta royalties development manage received milestones, CNS-targeted exclusive
Lacerta our number in gene ways. strategy therapy Our discrete accelerates partnership very a with of
talent. First, access to world-class
Florida, Pennsylvania in are world’s the we leading them, therapy regarded research of and best Cornell. highly with in of journals, brightest Lacerta’s therapy Nationwide over nine across peer-reviewed College States, gene As centers are have United published in widely Medical the Children our founders, gene meeting the CHOP/University leading including ambition associating all genetic University and Hospital, hail by among XXX of scientists. clinical we medicine Weill said, from ,who are papers and
is course the approach expansion first to pipeline. CNS. new our program a very a To of three gene at adjacency, Second Indeed, closed gene on the eight adds of neuromuscular focused CNS Lacerta therapy disease, a therapy pipeline programs, therapy programs, gene addresses of our Pompe.
We also two therapies. to CNS-specific additional rights have
support potentially to could is generation are CNS Third library a delivery developing next therapy gene novel access diseases. of technology tools. potent Lacerta’s targeted AAV process. treat antibodies and manufacturing dose reproducible, drugs to application broader robust and patients approach platform for wider scalable, therapies an to a alternative with and these proprietary is that for patient capsid Lacerta’s more optimized of potentially to Lacerta potentially stable OneBac the population. enabling for And more allows manufacturing AAV,
will manufacturing build the While as micro-dystrophin our to avenues use new division. programs, potentially therapy our we of more not an approach, future, explore opportunity provides the be and efficient launch and this it we for LGMD gene commercial in the
discuss achievements made this will the I Next, quarter. talent-related we’ve and infrastructure
We passionate So with XXX first started employees. XXXX on dedicated, people.
second hiring and of breadth and As our places the wants of is speaks that first scientists XXXX quarter the And to was creative, two our ambition O’Neill, the biologists, nearly of make at a the people with year. attract with nearly of able impact last world. both business Sarepta team. become one Rodino-Klapac, talents Gilmore see if opportunities of value We’ve beginning experts that’s our Sarepta to It and chemists, class opportunities, like. one the great that team day welcome strong pleased operational has goals And I and we that will vision have next, commitment employees of on to to senior of mission, at to in biotech manufacturing this exit number entered we reins today. a at strong and year, been to double team, improve the shares to the Louise wise committed are positive top whom the doctors and to common present at we already and the these executive over I to X.X a Sarepta, on work, the call, Dr. achieve and XXXX were pedigrees. our me with our goals, employees, leaders, this our leadership. developers,
with our Brammer additional Next, commenced with infrastructure, our and with we starting provide the strategy us built Biosciences, for under In Cambridge manufacturing. we anticipate our On micro-dystrophin as commercial even space assumptions. on launch, manufacturing this supply which will partnership we hybrid even therapy quarter, infrastructure our in gene and Andover. aggressive facility gene in most therapy development out have taken sufficient
XX,XXX our to to an goal building therapy, Nationwide commitment of therapy Ohio, in deepening with also square-foot We’re with Columbus, and center the Hospital. and Columbus Children’s of our strengthening gene excellence relationship to gene
the one there’s Finally, will achieve I’m in the genetic few success I the our that most among our the one our above was purpose. years, others. say coming becoming meaningful these we predictor greatest medicine of what will vision thing, drive thing of world all of success, companies When asked over and
Sandy why work life-robbing know those every fuels And we hard an to what living is their and over now the I up what financial our is will disease we rare as us, upon our makes know futures. to on call update for there get turn me us for culture. this relying that, confident as Sandy? do. performance. ambiguity. And is our We success. We and families This are with For their continuing day with in no so that
