March 16, 2023

Sarepta Therapeutics Announces Advisory Committee Meeting will be Held for SRP-9001

Sarepta Therapeutics today shared an update regarding SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, for which a Biologics License Application (BLA) is currently under review by the U.S. Food and Drug…

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November 28, 2022

Sarepta Announces Biologics License Application (BLA) Accepted For Filing by the FDA for SRP-9001

PPMD is thrilled to learn that the FDA has accepted for filing and granted priority review for the Biologics License Application (BLA) for Sarepta’s gene therapy product, SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant…

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September 29, 2022

Sarepta Therapeutics Submits Biologics License Application (BLA) for SRP-9001

Sarepta Therapeutics today announced it has submitted a Biologics License Application (BLA) to the FDA for the accelerated approval of SRP-9001 to treat ambulant patients with Duchenne. SRP-9001 is an investigational gene transfer therapy intended to deliver…

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September 13, 2022

Sarepta Shares EMBARK Study (SRP-9001-301) Enrollment Update

Sarepta Therapeutics, in partnership with Roche, has announced the EMBARK study of delandistrogene moxeparvovec (SRP-9001) in participants with Duchenne is now fully enrolled. Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy intended to deliver…

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July 29, 2022

Sarepta Announces Intent to Submit an Accelerated Approval Biologics License Application for SRP-9001

Sarepta Therapeutics today announced its intention to file for FDA approval of SRP-9001 this fall using the FDA’s accelerated approval pathway. SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle…

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July 6, 2022

Sarepta Shares New Functional Data From SRP-9001 Duchenne Gene Therapy Clinical Development Program

Sarepta Therapeutics has shared new functional data demonstrating functional improvements across multiple studies from the clinical development program for SRP-9001 including one-year functional results compared to an external control group, from Study SRP-9001-103 (ENDEAVOR) in…

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January 11, 2022

Sarepta Announces Topline Results from Part 2 of Study SRP-9001-102 Evaluating Duchenne Gene Therapy Candidate

Sarepta Therapeutics has shared an update from Part 2 of Study SRP-9001-102, an ongoing clinical trial evaluating Sarepta’s gene therapy candidate SRP-9001 in participants with Duchenne. SRP-9001 is an investigational gene transfer therapy intended to…

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October 11, 2021

Sarepta Therapeutics holds SRP-9001 R&D Day

This morning, Sarepta Therapeutics held SRP-9001 Micro-dystrophin R&D Day, during which Sarepta shared information from all their trials involving SRP-9001.  New data from the 103 Study of SRP-9001 (ENDEAVOR) using their commercial material was presented….

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October 8, 2021

Sarepta SRP-9001 Micro-dystrophin Gene Therapy Update and R&D Day

On October 4th, Sarepta Therapeutics announced the initiation, in partnership with Roche, of a pivotal study of SRP-9001-301 (rAAVrh74.MHCK7.micro-dystrophin) also known as EMBARK.  Simultaneously, the company announced plans to host a community webinar with PPMD…

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October 4, 2021

Sarepta Therapeutics in partnership with Roche announced the initiation of the EMBARK clinical trial for their investigational gene therapy product SRP-9001

Sarepta Therapeutics, in partnership with Roche, has announced the initiation of study SRP-9001-301, also known as EMBARK, a pivotal study of SRP-9001 (rAAVrh74.MHCK7. micro-dystrophin) for the treatment of Duchenne muscular dystrophy. It is important to…

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