srp-9001

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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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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November 8, 2021

Recap: Recent Research Updates

Welcome to FALL Time flies! Over the past few months, we have seen a number of announcements regarding drug development, changes to clinical trials, and new trials opening or on the horizon. For many, it…

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October 18, 2021

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (Webinar Recording)

Last week, Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar to discuss the details of the EMBARK clinical trial (SRP-9001-301). If you missed the live event, the recording can be found below.*…

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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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WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (Webinar Recording)

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar update on June 2, 2021. Sarepta was invited to present recent updates from their ongoing clinical…

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January 21, 2021

Postponed: SRP-9001 Update – Community Webinar

Postponed: SRP-9001 Update UPDATE: Sarepta will have to postpone the community webinar scheduled for today, January 21 at 12 PM EST. PPMD is deeply disappointed as we were hopeful to discuss current data and ask…

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January 7, 2021

Sarepta Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001 Gene Therapy

Sarepta Therapeutics today announced top-line results from Part 1 of Study SRP-9001-102 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)…

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