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 7, 2021

Dyne Therapeutics Presents New Preclinical Data on the Potential of FORCE Platform to Address the Genetic Basis of Duchenne

Dyne Therapeutics presents new preclinical data on the potential of FORCE platform to address the genetic basis of Duchenne. Dyne Therapeutics recently released new preclinical data from their Duchenne muscular dystrophy program, which was initially…

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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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October 1, 2021

Duchenne Gene Therapy Update

As many in the community are aware, on Monday, September 28, Pfizer released a statement to the public announcing a change in their Phase III gene therapy trial for Duchenne. We applaud their efforts to…

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September 29, 2021

Wave Life Sciences Announces Initiation of Dosing in Phase 1b/2a Duchenne Clinical Trial of WVE-N531 Starting in Canada and the UK

  This morning, Wave Life Sciences Ltd. announced the initiation of dosing in a Phase 1b/2a clinical trial expecting to enroll 15 boys, evaluating investigational WVE-N531 as a treatment for boys with Duchenne muscular dystrophy…

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September 28, 2021

Pfizer Announces Amendment to Ongoing Gene Therapy Phase III Trial

Today Pfizer announced that they have submitted a protocol amendment to their ongoing Phase III trial of their gene therapy candidate (fordadistrogene movaparvovec).  Across Pfizer’s studies, three Severe Adverse Events (SAE) have occurred leading Pfizer…

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September 27, 2021

ReveraGen and Santhera Announce FDA Orphan Grant Funding for Clinical Trial with Vamorolone in Becker Muscular Dystrophy

PPMD is pleased to share that ReveraGen Biopharma and Santhera Pharmaceuticals announced that ReveraGen has received a $1.2 million grant from the FDA under their “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare…

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September 27, 2021

Sarepta Therapeutics Plans to Initiate Part B of the MOMENTUM Study of SRP-5051 in Patients with Duchenne Following Positive Interactions with FDA

PPMD is pleased to share that Sarepta Therapeutics plans to initiate Part B of the MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy following positive interactions with the U.S. Food and Drug Administration…

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September 24, 2021

Capricor Therapeutics Announces Positive Final Data From its Phase II HOPE-2 Trial in Patients with Duchenne Muscular Dystrophy Treated with CAP-1002

PPMD is pleased to share that Capricor Therapeutics announced today positive final data from the HOPE-2 randomized, double-blind, placebo controlled clinical trial using CAP-1002 to treat patients with Duchenne. The HOPE-2 clinical trial met its…

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