gene therapy

A Safety Update on ELEVIDYS – June 2025

We are heartbroken to learn of the loss of another member of our community following treatment with ELEVIDYS. According to Sarepta, this represents the second reported case of acute liver failure in a non-ambulatory patient…

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REGENXBIO Shares Positive Functional Data from Phase I/II AFFINITY DUCHENNE Trial of RGX-202

Today, REGENXBIO announced new data from its ongoing Phase I/II AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy being developed for individuals with Duchenne muscular dystrophy. RGX-202 is designed to deliver microdystrophin via AAV8…

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Watch: Sarepta Therapeutics – Duchenne Gene Therapy Update (Webinar Recording)

Sarepta Therapeutics recently joined PPMD for a community webinar to share the latest updates on their gene therapy program. In addition to program updates, Dr. Chet Villa, cardiologist at Cincinnati Children’s Hospital, and Dr. Craig…

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REGENXBIO Shares Positive Biomarker Data from AFFINITY DUCHENNE Trial of RGX-202 Gene Therapy

REGENXBIO Inc. has reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy product delivering a micro-dystrophin transgene via AAV8…

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A Safety Update on ELEVIDYS

We are deeply saddened by the loss of a young member of our community following treatment with ELEVIDYS. According to Sarepta, the individual with Duchenne received ELEVIDYS and later suffered from complications of acute liver…

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February 28, 2025

WATCH: Solid Biosciences – Update on Gene Therapy Candidate SGT-003 (Webinar Recording)

Members of the Solid Biosciences team recently joined PPMD for a community webinar to review initial positive data from the INSPIRE DUCHENNE study of SGT-003, a next-generation microdystrophin gene therapy candidate. The Solid team provided…

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February 18, 2025

Solid Biosciences Shares Initial Data from INSPIRE DUCHENNE Study of Gene Therapy Candidate SGT-003

Solid Biosciences Inc. has announced positive initial data from the company’s Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their…

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January 27, 2025

Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYS

Sarepta Therapeutics, Inc. has announced positive topline results from Part 2 of EMBARK, the company’s Phase 3 clinical study of ELEVIDYS. ELEVIDYS is a micro-dystrophin gene therapy used for the treatment of individuals aged four…

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Pfizer Discontinues Development of Investigational Mini-Dystrophin Gene Therapy

PPMD is disappointed to learn that Pfizer Inc. has officially discontinued development of the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec. This announcement follows Pfizer’s June 2024 update on the Phase 3 CIFFREO study evaluating…

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Pfizer Shares Update on Phase 3 Study of Investigational Mini-Dystrophin Gene Therapy

PPMD is deeply disappointed to share that Pfizer Inc. announced today that CIFFREO, a Phase 3 randomized, double-blind, placebo-controlled study evaluating the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne did…

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