gene therapy

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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Sarepta Announces Positive 12-Week Expression and Safety Results from Study SRP-9001-103 (ENDEAVOR)

Sarepta Therapeutics this morning announced positive 12-week expression and safety results from the first 11 participants enrolled in Study SRP-9001-103, an open-label study known as ENDEAVOR being conducted in partnership with Roche. SRP-9001 demonstrated robust…

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Solid Provides IGNITE DMD Phase I/II Clinical Trial and Pipeline Update

Solid Biosciences has shared an update with the community this morning, including a dosing update in the IGNITE DMD Phase I/II clinical trial, promising long-term biopsy data from prior patients dosed with SGT-001 in the…

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Pfizer Shares Update on CIFFREO Phase 3 Gene Therapy Trial

Today Pfizer shared an update regarding their Phase 3 gene therapy trial, CIFFREO, with the community. While Pfizer has not yet opened sites within the U.S., the study is underway at 14 trial sites across…

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Webinar Q&A: Gene Therapy – Turning Obstacles into Possibilities

Last week, PPMD held a webinar to discuss the lessons learned in Gene Therapy, current challenges for our community, and what the future holds for these promising treatments. After opening with an insightful presentation from…

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PPMD Awards $31,500 Grant to Understand Immune Response to CRISPR/Cas-9 Gene Editing Strategies

PPMD is excited to award the University of Missouri School of Medicine a bridge grant for $31,500 to continue evaluating CRISPR therapy in a pre-clinical model of Duchenne. The project is led by Dongsheng Duan,…

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WATCH: Solid Biosciences Presents Safety & Efficacy Update (Webinar Recording)

On March 16, 2021, Solid Biosciences joined PPMD for a webinar to provide a safety and efficacy update on the ongoing IGNITE DMD phase I/II clinical study of SGT-001 microdystrophin gene therapy in patients with…

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Solid Reports Efficacy & Safety Data from Ongoing IGNITE DMD Clinical Trial & Resumption of Patient Dosing

Solid Biosciences has provided a safety and efficacy update on the ongoing IGNITE DMD phase I/II clinical study of SGT-001 microdystrophin gene therapy in patients with Duchenne. Upcoming Webinar: Join PPMD and Solid tomorrow, March…

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PPMD Awards University of Florida Wellstone Center $348,891 Grant for Duchenne Gene Therapy Research

PPMD is pleased to award the University of Florida (UF) Wellstone Muscular Dystrophy Cooperative Center a grant for $348,891. The project titled “Understanding and Improving AAV Gene Therapy for DMD” is led by H. Lee…

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February 17, 2021

Duchenne UK and PPMD Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK are excited to announce Professor Kanneboyina Nagaraju at Binghamton, the State University of New York, as the recipient of our Joint Research Grant Call of 2020. The…

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