gene therapy

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 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 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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August 17, 2021

Solid provides updates on IGNITE DMD and SGT-003, next generation gene therapy pipeline initiative

Solid Biosciences has shared an update with the community on dosing as part of the IGNITE DMD Phase I/II clinical trial, developments for next generation gene therapy pipeline initiative SGT-003 and their collaboration with Ultragenyx….

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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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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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