gene therapy

November 16, 2021

Pediatric Gene Therapy and Medical Ethics (PGTME) holds 2nd Annual Lecture Series

As gene therapy continues to advance through clinical trials, many new concepts, terms and issues arise, including those related to research design, patient autonomy, sibling and family involvement, fairness in access and the quality of…

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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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November 2, 2021

Pfizer’s Phase 3 Gene Therapy Trial, CIFFREO: Community Questions Answered

Gene Therapy is on everyone’s mind these days. We wonder about opportunities, clinical sites, neutralizing antibodies and most recently details of a small number of Serious Adverse Events (SAE) and what these learnings might mean…

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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 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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