February 10, 2021

Gene Therapy and Immune Response: Understanding Challenges and Strategies

PPMD recently announced the formation of the Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group to address the challenges involved in gene therapy. As part of their recent educational series, the PGTME held a…

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January 21, 2021

WATCH: PGTME Hosts Gene Therapy Lecture Series

As the Duchenne community enters the era of gene therapy, 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…

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January 21, 2021

Postponed: SRP-9001 Update – Community Webinar

Postponed: SRP-9001 Update UPDATE: Sarepta will have to postpone the community webinar scheduled for today, January 21 at 12 PM EST. PPMD is deeply disappointed as we were hopeful to discuss current data and ask…

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January 12, 2021

WATCH: PGTME Hosts Gene Therapy Lecture Series

As the Duchenne community enters the era of gene therapy, 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…

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January 7, 2021

Sarepta Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001 Gene Therapy

Sarepta Therapeutics today announced top-line results from Part 1 of Study SRP-9001-102 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)…

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January 7, 2021

Pfizer Doses First Participant in Phase 3 Study for Duchenne Investigational Gene Therapy

More exciting news for our community! Pfizer today announced that the first participant has been dosed in the Phase 3 CIFFREO study, which will evaluate the efficacy and safety of their investigational AAV9 mini-dystrophin gene…

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January 5, 2021

REGENXBIO Announces New Gene Therapy Program for Duchenne Muscular Dystrophy

PPMD is incredibly excited to share REGENXBIO’s announcement of their new program for Duchenne muscular dystrophy. REGENXBIO is seeking to treat Duchenne through a gene therapy approach delivering a novel microdystrophin transgene. Their gene therapy…

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December 18, 2020

Sarepta Community Bulletin: COVID-19 Vaccination and Gene Therapy

Sarepta Therapeutics has shared a community bulletin addressing questions about COVID-19 vaccination and gene therapy. This update confirms information presented during PPMD’s recent webinar, which will likely be consistent across all clinical trials. For more…

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December 3, 2020

WATCH: SRP-9001 Gene Therapy Path Forward (Webinar Recording)

Parent Project Muscular Dystrophy and Sarepta Therapeutics hosted a webinar on November 24, 2020 for a discussion about the SRP-9001 gene therapy development program. If you missed the live event, the recording can be found…

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November 6, 2020

Sarepta to Commence Dosing of Next Study with Commercial Process Material for SRP-9001 Gene Therapy

We are encouraged by the result of Sarepta’s Type C meeting with the FDA. Type C meetings are a “catch all” category and include any topics concerning the development and review of a product. This…

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