January 24, 2023

REGENXBIO Announces RGX-202 Gene Therapy Trial is Active & Recruiting

REGENXBIO has announced that the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202, an investigational gene therapy product for the potential treatment of Duchenne, is now active and recruiting trial participants. RGX-202 utilizes a novel adeno-associated virus (AAV8)…

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December 5, 2022

Solid Biosciences Announces Completion of AavantiBio Acquisition & Update on Duchenne Pipeline Program

Today Solid Biosciences shared an update with the Duchenne community regarding their strategic development. Solid announced they have completed the acquisition of AavantiBio, a gene therapy company. The combined company will focus on advancing a…

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November 28, 2022

Sarepta Announces Biologics License Application (BLA) Accepted For Filing by the FDA for SRP-9001

PPMD is thrilled to learn that the FDA has accepted for filing and granted priority review for the Biologics License Application (BLA) for Sarepta’s gene therapy product, SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant…

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November 18, 2022

Pfizer Announces First Participant Dosed in DAYLIGHT Phase 2 Gene Therapy Trial

Pfizer has announced that the first participant has been dosed in the Phase 2 DAYLIGHT study, which will evaluate the safety and tolerability of the company’s investigational mini-dystrophin gene therapy product (fordadistrogene movaparvovec) in very young…

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October 26, 2022

Duchenne UK and PPMD Announce 2022 Joint Call for Therapeutic Projects

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK, a UK-based patient organization, are pleased to announce the launch of their 2022 Joint Call for Therapeutic Projects inviting research proposals for submission and review. Up to $1…

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September 30, 2022

Solid Biosciences Acquires AavantiBio & Shares Update on Duchenne Gene Therapy Pipeline Programs

Today Solid Biosciences shared an update with the Duchenne community regarding their strategic development. Solid announced they have acquired AavantiBio, a gene therapy company with a focus on rare cardiomyopathies and Friedeich’s ataxia. While this…

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September 29, 2022

Sarepta Therapeutics Submits Biologics License Application (BLA) for SRP-9001

Sarepta Therapeutics today announced it has submitted a Biologics License Application (BLA) to the FDA for the accelerated approval of SRP-9001 to treat ambulant patients with Duchenne. SRP-9001 is an investigational gene transfer therapy intended to deliver…

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September 14, 2022

WATCH: SRP-9001 Data Update from Studies 101, 102, and 103 (August 2022)

On August 24, 2022, PPMD was joined by Sarepta Therapeutics for a community webinar to present a SRP-9001 gene therapy data update from Studies 101, 102, and 103, following their recent presentation at a medical…

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September 1, 2022

Pfizer Shares Update on DAYLIGHT Phase 2 Gene Therapy Trial

Pfizer has issued a community letter announcing that its Phase 2 DAYLIGHT clinical trial evaluating the company’s investigational mini-dystrophin gene therapy product (fordadistrogene movaparvovec) has opened a first trial site in Australia. Pfizer shares that…

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July 29, 2022

Sarepta Announces Intent to Submit an Accelerated Approval Biologics License Application for SRP-9001

Sarepta Therapeutics today announced its intention to file for FDA approval of SRP-9001 this fall using the FDA’s accelerated approval pathway. SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle…

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