April 21, 2023

Pfizer Completes Enrollment of CIFFERO Phase 3 and DAYLIGHT Phase 2 Gene Therapy Trials

Pfizer announced today that enrollment is now complete for both the Phase 3 CIFFERO study and Phase 2 DAYLIGHT study of PF-06939926 (fordadistrogene movaparvovec) in Duchenne. As always, we are so grateful to all of…

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April 20, 2023

How to Submit Written Testimony for the SRP-9001 FDA Advisory Committee Meeting – Deadline May 5th

Sarepta recently announced that the FDA Advisory Committee meeting for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, will be held on May 12th. The event will be completely virtual and a…

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April 10, 2023

Sarepta Announces Date for SRP-9001 FDA Advisory Committee Meeting: Make Your Voice Heard on May 12th

Today Sarepta shared that the FDA has announced that the planned Advisory Committee meeting for SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, will be held on May 12, 2023. The event…

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April 4, 2023

Leveraging Our United Voices: A Call To Collaborative Action Ahead of FDA’s Advisory Committee for SRP-9001

In September, Sarepta Therapeutics filed a BLA (Biologics License Application) for the approval of SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, using the FDA’s accelerated approval pathway. During the marketing application’s…

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March 22, 2023

How You Can Be Involved in the First FDA Ad Comm Meeting for a Gene Therapy Product for Duchenne

Last week, Sarepta announced that the FDA Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products had informed the company that an advisory committee meeting will be scheduled prior to the PDUFA (or…

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March 16, 2023

Sarepta Therapeutics Announces Advisory Committee Meeting will be Held for SRP-9001

Sarepta Therapeutics today shared an update regarding SRP-9001 (delandistrogene moxeparvovec), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, for which a Biologics License Application (BLA) is currently under review by the U.S. Food and Drug…

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January 26, 2023

PDC Submits White Paper Supporting Accelerated Approval for AAV Gene Therapies to FDA

PPMD is proud to contribute to a multi-stakeholder collaborative effort led by the Pathway Development Consortium (PDC) in the submission of a white paper to FDA on the use of the accelerated approval pathway for…

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January 25, 2023

PPMD Invests $500,000 in Myosana Therapeutics to Advance Non-Viral Gene Therapy Platform

Parent Project Muscular Dystrophy is excited to announce a $500,000 programmatic investment in Myosana Therapeutics, Inc. (Myosana) to support the company’s continuing development and translation of a non-viral gene therapy delivery platform aiming to slow…

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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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