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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October 23, 2020

Solid & Ultragenyx Announce Strategic Collaboration to Develop & Commercialize New Gene Therapies for Duchenne

We are pleased that Solid Biosciences has announced a strategic collaboration and license agreement with Ultragenyx Pharmaceutical. Emil Kaakis, CEO of Ultragenyx, has been focused on rare diseases across his career and has considerable experience…

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October 8, 2020

Sarepta Therapeutics Shares Fall 2020 Newsletter

Sarepta has shared the Fall 2020 issue of their newsletter, providing updates on community programs and highlighting their new class of Route 79 scholarship recipients. Click here to download.

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October 1, 2020

Pfizer Receives FDA Fast Track Designation for PF-06939926 Investigational Gene Therapy

Pfizer Inc. announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne has received Fast Track designation from the FDA. PF-06939926 is currently being evaluated to determine the safety and efficacy of…

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October 1, 2020

FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

PPMD is delighted to learn that the FDA has lifted the hold on Solid Biosciences’ IGNITE-DMD clinical trial for their gene therapy product (SGT-001) delivering a micro-dystrophin transgene via AAV9. Solid has worked diligently to…

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September 28, 2020

Sarepta Reports Sustained Functional Improvement Two Years After Treatment with SRP-9001 Gene Therapy

PPMD is excited to share today’s announcement from Sarepta Therapeutics regarding two-year follow up results from four clinical trial participants who received SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). Results presented at the Virtual 25th International Congress of the World…

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September 21, 2020

Pfizer Shares Update on PF-06939926 Gene Therapy Trial During Company’s Virtual Investor Day

Pfizer recently shared an update on their mini-dystrophin replacement gene therapy product PF-06939926 during their virtual Investor Day. In total 18 boys have received the gene therapy product as part of the Phase 1b study….

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September 10, 2020

Sarepta Therapeutics Provides Program Update for SRP-9001 Micro-Dystrophin Gene Therapy

Sarepta Therapeutics announced that it has completed a meeting (Type C) with FDA’s Office of Tissues and Advanced Therapies (OTAT), an office that is part of the Center for Biologics Evaluation and Research (CBER). The…

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August 20, 2020

Sarepta Therapeutics Shares Summer 2020 Newsletter

Sarepta has shared the Summer 2020 issue of their newsletter, including updates on current Sarepta programs. Click here to download.

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