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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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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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 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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July 24, 2020

Sarepta Therapeutics Receives Fast Track Designation for SRP-9001 Micro-Dystrophin Gene Therapy

PPMD was excited to learn at yesterday’s Virtual Annual Conference that Sarepta Therapeutics was granted Fast Track designation for SRP-9001 by the FDA. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding…

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July 2, 2020

Sarepta Therapeutics Signs Agreement with Hansa Biopharma for Imlifidase

PPMD is encouraged to learn that Sarepta Therapeutics announced an agreement with Hansa Biopharma, a company that has developed an IgG antibody-cleaving enzyme called imlifidase. Imlifidase is an investigational product initially developed to aid in…

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June 15, 2020

Sarepta Announces Positive Safety and Efficacy Data from SRP-9001 Gene Therapy Trial Published

Sarepta Therapeutics today announced safety and tolerability data at one year from four Duchenne clinical trial participants who received SRP-9001 micro-dystrophin (AAVrh74.MHCK7.micro-dystrophin) have been published in JAMA Neurology. PPMD is proud to have been an…

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