Sarepta Therapeutics receives fast track designation for SRP-9001 micro-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy

Sarepta Therapeutics

24 July 2020 - Sarepta Therapeutics today announced that the U.S. FDA has granted fast track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). 

SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. 

Safety and tolerability data at one year from four clinical trial participants who received SRP-9001 in Study 101 were recently published in JAMA Neurology, and Study 102, a randomised, double-blind, placebo-controlled study of SRP-9001, is ongoing with results expected in early 2021.

Read Sarepta Therapeutics press release

Michael Wonder

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Michael Wonder

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US , Gene therapy , Fast track