welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!

Sarepta Therapeutics to Commence Dosing of the Next Study With Commercial Process Material for the SRP-9001 Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced important updates related to the development plan for SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), the Company’s investigational gene transfer therapy for the treatment of Duchenne muscular dystrophy.

In light of the risks that the ongoing COVID-19 pandemic posed to the execution of a large, multi-center trial, Sarepta proposed a new clinical study in the September 2020 Type C meeting to support the Company’s clinical and regulatory efforts — Study SRP-9001-103 (Study 103). Study 103 is an open-label study in up to 10 patients evaluating the safety and expression of commercial process material for SRP-9001 and is intended to accelerate Sarepta’s validation of commercial process material.

Sarepta Therapeutics To Commence Dosing Of The Next Study With Commercial Process Material For The SRP-9001 Gene Therapy Program For The Treatment Of Duchenne Muscular Dystrophy