SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) developed by the Sarepta Therapeutics laboratory were announced in a press release dated January 7, 2021.
This double-blind, three-part trial evaluates the efficacy, tolerability and safety of one dose of SRP-9001 administered by infusion in 41 boys with DMD, aged 4 to 7 years, placebo controlled.
- In Part 1 now completed, 20 children received SRP-9001 and the other 21 received placebo, with a one-year follow-up.
- In part 2, which is still ongoing, the 21 children initially on placebo received, this time, SRP-9001 and the 20 others, placebo, with follow-up for one more year.
- In part 3, the trial is continuing in an open label with an additional two-year follow-up.
Other microdystrophin-encoding gene therapy products from other laboratories are being studied in Duchenne muscular dystrophy (DMD):
- Pfizer has just started a phase III clinical trial evaluating PF-0693399 with a first patient treated in Spain.
- Généthon (the AFM-Telethon laboratory) has just started the pre-inclusions of a clinical trial evaluating its own micro-dystrophin vector, in partnership with Sarepta Therapeutics.
Access Sarepta Therapeutics press release du 7 janvier 2021 : Sarepta Therapeutics Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
