Sarepta shares updates with the Duchenne community, including decision to close the ENVISION gene therapy clinical trial

ENVISION Gene Therapy

Sarepta Therapeutics, which received early funding from CureDuchenne, has provided a community letter outlining the status of ongoing research in their gene therapy and exon-skipping programs.

Of particular note is the decision of Sarepta to close the ENVISION gene therapy trial evaluating Elevidys in non-ambulatory and late-ambulatory individuals with Duchenne.  This trial has been on hold for over a year, as Sarepta has been evaluating the use of sirolimus with Elevidys to potentially improve safety outcomes in non-ambulatory individuals.  Sarepta determined that restarting the study after so much time and with required changes to the protocol would not be feasible.  Instead, Sarepta will close the study and analyze the blinded data from Part 1.  Important details about next steps for the participants in the ENVISION study are contained in the letter,

Letter to the community: