Sarepta Therapeutics announced screening and enrollment are underway in Cohort 8 of the ENDEAVOR study, evaluating the use of an enhanced immunosuppressive regimen as part of treatment with gene therapy Elevidys for non-ambulant individuals with Duchenne muscular dystrophy.
The cohort, which got the greenlight from the FDA in November 2025, will assess a prophylactic sirolimus treatment as part of an enhanced safety protocol during treatment with Elevidys.
Data from Cohort 8 will be used to determine whether administering sirolimus prior to and after Elevidys infusion can help reduce acute liver injury, a known risk associated with AAV gene therapy. The cohort is enrolling approximately 25 participants in the U.S. who are non-ambulatory.
Following the deaths of two non-ambulatory patients last year, the non-ambulatory indication was removed from the indication and usage section of the therapy’s prescribing information. A boxed warning indicating the risk of ALI and acute liver failure was added to the therapy’s label.
Sarepta aims to complete primary endpoint data collection for Cohort 8 in the second half of 2026.
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