FDA OKs Sarepta Study of New Immune Suppression Approach with Elevidys; Gilead Acquires Preclinical TREX1 Program
The FDA has approved Sarepta Therapeutics to begin Cohort 8 of the ENDEAVOR study, which will evaluate an enhanced immunosuppressive regimen using sirolimus before and after Elevidys infusion in non-ambulatory individuals with Duchenne muscular dystrophy.
The primary goals of Cohort 8 are to assess whether the new regimen can reduce the risk of acute liver injury (ALI) and acute liver failure (ALF), both known risks associated with AAV gene therapy.
Cohort 8 will enroll approximately 25 non-ambulatory participants in the U.S., with dosing expected to start before the end of 2025 and primary endpoint data collection completed in the second half of 2026.
Decisions on resuming commercial dosing for non-ambulatory patients will be made in collaboration with the FDA after reviewing study data.
Sarepta continues to work with the FDA on additional post-marketing studies to further assess the risk of serious liver injury.
Separately, Gilead Sciences has acquired a preclinical TREX1 program, which is unrelated to Elevidys but represents ongoing investment in gene therapy research for rare diseases.