Sarepta Therapeutics, Inc. has announced screening and enrollment are underway in Cohort 8 of the company’s ENDEAVOR study. ENDEAVOR is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple cohorts of individuals living with Duchenne, and Cohort 8 aims to assess prophylactic sirolimus treatment as part of an enhanced safety protocol during treatment with ELEVIDYS in non-ambulant individuals.
Initial clinical trial sites are now open and the cohort is enrolling approximately 25 participants in the U.S. who are non-ambulatory. This update comes after Sarepta shared in November 2025 that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8. Earlier that month, the FDA approved a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients treated with the product.
According to Sarepta, data from Cohort 8 will be used to determine whether administering sirolimus prior to and after ELEVIDYS infusion can help reduce acute liver injury (ALI), a known risk associated with AAV gene therapy. The immunosuppression regimen will include 14 days of peri-infusion sirolimus dosing (prior to ELEVIDYS administration) and will continue for 12 weeks after ELEVIDYS administration. Primary endpoints will evaluate safety–measured by incidence of ALI–and efficacy–measured by ELEVIDYS-dystrophin expression at 12 weeks.
PPMD is encouraged to see continued progress in efforts to deepen our understanding of how to minimize risk when administering gene therapy to individuals with more advanced disease following alignment with the FDA on an enhanced safety approach. We are hopeful that these efforts will help advance therapeutic options and improve safety for our community. We look forward to future updates as Cohort 8 of the ENDEAVOR study progresses and will share additional information with families as soon as it becomes available.
Read Sarepta’s press release here.
The post Sarepta Announces Initiation of Screening and Enrollment for ENDEAVOR Cohort 8 in Non-Ambulatory Individuals Living with Duchenne appeared first on Parent Project Muscular Dystrophy.