FDA Approves Sarepta to Begin New ELEVIDYS Gene Therapy Study for Non-Ambulant Duchenne Patients
Sarepta Therapeutics Inc. has received FDA approval to begin dosing in Cohort 8 of the ENDEAVOR study, evaluating an enhanced immunosuppressive regimen for ELEVIDYS gene therapy in non-ambulant Duchenne muscular dystrophy patients. The study aims to reduce the risk of liver injury associated with AAV gene therapy. Sarepta will work with the FDA to resume commercial dosing after reviewing study data. This news was generated by Public Technologies using AI and is for informational purposes only.
Sarepta Therapeutics Inc. has received approval from the U.S. Food and Drug Administration (FDA) to begin dosing in Cohort 8 of the ENDEAVOR study (Study 9001-103). This cohort will evaluate an enhanced immunosuppressive regimen, including sirolimus, as part of ELEVIDYS gene therapy for non-ambulant individuals with Duchenne muscular dystrophy. The study aims to mitigate the risk of acute liver injury and failure associated with AAV gene therapy. Sarepta will collaborate with the FDA to determine the resumption of commercial dosing for this patient population after reviewing study data. Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Sarepta Therapeutics Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20251125916203) on November 25, 2025, and is solely responsible for the information contained therein. © Copyright 2025 - Public Technologies (PUBT)
