Sarepta begins enrollment for enhanced safety protocol in gene therapy study
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Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duch
March 16, 2026 8:33 AM EDT- Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study
- The enhanced immunosuppressive regimen is designed to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF) associated with AAV gene therapy in non-ambulatory patients
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced screening and enrollment are underway in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to assess... More
