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Source document· March 16, 2026

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 Duchenne

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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 Duchenne - Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the END…
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  • Cohort 8 of ENDEAVOR is expected to build on dystrophin expression data while deepening understanding of safety profile in older patients with more advanced disease

    60% confidence
  • Individuals with non-ambulatory Duchenne face profound unmet need and fewer treatment options

    60% confidence
  • The approach for Cohort 8 is based on preclinical data and shaped by real-world clinical experience, including guidance from independent specialists in Duchenne and liver health

    60% confidence
  • Sarepta is on an urgent mission to engineer precision genetic medicine for rare diseases that devastate lives and cut futures short

    60% confidence
  • In non-ambulatory patients treated with ELEVIDYS, acute liver failure with fatal outcome has occurred in the clinical and post-marketing settings

    60% confidence
  • The enhanced immunosuppressive regimen with sirolimus is designed to mitigate the risk of acute liver injury and acute liver failure associated with AAV gene therapy in non-ambulatory patients

    60% confidence
  • Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS, particularly in non-ambulatory patients

    60% confidence
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Q4 2026 Biopharma Catalyst Cluster: Late-Stage Readouts and Regulatory Milestones
October 2026 brings a dense run of Phase 3 wins (ALOFT-IPF, RESTORE-3), new data presentations (Roche, Bayer, Lilly, Vertex, Galderma) and regulatory steps (FDA acceptance of Roche's vamikibart BLA, Scholar Rock's EMA resubmission). The next big catalyst is the ivonescimab FDA PDUFA date on 2026-11-14. Obesity combination studies (REDEFINE 4, TOGETHER-PsO) and the 2027 Maritime-1 completion point to more catalysts ahead. QAIAx's speculative AI-linked trial claims (the QIII pilot, targeted for 2027) are an unrelated micro-cap sideshow with weak evidence, and have little in common with the large-cap pipeline news.
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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 Duchenne — Source | Via News | ViaNews EU