Roundtable Meet the Expert track
MTE #24: Gene Therapies and Hepatotoxicity (Ticketed)
Sun, Nov 8, 2026 · 7:00–7:45 AM MST Convention Center: Room 1C, Colorado Convention Center
About this session
Multiple gene therapies have been approved in recent years by the US Food and Drug Administration (FDA) for a variety of inherited disorders, including: Duchenne muscular dystrophy (delandistrogene moxeparvovec-rokl, 2023) Hemophilia A (valoctocogene roxaparvovec-rvox, 2023) Hemophilia B (etranacogene dezaparvovec-drlb, 2022; fidanacogene elaparvovec-dzkt, 2024) Spinal muscular atrophy (onasemnogene abeparvovec-brve, 2025) Sickle cell disease (exagamglogene autotemcel, 2023).
