MTE #24: Gene Therapies and Hepatotoxicity (Ticketed)

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). Many other gene therapies are in the clinical trial phase of development. Join expert faculty to discuss this cutting-edge therapeutic modality. Speakers explore different mechanisms of delivery of gene therapy, including adeno-associated virus (AAV) vector delivery—currently the predominant delivery mechanism in approved gene therapeutics and those in clinical trials, although this may change in the future—as well as ex vivo gene editing, and lipid nanoparticles. Presenters highlight clinical challenges related to the potential for serious toxicity associated with gene therapy, particularly hepatotoxicity that may cause acute liver failure in a small number of patients.