Expanded Access use of Intravenous Gene Transfer with AAV9-GLB1 in a Juvenile GM1 Gangliosidosis Participant
Connor J. Lewis, Hera Akmal, Precilla D’Souza, Jean M. Johnston, Sumaiya Ashraf, Gilbert Vezina, Selby I. Chipman, Meghan Blackwood, Muhammad H. Yousef, Zenaide Quezado, William Allen Gahl, Barry J. Byrne, Terence R. Flotte, Xuntian Jiang, Audrey Thurm, , Cynthia J. Tifft
This study reports 5-year safety and 3-year clinical outcomes of AAV9-GLB1 gene therapy in a juvenile GM1 gangliosidosis patient, showing sustained seizure freedom and biochemical improvements.
medRxiv · Sep 10, 2026 · PreprintGenetic & Genomic Medicine