IW | Advancing AAV Gene Therapies: Insights from CURE Epilepsy-Funded Research

This workshop addresses the current state of AAV-based genetic therapies for epilepsy. CURE Epilepsy has invested in research focused on AAV-based genetic therapies through our various funding mechanisms, including the Rare Epilepsy Partnership Award Program.

The panel convenes experts working toward development of genetic therapies for epilepsy, with expertise spanning early development, pre-clinical testing, and clinical translation. It includes early-career investigators who discuss research related to designing and in vivo testing of AAV-based gene therapies for rare epilepsies, including Lafora Disease, SCN1B-related Dravet syndrome, and SLC6A1-related neurodevelopmental disorder. Additionally, the workshop highlights safety considerations for clinical translation.