Advancing AAV Gene Therapies: Insights from CURE Epilepsy-Funded Research
Sun, Dec 6, 2026
This investigators workshop examines adeno-associated virus gene therapies for epilepsy from design and animal testing through clinical translation. Research examples concern Lafora disease, SCN1B-related Dravet syndrome and SLC6A1-related neurodevelopmental disorder. The panel considers treatment efficacy, seizure-associated adverse effects and AAV toxicity, alongside strategies for overcoming current limitations. Discussion also covers biomarkers, scalability and the evidence needed to advance candidates beyond preclinical studies. Sunday 6 December, 14:00–15:30 Mountain Standard Time (Amer