Clinical ExpansionNeurogene has more than doubled its U.S. clinical footprint to 13 sites, positioning for rapid enrollment.
Regulatory ApprovalThe FDA formally cleared the registrational trial of NGN-401, offering earlier intervention and a broader label path.
Technological AdvantageICV delivery shows a consistent advantage across studies, achieving 10–100× greater transgene RNA expression in key brain regions compared to IT-L.