Neurogene Reports Second Quarter 2026 Financial Results and Highlights Recent Updates
Data from Phase 1/2 trial of NGN-401 for Rett syndrome showed 100% of participants improved in CGI-I score and gained ≥1 developmental milestone, with an average of 4.7 milestones per participant; no plateau and no milestone loss observed as of data cutoff date of June 16, 2026
Milestones gained in a progressive, stepwise sequence, suggesting a restart of development post-treatment
Topline data from Embolden™ registrational trial anticipated in 2H 2027
NGN-401 at the 1E15 vg dose remains generally well-tolerated (N=35) as of data cutoff date of August 10, 2026
