other material
confidence high
sentiment positive
materiality 0.85
Neurogene Reports Positive Initial Safety Data from Phase 1/2 Gene Therapy Trial for Rett Syndrome
Neurogene Inc.
- NGN-401 generally well-tolerated in three patients with multiple months of follow-up.
- No treatment-emergent or procedure-related serious adverse events or MeCP2 overexpression toxicity observed.
- Data presented at ASGCT Annual Meeting; includes one patient with mild variant predicted to retain residual MeCP2 function.
- Company expects interim efficacy readout in Q4 2024.