Neurogene's NGN-401 gene therapy well tolerated in first three Rett syndrome patients; no overexpression toxicity

Neurogene Inc.

All three pediatric patients (ages 4-7) treated with single ICV dose show mild, transient AEs; no serious adverse events.
No signs of MeCP2 overexpression toxicity in any patient, including one with a mild variant predicted to retain residual MeCP2 expression.
Follow-up ranges from ~3 to ~9 months; data cut-off April 19, 2024.
Interim efficacy data from Cohort 1 expected in Q4 2024; additional data including Cohort 2 in H2 2025.
NGN-401 uses EXACT transgene regulation technology to limit overexpression risk.

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