Neurogene completes dosing in NGN-401 Rett trial; no HLH, topline expected 2H 2027

Neurogene Inc.

Dosed 25 participants in Embolden trial (target 20, ITT up to 24) due to strong demand from Rett community.
NGN-401 generally well-tolerated; no HLH cases at 1E15 vg dose across Phase 1/2 and Embolden (n=35).
Topline data from registrational trial expected in 2H 2027; BLA submission planned.
Interim Phase 1/2 data with ≥12-month follow-up for all 10 participants expected mid-2026.

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