Neurogene reports ~$200M cash; DSMB clears third patient in Rett syndrome trial

Neurogene Inc.

DSMB cleared third pediatric patient for dosing in NGN-401 Phase 1/2 for Rett syndrome; dosing expected early Q1 2024.
UK MHRA approved CTA for NGN-401, enabling trial expansion beyond US.
Ended 2023 with ~$200M cash; cash runway into 2H 2026.
Plan to report interim Phase 1/2 data for NGN-401 in 4Q 2024 and NGN-101 in 2H 2024; FDA discussions for NGN-101 in 2H 2024.
Extended research collaboration with University of Edinburgh by three years; plan to advance one discovery product into clinic in 2025.

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