Neurogene's NGN-401 Rett syndrome gene therapy selected for FDA START Pilot Program

Neurogene Inc.

NGN-401 one of only three CBER programs chosen for FDA's START Pilot Program.
START provides enhanced communications with FDA to accelerate development of rare disease therapies.
Phase 1/2 trial of NGN-401 ongoing; favorable safety data from first three patients reported.
Interim efficacy data from the trial expected in Q4 2024.
Company aims to advance NGN-401 toward potential registrational study.

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