Neurogene receives FDA RMAT designation for NGN-401 gene therapy in Rett syndrome

Neurogene Inc.

RMAT designation based on preliminary clinical evidence from ongoing Phase 1/2 trial showing potential to address unmet medical needs.
Designation provides Fast Track, Breakthrough Therapy benefits, and eligibility for Accelerated Approval and Priority Review.
NGN-401 previously selected for FDA START Pilot Program; interim low-dose efficacy data expected Q4 2024, high-dose data H2 2025.
CEO notes FDA commitment to expedite development; designation underscores therapeutic potential of NGN-401.

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