Clinical Trial SuccessNGN-401 is poised to be best-in-class for Rett Syndrome due to its broad and durable clinical benefits seen in the Phase I/II study.
Financial StabilityNeurogene has a cash runway sufficient to fund operations into the first quarter of 2028, providing financial stability.
Regulatory AdvantagesNGN-401 was selected by the FDA for its START Pilot Program and has also received Regenerative Medicine Advance Therapy (RMAT) designation, Orphan Drug Designation (ODD), Fast Track designation, and Rare Pediatric Disease Designation (RPDD) from the FDA.