Neurogene’s NGN-401, a gene therapy, has obtained Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for treating Rett syndrome. This designation stems from promising clinical evidence in an ongoing trial, suggesting NGN-401’s potential to fulfill unmet medical needs in this disorder.
RMAT designation grants NGN-401 eligibility for an Accelerated Approval pathway under the 21st Century Cures Act. It also aligns with NGN-401’s prior selection for the FDA’s START Pilot Program, recognizing its therapeutic potential for Rett syndrome.
Neurogene’s CEO, Rachel McMinn, commends the FDA’s support in expediting NGN-401’s development through these initiatives. The company anticipates sharing interim efficacy data from the low-dose cohort later this year, followed by additional data from the high-dose cohort in 2025.
NGN-401 is an AAV9 gene therapy designed as a single-administration treatment for Rett syndrome. It utilizes Neurogene’s EXACT transgene regulation technology to deliver the full-length human MECP2 gene without causing overexpression-related toxic effects. This advancement addresses the need for a controlled treatment approach in Rett syndrome.
Apart from RMAT designation, NGN-401 had been previously selected for the FDA’s START Pilot Program due to its favorable clinical potential and development readiness. This program facilitates frequent interaction between the sponsor and FDA staff to support program development and ensure high-quality data for future marketing applications.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
