Neurogene Expands Rett Syndrome Gene Therapy Trial for Swifter Patient Enrollment

Neurogene Inc., a company specializing in genetic medicines for rare neurological disorders, is advancing its Phase 1/2 clinical trial for NGN-401, a gene therapy for Rett syndrome in female pediatric patients. The trial will now include more participants in Cohort 1 and introduce a new high-dose group, aiming to accelerate patient enrollment and provide a more comprehensive data set for future trial planning.

The company has successfully dosed a third patient in Cohort 1 and anticipates completing dosing for this group by the end of 2024. The clinical protocol has been revised to expand Cohort 1 from five to eight patients and to eliminate the staggered dosing approach. Additionally, a new Cohort 2 has been introduced, also consisting of eight patients, with the first three to be dosed sequentially, pending safety reviews.

Neurogene's EXACT gene regulation platform, featured in NGN-401, is designed to control transgene expression levels, potentially allowing for higher doses without increasing the risk of overexpression-related toxicities. The company's manufacturing facility in Houston, Texas, has produced GMP-quality NGN-401 to support pivotal clinical development.

The trial's third patient has shown good tolerance for NGN-401, with no serious adverse events or signs of toxicity reported. Neurogene anticipates releasing interim data from Cohort 1 in Q4 of 2024 and additional data from Cohort 2 in H2 of 2025.

NGN-401 is an experimental AAV9-based gene therapy developed to deliver the MECP2 gene, which is crucial for treating Rett syndrome. The company's mission is to develop innovative treatments for severe neurological diseases, focusing on maximizing efficacy and safety while minimizing the limitations of traditional gene therapy.

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