Positive Initial Safety Results for NGN-401 Gene Therapy in Rett Syndrome Phase 1/2 Trial

Neurogene Inc., a company dedicated to developing genetic medicines for rare neurological disorders, has reported positive initial findings from its Phase 1/2 clinical trial of a gene therapy for Rett syndrome. The therapy, named NGN-401, has shown good tolerability in three pediatric patients who have been monitored for three to nine months post-treatment. Notably, there were no indications of overexpression toxicity, even in a patient with a mild genetic variant expected to allow some MeCP2 protein to remain.

The study, which is still enrolling female patients aged 4-10 with classic Rett Syndrome, involves a one-time infusion of NGN-401 at varying doses. The first cohort received a lower dose, and the second cohort is set to receive a higher dose. The patients' baseline demographics varied in terms of age, race, and severity of their MECP2 mutation, but all have shown a similar response to the treatment.

Rachel McMinn, Neurogene's CEO, highlighted the incorporation of the company's EXACT™ transgene regulation technology into NGN-401, which is designed to deliver therapeutic protein levels to critical areas of the brain and nervous system without causing toxicity. The interim safety data presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting indicate that NGN-401 has been well-tolerated, with all adverse events being mild and transient.

Bernhard Suter, MD, principal investigator of the NGN-401 clinical trial and Medical Director of the Blue Bird Circle Rett Center at Texas Children’s Hospital, emphasized the significant unmet need for treatments that can alleviate the daily burdens faced by individuals with Rett syndrome and their families. He expressed optimism about the potential of gene therapy to provide a one-time treatment addressing the root cause of the disease.

Neurogene anticipates releasing interim clinical data, including efficacy results, from the first cohort in the fourth quarter of 2024, with additional data from the second cohort expected in the latter half of 2025. The company's mission is to improve the lives of patients and families affected by rare neurological diseases through innovative approaches and treatments that overcome the limitations of traditional gene therapy.

Neurogene's EXACT transgene regulation platform is a key component of their strategy, enabling the delivery of therapeutic levels of medication while minimizing the risk of transgene toxicity. The company has also established a cutting-edge gene therapy manufacturing facility in Houston, Texas, which will support the pivotal clinical development of NGN-401.

The company's commitment to advancing the field of gene therapy for neurological disorders is evident in their ongoing clinical trials and the development of their proprietary technologies. As they continue to work towards providing life-changing treatments, the initial safety and tolerability data from the NGN-401 trial represent a promising step forward in the quest to treat Rett syndrome and other rare neurological conditions.