Neurogene to Present Interim Phase 1/2 Data on NGN-401 for Rett Syndrome

Neurogene Inc., a company specializing in developing genetic medicines for rare neurological disorders, has announced an upcoming webcast to present interim efficacy data from its ongoing Phase 1/2 clinical trial of NGN-401 gene therapy for children with Rett syndrome. The webcast is scheduled for November 11, 2024, at 4:30 p.m. ET. During this event, Neurogene will share safety data from both the low- and high-dose cohorts involved in the trial.

The following day, on November 12, additional data will be shared in a late-breaking poster presentation at the 53rd Annual Child Neurology Society (CNS) Annual Meeting in San Diego, California. This presentation will take place at 12:30 p.m. PT.

The webcast, including a live conference call, will be accessible through the Investor Relations section of the Neurogene website under Events. A replay of the webcast will also be available for a limited period.

The poster presented at the CNS meeting is titled "NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome: Preliminary Results from the First-in-Human Study." It will be presented by Dr. Bernhard Suter, who is the Medical Director of the Blue Bird Circle Rett Center at Texas Children’s Hospital, an Associate Professor of Pediatrics and Neurology at Baylor College of Medicine, and the principal investigator in the NGN-401 clinical trial.

Neurogene's mission is to address severe neurological diseases, aiming to improve the lives of patients and their families. The company's innovative strategies in gene therapy seek to overcome the limitations of traditional methods used for treating central nervous system disorders. This includes optimizing delivery methods to ensure maximum distribution to target tissues and designing products for enhanced efficacy and safety. Neurogene's proprietary EXACT transgene regulation platform technology is a significant advancement, enabling the delivery of therapeutic levels while minimizing the toxicity often associated with conventional gene therapies.

In support of its clinical development activities, Neurogene has established a leading-edge gene therapy manufacturing facility in Houston, Texas. This facility was utilized for the CGMP production of NGN-401, which is intended to support crucial clinical development phases.