Neurogene's ngn-401 Gene Therapy for Rett Syndrome Accepted into FDA START Program

Neurogene Inc. (NASDAQ: NGNE) has announced that its ngn-401 gene therapy for Rett syndrome has been selected to participate in the U.S. Food and Drug Administration (FDA) Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. This program aims to accelerate the development of rare disease therapies through enhanced communications with the FDA.

The ngn-401 gene therapy is being evaluated in a phase 1/2 clinical trial, with two dose levels being studied. The trial is assessing the safety, tolerability, and preliminary efficacy of a one-time intracerebroventricular (ICV) administration of ngn-401 in female pediatric patients with Rett syndrome. Neurogene recently presented favorable safety data from the first three patients dosed with ngn-401 and is on track to report interim efficacy data from the trial in the fourth quarter of 2024.

Neurogene's CEO, Rachel Mcminn, Ph.D., expressed gratitude for the FDA's commitment to investing significant agency resources to accelerate the development of ngn-401 and highlighted the potential clinical benefits of the gene therapy as one of the factors evaluated by the FDA in its selection.

The START program, designed to facilitate the development of novel drug and biological product candidates for rare diseases, requires clinical, chemistry, manufacturing, and controls (CMC), and non-clinical development plans. Selected sponsors are expected to receive more frequent advice from FDA staff to facilitate program development and generate high-quality and reliable data to support potential future marketing applications.

The ngn-401 gene therapy, an investigational AAV9 gene therapy, aims to deliver the full-length human MECP2 gene under the control of Neurogene’s EXACT technology. This technology is a significant advancement in gene therapy for Rett syndrome, addressing the need for a treatment approach that enables targeted levels of MECP2 transgene expression without causing overexpression-related toxic effects associated with conventional gene therapy.

Neurogene's mission is to develop novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. The company's proprietary EXACT transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy.

The company has also constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas, where cGMP production of ngn-401 was conducted and is expected to support pivotal clinical development activities. As a result of these announcements, the company's shares have moved 1.4% on the market, and are now trading at a price of $38.13. For the full picture, make sure to review Neurogene's 8-K report.

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