NGN-401 has been generally well-tolerated by three patients with multiple months of follow-up

NEW YORK, NY, USA I April 22, 2024 I Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that initial safety and tolerability data from its ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome will be presented at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting. The data show that the NGN-401 gene therapy candidate has been generally well-tolerated, and there have been no treatment-emergent or procedure-related serious adverse events or signs of MeCP2 overexpression-related toxicity observed in any patient, including a patient with a mild variant predicted to result in residual MeCP2 function.

“We look forward to sharing the initial, favorable safety data of NGN-401, which is designed to use our differentiated EXACT transgene regulation technology and deliver a full-length MECP2 gene to key areas of the brain underlying the pathophysiology of Rett syndrome,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “Rett syndrome is a complex neurological disorder with a narrow therapeutic window, and conventional gene therapy approaches have been unable to provide therapeutic protein levels without detrimental overexpression. Therefore, we believe it is important to share this safety update at the ASGCT Meeting, in advance of our expected fourth quarter 2024 interim efficacy read-out, as we have multiple months of data from three patients showing NGN-401 has been generally well-tolerated.”

Neurogene’s ASGCT Meeting Poster Presentation Details

  • Title: Preliminary Safety Results from the Ph1/2 study of NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome
  • Date: May 9, 2024
  • Time: 12:00 p.m. ET
  • Location: Exhibit Hall

The abstract can be accessed at this link.

About Neurogene

The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit

SOURCE: Neurogene