The oral presentation, titled 'Hemophagocytic Lymphohistiocytosis (HLH)/Hyperinflammatory Syndrome Following High Dose AAV9 Therapy,' is scheduled for May 16 at 3:00 p.m. CT in NOLA Theater C.

In their Phase 1/2 clinical trial of NGN-401 gene therapy for Rett syndrome, Neurogene is utilizing a dose that has not been associated with HLH cases, specifically at the 1E15 vg level, translating to the E13 vg/kg range.

In addition to the main presentation, Dr. McMinn will participate in two other sessions at the conference, including a fireside chat on May 14 and a discussion on the FDA's START Pilot Program on May 15.

Neurogene's mission is to enhance the lives of patients suffering from rare neurological diseases through innovative gene therapies, with a strong emphasis on optimizing both efficacy and safety.

Neurogene Inc. is set to present an oral session at the upcoming ASGCT Annual Meeting, taking place from May 13-17, 2025, in New Orleans, where they will focus on hemophagocytic lymphohistiocytosis (HLH) related to high-dose gene therapy.

The company employs a proprietary technology known as the EXACT™ transgene regulation platform, which is designed to enhance therapeutic delivery while minimizing toxicity.

Neurogene operates a state-of-the-art gene therapy manufacturing facility in Houston, Texas, where they produce NGN-401 under CGMP conditions to support their clinical development activities.

During the presentation, Neurogene will share successful strategies for monitoring and treating HLH, a rare syndrome associated with high doses of adeno-associated virus (AAV) gene therapy.

Rachel McMinn, Ph.D., CEO of Neurogene, highlighted the necessity for effective monitoring protocols due to the limited existing research on HLH in the context of gene therapy.