Neurogene Inc. delivered an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting on May 16, 2025, detailing an evidence-based monitoring and treatment algorithm. This protocol is designed to reverse hemophagocytic lymphohistiocytosis (HLH), a rare and severe hyperinflammatory syndrome associated with high-dose adeno-associated virus (AAV) gene therapy.
The company highlighted that early monitoring and prompt treatment have proven effective in reversing the course of HLH in higher-dose AAV gene therapy settings. Neurogene has incorporated this comprehensive monitoring and treatment protocol into its Phase 1/2 clinical trial of NGN-401 for Rett syndrome.
Neurogene emphasized that its NGN-401 1E15 vg dose level, which translates into the E13 vg/kg range, has not been associated with any reported cases of HLH. The presentation aims to encourage the adoption of this early monitoring and treatment algorithm across the gene therapy community, fostering a safer development environment.
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