Neurogene Inc. announced on April 28, 2025, that it will deliver an oral presentation at the American Society for Gene and Cell Therapy (ASGCT) 28th Annual Meeting. The presentation will focus on approaches for monitoring and managing hemophagocytic lymphohistiocytosis (HLH), a rare hyperinflammatory syndrome linked to high doses of adeno-associated virus (AAV) gene therapy.
The company emphasized the importance of providing information to the AAV gene therapy community on effective strategies to monitor, treat, and reverse cases of HLH, given the limited published research on this complication. Neurogene has already incorporated early monitoring and a treatment plan into its NGN-401 Phase 1/2 trial protocol for Rett syndrome.
Neurogene noted that the 1E15 vg dose level of NGN-401, which translates into the E13 vg/kg range, has not been associated with any reported cases of HLH. This proactive communication aims to share best practices and potentially mitigate risks across the gene therapy field.
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