Neurogene (NGNE) Provides Update on NGN-401 Gene Therapy Trial

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Go back to Neurogene (NGNE) Provides Update on NGN-401 Gene Therapy Trial

Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome

November 18, 2024 6:45 AM EST

NEW YORK--(BUSINESS WIRE)-- 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 an update on its ongoing Phase 1/2 open-label clinical trial evaluating NGN-401 gene therapy for the treatment of Rett syndrome.

As previously disclosed, on November 11, 2024, Neurogene became aware of an emerging treatment-related serious adverse event (SAE) in a trial participant who received NGN-401 at a dose of 3E15 vg (high-dose cohort). This participant, who was dosed on November 5, subsequently experienced signs of a systemic hyperinflammatory... More