Neurogene (NGNE) announced an update on its ongoing Phase 1/2 open-label clinical trial evaluating NGN-401 gene therapy for the treatment of Rett syndrome. On November 11, Neurogene became aware of an emerging treatment-related serious adverse event, or 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 syndrome, a rare and life-threatening immune response that has been reported with systemic exposure to high doses of AAV. The participant is in critical condition, and the case is continuing to evolve. The FDA completed a review of the safety data for NGN-401 and allowed Neurogene to proceed with the Phase 1/2 trial using the 1E15 vg dose – low-dose cohort -. Neurogene paused further use of the 3E15 vg dose upon initial notification of the SAE and does not plan to enroll any further participants at the 3E15 vg dose level. To date, there have been no other treatment-related SAEs in the clinical trial. All treatment-related AEs in the low-dose cohort have been mild. There have been no signs or symptoms indicative of MeCP2 overexpression toxicity. Neurogene no longer anticipates completing enrollment in the low-dose cohort of NGN-401 in Q4 as the company updates the protocol to reflect the discontinuation of the 3E15 vg dose.
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