A participant has died in the high-dose cohort of Neurogene’s phase 1/2 clinical trial evaluating its investigational AAV9 gene therapy as a one-time treatment for Rett syndrome.
Earlier this week, Neurogene announced that it had halted the high-dose cohort of the trial and that the patient in critical condition. The patient, who had received NGN-401 at a dose of 3E15 vg on November 5, has 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.
Neurogene provided the update in an SEC filing. The company also said that the FDA has given Neurogene the go-ahead to proceed with the phase 1/2 trial using the lower dose — 1E15 vg — for both the pediatric and adolescent/adult cohorts. Neurogene will also incorporate the 1E15 vg dose in its future registrational trial design planning.
Subscribe to our e-Newsletters
Stay up to date with news, articles and insights relevant to cell and gene therapy development and manufacturing. Plus, get special offers from Cell & Gene Therapy Review delivered right to your inbox!
Sign up now!