10:43 AM EDT, 06/18/2024 (MT Newswires) -- Neurogene ( NGNE ) said Tuesday that the first patient in cohort 2 received high-dose NGN-401 gene therapy in a phase 1/2 trial for female pediatric patients with the neurodevelopmental disorder Rett syndrome.

The company said that high-dose NGN-401 has been well-tolerated following dosing in May. NGN-401 is an investigational adeno-associated virus, or AAV, 9 gene therapy being developed as a one-time treatment for Rett syndrome.

The company also said that updated interim data on the first three patients in the low-dose group show that NGN-401 has continued to have a favorable safety profile with no new treatment-related adverse events.

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