Neurogene announced that the first patient in Cohort 2 received high-dose NGN-401 gene therapy in the Phase 1/2 trial for female pediatric patients with Rett syndrome, and high-dose NGN-401 has been well-tolerated following dosing in May 2024. The Company also provided an update on interim safety data on the first three patients in the low-dose cohort during the IRSF ASCEND 2024 Rett Syndrome National Summit, which shows that NGN-401 has continued to have a favorable safety profile with no new treatment-related adverse events.
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