Neurogene Reports First Patient Dosed In High-Dose Cohort Of NGN-401 Gene Therapy Clinical Trial For Rett Syndrome
High-dose NGN-401 has been well-tolerated, and low-dose NGN-401 continues to show a favorable safety profile
Interim safety data presented at the International Rett Syndrome Foundation (IRSF) ASCEND Summit
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 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 (AEs).