Shots:
- Neurogene has reported interim data from the pediatric cohort (4-10yrs.) of the ongoing P-I/II trial assessing NGN-401 for the treatment of females with Rett syndrome
- As of Oct 30, 2025, all pediatric pts (n=8) had functional gains across Rett syndrome domains, achieving 35 developmental milestones in fine motor, communication & ambulation. These gains were durable, with multidomain improvements observed in key domains
- Pts with longer follow-up continued to gain developmental milestones, consistent with recently dosed pts with 6mos. of follow-up. CGI-I improvements were durable, with additional data expected in 2026
Ref: Neurogene | Image: Neurogene | Press Release
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