Neurogene says NGN-401 gene therapy shows sustained Rett syndrome milestone gains in Phase 1/2 trial through 30 months

Neurogene Inc.

Neurogene Inc.

NGNE

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  • Neurogene reported positive long-term Phase 1/2 data for NGN-401 gene therapy in females with Rett syndrome.
  • Results were discussed in a webcast today; additional data are scheduled for presentation at the IRSF scientific meeting June 29–July 1, 2026.
  • All treated participants showed overall clinical improvement, with durable gains in everyday developmental function that continued to build over time.
  • Benefits were seen across ages and disease severity, with no sign of losing previously gained abilities during follow-up.
  • Safety profile remained broadly consistent with expectations, with no new serious treatment-related events reported since the prior update.


Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Neurogene Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20260629170706) on June 29, 2026, and is solely responsible for the information contained therein.