Neurogene says NGN-401 Rett trial shows milestone gains up to 24 months

Neurogene Inc.

Neurogene Inc.

NGNE

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  • Neurogene highlighted data at ASGCT supporting intracerebroventricular delivery as a practical route for CNS gene therapy, with results already presented at the meeting.
  • Preclinical findings indicated broader distribution in brain regions tied to Rett syndrome versus an alternative spinal-fluid delivery approach, supporting strategy for its NGN-401 program.
  • Phase 1/2 Rett syndrome readout described functional gains that continued over time, with treatment generally well tolerated through an October 30, 2025 data cutoff.
  • Company is using same delivery method in Embolden registrational trial, positioning it as a scalable clinical approach that could support outpatient-style monitoring.


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: 202605120600BIZWIRE_USPR_____20260512_BW663376) on May 12, 2026, and is solely responsible for the information contained therein.

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