Neurogene posts NGN-401 Rett syndrome Phase 1/2 clinical data update presentation

Neurogene Inc.

Neurogene Inc.

NGNE

0.00
  • Neurogene posted a Phase 1/2 clinical data update on NGN-401 gene therapy for Rett syndrome.
  • All 10 Phase 1/2 participants gained at least 1 developmental milestone, improved on CGI-I, followed for up to 30 months.
  • 80% met the Embolden composite responder definition at 12 months; milestones continued to accumulate with no plateau reported.
  • Safety update: 1E15 vg dose generally well tolerated; no treatment-related serious adverse events or dose-limiting toxicities reported in Embolden.
  • Embolden registrational trial dosed 25 participants; topline results expected in 2H 2027.


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 on June 29, 2026, and is solely responsible for the information contained therein.

This page is machine-translated. Sahm tries to improve but does not guarantee the accuracy and reliability of the translation, and will not be liable for any loss or damage caused by any inaccuracy or omission of the translation. *Disclaimer: The above content only represents the author's personal position and opinion and does not represent any position of Sahm Capital Financial Company and Sahm cannot confirm the authenticity, accuracy, and originality of the above content. Investors should consider the risks of investment products in light of their circumstances before making any investment decisions. When necessary, please consult a professional investment advisor. Sahm does not provide any investment advice, nor does it make any commitments and guarantees.