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Neurogene presents NGN-401 data showing developmental milestone gains in all 10 Phase 1/2 participants

PUBT·11/09/2026 12:51:06
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Neurogene presents NGN-401 data showing developmental milestone gains in all 10 Phase 1/2 participants
  • Neurogene completed dosing 25 participants in its Embolden registrational trial of NGN-401 for Rett syndrome, exceeding its enrollment target by 25%.
  • Topline Embolden results are expected in the second half of 2027.
  • In the Phase 1/2 trial, 80% of 10 participants met Embolden's composite responder definition at 12 months.
  • All 10 participants gained developmental milestones; no milestone losses were observed through 30 months of follow-up.
  • Completion of BLA-enabling Process Performance Qualification runs is expected by year-end 2026.


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 November 09, 2026, and is solely responsible for the information contained therein.