• Neurogene highlighted NGN-401 as a one-time gene therapy candidate for Rett syndrome, supported by interim Phase 1/2 data showing functional improvements across core domains in 100% of participants, with 35 total developmental milestones gained and no plateau reported out to 24 months.
• Safety profile at 1E15 vg dose described as generally well tolerated in 10 participants, with treatment-emergent adverse events related to NGN-401 limited to Grade 1 or Grade 2; most participants had mild liver enzyme elevations.
• Registration plan centers on Embolden, a single-arm, baseline-controlled, open-label trial in females aged ≥3 years (n=20) using a responder-based composite endpoint at 12 months; success threshold set at 35%, or 7 of 20 participants.
• Program timeline calls for completion of Embolden dosing in 2Q'26, presentation of 12+ months Phase 1/2 data for all 10 participants in mid-2026, followed by a process performance qualification campaign starting mid-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 May 11, 2026, and is solely responsible for the information contained therein.