Neurogene says Embolden CRAO... no, Rett trial data due 2H 2027, cash runs to Q1 2029

Neurogene Inc. said on November 9, 2026 that it still expects to report topline data from the Embolden registrational trial in the second half of 2027¹ for NGN-401, its gene therapy candidate for Rett syndrome.

The company disclosed that the Embolden trial was overenrolled due to strong demand from the Rett syndrome community, and Neurogene elected to include all 25 participants dosed in the primary efficacy analysis, with the update to the statistical analysis plan aligned with the FDA through the START Pilot Program; the corresponding threshold for success to reject the null hypothesis is 9 of 25 participants (36%)¹. The company said all other previously aligned key elements of the trial design remain unchanged¹.

On safety, Neurogene reported that NGN-401 at the 1E15 vg dose has been generally well-tolerated in the Phase 1/2 trial and Embolden (N=35) as of November 8, 2026¹. Separately, the company presented an encore of positive long-term Phase 1/2 data, as of a data cutoff of June 16, 2026, at the 55th Child Neurology Society Annual Meeting¹, showing that developmental milestones were gained in a progressive, stepwise sequence, suggesting a restart of development post-treatment¹.

On manufacturing, Neurogene said it remains on track to complete Process Performance Qualification (PPQ) runs by the end of 2026 to support the planned Biologics License Application (BLA) submission¹.

Financially, the company reported cash, cash equivalents and short-term investments as of September 30, 2026 were $359.7 million and are expected to fund planned operations into the first quarter of 2029¹. Quarterly R&D expense rose to $23.5 million for the three months ended September 30, 2026, compared to $17.2 million for the three months ended September 30, 2025¹, while net loss was $29.0 million for the three months ended September 30, 2026, compared to $21.0 million for the three months ended September 30, 2025¹.