Neurogene provided an update on recent achievements for NGN-401 gene therapy for Rett syndrome and outlined plans for 2026.
Multiple participants have been dosed in the Embolden registrational trial of NGN-401.
The company plans to present interim data on pediatric and adolescent/adult cohorts from Phase 1/2 trial in mid-2026.
Dosing Progress
Multiple participants dosed in Embolden trial in Q4 2025.
Interim Data
Plans to report updated safety and efficacy data by mid-year.
Commercialization Focus
Engaging with payors and stakeholders for future launch readiness.
Cash Runway
Expected through Q1 2028 to achieve key milestones.
- Neurogene is progressing towards completing dosing in the Embolden trial by Q2 2026.
- Positive interim data from the Phase 1/2 trial shows durable improvements in females with Rett syndrome.
- The company's approach aims to maximize therapy distribution and potency for optimized efficacy and safety.
- Neurogene's manufacturing facility in Houston supports pivotal activities for the development of NGN-401.
Neurogene is focused on advancing NGN-401 towards commercialization and achieving key milestones in 2026. With promising data and a clear strategy, the company aims to make a significant impact in the field of gene therapy for Rett syndrome.