Business
Neurogene Announces Recent Achievements and Anticipated 2026 Key Milestones for NGN-401, a Potential Best-in-Class Gene Therapy for Rett Syndrome
NEW YORK, January 12, 2026--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today provided an update on recent achievements for NGN-401 gene therapy for the treatment of Rett syndrome and plans for multiple value-creating catalysts anticipated in 2026.
About this update from Neurogene Inc.
[{"type":"text","content":"Dosed multiple participants in Embolden™ registrational trial in fourth quarter of 2025","length":87,"tagName":"p","attribs":{}},{"type":"text","content":"Completion of dosing in Embolden expected in second quarter of 2026","length":67,"tagName":"p","attribs":{}},{"type":"text","content":"Plan to present interim data on pediatric and adolescent/adult cohorts from Phase 1/2 trial in mid-2026","length":103,"tagName":"p","attribs":{}},{"type":"text","content":"Early commercial-readiness activities underway","length":46,"tagName":"p","attribs":{}},{"type":"text","content":"Neurogene to present at J.P. Morgan Healthcare Conference on January 14 at 7:30 a.m. PT","length":87,"tagName":"p","attribs":{}},{"type":"text","content":"NEW YORK, January 12, 2026--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today provided an update on recent achievements for NGN-401 gene therapy for the treatment of Rett syndrome and plans for multiple value-creating catalysts anticipated in 2026.","length":391,"tagName":"p"},{"type":"text","content":""We are pleased to share that multiple participants have been dosed in our Embolden registrational trial of NGN-401 gene therapy for Rett syndrome, an important step toward completing dosing in the second quarter," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "We continue to track the progress of the participants in our Phase 1/2 trial, with plans to report updated interim safety and efficacy data by mid-year, including at least 12 months of data for all participants. As we rapidly advance NGN-401 towards commercialization, we are actively engaging with payors and other key stakeholders while leveraging our internal CMC capabilities for commercial-grade production to ensure future launch readiness. With cash runway expected through the first quarter of 2028, we believe we are well-positioned to advance NGN-401 through key upcoming milestones, including a BLA submission, to achieve its potential as a best-in-class gene therapy for Rett syndrome."","length":1013,"tagName":"p"},{"type":"text","content":"NGN-401 Program Updates and Recent Accomplishments","length":50,"tagName":"p"},{"type":"list","items":[{"val":[{"type":"te...