NGN-401
Rett Syndrome
Phase 1/2Active; Registrational trial (Embolden™); FDA Breakthrough Therapy Designation granted Feb 2026
Key Facts
Indication
Rett Syndrome
Phase
Phase 1/2
Status
Active; Registrational trial (Embolden™); FDA Breakthrough Therapy Designation granted Feb 2026
Company
About Neurogene
Neurogene is a public, clinical-stage biotech (Nasdaq: NGNE) founded in 2018 with a mission to turn devastating neurological diseases into treatable conditions. The company's core innovation is the EXACT technology, a microRNA-based platform for controlled transgene expression, which underpins its lead candidate NGN-401 for Rett syndrome, currently in a registrational Phase 1/2 trial. With a seasoned leadership team combining deep scientific, clinical, and financial expertise, Neurogene is advancing a pipeline focused on high-unmet-need rare neurological disorders.
View full company profileTherapeutic Areas
Other Rett Syndrome Drugs
| Drug | Company | Phase |
|---|---|---|
| DAYBUE® (trofinetide) | Acadia Pharmaceuticals | Approved/Commercial |
| TSHA-102 | Taysha Gene Therapies | Phase 1/2 |
| Trofinetide (NNZ-2566) / DAYBUE™ | Neuren Pharmaceuticals | Approved |
| ANAVEX®2-73 (blarcamesine) | Anavex Life Sciences | Phase 2/3 |