NGN-401
Rett Syndrome
Phase 1/2 & RegistrationalActive (Embolden Trial)
Key Facts
About Neurogene
Neurogene's mission is to develop life-changing genetic medicines for patients and families impacted by rare neurological diseases. Its key achievement is advancing NGN-401, a gene therapy for Rett syndrome, into the Embolden™ registrational trial, with promising early safety data. The company's strategy is built on its proprietary EXACT™ technology platform, designed to overcome overexpression toxicity, and a fully integrated approach that includes internal manufacturing to accelerate development.
View full company profileTherapeutic Areas
Other Rett Syndrome Drugs
| Drug | Company | Phase |
|---|---|---|
| NTI164 | Neurotech International | Phase 2 |
| RTT1 | Grann Pharmaceuticals | Pre-clinical |
| NEX-1 / NEX-2 | Nexien BioPharma | Pre-clinical / Phase 1 |
| KIT-13 | NeuroCores | Pre-clinical |
| DPM-1003 | DepYmed | Phase 1 |
| ExoEdit® for Rett Syndrome | Evox Therapeutics | Pre-clinical |
| RVL-001 (vorinostat) | Unravel Biosciences | Clinical Trial Initiated |
| Rett Syndrome Program | Herophilus | Discovery |
| Glutamate Modulator for Rett Syndrome | Numedicus | Licensed/Orphan Designation |
| TTI-0102 | Thiogenesis Therapeutics | Phase 1 |
| DAYBUE® (trofinetide) | Neuren Pharmaceuticals | Approved |
| TSHA-102 | Taysha Gene Therapies | Phase 1/2 |