AMT-130
Huntington's Disease
Phase 1/2Active
Key Facts
About uniQure
uniQure is a clinical-stage gene therapy company with over two decades of experience, dedicated to reimagining medicine through innovative genomic treatments. The company has built a modular AAV platform, featuring proprietary technologies like miQURE for gene silencing, and is advancing a pipeline targeting CNS and liver diseases. With a lead program, AMT-130, in late-stage development for Huntington's disease and a commercial-stage partnership for hemophilia B gene therapy, uniQure is positioned at the forefront of the gene therapy revolution.
View full company profileTherapeutic Areas
Other Huntington's Disease Drugs
| Drug | Company | Phase |
|---|---|---|
| SRP-1005 (ARO-HTT) | Arrowhead Pharmaceuticals | Phase 1 |
| PTC518 | PTC Therapeutics | Phase 2 |
| Undisclosed HD Program(s) | WaVe Life Sciences | Preclinical |
| Undisclosed Program | Sangamo Therapeutics | Preclinical |
| Pepinemab (VX15/2503) | Vaccinex | Phase 2 |