Etranacogene Dezaparvovec (AMT-061)
Hemophilia B
ApprovedCommercial (via partner CSL Behring)
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 Hemophilia B Drugs
| Drug | Company | Phase |
|---|---|---|
| Hemgenix (CSL222) | CSL | Commercial |