A focused pipeline of innovative gene therapies.
We have built a focused pipeline of innovative gene therapies to treat patients with hemophilia and Huntington’s disease as well as early-stage research into opportunities for gene therapies in other severe diseases.
- uniQure Proprietary programs
- CSL Behring partnership
- Program Name
- Phase 1/2
- Phase 3
Hemophilia B etranacogene dezaparvovec (AMT‑061)
Huntington’s Disease (AMT-130)
Amyotrophic Lateral Sclerosis – SOD1 (AMT-162)
Temporal Lobe Epilepsy (AMT-260)
Fabry Disease (AMT‑191)
Amyotrophic Lateral Sclerosis – c9orf72 (AMT-161)
Autosomal Dominant Alzheimer’s Disease (AMT-240)
Other undisclosed programs