Pipeline
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
- Preclinical
- Phase 1/2
- Phase 3
Indications
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Hemophilia B etranacogene dezaparvovec (AMT‑061)
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Huntington’s Disease (AMT-130)
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Amyotrophic Lateral Sclerosis – SOD1 (AMT-162)
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Temporal Lobe Epilepsy (AMT-260)
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Fabry Disease (AMT‑191)
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Amyotrophic Lateral Sclerosis – c9orf72 (AMT-161)
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Autosomal Dominant Alzheimer’s Disease (AMT-240)
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Other undisclosed programs