Hero 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
  • Bristol-Myers Squibb partnership
  • Program Name
  • Preclinical
  • Phase 1/2
  • Phase 3

Liver-Directed/Rare Diseases

  • Hemophilia B etranacogene dezaparvovec (AMT‑061)

  • Fabry Disease (AMT‑191)

  • Other undisclosed programs

Central Nervous System Diseases

  • Huntington’s Disease (AMT‑130)

  • Temporal Lobe Epilepsy (AMT‑260)

  • Parkinson’s Disease (AMT‑210)

  • Autosomal Dominant Alzheimer’s Disease (AMT‑240)

  • Amyotrophic Lateral Sclerosis (AMT‑161)

  • Other undisclosed programs

Cardiovascular and Muscle Diseases

  • Four undisclosed programs

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