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
  • Program Name
  • Preclinical
  • 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

uniQure Logo Twitter Icon LinkedIn Icon YouTube Icon Twitter Icon Arrow Slideshow Next Button