uniQure
Updated: April 08, 2026
Matt Kapusta, CEO
Country: Netherlands | Funding: $1.3B (+)
Founded: 1998
Website: https://www.uniqure.com/
uniQure has developed an industry-leading AAV gene therapy platform focused on liver and central nervous system diseases. The company's lead candidate, AMT-130, for the treatment of Huntington's disease consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein. The company is also developing candidate AMT-191 for Fabry disease (it delivers a GLA transgene to the liver to enhance GLA protein production), AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene (it is a recombinant AAVrh10 vector expressing microRNA designed to suppress SOD1 expression) and HEMGENIX - the world's first FDA-approved gene therapy for hemophilia B.
Founded: 1998
Website: https://www.uniqure.com/
uniQure has developed an industry-leading AAV gene therapy platform focused on liver and central nervous system diseases. The company's lead candidate, AMT-130, for the treatment of Huntington's disease consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein. The company is also developing candidate AMT-191 for Fabry disease (it delivers a GLA transgene to the liver to enhance GLA protein production), AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene (it is a recombinant AAVrh10 vector expressing microRNA designed to suppress SOD1 expression) and HEMGENIX - the world's first FDA-approved gene therapy for hemophilia B.
