Address: Paasheuvelweg 25a, 1105 BP, Amsterdam Chamber of Commerce 34275365 , Netherlands
Phone: +31 20 240 6000
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with severe genetic diseases of the central nervous system (CNS) and liver, including clinical programs in hemophilia B and Huntington’s disease and preclinical candidates in Fabry disease, spinocerebellar ataxia Type 3, temporal lobe epilepsy, Alzheimer’s, Parkinson’s, and ALS.
Our pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities.