Delivering on the promise of gene therapy.

Our focus is to advance the future of medicine through gene therapy
Leveraging our more than 20 years of gene therapy leadership.

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. 

 

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Learn more about uniQure's top priorities as we work to deliver curative gene therapies that transform the lives of patients.