uniQure N.V. develops genomic medicines and is known for creating the first gene therapy approved in Europe, as well as an FDA-approved gene therapy for hemophilia B that is now commercialized in partnership with CSL Behring. The company's proprietary technology platform encompasses adeno-associated virus (AAV) vectors, gene-silencing constructs, and commercial-scale manufacturing capabilities. Its lead clinical program is AMT-130, an investigational gene therapy for the treatment of Huntington's disease that utilizes the company's miQURE gene-silencing technology. Additionally, uniQure is advancing a pipeline of preclinical and early-stage clinical candidates targeting other severe neurological and genetic disorders, including refractory mesial temporal lobe epilepsy and Fabry disease. The company operates primarily out of its research headquarters in the Netherlands and its manufacturing facility in the United States.
