uniQure N.V. operates as a specialized gene therapy company, dedicated to pioneering therapeutic solutions for individuals grappling with severe genetic and other debilitating illnesses. The firm's most advanced project, Etranacogene dezaparvovec (designated as AMT-061), is currently progressing through a crucial Phase III pivotal trial, known as HOPE-B, targeting the treatment of hemophilia B. In addition to its flagship program, uniQure is actively developing several other candidates. This includes AMT-130, a gene therapy undergoing Phase I/II clinical assessment for Huntington's disease, and AMT-060, also in Phase I/II clinical trials for hemophilia B. The company's pipeline further extends to AMT-210 for Parkinson's disease, AMT-260 aimed at temporal lobe epilepsy, and AMT-161, which addresses amyotrophic lateral sclerosis. Furthermore, AMT-240 is an early-stage, preclinical product candidate focused on autosomal dominant Alzheimer's disease. Established in 1998, uniQure N.V. maintains its corporate headquarters in Amsterdam, Netherlands.
