About:
uniQure N.V., a gene therapy company, engages in the development of treatments for patients suffering from genetic and other devastating diseases. Its lead program is Etranacogene dezaparvovec (AMT-061), which is in Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also engages in developing AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington’s disease; AMT-060, which is in Phase I/II clinical trial for the treatment of hemophilia B; AMT-180 for the treatment of hemophilia A; AMT-190, an IV-administered adeno-associated virus (AAV) 5-based gene therapy for the treatment of Fabry disease; and AMT-150, a one-time, intrathecally-administered, AAV gene therapy for treating spinocerebellar ataxia type 3. The company has collaboration and license agreements with Bristol Myers-Squibb Company; Gen-X; and Synpromics Limited. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.