UniQure is a world leader in the field of gene therapy. The company has developed a proprietary technology platform that enables the delivery of therapeutic genes to patients with serious and life-threatening diseases. UniQure’s first approved product, Glybera (alipogene tiparvovec), is a treatment for lipoprotein lipase deficiency (LPLD), an ultra-rare genetic disorder that results in disabling pancreatitis. UniQure is also advancing a broad pipeline of products in various stages of development for hemophilia B, Huntington’s disease, congestive heart failure and other cardiovascular diseases, using either its adeno-associated viral vector (AAV) or lentiviral vector (LV) technology platforms.
In 2012, UniQure became the first company to receive approval from the European Commission for a gene therapy product. This was followed by approval from the U.S. Food and Drug Administration (FDA) in 2015. In 2016, UniQure received FDA approval for its second product, Amgen’s Hemlibra (emicizumab-kxwh), which treats people with hemophilia A who have inhibitors to factor VIII replacement therapies. Hemlibra is jointly commercialized by UniQure and Amgen in the United States and other countries around the world.
UniQure has built an integrated organization with global reach to support the development and commercialization of its expanding portfolio of innovative gene therapies designed to improve patient lives worldwide suffering from severe genetic diseases with currently no adequate treatment options available.