Uniqure

uniQure is a leader in the field of gene therapy and had developed the first and currently the only gene therapy product to receive regulatory approval in the European Union. Gene therapy offers the prospect of long-term and potentially curative benefit to patients with genetic or acquired diseases by directing the expression of a therapeutic protein or restoring the expression of a missing protein through a single administration. Our first product, Glybera, was approved by the European Commission in October 2012 under exceptional circumstances for the treatment of a subset of patients with lipoprotein lipase deficiency, or LPLD, a potentially life-threatening, orphan metabolic disease. We are also developing a pipeline of additional adeno-associated virus (AAV)-based gene therapies through multiple collaborations. We develop our gene therapies using our innovative, modular technology platform, including our proprietary manufacturing process. We initially are focused on orphan diseases but believe that we will also be able to develop gene therapies targeting chronic and degenerative diseases that affect larger populations.

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