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.
We are developing a pipeline of adeno-associated virus (AAV)-based gene therapies both internally and through multiple collaborations. We develop our gene therapies using our innovative, modular technology platform, including our proprietary manufacturing process. We initially focus on orphan diseases, but through our multiple collaborations and the recent acquisition of the cardiology gene therapy company InoCard we have made the next step towards developing gene therapies targeting chronic and degenerative diseases that affect larger populations.