uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe genetic diseases.
We are advancing a focused pipeline of innovative gene therapies and have established clinical proof-of-concept in our lead indication, hemophilia B, and preclinical proof-of-concept in Huntington’s disease. Our pipeline of adeno-associated virus (AAV)-based gene therapies is developed both internally and through multiple collaborations using our innovative, modular technology platform, including our proprietary commercial-grade industry-leading manufacturing capabilities in two geographical locations. Through our collaborations and the strategic partnership with Bristol-Myers Squibb to develop gene therapies for cardiovascular diseases, we have made the next step towards developing gene therapies targeting chronic and degenerative diseases that affect larger populations.