uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular technology 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 has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities. Through recent collaborations and our strategic partnership with Bristol-Myers Squibb to develop gene therapies for cardiovascular diseases, we have taken the next steps toward developing gene therapies targeting chronic and degenerative diseases that affect larger populations.