Company name



Academic Medical Center Amsterdam

The company

uniQures focus is to advance the future of medicine through gene therapy. They  have the resources and experience to achieve this.

uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. They have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe genetic diseases.

They 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. Their pipeline of adeno-associated virus (AAV)-based gene therapies is developed both internally and through multiple collaborations using their innovative, modular technology platform, including the proprietary commercial-grade industry-leading manufacturing capabilities in two geographical locations. Through the collaborations and the strategic partnership with Bristol-Myers Squibb to develop gene therapies for cardiovascular diseases, they have made the next step towards developing gene therapies targeting chronic and degenerative diseases that affect larger populations.