Company Profile:

Delivering on the promise of gene therapy.

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

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. 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.

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