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 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.

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Learn more about our work to deliver curative gene therapies that transform the lives of patients.