Collaborating with medical professionals to bring gene therapy to patients.

uniQure works together with healthcare professionals to treat patients with severe genetic diseases.

We are advancing a focused pipeline of innovative gene therapies. We are currently conducting a pivotal Phase III trial in our lead indication, hemophilia B, and are preparing to begin a Phase I/II trial in Huntington’s disease this year.  Learn more about our pivotal HOPE-B study for patients with severe and moderately-severe hemophilia B here and see below for helpful gene therapy educational resources. 

Supplement: Fundamentals of Gene Therapy: Addressing Gaps in Physician Education

Miesbach W, O’Mahony B, Key NS, Makris M. How to discuss gene therapy for haemophilia? A patient and physician perspective. Haemophilia. 2019;00: 1–13

An animated demonstration of how gene transfer technology functions within the body:

Contact Us

For physicians:
Eileen Sawyer, VP, Global Medical Affairs

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