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 fully-enrolled pivotal Phase III trial in our lead indication, hemophilia B, and are now enrolling patients in our Phase I/II trial in Huntington’s disease, and have treated the first two patients in the trial.  Learn more about our ongoing clinical trials here and 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:

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