UniQure

By bringing life-changing benefits to people with rare diseases and cancers, cell and gene therapies are reshaping the field of medicine. These treatments have recently gained tremendous publicity for meeting long-standing unmet needs and being extremely costly. But high production costs are limiting patient access...

An X-linked recessive disorder, Hemophilia occurs predominantly in males with females being the carriers. Patients diagnosed with Hemophilia usually lack one of the two proteins essential for the blood coagulation cascade namely factor VIII (FVIII) and factor IX (FIX). On the basis of the different blood coagulatio...