uniQure N.V.

uniQure is a biotechnology company developing gene therapies, which are treatments that work by modifying genes to help the body fight disease. The company has one approved product called HEMGENIX that helps people with hemophilia B, a bleeding disorder, by enabling their bodies to produce a crucial clotting factor. The rest of uniQure's pipeline consists of experimental gene therapies still being tested in patients for rare conditions including Huntington's disease, epilepsy, ALS, and Fabry disease.