QURE

Uniqure N.v.

A gene therapy company developing one-time treatments for rare diseases, uniQure produced HEMGENIX, the first FDA-approved gene therapy for hemophilia B, now sold worldwide by partner CSL Behring. Founded in 1998 as Amsterdam Molecular Therapeutics by scientists from Amsterdam's Academic Medical Center, it renamed itself uniQure—a play on "unique cures." It also developed Glybera, the first gene therapy approved in the Western world, and its lead experimental candidate targets Huntington's disease.