Multi-national study to evaluate the safety and efficacy of a type of AAV5-based gene therapy.
New options to treat Hemophilia B are needed
*Etranacogene dezaparvovec (e tra nak’ oh jeen dez ah par’ voe vek) is an investigational product being studied in a phase 3 trial for the treatment of adults with hemophilia B.
Learn more about the development of a new therapy for hemophilia B
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The company is developing several gene therapies for the treatment of patients with liver/metabolic, central nervous system, and other severe genetic diseases.