Is gene therapy science fiction?
No. Gene therapy has been studied for more than half a century. In 2012, uniQure was the first company to secure approval for a gene therapy in Europe for a metabolic disease, and thus has been an important contributor to the field. Currently there is one AAV-based gene therapy approved and available for treatment of patients with a rare form of blindness.
Are there any gene therapies approved to treat patients with hemophilia?
There are currently no approved gene therapies for the treatment of hemophilia.
Is gene therapy a cure for hemophilia B?
How to define ‘cure’ is complicated, since different people may consider a cure to be different things. The goal of gene therapy for hemophilia B is to enable persons with hemophilia B to live their lives as they’d like to do without bleeds or need for routine treatments. HOPE-B will investigate whether or not AMT-061 is able to provide these effects.
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.