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US FDA grants uniQure breakthrough therapy designation for hemophilia drug

The U.S. FDA granted uniQure NV breakthrough therapy designation for AMT-060, an investigational gene therapy for patients with severe hemophilia B.

Hemophilia B is a serious and rare inherited disease in males characterized by insufficient blood clotting.

The designation is based on the ongoing phase 1 and 2 studies of the drug that show sustained increases in Factor IX, reductions in Factor IX replacement usage and a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up.