聽uniQure recently received the designation of a “priority medicine” from the European Union for a potential gene therapy treatment for Hemophilia B.
From the press release:
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy聽in patients with severe hemophilia B, has received PRIME designation by the European Medicines Agency (EMA). This designation is based on results from the ongoing, dose-ranging Phase 1-2 study that show a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up, clinically significant and sustained increases in Factor IX (FIX) and substantial reductions in FIX replacement聽usage.
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