Â 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 therapyÂ 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 replacementÂ usage.
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