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UniQure Receives FDA Breakthrough Designation of AMT-060 Investigational Gene Therapy

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Note: The following is edited from a press release from UniQure. Read the full press release in it’s entirety here.

UniQure announced that AMT-060,  its proprietary, investigational gene therapy in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).  This designation is based on results from the ongoing, dose-ranging Phase 1-2 study that show sustained increases in Factor IX (FIX), reductions in FIX replacement usage and a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up.

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