Successful results were announced by uniQure for its gene therapy, AMT-061, on hemophilia B patients
February 11, 2019
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The United States FDA granted Breakthrough Therapy Designation to AMT-061. The European Medicines Agency gave it access to the Priority Medicine (PRIME) regulatory initiative. Gene therapy firm, uniQure (QURE), announced updated clinical data in patients treated in the ongoing Phase 2b study of AMT-061; an investigational AAV5-based gene therapy containing a patent-protected FIX9-Padua (Factor 9) for the treatment of patients with severe and moderately severe hemophilia B. The announced data, which …