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uniQure announced that the third patient has been treated in the company's Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severehemophilia B. AMT-061 has been granted Breakthrough Therapy Designation by the FDA and access to Priority Medicine regulatory initiative by the European Medicines Agency, the company points out. The Phase IIb dose-confirmation study is an open-label, single-arm, single-dose trial being conducted in the United States to confirm the dose of AMT-061 prior to patient treatment in the ongoing Phase IIIHOPE-Bpivotal trial. Three patients received a single intravenous infusion of 2x1013vc/kg over the past month and are currently under evaluation to assess safety and Factor IX activity. "We are very pleased with the progress we have made in the clinical development of AMT-061, our lead gene therapy product candidate for hemophilia B," statedMatthew Kapusta, CEO of uniQure. "The treatment of the third patient puts us in the position to present topline data and confirm dosing for our Phase III HOPE-B pivotal study by the end of the year."