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uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

desember 11, 2017

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uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its investigational gene therapy in patients with severe hemophilia B. The data includes up to two years of follow-up from the low-dose cohort and up to 18 months of follow-up from the second, higher-dose cohort. The AAV5-based AMT-060 remains safe and well-tolerated with up to two years of follow-up, with no new serious adverse events and no development of inhibitors. No patient in the study has had any loss of Factor IX (FIX) activity or capsid-specific, T-cell-mediated immune response.