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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

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Note: The following is an edited version of a press release from uniQure. Read the press release from uniQure in it’s entirety here.


uniQure N.V. has announced updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose cohort and up to one year of follow-up from the second dose cohort.

The AAV5-based AMT-060 remains safe and well-tolerated with up to a year and a half of follow-up, with no serious adverse events and no development of inhibitors. All patients are now past one year of follow up with no loss of Factor IX (FIX) activity and no capsid-specific T-cell activation.

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