Note: The following is edited from a press release from UniQure. Read the full press release in it’s entirety here.
UniQure announced new and updated results during the American Society of Hematology (ASH) meetingÂ in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure’s proprietary, investigational gene therapyÂ for patients with severe hemophilia B. The data includes demonstration of sustained levels of factor IX activity for up to a year with reductions in bleeding over time for those in the low-dose cohort. Data presented at ASH also showed that the cohort receiving a second dose of AMT-060 were able to stop prophylactic factor IX therapy.