uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the online publication in Blood of clinical data from the ongoing Phase I/II trial of AMT-060 in patients with severe hemophilia B. The manuscript reports data on up to one year of follow-up from the low-dose cohort of patients in the trial and up to six months of follow-up on patients in the higher-dose cohort. The published manuscript, entitled “Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B,” shows that AAV5 liver-directed wildtype hFIX gene transfer was well tolerated and clinically effective in patients with severe and moderate-severe hemophilia B. No cellular immune responses to the AAV5 vector were detected and Factor IX (FIX) expression levels were stable over the entire observation period. The manuscript is available online today and will be included in a future print edition of Blood.