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uniQure Announces Oral Presentation of Updated Results for AMT-060 at the World Federation of Hemophilia (WFH) 2016 World Congress

AMSTERDAM, The Netherlands, July 11, 2016 (GLOBE NEWSWIRE) -- uniQure N.V. (QURE), a leader in human gene therapy, today announced that updated data from its ongoing Phase I/II clinical study of AMT-060 in hemophilia B patients will be presented at the World Federation of Hemophilia 2016 World Congress being held July 24-28, 2016 in Orlando, Florida.

The data will be presented by Dr. Wolfgang Miesbach, Professor at the Universitats Klinikum in Frankfurt, Germany, the principal investigator of the trial, on Wednesday July 27, 2016 during the Late Breaking Session on Gene Therapy. The updated results will include 39 weeks of follow-up and immunogenic data from the five-patient low-dose cohort. AMT-060 is an AAV5-FIX gene therapy manufactured by uniQure using its proprietary insect-cell based technology platform.

The presentation will take place at 2:45 pm EDT in Hall A4 of the Orange County Convention Center.

AMT-060 Phase I/II Trial Overview

The Phase I/II, open-label, multi-center study includes 10 patients each receiving a one-time, 30-minute, intravenous administration of AMT-060, without the use of immunosuppression therapy. The study includes two dose cohorts of five patients each, with the first cohort receiving 5x1012 gc/kg and the second cohort receiving 2x1013 gc/kg. All patients in the trial had documented severe or moderately-severe hemophilia, including documented FIX levels less than...


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