uniQure Announces Clinical Data Presentations at the Upcoming International Society on Thrombosis and Hemostasis (ISTH) Congress

~ Updated, Long-term Results from the Phase I/II Dose-Escalating Study of AMT-060 to be Presented on Monday, July 10, 2017 ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, June 23, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that two abstracts have been accepted for presentation at the International Society on Thrombosis and Hemostasis (ISTH) Congress held in Berlin, Germany, July 8 - 13, 2017.

"The data to be featured in an oral presentation at ISTH now demonstrate long-term safety, efficacy and the potential for broad application of AAV5 in hemophilia B," stated Matthew Kapusta, chief executive officer of uniQure.  "We also look forward to the presentation of clinical data demonstrating successful and effective transduction in patients with pre-existing neutralizing antibodies to AAV5, further supporting our belief that our AAV5-based gene therapies may provide clinical benefits to nearly all patients suffering from hemophilia B and other severe diseases."

Specific details on uniQure's presentations at ISTH include:

• Title: Updated Results from a Dose-escalating Study in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy: Up to 1.5 Years Follow-up
  Oral Session Title: Gene Therapy for Hemophilia: Clinical
  Date and Time: Monday, July 10, 2017, 6:00 p.m. CET / 12:00 p.m. EDT
  Location: Hall B, CityCube Berlin, Messedamm 26, 14055, Berlin
  
  Updated data on AMT-060 will be presented by study investigator Professor Frank W. Miesbach, M.D. Ph.D. of the Universitäts Klinikum Frankfurt, Germany. AMT-060 is an AAV5-FIX gene therapy developed by uniQure using its proprietary insect-cell based technology platform. The updated data will include up to 1.5 years of follow-up on the first patient cohort receiving 5x10¹² gc/kg and up to a year of follow-up on the second patient cohort receiving a higher dose of 2x10¹³ gc/kg.
  
  
• Title: Pre-existing anti-AAV5 neutralizing antibodies measured using a highly sensitive assay in sera of Hemophilia B patients in a Phase I/II clinical trial of AMT-060 do not predict efficacy of AAV5-mediated liver-directed gene transfer
  Poster Session Date and Time: Tuesday, July 11, 2017, 12:00 pm CET / 6:00 a.m. EDT
  Location: Exhibition Hall 4.2, CityCube, Messedamm 26, 14055, Berlin

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