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uniQure Announces Second Quarter 2020 Financial Results and Highlights Recent Company Progress

~ Entered into Exclusive Global License Agreement with CSL Behring for Development and Commercialization of uniQure’s Gene Therapy Candidate for Hemophilia B ~~ Completed First Patient Dosing in Phase I/II Clinical Trial of AMT-130 in Huntington’s Disease ~LEXINGTON, Mass. and AMSTERDAM, July 30, 2020 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2020 and highlighted recent progress across its business.“The first half of 2020 has been extremely productive for uniQure with significant achievements across our clinical-stage programs,” stated Matt Kapusta, chief executive officer at uniQure. “The initiation of patient dosing in our Phase I/II study of AMT-130 marked the first AAV gene therapy for patients with Huntington’s disease to enter the clinic, and we remain highly focused on advancing development of this important program. We also completed the dosing phase of our HOPE-B pivotal study for etranacogene dezaparvovec in patients with hemophilia B and remain on track to present topline data on all patients by the end of 2020 and submit a BLA in 2021.”“Importantly, in June we announced a landmark global license agreement with CSL Behring to develop and commercialize our hemophilia B gene therapy candidate, with uniQure eligible following regulatory approval to receive more than $2 billion of economics, including upfront cash, milestone, and royalty payments,” he added. “The collaboration ideally positions us to deliver our highly innovative gene therapy to the largest number of hemophilia B patients as quickly as possible, and we expect it will provide an exceptionally strong balance sheet to aggressively expand our pipeline, invest in technology innovation and further scale our manufacturing capabilities.”  Recent Company ProgressAdvancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B

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