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uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that four data presentations, including one oral presentation and three poster presentations, will be delivered at the 62nd American Society of Hematology (ASH) Annual Meeting, which will be held December 5-8 asa virtual event.


GlobeNewswire Inc | Nov 4, 2020 10:47AM EST

November 04, 2020

LEXINGTON, Mass. and AMSTERDAM, Nov. 04, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that four data presentations, including one oral presentation and three poster presentations, will be delivered at the 62nd American Society of Hematology (ASH) Annual Meeting, which will be held December 5-8 asa virtual event.

We expect to have a strong presence at the virtual ASH annual meeting next month, and look forward to having long-term follow-up data presented from our hemophilia B gene therapy studies, including two years of follow-up on the Phase IIb clinical trial of etranacogene dezaparvovec (AMT-061) and up to five years of follow-up from the Phase I/II clinical trial of AMT-060, stated Matt Kapusta, chief executive officer at uniQure. We also remain on track to announce top-line data from the pivotal HOPE-B study of etranacogene dezaparvovec before the end of this year, either at ASH or through a company presentation.

Oral Presentation

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an EnhancedTitle: Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b TrialPresenter: Annette von Drygalski, M.D., PharmDSession 801. Gene Editing, Therapy and Transfer IName:Date: Monday, December 7, 2020Presentation 2:45 p.m. ET (11:45 a.m. PT)Time:

Poster Presentations

AMT-060 Gene Therapy in Adults with Severe or Moderate-SevereTitle: Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 YearsSession 801. Gene Editing, Therapy and Transfer: Poster IIIName:Date: Monday, December 7, 2020Presentation 10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)Time: A Single Administration of AAV5-hFIX in Newborn, Juvenile andTitle: Adult Mice Leads to Stable hFIX Expression up to 18 Months after DosingSession 801. Gene Editing, Therapy and Transfer: Poster IIName:Date: Sunday, December 6, 2020Presentation 10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)Time: Title: Examining the Hemophilia Disability ParadoxSession 904. Outcomes Research?Non-Malignant Conditions: Poster IIName:Date: Sunday, December 6, 2020Presentation 10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)Time:

The conference abstracts were made available today and can be accessed through this link: ASH abstracts.

About uniQure

uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.www.uniQure.com

uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether we will present long-term follow-up data from our hemophilia B gene therapy studies, including two years of follow-up on the Phase IIb clinical trial of etranacogene dezaparvovec (AMT-061) and up to five years of follow-up from the Phase I/II clinical trial of AMT-060, and whether we will announce top-line data from the pivotal HOPE-B study of etranacogene dezaparvovec before the end of this year. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQures Quarterly Report on Form 10-Q filed on October 27, 2020. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure Contacts:

FOR INVESTORS: FOR MEDIA: Maria E. Cantor Chiara Russo Tom MaloneDirect: 339-970-7536 Direct: 617-306-9137 Direct: 339-970-7558Mobile: 617-680-9452 Mobile: 617-306-9137 Mobile:339-223-8541m.cantor@uniQure.com c.russo@uniQure.com t.malone@uniQure.com







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