uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT)

New Data Further Demonstrating Favorable Immunogenicity Profile of AAV5 to be Featured in Oral Presentation

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 30, 2018 -- 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 in a plenary session, will be delivered at the upcoming American Society of Gene and Cell Therapy (ASGCT) 21^st Annual Meeting held in Chicago, Illinois, May 16-19, 2018.

“uniQure has developed a toolkit of technologies that are critical for therapeutic efficacy of gene therapy, including non-immunogenic vectors, effective and safe gene silencing technologies, and methods that lead to efficient transduction of target cells within the affected organ,” stated Sander van Deventer, chief scientific officer of uniQure. “These technologies are now broadly applicable to treat patients with liver and CNS disorders.”

Specific details on uniQure’s presentations at ASGCT include:

• Title: Therapeutic factor IX (FIX) activity after single treatment with AMT-060 (AAV5-FIX) in Hemophilia B patients with pre-existing anti-AAV5 Humoral immunity (Abstract #1002).
  Oral Session Title: Immunological Aspects of Gene Therapy and Vaccines
  Date and Time: Saturday, May 19, 2018, 11:45 a.m. - 12:00 p.m. CDT
  Location: Salon A-5

• Title: Evaluation of different AAV5 gene delivery methods to the central nervous system and application in rats (Abstract # 398).
  Poster Session Title: AAV Vectors II  
  Session Date and Time: Thursday, May 17, 2018, 5:15 p.m. - 7:15 p.m. CDT
  Location: Stevens Salon C, D

• Title: Lipid nanoparticle pre-treatment improves rAAV diffusion in the primate liver and enables an increase of therapeutic transgene expression (Abstract #533).
  Poster Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases II
  Session Date and Time: Thursday, May 17, 2018, 5:15 p.m. - 7:15 p.m. CDT
  Location: Stevens Salon C, D 

• Title: AAV-serotype-specific Transduction Patterns In Mice And Non-human Primates (NHPs) Liver Tissue: Implications For Therapeutic Efficacy  (Abstract #706).
  Poster Session Title: AAV Vectors III
  Session Date and Time: Friday, May 18, 2018, 5:45 p.m. - 7:45 p.m. CDT
  Location: Stevens Salon C, D

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 and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and cardiovascular 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, our upcoming anticipated milestones, the development of our gene therapy product candidates, the transition to our AMT-061 product candidate, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. 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, 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 uniQure’s Annual Report on Form 10-K filed on March 14, 2018. 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.