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

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 24, 2017 -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that five abstracts have been accepted for presentation at the American Society of Gene and Cell Therapy (ASGCT) 20^th Annual Meeting held in Washington, D.C., May 10-13, 2017. Together with their collaborators, members of uniQure’s research and development team will present data focused on progress in re-administration protocols, gene therapy delivery systems and optimized vector distribution technologies in two of uniQure’s key programs, hemophilia B and Huntington’s disease. An investor and analyst breakfast meeting featuring senior members of uniQure’s research and development team will be held on Friday, May 12 at 7 a.m.

“The data to be presented at the ASGCT meeting demonstrate the development and validation of uniQure’s technologies to improve gene therapy as a therapeutic approach, and supports our aim to rapidly bring new disease-modifying therapies to patients with severe genetic diseases,” stated Matthew Kapusta, chief executive officer of uniQure.

Harald Petry, Ph.D., chief scientific officer at uniQure, added, “We are making strong progress in advancing the preclinical development of AMT-130 in Huntington’s disease, and further enhancing AAV vector technology and the potential for re-administration protocols, which continue to provide compelling evidence of our modular platform.”

Specific details on uniQure’s presentations at ASGCT include:

• Title: Novel AAV Vector Reservoirs: peripheral Blood Cells and Hematopoietic Progenitors. (collaborator presentation)
  Oral Session Title: AAV Vector Biology
  Date and Time: Wednesday, May 10, 2017, 10:30 a.m. EDT
  Location: Marriott Salon 1
  
  
• Title: Circulating Anti-AAV5 Neutralizing Antibody Titers up to 1:1031 Do Not Affect Liver Transduction Efficacy of AAV5 Vectors in Non-Human Primates (Poster 198).
  Poster Session Title: Immunological Aspects of Gene Therapy and Vaccines I
  Session Date and Time: Wednesday, May 10, 2017, 5:30 – 7:30 p.m. EDT
  Location: Hall A&B South
  
  
• Title: Successful Repeated Hepatic Gene Delivery in Non-Human Primates Achieved with AAV5 by Use of Immune Adsorption (Poster 395).
  Poster Session Title: Immunological Aspects of Gene Therapy and Vaccines II
  Session Date and Time: Thursday, May 11, 2017, 5:15 – 7:15 p.m. EDT
  Location: Hall A&B South
  
  
• Title: AAV5-miHTT Gene Therapy Demonstrates Broad Vector Distribution and Strong Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model.
  Oral Session Title: Preclinical Progress Towards Therapies for Neurologic Disorders
  Date and Time: Friday, May 12, 2017, 4:30 p.m. EDT
  Location: Delaware AB
  
  
• Title: Detection of AAV Vector DNA and Transgene RNA in Liver Tissue by Fluorescent In Situ Hybridization (Poster 567).
  Poster Session Title: AAV Vectors III
  Session Date and Time: Friday, May 12, 2017, 5:45 – 7:45 p.m. EDT
  Location: Hall A&B South

Investor and Analyst Breakfast
uniQure management will host an investor and analyst breakfast meeting featuring members of the research and development team to review the data presented during ASGCT.

To request attendance at the meeting, please RSVP to [email protected] by May 5, 2017, as space is limited.

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 liver/metabolic, central nervous system 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, statements regarding the  development of our gene therapy 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 collaboration arrangements, our and our collaborators' clinical development activities, 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 2016 Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 15, 2017. 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:

Maria E. Cantor
Direct: 339-970-7536
Mobile:  617-680-9452
[email protected]

Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
[email protected]

Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
[email protected]