uniQure To Present Data On Gene Therapy Technology Advances At Annual Meeting Of The American Society Of Gene And Cell Therapy

AMSTERDAM, The Netherlands, April 19, 2016 -- uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced that six abstracts have been accepted for poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 19^th Annual Meeting held in Washington D.C., May 4-7, 2016. Together with their collaborators, members of uniQure's research and development team will present data focused on progress in improving gene therapy delivery systems, re-administration protocols and vector technologies in two of uniQure's key focus areas, liver/metabolism and CNS.

"Innovation in gene therapy technology is an important focus at uniQure and we believe that enhancing the functionality and applicability of gene therapy will significantly improve potential benefit for patients," said Daniel Soland, Chief Executive Officer of uniQure.

"The data we will present at the ASGCT meeting specifically highlight novel therapeutic strategies for gene transduction and gene therapy re-administration providing insights into pioneering technologies that will impact our development programs in the future," added Harald Petry, PhD, Chief Scientific Officer of uniQure.

The following presentations are scheduled at the ASGCT meeting sessions:

Liver/Metabolism

• Changing the Route of Administration to Improve Liver Transduction by Recombinant AAV-Based Vectors – Abstract #300
  Session: AAV Vectors II
  Poster Presentation – Thursday, May 5, 2016, 6.00 – 8.00 pm
• Development of MFP-Inducible System for AAV5 Gene Therapy of Chronic Diseases in the Liver – Abstract #546
  Session: AAV Vectors III
  Poster Presentation – Friday, May 6, 2016, 6.00 – 8.00 pm
• Successful Repeated Hepatic Gene Delivery in Mice and Non-Human Primates Achieved by Sequential Administration of AAV5 and AAV1 Vector Serotypes – Abstract #700
  Session: Immunological Aspects of Gene Therapy II: AAV Vectors
  Poster Presentation – Friday, May 6, 2016, 6.00 – 8.00 pm

CNS

• Directional Transduction of AAV5 Vectors in the NHP Brain – Abstract #618
  Session: Delivery Questions for Neuronal Applications
  Poster Presentation – Friday, May 6, 2016, 6.00 – 8.00 pm

Gene Therapy Technology

• Analysis of AAV5 Biodistribution and Viral Shedding in the Presence or Absence of Neutralizing Antibodies – Abstract #310
  Session: AAV Vectors II
  Poster Presentation – Thursday, May 5, 2016, 6.00 – 8.00 pm
• Successful In Vivo Re-Administration of AAV with the Use of Two-Step AAV injection – Abstract #701
  Session: Immunological Aspects of Gene Therapy II: AAV Vectors
  Poster Presentation – Friday, May 6, 2016, 6.00 – 8.00 pm

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 CNS, liver/metabolic 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 therapies 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 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 2014 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 25, 2014 and its 2015 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2016. 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.

CONTACT: uniQure:
         Eva Mulder
         Direct : +31 20 240 6103
         Main: +31 20 240 6000
         [email protected]
         
         Media inquiries:
         Gretchen Schweitzer
         MacDougall Biomedical Communications
         Direct: +49 172 861 8540
         Main: +49 89 2424 3494 or +1 781 235 3060
         [email protected]