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) 19th 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

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