uniQure Publishes Data Further Demonstrating Favorable Immunogenicity Profile and Broad Utilization of AAV5-Based Gene Therapies

-- Study published in Molecular Therapy provides proof of concept of successful cross-administration of AAV5- and AAV1-based gene therapies --

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, June 15, 2017 -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the online publication in Molecular Therapy of data demonstrating successful repeated liver-targeted gene delivery in non-human primates (NHPs) using sequential administration of AAV5 and AAV1 vector serotypes. In this study, two different proteins have been shown to be effectively produced in the NHP liver after sequential administration of high, clinically relevant doses of both AAV5 and AAV1, and suggests that cross-administration of AAV5 gene therapies with other vectors may be possible in humans.

A major hurdle in achieving successful delivery of an AAV-based therapeutic is the presence of circulating neutralizing antibodies (NABs), which can develop after a single administration of gene therapy. These neutralizing antibodies may prevent successful gene transfer in patients who have previously received gene therapy.

In the on-line publication, entitled “Successful repeated hepatic gene delivery in mice and non-human primates achieved by sequential administration of AAV5^ch and AAV1 vectors,” uniQure researchers in collaboration with the Gene Therapy and Hepatology Department at CIMA, University of Navarra, Pamplona describe the feasibility of using AAV5 and AAV1 for repeated liver-targeted gene delivery. Animals were first immunized with a high dose of AAV5-hSEAP in order to generate high levels of anti-AAV5 NAB. They were then given the same dose of AAV1-hFIX.  Transduction with AAV1 proved to be successful despite high levels of anti-AAV5 NAB raised after the first gene transfer, as expression of both reporter proteins was observed.

“These data show that gene therapies do not necessarily have to be limited to a one-time administration, and that successful cross-administration of AAV5 gene therapies with other vectors may be possible in humans,” stated Valerie Sier-Ferreira, Ph.D., head of immunology at uniQure. “The study provides additional support to our belief that AAV5 has a superior immunogenicity profile and can be made accessible to nearly all patients.”

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

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uniQure Contacts:

Maria E. Cantor
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Tom Malone
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Eva M. Mulder
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