-- 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 (GLOBE NEWSWIRE) -- 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 AAV5ch 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."
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, 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 corporate reorganizations and strategic shifts, 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 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@example.com Tom Malone Direct: 339-970-7558 Mobile: 339-223-8541 t.malone@uniQure.com Eva M. Mulder Direct: +31 20 240 6103 Mobile: +31 6 52 33 15 79 e.mulder@uniQure.com