uniQure Publishes Data Further Demonstrating Favorable Immunogenicity Profile and Broad Utilization of AAV5-Based Gene Therap...
June 15 2017 - 7:00AM
-- Study published in Molecular Therapy provides
proof of concept of successful
cross-administration of AAV5- and AAV1-based gene
therapies --
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.”
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
uniQure Forward-Looking
StatementsThis 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,"
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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
m.cantor@uniqure.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
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