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 --
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."
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
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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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The issuer of this announcement warrants that they are solely
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information contained therein.
Source: uniQure N.V. via Globenewswire
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