uniQure Publishes Data Further Demonstrating Favorable Immunogenicity Profile and Broad Utilization of AAV5-Based Gene Therapies
(Thomson Reuters ONE) -
-- 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
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
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
m.cantor(at)uniqure.com
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone(at)uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder(at)uniQure.com
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: uniQure N.V. via GlobeNewswire
Datum: 15.06.2017 - 13:00 Uhr
Sprache: Deutsch
News-ID 548165
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