uniQure Publishes Data Demonstrating Wide Distribution of AAV5 in the Central Nervous System
April 04 2017 - 7:42AM
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 Gene Therapy of
data demonstrating widespread transduction in the CNS following
direct injection of uniQure’s AAV5 vector in a large animal model.
uniQure has exclusive, worldwide rights to AAV5 for use in
therapeutic products delivered to the brain or liver.
The method of injection used was found to result
in very controlled and accurate administration with no adverse
events observed in the non-human primates. Varying doses of AAV5
achieved predictable transduction of connected areas of the brain.
The data demonstrate that AAV5 is a very effective vector for the
central nervous system and has potential for the treatment of a
wide range of neurological pathologies. These data will help guide
the clinical development of uniQure’s gene therapy product
candidate AMT-130, which consists of an AAV5 vector carrying an
artificial micro-RNA that silences the huntingtin gene for the
treatment of Huntington’s disease.
In the publication entitled “MR-guided
parenchymal delivery of adeno-associated viral vector serotype 5 in
nonhuman primate brain,” uniQure researchers in collaboration with
experts from the University of California, San Francisco, describe
the predictable and dose-dependent axonal transport of AAV5 after
MRI-guided, convection-enhanced, parenchymal infusion. The results
indicate that AAV5 can effectively achieve wide distribution
throughout the central nervous system, which is crucial for the
treatment of many CNS disorders, such as Huntington’s disease.
“This study further supports our belief that
AAV5 is an optimal vector for CNS-delivery of gene therapy, and is
an important step forward in defining the parameters for direct
injection of AAV5 into the brain for future clinical applications
in humans,” stated Harald Petry, uniQure’s chief scientific
officer. “In particular, these data will help guide the
design and development of the planned clinical trials of AMT-130.
We look forward to making several preclinical data presentations at
scientific meetings this year and to file an Investigational New
Drug (IND) application for AMT-130 in 2018.”
About uniQureuniQure 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,"
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"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, statements regarding
the development of our gene therapy product candidate
AMT-130. Our actual results could differ materially from those
anticipated in these forward-looking statements for many reasons,
including, without limitation, risks associated with 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 with the Securities
and Exchange Commission 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
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
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