uniQure Publishes Data Demonstrating Wide Distribution of AAV5 in the Central Nervous System
(Thomson Reuters ONE) -
Study published in Gene Therapy will help guide clinical development of gene
therapy product candidate AMT-130 in Huntington's disease
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 04, 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 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 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,
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(at)uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder(at)uniQure.com
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone(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: 04.04.2017 - 13:49 Uhr
Sprache: Deutsch
News-ID 534387
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