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@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