uniQure Announces the Successful Development and Scale-up of Manufacturing Processes for Its Hemophilia B Gene Therapy Progra...
July 21 2017 - 7:00AM
-- Commercial-Scale Batches of AMT-060
Successfully Produced in Accordance with Good Manufacturing
Practices in Lexington, MA Facility --
uniQure N.V. (Nasdaq:QURE), a leader in human gene therapy and
AAV manufacturing, today announced that it has successfully
developed and optimized a reproducible and scaled-up manufacturing
process for producing its lead gene therapy candidate for patients
with hemophilia B at its state-of-the-art manufacturing facility in
Lexington, Massachusetts. This progress includes the
validation of analytical methods required to manufacture and test
product in accordance with Good Manufacturing Practices
(GMP). The Company has also finalized its comparability
protocol and is making progress on its execution.
“We have now achieved the successful manufacturing of AMT-060 at
our Lexington facility at a scale sufficient to support pivotal
clinical trials and commercial supply,” stated Matt Kapusta, chief
executive officer of uniQure. “We have made significant
progress over the past several months to now be in a position where
we have developed a commercial-scale process and are evaluating our
completed batches to assess comparability. We look forward to
finalizing this work in anticipation of meetings with regulators to
further discuss plans to advance our hemophilia B program into a
pivotal study next year.”
As a result of this progress, the Company
expects to meet with the U.S. Food and Drug Administration and
European Medicines Agency in the early fall. At the same
time, uniQure expects to leverage its fully developed,
commercial-scale, manufacturing process to begin producing GMP
material associated with a Phase I/II trial of AMT-130 in
Huntington’s disease by the end of the year.
About Gene Therapy Manufacturing at uniQure
uniQure’s facility in Lexington, Massachusetts is one of the
largest, most versatile gene therapy manufacturing plants in the
world. uniQure made significant investments in designing,
constructing and equipping the 55,000-square foot facility with
state-of-the-art laboratories and commercial-scale, GMP
production capabilities to support all of its existing
programs, with flexibility to expand further.
uniQure produces its AAV-based gene therapies in its own
facilities with its proprietary manufacturing process. In
aggregate, uniQure owns seven AAV manufacturing patent families in
which 130 patents have been granted worldwide, and 55 applications
are pending, including in the U.S., Europe, Asia and South America.
A comprehensive summary of uniQure’s AAV manufacturing technology
is available on the Company’s website.
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 CNS,
liver/metabolic 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 and manufacture of
our gene therapy product candidates, the success of our
collaborations and the risk of cessation, delay or lack of success
of any of our ongoing or planned clinical studies and/or
development of our 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 Quarterly Report on Form 10-Q filed on
May 9, 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:
FOR INVESTORS:
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
FOR MEDIA:
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com
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