uniQure Announces the Successful Development and Scale-up of Manufacturing Processes for Its Hemophilia B Gene Therapy Program
July 21 2017 - 7:00AM
-- Commercial-Scale Batches of
AMT-060 Successfully Produced in Accordance with Good Manufacturing
Practices in Lexington, MA Facility --
-- Manufacturing Process to be
Leveraged in Huntington's Program, With Production Expected to
Begin Before Year End --
LEXINGTON, Mass. and AMSTERDAM, the Netherlands,
July 21, 2017 (GLOBE NEWSWIRE) -- 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
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 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
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
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