uniQure Announces the Successful Development and Scale-up of Manufacturing Processes for Its Hemophilia B Gene Therapy Program
(Thomson Reuters ONE) -
-- 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(at)uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder(at)uniQure.com
FOR MEDIA:
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: 21.07.2017 - 13:00 Uhr
Sprache: Deutsch
News-ID 553376
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contact information:
Town:
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Kategorie:
Business News
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