uniQure Expands Intellectual Property Portfolio with Newly Issued Patent Providing Broad Protection

uniQure Expands Intellectual Property Portfolio with Newly Issued Patent Providing Broad Protection of Insect Cell-Based AAV Manufacturing

ID: 552791

(Thomson Reuters ONE) -


Patent Covers Overlapping Rep78/52 Nucleotide Sequences for Robust, Large-Scale
Production of AAV Constructs

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, July 18, 2017 (GLOBE NEWSWIRE)
-- uniQure N.V. (Nasdaq:QURE), a leader in human gene therapy and AAV
manufacturing, today announced that the United States Patent and Trademark
Office (USPTO) has granted a patent for a proprietary invention by uniQure which
broadly covers an important component of insect cell-based AAV manufacturing
technology. The USPTO issued patent number US 9,708,627, a member of one of
uniQure's Hermens patent families, with claims covering the expression of both
Rep78 and Rep52 proteins from a Rep78 nucleic acid sequence in insect cells.

The newly issued Hermens '627 patent significantly expands uniQure's leading
intellectual property portfolio related to large-scale, highly reproducible
manufacturing of AAV in insect cells. This patent, which broadens earlier claims
granted in this patent family, is based on years of research by uniQure
scientists focused on enhancing the genetic stability of the Rep78/52 encoding
sequences used to produce AAV vectors in insect cells. The technology covered in
the Hermens '627 patent family is currently widely applied in insect cell-based
AAV manufacturing.

"As an early pioneer of insect cell-based AAV manufacturing, we have continued
to innovate and protect all aspects of our proprietary technology that are
essential for the efficient and large-scale production of AAV vectors for
commercial use," stated Jonathan Garen, chief business officer for uniQure.
"With the issuance of this patent, we have significantly increased the value of
our patent portfolio and advanced our leadership in the manufacturing of AAV-
based gene therapies."

uniQure's intellectual property portfolio for its manufacturing platform




includes multiple fundamental molecular and process-related patents and
extensive know how covering essential production, purification, and processing
steps that are necessary for the large-scale insect cell-based manufacturing and
for compliance with the regulatory authorities.

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 Gene Therapy Manufacturing at uniQure
uniQure produces its AAV-based gene therapies in its own facilities with its
proprietary manufacturing process, which uses insect cells and baculoviruses, a
common family of viruses found in invertebrates. The Company's manufacturing
capabilities enable it to be an attractive collaborator for academic research
institutions and biotechnology and pharmaceutical companies seeking to advance
their programs into larger, late-stage clinical trials that require commercial-
scale manufacturing.

uniQure's facility in Lexington, Massachusetts is one of the largest, most
versatile gene therapy manufacturing plants in the world.  uniQure invested more
than $25 million in designing, constructing and equipping the 55,000-square foot
facility with state-of-the-art laboratories and commercial-scale
production capabilities. The facility offers 500-liter capacity with the ability
to expand to up to 2,000 liters when needed.

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 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 2016 Annual
Report on Form 10-K filed 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:

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




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Bereitgestellt von Benutzer: hugin
Datum: 18.07.2017 - 13:00 Uhr
Sprache: Deutsch
News-ID 552791
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