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@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