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

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, July 18, 2017 -- 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
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Eva M. Mulder
Direct: +31 20 240 6103
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FOR MEDIA:

Tom Malone
Direct: 339-970-7558
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