uniQure Announces the Issuance of New Patents Providing Broad Protection of the Padua Variant of Factor IX in Gene Therapy

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uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, such as hemophilia B, Huntington's disease and congestive heart failure, today announced that the U.S. Patent and Trademark Office has granted Patent Number 9,982,248 which broadly covers methods of treating coagulopathies (bleeding disorders), including hemophilia B, using AAV gene therapy with nucleic acid encoding the hyperactive Factor IX (FIX) Padua variant. This is the second U.S patent to issue, along with the previously issued U.S. Patent Number 9,249,405, in a patent family that uniQure acquired from the inventor, Professor Paolo Simioni, a renowned hemophilia expert at the University of Padua, Italy.

"We are pleased with the ongoing progress related to our FIX-Padua patent portfolio," said Jonathan Garen, chief business officer at uniQure. "Our new U.S. patent, which was prosecuted by uniQure and granted after consideration of the prior art, further confirms the patentability in the U.S of Professor Simioni's groundbreaking invention. We believe this patent significantly strengthens our intellectual property portfolio and covers the use of the FIX-Padua variant in AAV gene therapy to treat hemophilia B."

The Factor IX Padua variant is a Factor IX protein carrying a leucine at the R338 position, often called the "FIX-Padua" or "Padua mutant". The newly issued claims of the U.S. '248 patent focus on the use of AAV gene therapy with FIX-Padua to treat hemophilia B and other bleeding disorders. The previously issued U.S. '405 patent acquired from Professor Simioni in 2017 covers compositions of FIX-Padua nucleic acids and polypeptides (proteins), as well as their therapeutic uses.

In addition to the U.S. patent, on February 20, 2018 the Canadian Intellectual Property Office granted Patent Number 2,737,094, which broadly covers FIX-Padua nucleic acids for use in gene therapy and FIX-Padua polypeptides for use in FIX replacement therapy. uniQure also is currently pursuing European patents directed toward therapeutic uses of FIX-Padua nucleic acids and polypeptides.

Expanding Intellectual Property Portfolio in Manufacturing

uniQure also announced today the issuance of a new patent further strengthening the Company's intellectual property related to its proprietary insect cell-based AAV manufacturing process. U.S. Patent Number 9,840,694 includes claims covering nanofiltration to selectively remove potential residual baculovirus from the product. The Company believes this nanofiltration step is important for product quality and safety and that nanofiltration generally may be required to comply with viral clearance standards established by global regulatory authorities. Related patents were previously granted in Europe, Japan and several other jurisdictions.

The '649 patent expands uniQure's significant intellectual property portfolio directed to large-scale, highly-reproducible manufacturing of AAV in insect cells using baculovirus vectors. The portfolio includes multiple important molecular and process-related patents as well as 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.