uniQure Strengthens Intellectual Property Portfolio with Acquisition of Patent Family Providing Broad Protection of the Hyperactive Padua Variant of Factor IX (FIX-Padua)

  • Issued Patent in the U.S. Covers Use of FIX-Padua in Gene Therapy
  • Divisional patent applications filed to further strengthen Padua intellectual property position

LEXINGTON, Mass. and AMSTERDAM, the Netherlands (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the Company has acquired a patent family, with claims issued in the U.S., that broadly covers a hyperactive variant of Factor IX carrying an R338L mutation (often referred to as "FIX-Padua") and its use in gene therapy for the treatment of coagulopathies, including hemophilia B. This patent family was acquired from the inventor, Professor Paolo Simioni, a renowned hemophilia expert at the University of Padua, Italy, who is widely recognized as the first to identify this mutant.

Professor Simioni filed a PCT application on September 15, 2009, and patent applications are pending in the U.S., Europe, and Canada. The U.S. Patent and Trademark Office issued U.S. Patent 9,249,405 on February 2, 2016, which includes claims directed to Factor IX protein with a leucine at the R338 position of the protein sequence, nucleic acid sequences coding for this protein, and therapeutic applications, including gene therapy. Additional fast track divisional patent applications have also been filed in the U.S. and in Europe that would further strengthen uniQure's intellectual property position.

"I have worked my entire career in the field of coagulopathies, and in my experience the innovation of FIX-Padua holds great promise for the treatment of hemophilia B," said Professor Simioni. "Combined with the known safety profile of the AAV5 vector, this gene has the potential to significantly improve the health of hemophilia B patients.  I look forward to working with uniQure as they advance what I believe will be the most effective gene therapy for patients suffering from hemophilia B."

Professor Simioni is serving as advisor and consultant to uniQure for the development of therapeutic products using his invention of FIX-Padua. He will assist in the Company's discussions with regulators, investigators, and key opinion leaders throughout the clinical development of AMT-061.

"uniQure is very pleased to have Professor Simioni, a leading expert in hemophilia and FIX-Padua, as a collaborator and to have acquired his patents on the invention of FIX-Padua for gene therapy," said Jonathan Garen, chief business officer of uniQure. "These patents provide uniQure with an enhanced proprietary position in hemophilia B gene therapy that we can further leverage for additional patent protection."