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The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.


uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced that uniQure and CSL Behring have entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene dezaparvovec, uniQure鈥檚 investigational gene therapy for patients with hemophilia B. Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). Under the terms of the agreement, uniQure will receive a $450 million upfront cash payment and be eligible to receive up to $1.6 billion in payments based on regulatory and commercial milestones. uniQure will also be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percentage of net product sales arising from the collaboration.

The collaboration leverages CSL Behring鈥檚 strong global reach and commercial infrastructure in hematology to accelerate access of etranacogene dezaparvovec to hemophilia B patients around the world.

鈥淲e are thrilled to enter into this commercialization and license agreement with CSL Behring, an ideal commercial partner with global reach and decades of expertise in hemophilia,鈥 stated Matt Kapusta, chief executive officer of uniQure. 鈥淲e believe that through this arrangement, we are ideally positioned to deliver globally our innovative gene therapy to the largest number of hemophilia B patients as quickly as possible.鈥

As a CSL Limited聽company, CSL Behring is a global biotherapeutics leader delivering lifesaving medicines to patients with rare and serious diseases. A global leader in treating bleeding disorders, CSL Behring has been delivering innovations for the hemophilia patient community for more than 30 years. The company reported more than $1 billion in sales of hemophilia-related medicines in 2019.

鈥淥ur vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,鈥 said CSL鈥檚 CEO and Managing Director Paul Perreault. 鈥淲ith more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximize the potential benefit of this therapy. Upon approval, we believe this next-generation therapy will be highly complementary to our existing best-in-class hemophilia B product portfolio with an alternate best-in-class treatment option.鈥

Under the terms of the agreement, uniQure will be responsible for the completion of the HOPE-B pivotal study, manufacturing process validation, and the manufacturing supply of etranacogene dezaparvovec until such time that these capabilities are transferred to CSL Behring. Clinical development and regulatory activities performed by uniQure under the agreement will be reimbursed by CSL Behring. CSL Behring will be responsible for regulatory submissions and commercialization of etranacogene dezaparvovec.

The closing of the transaction is contingent on completion of review under antitrust laws in the United States, Australia and the United Kingdom.

Read the press release in its entirety here.

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