CSL Behring has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy program, AMT-061 (etranacogene dezaparvovec), for the treatment of hemophilia B from uniQure. The AMT-061 program, currently in Phase III trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B.
One dose of AMT-061 has shown to increase Factor IX (FIX) plasma levels – the blood clotting protein lacking in people with hemophilia B – to a degree that reduces or eliminates the tendency for bleeding for many years. Should AMT-061 be successful, appropriate candidate hemophilia B patients would be able to have a one-time treatment to restore FIX activity to functional levels capable of eliminating the need for frequent and ongoing replacement therapies.
uniQure will receive an upfront payment of $450 million followed by regulatory and commercial sales milestones and royalties. Under the terms of the agreement, uniQure will complete the Phase III trial and scale up manufacture for early commercial supply under an agreement with CSL Behring. The transaction is subject to customary closing conditions.
This acquisition expands CSL's capabilities in its gene therapy portfolio. The company is currently developing a stem cell gene therapy (CSL200) for the treatment of sickle cell disease and has recently established an alliance with Seattle Children's Research Institute to develop a stem cell gene therapy for primary immunodeficiency diseases.