CSL Behring strengthens gene therapy ambition in $450m deal

UniQure will continue manufacturing hemophilia gene therapy candidate AMT-061 after selling the licensing rights to CSL Behring.

CSL Behring has paid uniQure $450 million for the global rights to commercialize the adeno-associated virus (AAV) gene therapy candidate AMT-061 (etranacogene dezaparvovec), in late stage studies for the treatment of hemophilia B. UniQure could be entitled to up to $1.6 billion depending on the progress of the candidate.

The addition to CSL Behring supports the firm’s advanced therapy ambitions, according to company spokesperson Natalie de Vane.

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“As a company we are growing our capabilities in the gene and cell therapy, as it is one of our strategic research platforms,” she told this publication. “AMT-061 for hemophilia B would join CSL200 for sickle cell disease and the company’s recent strategic alliance with Seattle Children’s Research Institute to accelerate the development of stem cell gene therapies for primary immunodeficiency diseases.”

CSL Behring added the CSL200 program through its acquisition of Calimmune in 2017 for $91 million. The candidate is an ex vivo gene therapy, meaning the product aims to work by removing cells from the body and treating them before injecting them back into the patient.

“AMT-061 is an example of in vivo gene therapy,” de Vane explained. “In this case, the cells are not removed from the body. Instead, the new gene is inserted into the cells within the body. The new gene allows the cells to produce the clotting factor they previously could not make due to an inherited dysfunctional gene. So AMT-061 would diversify our portfolio.”

Initially, AMT-061 will continue to be manufactured at uniQure’s Lexington, Massachusetts, site, she confirmed. No future manufacturing plans have been confirmed.

The acquisition of AMT-061 puts CSL Behring in the mix of companies vying to commercialize gene therapies for hemophilia. Valoctocagene roxaparvovec, a BioMarin Pharmaceutical gene therapy developed to treat the more common hemophilia A, is expected to receive an FDA decision by late August. Meanwhile, Pfizer and partner Sangamo Therapeutics last week reported positive Phase I/II results for their hemophilia A gene therapy, giroctocogene fitelparvovec.