CSL Behring in $450m spend on hemophilia gene therapy

By Ben Hargreaves

- Last updated on GMT

(Image: Getty/Vladimir_Timofeev)
(Image: Getty/Vladimir_Timofeev)

Related tags CSL Behring Hemophilia Gene therapy

The company buys the global rights to a gene therapy currently going through Phase III trials.

CSL Behring agreed a deal with uniQure for the exclusive global rights to AMT-061 (etranacogene dezaparvovec), a gene therapy being tested for the treatment of hemophilia B.

To gain access to the gene therapy program, CSL Behring will pay $450m (€400m) upfront, as well as regulatory and commercial milestone payments to uniQure, in addition to royalties.

Despite now having a major stake in the program, CSL Behring has agreed that uniQure will be responsible for completing Phase III trials and for the scale up of the manufacture of the gene therapy for ‘early’ commercial supply.

Previous clinical trials have shown that a single dose of the adeno-associated virus gene therapy is able to increase levels of Factor IX, which is the blood clotting protein lacking in people with hemophilia B.

As a result, patients receiving the therapy saw a reduced tendency for bleeding. In Phase IIb trials, all patients receiving the gene therapy showed stabilized and sustained Factor IX activity at higher levels one year after being dosed.

The product, if approved, would slot neatly into CSL Behring’s product portfolio, which is predominantly made up of products in hematology and immunology.

Alongside this gene therapy candidate, the company is also developing a stem cell gene therapy (CSL200) for the treatment of sickle cell disease.

Last month, CSL Behring managed to free up capital by leasing its future site in Switzerland to Thermo Fisher Scientific​.

The deal will see Thermo Fisher responsible for manufacturing CSL Behring’s hemophilia products.

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