CSL buys gene therapy treatment for haemophilia

Biotechnology company CSL has completed the acquisition of a novel gene therapy for the treatment of haemophilia B from NASDAQ listed uniQure.

The company will pay US$450 million initially with further milestone payments and royalties to follow as the product etranacogene dezaparvovec (AMT-061) is further developed and commercialised.

uniQure will complete the Phase three trial already underway and scale up for manufacture of initial commercial supplies while CSL Behring will handle regulatory submissions and commercialisation.

The closing comes following completion of antitrust reviews of the purchase in Australia, the US and the UK.

AMT-061 works by delivering a gene variant which stimulates the production of the blood clotting agent, Factor IX.

Haemophilia B is less common that haemophilia B and is either hereditary or the result of genetic mutation.

CSL CEO Paul Perreault said the purchase built on the company’s legacy of lifesaving innovation in haemophilia.

“This agreement enables us to take forward a gene therapy that, if approved, has the potential to transform the lives of haemophilia B patients.

“Etranacogene dezaparvovec has the potential to be the first-ever gene therapy approved for haemophilia B.”

Picture: CSL

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