
In an unexpected twist, CSL buys worldwide rights to uniQure's PhIII hemophilia B gene therapy in $2B-plus deal
In a surprise move, CSL Behring reported after the market closed on Wednesday that it is acquiring uniQure’s Phase III hemophilia B gene therapy with a $2 billion-plus deal fronted with $450 million in cash.
The money — which includes $1.6 billion in milestones — gives the Australian pharma giant global rights to the pivotal stage AMT-061 (etranacogene dezaparvovec), an AAV5 viral vector carrying a gene cassette with the Padua variant of Factor IX, which hemophilia B patients lack. Top-line Phase III data are expected before the end of this year from the HOPE-B study.
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