- Biotech giant CSL (CSL) has closed its global commercialisation and licence agreement with uniQure for AMT-061
- Mid last year, CSL agreed to acquire the exclusive global licence rights to commercialise AMT-061 from uniQure for $656 million
- AMT-061 is uniQure’s novel gene therapy for the treatment of haemophilia B, a blood clotting disorder
- One dose of AMT-061 has shown to increase Factor IX plasma levels which is a blood clotting protein lacking in people with haemophilia B
- CSL has ended the day a slight 0.28 per cent in the red with shares trading at $276.89
Biotech giant CSL (CSL) has closed its global commercialisation and licence agreement with uniQure for AMT-061.
Mid last year, CSL agreed to acquire the exclusive global licence rights to commercialise AMT-061 from uniQure for $656 million.
AMT-061, or etranacogene dezaparvovec, is uniQure’s novel gene therapy for the treatment of haemophilia B (blood clotting).
It contains an AAV5 viral vector which has shown to be safe and well-tolerated in many clinical trials. AAV5 moderates gene transfer in an independent manner.
One dose of AMT-061 has shown to increase Factor IX plasma levels which is a naturally produced protein that forms blood clots.
Unfortunately, this protein is highly lacking in haemophilia B patients.
AMT-061 is currently undergoing phase three clinical trials.
“This agreement enables us to take forward a gene therapy, that, if approved, has the potential to transform the lives of haemophilia B patients,” CEO and Managing Director Paul Perreault said.
“Etranacogene dezaparvovec has the potential to be the first-ever gene therapy approved for haemophilia B and help CSL deliver on our ongoing commitment to improving the lives of those living with haemophilia B,” he added.
CSL has ended the day a slight 0.28 per cent in the red with shares trading at $276.89 in a $126.3 billion market cap.