CSL cracks the genetic code of haemophilia B treatment
At the launch of the world’s first gene therapy for haemophilia B in late 2022, there were tears.
Patients had waited decades for the therapy named Hemgenix – created by local biotech giant CSL and uniQure – which promised to give them more freedom than they had ever known.
The CSL therapy has won a special award for Best Product Innovation in the AFR BOSS Most Innovative Companies list.
Before Hemgenix, people with the rare lifelong bleeding disorder (caused by a single gene defect) had to receive prophylactic infusions of a protein called factor IX. People with the condition do not produce enough of the protein naturally and it is infused twice-weekly, or fortnightly.
CSL’s head of research and development Bill Mezzanotte says Hemgenix is life-changing for haemophilia b patients. James Brickwood
The impact of the disorder on their lifestyles was significant, affecting their ability to work, travel and live unencumbered.
But with Hemgenix, that frequent infusion schedule becomes a one-off treatment, which restores their factor IX activity in their blood to functional levels, eliminating the need for frequent replacement therapies.
“You can’t even measure the difference in a person’s life taking one treatment and not need to think abut it for at least 10 to 20 years [versus multiple times a week],” CSL’s head of R&D Bill Mezzanotte told The Australian Financial Review following the US Food and Drug Administration approval of the therapy in November last year.
“It’s not just their convenience, it’s their sense of mental security, and of course, it saves the healthcare system a lot of money in both the cost of therapy, rescue therapies, the cost of treating a bleed and also the lost productivity of these patients.”
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