Ground-breaking gene therapy trial for haemophilia A

People with haemophilia A are born with a mutation in the gene responsible for making Factor VIII, the protein needed for blood clotting. This leads to uncontrolled bleeding episodes, crippling joint disease, and increased risk of death.

In 2019, Prof Rasko, along with international colleagues, performed a ground-breaking gene therapy clinical trial for hemophilia A. This involves engineering a virus to deliver a working copy of the gene into the body. This work was published today in the prestigious New England Journal of Medicine.

The trial found 91.5 per cent of participants had reduced bleeds and 96.4 per cent had fewer Factor VIII infusions. This work brings us one step closer to a cure.

Prof. Rasko on Channel 9 news - Watch here!

Read the New England Journal of Medicine article entitled - Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.

For more on this cutting-edge work, read this RPA hospital facebook post and watch the Channel 9 news story.