Australian-developed gene therapy for haemophilia B is safe and effective

Embargoed until: Publicly released: 2025-04-17 07:00

Australia; NSW

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An Australian-led trial has found that a gene therapy treatment for haemophilia B remains safe and effective three to six years after treatment.  The gene therapy, called fidanacogene elaparvovec, uses a harmless virus to carry a corrective gene into the liver. In the study, 15 people with severe or moderately severe hemophilia B received the gene therapy and were followed for 3-6 years. The researchers say the overall safety results suggest that the gene therapy is generally safe and the patients' disease levels went from severe or moderate to mild and remained at that level, with a low annual rate of bleeds.