Gene therapy shows positive results for patients with haemophilia

Photo: Colourbox
In January 2020, the first gene therapy treatment for haemophilia was administered at Skåne University Hospital as part of an international study. The results thus far have been exclusively positive, with all patients responding favourably to the treatment. Approval processes are now underway worldwide for the drugs.

Gene therapy for haemophilia involves replacing the defective gene with healthy genetic material, which is delivered to the patient's liver cells via a carrier molecule composed of a specific virus. The treatment is administered through a drip, enabling the virus vector to target the intended cell.

Within the cell, the viral envelope is shed, allowing the gene to commence production of the missing coagulation factor, be it factor 8 for haemophilia A or factor 9 for haemophilia B.