The Supply of a Factor IX Gene Therapy Product

factor concentrate which is given intravenously on a regular weekly schedule (prophylaxis). The current standard of care for people with severe or moderately-severe factor IX deficiency in Ireland is that all patients are treated with factor IX prophylaxis. Despite regular prophylaxis, certain pati

ents do not achieve adequate bleed control and require increased amounts of FIX replacement leading to higher-than-average FIX consumption. In addition, some patients have difficulty with venous access meaning that an intravenous treatment