Hemophilia is a rare disorder in which blood doesn’t clot in the typical way because it doesn’t have enough blood-clotting proteins (clotting factors).
The two most common types of hemophilia are A and B.
Hemophilia A is the most common type of hemophilia and is caused by a lack of blood clotting factor VIII, whereas hemophilia B is caused by a lack of factor IX (FIX).
The more severe the condition, the less able a person is to form blood clots, making them more vulnerable to bleeding.
ABOUT GENE THERAPY
In general, gene therapies are an innovative approach to treating medical conditions by introducing a functional, or working, gene into the body or by turning off or changing the gene that is causing the condition.
Current treatments for hemophilia work to temporarily replace or supplement low levels of blood-clotting factor. But, gene therapy has the potential to restore near-normal blood clotting ability.
THROUGH A ONE-TIME INFUSION,
gene therapy aims to enable patients to create their own factor, potentially delivering lasting bleed protection for years. Gene therapy can potentially provide long-term benefits of sustained factor activity levels from a single administration of treatment, potentially reducing or even eliminating the need for regular, long-term prophylaxis.