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SVac based therapeutics promise a long-term solution to hemophilia"

Hematology

Hemophilia comprises a number of genetic disorders that stop the blood clotting properly, leading to uncontrolled bleeding. Worldwide, an estimated 500,000 people live with hemophilia. The two most common types are hemophilia A and B. Although the two types have very similar symptoms, they are caused by mutations in different genes. Hemophilia A is caused by a mutation in the Factor IX gene, whereas hemophilia B is caused by mutations in the Factor VIII gene.

There is currently no cure for hemophilia. The disease can be managed through regular infusions of the blood clotting factors which are missing. However, a significant number of patients develop an immune response to the infused factors, rendering the therapy ineffective.

Amarna is developing SVac vector particles encoding activated blood clotting Factor VII (SVFVIIa). Expression of activated Factor VII in hemophiliacs to overcome Factor VIII and IX deficiencies can be used to treat hemophilia A and B. Unlike the currently used therapeutics, SVFVIIa secures a long-lasting therapeutic effect in the blood and restores the blood clotting process to normal levels.