Gene Therapy for Hemophilia A - Patient Education

This educational video describes an investigational gene therapy for hemophilia A cleared exclusively for clinical trials in the U.S. by the FDA, Food and Drug Administration. Hemophilia A is a rare genetic disorder caused by defects in a gene that is essential for the production of the clotting protein factor VIII. Lack of factor VIII leads to uncontrolled bleeding. ASC Therapeutics is investigating a second-generation gene therapy that, in extensive preclinical studies, showed a significant increase in the secretion of factor VIII from the liver into the bloodstream, potentially reducing the dose of gene therapy treatment and increasing durability.

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