Hemgenix

Hemgenix (etranacogene dezaparvovec) is an advanced gene therapy used to treat adults with Hemophilia B, a rare genetic bleeding disorder caused by a deficiency of clotting factor IX. It works by delivering a functional copy of the F9 gene into liver cells, enabling the body to produce its own clotting factor naturally after a single intravenous infusion. As a result, patients may experience fewer bleeding episodes and reduced dependence on regular factor IX replacement therapy. This innovative treatment represents a major breakthrough in rare disease gene therapy, precision medicine, and modern biotechnology for long-term bleeding control.