Loma Linda University Health has made a groundbreaking discovery in gene therapy for hemophilia B, alongside the development of "dirloctocogene samoparvovec," an advanced second-generation gene therapy for hemophilia A.
These breakthroughs have the potential to transform the lives of numerous children and adults suffering from these debilitating conditions.
Hemophilia, a genetic disorder that impairs the blood's ability to clot, has long been life-altering for those affected. Minor injuries can pose serious health risks due to uncontrolled bleeding. The disorder is typically inherited through the X chromosome, primarily affecting males while females are usually carriers.
Traditionally, hemophilia treatment has required patients to undergo frequent infusions of clotting factors, often involving multiple self-injections each week to prevent dangerous bleeding episodes. This regimen can be both painful and restrictive.
HEMGENIX, a novel treatment, enables patients' bodies to produce their own missing clotting factors. This single-session therapy involves a three-hour infusion that delivers a modified gene to the liver, facilitating the natural production of clotting factors.
