Pharma Focus Asia

Loma Linda University Health Developed First-of-its-kind Gene Therapy for Hemophilia

Loma Linda University Health has achieved a groundbreaking milestone with the development of a pioneering gene therapy for hemophilia B, complemented by dirloctocogene samoparvovec, an advanced second-generation therapy for hemophilia A.

HEMGENIX represents a groundbreaking treatment that enables patients to naturally produce vital clotting factors. This therapy involves a single three-hour infusion, during which a modified gene is delivered directly into the liver, allowing for continuous production of these essential factors.

This breakthrough holds immense promise in profoundly improving the quality of life for countless children and adults grappling with these debilitating conditions.

Hemophilia, a genetic disorder impairing the blood's clotting ability, has long presented significant challenges for those affected, turning even minor injuries into potentially life-threatening situations due to uncontrollable bleeding.

Typically inherited through the X chromosome, hemophilia predominantly affects males, with females often serving as carriers. Managing the disorder traditionally involves frequent infusions of clotting factors, often requiring multiple self-injections each week to prevent hazardous bleeding episodes. This treatment regimen can impose considerable pain and limitations on patients' daily lives.
 

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