Scientists at the Christian Medical College (CMC) Vellore have achieved a significant milestone by conducting India’s inaugural human clinical trial of gene therapy for haemophilia A (FVIII deficiency), as announced by Union Minister Dr. Jitendra Singh during the “National Science Day 2024” program at Vigyan Bhawan in New Delhi on Wednesday.
Hemophilia A, a genetic disorder characterized by the absence or malfunction of clotting protein factor VIII (FVIII), leads to prolonged bleeding in affected individuals. The groundbreaking clinical trials employed an innovative approach utilizing a lentiviral vector to introduce a FVIII transgene into the patient’s hematopoietic stem cells. Subsequently, these modified cells expressed FVIII in specific differentiated blood cells.
India, hosting the second-largest population of individuals with Haemophilia, estimates around 1,36,000 individuals grappling with hemophilia A