India Achieves Breakthrough in Gene Therapy for Hemophilia A

India has achieved a significant breakthrough in treating severe Hemophilia A through its first-in-human gene therapy using lentiviral vectors, conducted by the Centre for Stem Cell Research (CSCR) at Christian Medical College, Vellore. Five participants in the trial experienced zero bleeding episodes and prolonged production of Factor VIII, eliminating the need for repeated infusions. This innovative therapy addresses the root cause of Hemophilia A, a genetic disorder affecting around 136,000 individuals in India, by delivering a functional gene into the patients' blood stem cells. The results, published in the New England Journal of Medicine, indicate a transformative step forward in the management of this condition, which traditionally relies on costly and invasive treatments. Gene therapy offers hope amidst challenges like high treatment costs and difficulties in accessing veins for infusions, particularly in children. This advancement not only promises improved quality of life for patients but also paves the way for future treatments for rare genetic disorders.