In a landmark achievement, Indian scientists have successfully trialed a novel gene therapy for severe Hemophilia A, potentially eliminating the need for frequent and costly clotting factor injections. The trial, conducted by Dr. Alok Srivastava and his team at the Centre for Stem Cell Research (CSCR), Christian Medical College, Vellore, involved five patients who reported no bleeding episodes over 14 months. The study was recently published in the prestigious New England Journal of Medicine (NEJM).
Hemophilia A, a hereditary condition caused by a deficiency of Factor VIII, affects up to 100,000 patients in India. Severe cases require regular and expensive Factor VIII replacement therapies, costing an estimated ₹2.54 crore per patient over a decade. This gene therapy, funded by the Union Department of Biotechnology, uses lentiviral vectors to introduce a corrective gene, enabling patients’ bodies to produce Factor VIII on their own.
“This ground-breaking study is notable for several reasons,” said Dr. Johny Mahlangu in an editorial in NEJM. “It demonstrates the feasibility of conducting advanced gene therapy research in resource-constrained settings like India and has the potential to reduce costs and increase accessibility globally.”
The approach, considered safer than using adenoviral vectors, may also extend treatment access to children and eliminate the need for immunosuppressive therapy. This breakthrough positions India as a hub for affordable and innovative gene therapy solutions.
