Gene Therapy Treatment for Haemophilia | 13 Dec 2024
Indian scientists have developed a novel gene therapy for haemophilia A, offering a one-time treatment to replace frequent clotting factor injections.
- In a trial at Christian Medical College, Vellore, five patients have been free of bleeding episodes for over a year.
- Haemophilia A is a genetic disorder caused by insufficient Factor VIII, preventing proper blood clotting. India, with 40,000 to 100,000 affected patients, has the second-largest haemophilia population globally.
- Haemophilia A is inherited in an X-linked recessive pattern. Males with a defective X chromosome have hemophilia, while females need two defective X chromosomes to be affected.
- Current treatments are lifelong and expensive, costing up to Rs 2.54 crore over ten years, making gene therapy a cost-effective alternative.
- Gene therapy replaces defective genes in a patient’s cells with healthy ones.
- Roctavian, the only USFood and Drug Administration approved gene therapy, uses an adenovirus vector to deliver a gene for Factor VIII production in the liver but is not approved for children.
- The Vellore trial used a lentivirus vector, considered safer and potentially suitable for children, offering new possibilities for gene therapy in resource-constrained settings.
Read more: World Haemophilia Day