Context : Scientists in India have made significant progress in using gene therapy to treat haemophilia A.
Relevance: GS 3 (Science)
Successful Gene Therapy Trial.
- Trial conducted on five patients in Tamil Nadu, with none reporting bleeding episodes over an average follow-up of 14 months.
- Results published in the New England Journal of Medicine (NEJM).
Current Treatments.
- Involve frequent injections of clotting factors to prevent bleeding.
- Gene therapy offers a one-time solution, enabling the body to produce the missing clotting factor.
Haemophilia A basics.
- Caused by the absence of Factor VIII, a critical blood-clotting protein.
- India has the world’s second-largest haemophilia A patient population (40,000–100,000 individuals).
- Severe cases currently managed with expensive treatments like Factor VIII replacement and monoclonal antibodies.
Cost of Treatment.
- Managing haemophilia in India costs around $300,000 (₹2.54 crore) per patient over 10 years.
