Novel Gene Therapy to Provide Solution For Haemophilia

Syllabus: GS3/ S&T

In News

• Indian scientists have successfully tested gene therapy to treat severe hemophilia A, a rare hereditary condition that leads to spontaneous and potentially fatal bleeding episodes. 

What is Hemophilia A?

• A hereditary disorder caused by the absence or dysfunction of Factor VIII, a protein critical for blood clotting.
• Severe hemophilia A patients have less than 1% of the clotting factor, leading to frequent, spontaneous bleeding episodes.
• India has the world’s second-largest hemophilia patient pool, with an estimated 40,000–100,000 individuals affected.

Traditional Treatment

• Requires frequent injections of Factor VIII or other substitutes, making it expensive and burdensome.
• Estimated cost: ₹2.54 crore ($300,000) per patient over 10 years in India.

Gene Therapy as a One-Time Solution

• A therapeutic gene is introduced into the body, enabling it to produce sufficient levels of Factor VIII to prevent bleeding.
• The CMC Vellore trial used lentivirus vectors to integrate the clotting factor gene into the patient’s stem cells, avoiding the use of adenovirus vectors, which can trigger immune responses.
  • Lentivirus is a type of viral vector capable of transferring genetic material into host cells.

Accessibility and Affordability

• Developing gene therapy in India opens up the possibility of local manufacturing, which could significantly reduce costs and make the treatment more accessible to patients in India and other developing countries.

Source: TH