India’s First Gene Therapy Trial for Haemophilia Completed

BRIC-inStem, based in Bengaluru, in collaboration with CMC Vellore, has successfully completed India’s inaugural human gene therapy trial for Haemophilia—a significant milestone in the country’s medical research.

Understanding Gene Therapy

Gene therapy is an advanced biomedical strategy aimed at correcting or replacing defective genes to treat or prevent diseases. Instead of managing symptoms, this method tackles the root genetic cause within cells.
Key approaches to gene therapy include:

• Replacing faulty genes with healthy versions
• Deactivating genes that function improperly
• Adding entirely new genes to aid in treatment

Current clinical trials explore its potential in both inherited and acquired diseases, using techniques such as:

• Modifying hematopoietic stem cells and T-lymphocytes outside the body (ex vivo)
• Delivering gene-editing tools or therapeutic genes directly into the patient (in vivo)

About Haemophilia

Haemophilia is a rare inherited disorder that disrupts the blood’s ability to clot due to mutations in the genes responsible for clotting proteins. These genes are found on the X chromosome, making males predominantly affected. It occurs in approximately 1 in 10,000 individuals, with India home to a substantial number of cases.

About BRIC-inStem

BRIC-inStem operates under the Biotechnology Research and Innovation Council (BRIC), bringing together 14 autonomous institutions into a single national framework. The institute leads cutting-edge research in areas such as:

• Gene therapy
• Regenerative medicine
• Anti-viral protective masks (notably during the COVID-19 pandemic)
• ‘Kisan Kavach’—a pesticide safety solution for farmers

Its state-of-the-art Biosafety Level III laboratory plays a crucial role in researching high-risk pathogens as part of the One Health initiative.