India Conducts First Human Gene Therapy Trial for Hemophilia.

National | Dated: 05 May 2025

India achieved a significant milestone in medical science in April 2025, by conducting its first human gene therapy trial for haemophilia.

🎯 Key Highlights:

  • - The trial was carried out through a collaboration between Bengaluru-based Biotechnology Research and Innovation Council's (BRIC's) Institute for Cell Science and Regenerative Medicine (BRIC-inStem) and Christian Medical College (CMC) Vellore in Vellore, Tamil Nadu (TN).
  • - This achievement was announced by Union Minister Jitendra Singh, Ministry of Science and Technology (MoS&T), during his inspection of facilities at BRIC-inStem.
  • - Haemophilia is a genetic blood disorder where blood does not clot normally, leading to abnormal bleeding.

💡 Other Important Facts:

  • (i) In this trial, patients with severe haemophilia A received autologous Hematopoietic Stem Cells (HSCs) that were genetically modified using lentiviral vectors to express the Factor VIII gene.
  • (ii) These modified cells were transplanted back into the patients, enabling their bodies to naturally produce the necessary clotting factor.
  • (iii) India’s Biotechnology sector experienced remarkable growth, expanding 16-fold to reach USD 165.7 billion in 2024, with a target to reach USD 300 billion by 2030.

📚 Test Your Knowledge:

Who inaugurated India's first human gene therapy trial for haemophilia?

Correct Answer: Dr. Jitendra Singh

🚀 Quick Recap: