India Conducts First Human Gene Therapy Trial for Hemophilia.

National

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


      - 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.

Main Point :-   (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.

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