(THE GIST OF PIB) Human Gene Therapy Trial for Haemophilia

(APRIL-2025)

BRIC-inStem, Bengaluru, has successfully conducted India’s first-in-human gene therapy trial for Haemophilia in collaboration with CMC Vellore.

Gene therapy is a biomedical technique that involves modifying or replacing faulty genes in a person’s cells to treat or prevent diseases.
The primary objective is to introduce a functional gene to compensate for a mutated or disease-causing gene.
Gene therapy approaches include:
Replacing a mutated gene with a healthy copy.
Inactivating a malfunctioning gene.
Introducing a completely new gene into the body.
Unlike conventional medicines, gene therapy targets the root genetic causes inside cells, rather than just addressing symptoms.

Ex vivo modification of hematopoietic stem cells and T-lymphocytes.
In vivo gene delivery or use of gene-editing reagents directly into the patient’s body.

Haemophilia is a rare genetic bleeding disorder where blood clotting is impaired due to mutations in genes encoding clotting proteins.
These genes are located on the X chromosome, making males more susceptible.
It affects about 1 in 10,000 people, with India having a significant patient load.

BRIC-inStem, part of the Biotechnology Research and Innovation Council (BRIC), integrates 14 autonomous research institutions under one national umbrella.

It has pioneered translational and regenerative research, including:

Gene therapy
Anti-viral germicidal masks (developed during COVID-19)
‘Kisan Kavach’ pesticide shield for farmers
Biosafety Level III Lab at BRIC-inStem is essential for studying high-risk pathogens under the One Health Mission.