Human Gene Therapy Trial for Haemophilia

Human Gene Therapy Trial for Haemophilia Latest News

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

About Gene Therapy

• 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.
• Current clinical trials focus on both inherited and acquired disorders, using methods such as:
  • 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.

About Haemophilia

• 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.

About BRIC-inStem

• 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.

Source: PIB