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.

Human Gene Therapy Trial for Haemophilia FAQs

Q1. What is gene therapy in the context of Haemophilia?
Ans. Gene therapy involves delivering a functional copy of the faulty gene responsible for Haemophilia to the patient’s liver cells to enable the production of clotting factors.

Q2. Which clotting factor is deficient in Haemophilia A?
Ans. Factor VIII is deficient in Haemophilia A, while Factor IX is deficient in Haemophilia B.

Source: PIB