Haemophilia Gene Therapy Trial

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  • India’s First Gene Therapy Trial for Haemophilia: BRIC-inStem, Bengaluru, in collaboration with CMC Vellore, successfully conducted India’s first-in-human gene therapy trial for Haemophilia.
  • Gene Therapy Basics: Gene therapy modifies or replaces faulty genes to treat or prevent diseases, addressing the root genetic cause instead of just symptoms. It involves replacing, inactivating, or introducing new genes.
  • Haemophilia Explained: Haemophilia is a genetic bleeding disorder due to mutations in genes encoding clotting proteins, primarily affecting males.
  • BRIC-inStem’s Role: BRIC-inStem integrates 14 research institutions and pioneers translational research, including gene therapy. They also developed solutions like anti-viral masks and pesticide shields.
  • Minister’s Commendation: Dr. Jitendra Singh hailed the gene therapy trial as a milestone and emphasized biotechnology’s role in nation-building and the future economy.
  • Biotech Sector Growth: India’s biotechnology sector has grown significantly, with a vision to reach $300 billion by 2030, supported by policy reforms like the BIO-E3 Policy.
  • Startup Ecosystem: The number of biotech startups has increased dramatically, highlighting the sector’s dynamism.
  • Biosafety
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    Lab:     BRIC-inStem’s Biosafety Level III lab is crucial for studying high-risk pathogens under the One Health Mission, enhancing pandemic preparedness.
  • Focus on Birth Defects: The newly launched CReATE addresses birth defects and infertility through developmental biology research.
  • Call for Collaboration: Dr. Singh urged greater collaboration between scientific and medical institutions.
  • Bio-Driven Economy: India’s future economy will be bio-driven, with institutions like BRIC-inStem leading the transformation.
  • Gene therapy in Haemophilia Gene therapy in
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    Haemophilia involves delivering a functional copy of the faulty gene to the patient’s liver cells to enable the production of clotting factors.
  • Clotting factor Factor VIII is deficient in Haemophilia A, while Factor IX is deficient in Haemophilia B.