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 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 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.
