Context

In a significant stride for medical science, Indian researchers have achieved ground breaking advancement in the treatment of Haemophilia A through gene therapy. This achievement not only marks a pivotal moment in the global fight against genetic disorders but also underscores India's growing prominence in the biotechnology sector.

About Haemophilia A

Haemophilia A is a rare genetic disorder characterized by a deficiency in clotting Factor VIII, leading to spontaneous and prolonged bleeding episodes.

·    This condition significantly impairs the quality of life and poses treatment challenges, especially in resource-limited settings.

·     India, with approximately 136,000 individuals affected, bears the second-highest global burden of Haemophilia.​

Breakthrough Gene Therapy:

Scientists at the Centre for Stem Cell Research (CSCR) at Christian Medical College (CMC), Vellore, in collaboration with the Biotechnology Research and Innovation Council's Institute for Stem Cell Science and Regenerative Medicine (BRIC-inStem) Bengaluru, have developed the country's first human gene therapy for Haemophilia A.

·    This innovative therapy utilizes lentiviral vectors to introduce a functional copy of the Factor VIII gene into patients' blood stem cells. The modified cells are then transplanted back into the patients, enabling them to produce the clotting factor naturally.

·    The trial involved participants aged between 22 and 41 years. Over a cumulative follow-up period of 81 months, all participants exhibited zero annualized bleeding rates and sustained production of Factor VIII, eliminating the need for regular infusions.

Significance and Implications

This development holds profound implications for public health and the biotechnology sector in India especially in nation-building and the transformative potential of biotechnology in shaping India's economy and public health infrastructure.

India’s Biotechnology sector has witnessed remarkable growth, expanding 16-fold over the past decade to reach $165.7 billion in 2024, with projections of $300 billion by 2030.​

Challenges and Future Prospects

Despite the promising results, challenges remain in scaling this therapy for widespread use. The high cost of gene therapy, limited access to specialized medical facilities, and the need for advanced infrastructure pose significant barriers.

Addressing these challenges requires concerted efforts in policy-making, healthcare infrastructure development, and public-private partnerships.​

Looking ahead, the success of this gene therapy opens avenues for further research and development in the treatment of other genetic disorders. With on-going support from the Department of Biotechnology and other stakeholders, India is poised to become a global leader in biotechnology innovation, offering hope to millions affected by genetic diseases.

Conclusion

This breakthrough in gene therapy for Haemophilia A is not just a scientific achievement but a testament to India's growing capabilities in biomedical research and its commitment to improving public health outcomes.