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India Takes a Leap in Medical Science: First Human Clinical Trial of Gene Therapy for Haemophilia A Conducted

In a groundbreaking milestone for medical science, India has achieved a remarkable feat by conducting the first human clinical trial of gene therapy for haemophilia A (FVIII deficiency). The pioneering trial took place at the prestigious Christian Medical College (CMC) in Vellore, marking a significant advancement in the country’s healthcare landscape.

The announcement was made by Union Science & Technology Minister Dr. Jitendra Singh during his address at the prestigious “National Science Day 2024” programme held at Vigyan Bhavan in New Delhi. Dr. Singh revealed that the trial, a collaboration between the Department of Biotechnology, the Centre for Stem Cell Research (a unit of InStem Bengaluru), and Emory University, USA, showcased the deployment of cutting-edge technology using a lentiviral vector to express a FVIII transgene in the patient’s own haematopoietic stem cell.

Expressing optimism, Dr. Singh highlighted the potential commencement of vector manufacturing in India, paving the way for further clinical trials and potentially revolutionizing treatment for haemophilia A patients.

The event, commemorating the discovery of the “Raman Effect” by Nobel Laureate Sir CV Raman, saw Dr. Singh underscore India’s commitment to scientific progress. He emphasized Prime Minister Shri Narendra Modi’s vision, aligning with Raman’s belief that science is the key to India’s advancement. Under PM Modi’s leadership, India has witnessed remarkable growth in its scientific and technological capabilities, with the bio-economy soaring from $10 billion in 2014 to over $130 billion in 2024.

Dr. Singh also lauded India’s prowess in advanced technologies, asserting the nation’s readiness to compete globally in Quantum Technology, Artificial Intelligence, and Machine Learning. He emphasized the increasing role of women scientists, empowered under PM Modi’s initiatives, and expressed confidence in the Anusandhan National Research Foundation as a catalyst for research and development.

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The event witnessed the unveiling of a compendium of SATHI Clusters and the release of a White paper on ‘Evolution: Catalysing technology led ecosystem for Bharat e-Mobility’. Dr. V.K Saraswat, Member Niti Aayog, highlighted indigenous efforts in producing Bullet proof vests for soldiers, echoing PM Modi’s commitment to ensuring their safety.

Professor Abhay Karandikar, Secretary DST, emphasized the transformative potential of India’s scientific endeavors, while Dr. Rajesh Gokhale, Secretary, Department of Biotechnology, and other dignitaries addressed the gathering, emphasizing India’s prowess in scientific research and development.

National Science Day, celebrated annually on 28 February, commemorates the ‘Raman Effect’, signifying India’s commitment to scientific exploration. This year’s theme, ‘Indigenous Technologies for Viksit Bharat’, underscores India’s strides towards self-reliance in science and technology.

As the event concluded, Dr. Singh acknowledged India’s robust vaccine development capacity, highlighting the nation’s leadership in preventive healthcare, particularly evident during the COVID-19 pandemic.

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