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India Leads First Human Gene Therapy Trial for Haemophilia A

In a groundbreaking development, India has marked a significant milestone in the field of medical science by conducting the first-ever human clinical trial of gene therapy for haemophilia A (FVIII deficiency). This revolutionary trial took place at the prestigious Christian Medical College (CMC) in Vellore.

The announcement was made by Union Science & Technology Minister, Dr. Jitendra Singh, during his address at the "National Science Day 2024" programme held at Vigyan Bhavan. Dr. Singh revealed that the pioneering trial was made possible through a collaborative effort involving the Department of Biotechnology, the Centre for Stem Cell Research (a unit of InStem Bengaluru), and Emory University, USA, in conjunction with Christian Medical College, Vellore.

The trial employed an innovative approach utilizing a lentiviral vector to express a FVIII transgene in the patient's own haematopoietic stem cells, which subsequently express FVIII from specific differentiated blood cells.

Dr. Singh expressed optimism regarding the future prospects of this therapy, anticipating the commencement of vector manufacturing in India and subsequent phases of clinical trials.

The significance of this achievement was underscored by Dr. Singh in the context of India's commitment to scientific progress, echoing the sentiments of Nobel Laureate Sir CV Raman, who emphasized the pivotal role of science in India's advancement. Dr. Singh highlighted the government's unwavering support for scientific endeavors under the leadership of Prime Minister Narendra Modi, citing substantial growth in India's bio-economy and its emergence as a global player in startup innovation.

Furthermore, Dr. Singh emphasized India's strides in advanced technologies such as Quantum Technology, Artificial Intelligence, and Machine Learning, positioning the country at the forefront of global innovation.

Recognizing the role of women in science, Dr. Singh highlighted empowerment initiatives under the Modi administration, noting the leadership of women scientists in various scientific institutions and programs, including space missions.

The event also saw the unveiling of key initiatives and discussions on the transformative potential of indigenous technologies for India's development. Dr. Singh lauded India's robust vaccine development capacity, particularly evident during the COVID-19 pandemic, reaffirming India's status as a global leader in preventive healthcare.

The National Science Day celebration, commemorating the discovery of the "Raman Effect" by Sir CV Raman, served as a platform to showcase India's scientific prowess and reaffirm its commitment to technological innovation for a prosperous future.

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