India's first human gene therapy trial for hemophilia has been successfully conducted through a collaboration between the Biotechnology Research and Innovation Council's Institute for Stem Cell Science and Regenerative Medicine (BRIC-inStem) in Bengaluru and Christian Medical College (CMC) Vellore, officials announced on April 25, 2025.
Dr. Jitendra Singh, Union Minister of State for Science and Technology, visited BRIC-inStem to review the breakthrough trial and inspect the institute's facilities. During his visit, he described the achievement as a "milestone in India's scientific journey" and emphasized its significance for both medical science and national development.
Breakthrough in Hemophilia Treatment
The gene therapy trial represents a significant advancement in treating hemophilia, a rare genetic bleeding disorder that affects approximately 20,000 people in India. Patients with hemophilia lack essential clotting factors, leading to prolonged bleeding and potential joint damage or life-threatening hemorrhages.
While conventional treatment requires regular infusions of clotting factors, gene therapy aims to provide a long-lasting solution by delivering functional copies of the defective gene to patients' cells, potentially eliminating the need for ongoing treatment.
The clinical trial, the first of its kind in India, demonstrates the country's growing capabilities in advanced genetic medicine and regenerative healthcare. Details regarding the specific vector used, patient outcomes, and efficacy data are expected to be published in peer-reviewed journals in the coming months.
National Biotechnology Growth
Dr. Singh highlighted the extraordinary growth of India's biotechnology sector, which has expanded 16-fold over the past decade to reach $165.7 billion in 2024. The government has set an ambitious target of $300 billion by 2030.
"We now have over 10,000 biotech startups compared to just 50 a decade ago," Dr. Singh noted, attributing this growth to enabling policy reforms, including the recently approved BIO-E3 Policy that aims to boost the economy, employment, and environment through biotechnology.
The formation of the Biotechnology Research and Innovation Council (BRIC), which unified 14 autonomous institutions under one umbrella, has been instrumental in streamlining research efforts and accelerating innovation in the field.
Advanced Research Infrastructure
During his visit, Dr. Singh toured BRIC-inStem's Biosafety Level III laboratory, a critical national facility for studying high-risk pathogens under India's One Health Mission.
"The recent pandemic taught us that we must always be prepared. Facilities like this will help us stay a step ahead," Dr. Singh stated, emphasizing the importance of such infrastructure for national health security.
The Minister also praised the newly launched Centre for Research Application and Training in Embryology (CReATE), which addresses birth defects and infertility through developmental biology research. "With about 3 to 4 percent of babies born with some form of defect, this centre is vital for improving maternal and neonatal health outcomes," he said.
Future Directions and Collaborations
Dr. Singh called for greater collaboration between scientific and medical institutions, suggesting that BRIC-inStem explore MD-PhD programs and integrate more deeply with clinical research.
"This is not just about science—it's about nation-building," he said, commending the Department of Biotechnology's emergence from relative obscurity into national relevance.
The Minister emphasized that India's future economy would be bio-driven, with institutions like BRIC-inStem serving as torchbearers of this transformation. "Biotechnology is not just a science anymore—it is a pillar of our national strategy," he concluded.
The successful gene therapy trial for hemophilia represents not only a medical breakthrough but also a testament to India's growing capabilities in advanced biotechnology and its potential to address critical healthcare challenges through indigenous research and innovation.
