India makes significant strides in Cell and Gene Therapy

In May this year, the Global Observatory for Genome Editing’s International Summit convened the largest ever gathering of scientists, researchers, bioethicists, and policymakers to deliberate on shaping a policy framework for cell and gene testing and discuss its ethical boundaries. As advances in Cell and Gene Therapy (CGT) rapidly reshape medical landscapes, the Summit highlighted a crucial point: emerging technologies such as genome editing and CRISPR must be guided not merely by innovation, but also by principles of equity, human dignity, and ethical governance.

Cell and Gene Therapy is a groundbreaking biomedical approach designed to treat or potentially cure diseases by addressing their root causes at the cellular or genetic levels. It has already revolutionized treatment possibilities for previously incurable diseases, such as Sickle Cell Disease. With numerous US FDA-approved therapies and over 4,000 gene, cell, and RNA-based treatments currently in the development pipeline, according to the American Society of Gene & Cell Therapy (ASGCT), global momentum behind CGT underscores both its immense promise and the critical need for ethical and inclusive governance.

In India, significant strides are already underway with affordable, homegrown innovations. NexCAR19, developed by ImmunoACT (an IIT Bombay spin-off) in collaboration with Tata Memorial Hospital, became the first indigenously developed CAR-T therapy to receive DCGI approval. Immuneel Therapeutics is also advancing personalized CAR-T therapies for difficult-to-treat cancers, marking India’s commitment to inclusive frameworks that ensure affordable access to life-saving therapies.

Beyond cancer and genetic diseases, CGT holds enormous potential in addressing Antimicrobial Resistance (AMR)—one of the most serious global public health threats today. Research increasingly suggests that gene-editing technologies, particularly CRISPR–Cas systems, can effectively combat AMR by targeting and disabling resistance genes within pathogenic bacteria. A review of 48 studies in BMC highlights the efficacy of CRISPR–Cas systems, such as Cas9, Cas12a, and Cas3, in disrupting key resistance genes like blaOXA-232, blaNDM, mecA, and mcr-1, restoring bacterial susceptibility to antibiotics.

At Venus Medicine Research Centre (VMRC), we have been actively exploring this promising frontier for several years. Our internal research has achieved remarkable results in utilizing CRISPR-based genetic constructs to successfully target and eliminate some of the deadliest resistance genes—including those encoding metallo-beta-lactamases (MBLs), efflux pumps, and outer membrane porins. By targeting conserved gene regions, our approach has shown the potential to immunize bacteria against re-acquiring, potentially with a significant degree of permanence, these resistance mechanisms, offering a potentially transformative tool against AMR.

Despite such breakthroughs, several technical and translational challenges remain. Effective in vivo delivery, managing potential resistance evolution, scalability, and cost-effectiveness are critical hurdles that researchers must overcome. Nonetheless, India’s proactive approach, combining robust policy support, clear regulatory frameworks, and public–private collaboration, provides a strong foundation to address these barriers.

The Indian government’s commitment to fostering innovation, exemplified by initiatives such as the Prime Minister’s Science, Technology & Innovation Advisory Council (PM-STIAC), recognizes CGT as vital for biopharmaceutical development. Complemented by efforts from the Biotechnology Industry Research Assistance Council (BIRAC) and the Department of Biotechnology (DBT), India is positioning itself as a significant player in global CGT research, particularly in combating AMR.

Looking ahead, the successful application of CGT in fighting AMR will depend on collaborative efforts across synthetic biology, microbiology, and public health. Given the interdisciplinary nature of CGT—requiring expertise in genetic engineering, bioinformatics, molecular biology, and regulatory affairs—developing specialized training programs will be crucial. Streamlined approval processes for clinical trials and clear intellectual property frameworks will further support India’s ambitions in this sector.

With its strong capabilities in affordable manufacturing, innovative approaches already demonstrated by therapies like NexCAR19, and pioneering research conducted by institutes like VMRC, India is well-positioned to leverage CGT’s full potential in addressing AMR. Sustained governmental and policy support will be essential to drive these innovative solutions from the lab to the patient bedside, ultimately transforming public health outcomes.