40 million in India affected by thalassemia, sickle cell anaemia

India, with its diverse population, has long grappled with the burden of genetic disorders. Over 40 million individuals in India are estimated to be affected by haemoglobinopathies which include thalassemia and sickle cell anaemia

Gene therapy is not just about managing symptoms — it is about rewriting the genetic code that underlies  thalassemia and sickle cell anaemia that have cast a long shadow over the lives of individuals and families.

Traditional Treatment

Traditional treatments often involve lifelong management, such as regular blood transfusions.

By introducing functional genes and repairing the faulty ones, gene therapy offers not just treatment but the potential for a cure. The development of targeted therapies for rare diseases not only transforms individual lives of the millions in India that suffer but also addresses a critical gap in healthcare — providing treatments for conditions that are, by definition, rare. The imperative, therefore, lies in investing in research and infrastructure to make these transformative therapies accessible to those who need them.

As demographic and lifestyle changes shape new health challenges, gene and cell therapies go beyond treatment and embody a vision for the future of healthcare in the nation. These therapies anticipate and prepare for a future where precise and personalised treatments become the norm.


Cancer, a formidable adversary in India’s healthcare landscape, demands more than traditional treatment approaches at affordable cost. With over 1.16 million new cancer cases reported annually, the imperative for gene and cell therapy becomes glaringly apparent. The promise lies in precision medicine, a tailored approach that considers the unique genetic makeup of each patient. Chimeric Antigen Receptor T-cell therapy, or CAR-T therapy, exemplifies this precision. By modifying a patient’s own immune cells to recognise and target cancer cells, CAR-T therapy opens new frontiers in personalised cancer treatment. India’s genetic diversity adds complexity to cancer treatment, and here lies the beauty of gene/cell therapy — it can be customised to the individual’s unique genetic profile, potentially making treatments more effective and reducing the side effects associated with conventional approaches.

Infectious diseases always pose a major challenge to public health just like the Covid-19 pandemic resulted in unprecedented dire consequences. Subtropical India has always been prone to infections ranging from viral pathogens like dengue to tuberculosis. The anti-Covid-19 mRNA vaccines, such as the Pfizer-BioNTech and Moderna Covid-19 vaccines, which are, theoretically, gene-therapy products, have been ground-breaking.

CAR-T cell therapy is also being investigated as a potential treatment for many viral diseases like dengue, HIV/AIDS, cytomegalovirus (CMV) and Epstein-Barr virus (EBV). By modifying immune cells to better recognise and eliminate viral threats, genetic medicine becomes a potent weapon in the fight against infectious diseases.

Gene Therapy

Gene therapy holds the potential to alleviate economic burdens associated with chronic genetic conditions. By moving towards curative solutions, long-term healthcare costs can be reduced, paving the way for a more sustainable and inclusive healthcare model.

Gene therapy offers a beacon of hope by addressing the root cause — the genetic mutation is responsible for these conditions.

Gene and cell therapy are the cutting-edge medical advancements poised to revolutionise our approach to and treatment of various health conditions, from sickle cell disease and dengue to, cancer and HIV/AIDS. Despite the immense potential, the integration of gene and cell therapy into mainstream healthcare in India comes with its own set of challenges. From infrastructure limitations to ethical considerations, the journey is not without hurdles. However, these challenges present opportunities for collaborative efforts — bringing together scientific communities, industries, policymakers, and healthcare providers. Investing in research and development, creating regulatory frameworks that balance innovation with ethical considerations, and fostering a supportive ecosystem for biotechnology are crucial steps.

(Dr Naresh Purohit is Executive Member – Association of Studies for Bleeding Disorders. The views and opinion expressed in this article are those of the author)


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