Editing the Future: India’s Quest for Indigenous and Affordable Gene Therapy
| AUTHOR | Aditi Palo |
| DATE | July 31, 2026 |
| DOCUMENT | Blog |
| CATEGORIES | Advanced Biology Public Health Bioeconomy |
Zolgensma, a gene therapy for Spinal Muscular Atrophy (SMA), costs around ₹17 crore for a single-dose infusion, making it one of the world’s costliest treatments. As with most other current Cell and Gene Therapies (CGT), India relies entirely on imports of Zolgensma from the US. Some relief is provided by the Indian government — financial assistance up to ₹50 lakh per patient under the National Policy for Rare Diseases (NPRD), treatment cost waivers for special cases, and a tax rate cut from 12% to 0% for life-saving drugs. Despite these measures, the patient’s cost is barely covered. Novartis heavily discounts access to treatment for ‘compassionate use’, but this comes with the risk of being left to a foreign company’s mercy. The need of the hour is a domestic self-sustained CGT ecosystem. NITI Aayog’s recently released Bioeconomy Roadmap includes a mission called “GeneIndia”, which promises to build an affordable cell and gene therapy ecosystem by 2035. Its key recommendations include expansion of India’s genomic foundation, stronger early detection measures, acceleration of translation from laboratory-to-clinic-to-manufacturing, and the creation of a genome-to-therapy digital innovation pipeline. While these measures are necessary to build the ecosystem further, we must look at how the ecosystem has developed so far and what is missing in its growth.
Among recent CGTs, CAR T-cell therapy is a major breakthrough that targets blood and bone marrow cancers. If we take a snapshot of the status of CAR T-cell therapy ecosystem in different economies, China has several approved domestic treatments that are significantly cheaper than what the US offers. It derives its competitive edge from its recent biotechnology-centric five-year plans. Brazil has adopted a state-run manufacturing model, aiming to deliver CAR-T cell therapies via its public healthcare network.
India, on the other hand, has adopted a private-biotech-led model, enabling itself to roll out cost-effective alternatives. NexCAR19, India’s first approved indigenous CAR-T cell therapy, developed by ImmunoACT, provides over 90% cost reduction in comparison to the US and European alternatives, making it one of the most affordable CAR-T cell treatments in the world. This success story gives us confidence that India can generate similar indigenous CGTs for other genetic diseases. It also seeds ambitious dreams that India can establish itself as a strategic complement in global China+1 models for CGT export manufacturing.
GeneIndia is a timely intervention that aims to integrate existing national strengths to build a domestic CGT ecosystem. This mission includes expansion of GenomeIndia’s reference genome from 50,000 to 1 lakh individuals, thus guiding research for precise therapeutic constructs. Strengthening early detection by expanding DBT’s “UMMID” (Unique Methods of Management and Treatment of Inherited Disorders) initiative for newborn screening is a pivotal recommendation. GeneIndia provides a blueprint for building diagnostics via national translational trial platforms, and also for taking diagnostics from the lab to large-scale manufacturing via DBT–BIRAC’s biomanufacturing framework.
Despite GeneIndia’s commendable plan, it must be noted that it treats only the approval of gene therapies as the finish line. Creating affordable gene therapy does not automatically yield a robust CGT ecosystem for India. To build such an ecosystem, the first focus must be on the fragmented and incomplete registry for genetic disorders. ICMR launched a National Registry for Rare and Other Inherited Disorders (NRROID) back in 2019, with roughly 15,000 patients registered for 231 rare genetic diseases in India. However, the country’s actual rare genetic disease burden is estimated at 70 million to 96 million people, indicating that the registry falls far short of adequate coverage.
One of the main reasons behind this is the “Diagnostic Odyssey” — a lengthy, financially exhaustive journey that most patients of rare diseases face before receiving an accurate diagnosis. The other reason could be a lack of point-of-care screening and treatment. Correlational studies have shown that genetic diseases are concentrated in tribal communities, most probably due to strict endogamy practices and prolonged geographic isolation. BIRSA 101, India’s first indigenously developed CRISPR gene-editing therapy, is being designed to cure Sickle Cell Disease, highly prevalent in tribal communities in Madhya Pradesh, Chhattisgarh, and Jharkhand. Ironically, the required CRISPR editing, cell processing, and infusion infrastructure exists almost exclusively in metro cities.
To tackle this challenge, tribal populations must be kept in mind while implementing DBT’s UMMID initiative and expanding GenomeIndia’s reference genome dataset. Further research must also be done on emerging prototypes of AI-based screening as well as non-invasive screening.
Not every hospital is equipped to offer CGT treatment. It took several years for ImmunoACT, NexCAR19’s company, to create a network of over 80 authorised hospitals across India. Treatment centres need trained staff, monitoring capabilities, specific medicines and equipment to deliver the treatment and safely manage complications. India needs a clear pipeline to build on the above infrastructure if GeneIndia is to succeed.
The ambitious goals of GeneIndia are not unattainable, given the breakthroughs of NexCAR19 and BIRSA 101. But affordability must not be equated with accessibility. Until the fragmented registry, the diagnostic odyssey, and the lack of point-of-care facilities and hospital infrastructure for CGTs are addressed, the blueprint is incomplete. Thus, the 2035 target achievement must be measured by lives treated, and not by the approvals attained.
( I would like to thank Shambhavi Naik and Anupam Manur for reviewing this blog! )