Not Just Catching Up: How India Can Democratize Gene and Cell Therapies
NexCAR19, approved in 2023, is India’s first home-grown CAR-T cell therapy for blood cancers. NexCAR19 is not an isolated success story. Indian institutions are working on gene and cell therapies for haemophilia A, sickle cell disease, and age-related macular degeneration (AMD)
In 2024, an Indian child was treated with a ₹17.5 crore gene therapy for spinal muscular atrophy (SMA) imported from the U.S. A few months earlier, in October 2023, the Indian drug regulator approved the first indigenously developed CAR-T therapy for blood cancers, priced at under ₹45 lakh — less than one-tenth the cost of similar treatments in the U.S. These examples demonstrate how the science behind the development of cell and gene therapies has advanced. That said, the access to treatment is limited to the few who can afford it. The inequalities in access to healthcare has expanded over the years and such sophisticated therapies may remain elusive to the public at large. However, India finds itself in a unique position, it has demonstrated the potential to emerge as a pioneer in designing affordable, scalable, and inclusive alternatives.
Patients struggling with diseases that manifest due to errors in the DNA and cannot be cured with traditional medicine, have found hope in gene and cell therapies that could correct the DNA defects. These therapies are redefining what it means to “cure” a disease for various conditions including retinal diseases and blood disorders to muscular dystrophies and rare cancers. However, with the cost exceeding a few crores the access to these therapies are expected to deepen the healthcare divide. According to recent FDA data, over 40 cell and gene therapies have been approved, yet the access to these remains overwhelmingly concentrated in high-income countries. The economic barrier associated with these therapies excludes millions of people in low- and middle-income countries behind.
India bears a disproportionate disease burden of thalassemia, sickle cell disease, haemophilia, and Duchenne muscular dystrophy that gene therapy could potentially address. India being the most populous country in the world with over 4,600 population groups and endogamy is expected to have a large population of patients with rare genetic disorders. The cost of replacement therapy in India for haemophilia alone runs into hundreds of millions of dollars while the insurance coverage for these disorders remains largely absent. There is both a humanitarian and strategic imperative to act.
What makes India’s role crucial is not such the disease burden, but its proven capacity to innovate under constraints and creating process innovations to reduce cost of technology. India has repeatedly demonstrated that cutting edge science can be achieved at lower costs with stellar examples in low-cost vaccine production, affordable diagnostics, and generic drug manufacturing. The same Indian mindset is now driving the research in gene and cell therapy space — with encouraging early results.
Leading the way
NexCAR19, approved in 2023, is India’s first home-grown CAR-T cell therapy developed under a collaboration between IIT Bombay, Tata Memorial Centre, and ImmunoACR. The therapy was approved for relapsed B-cell leukaemia and lymphomas. The therapy is available at one tenth of the cost while the similar therapies abroad are priced between ₹3 and ₹4 crore. The development of NexCAR19 not only relied on scientific rigor but on rethinking how cell therapies are designed, manufactured, and delivered. The team localised the vector production, simplified manufacturing protocols, and deplored modular Good Manufacturing Practices infrastructure to drastically reduce the fixed and variable cost. This was all achieved as Indian innovators are capable of designing for masses without compromising on safety or efficacy. In early trials, NexCAR19 recipients have shown a 67% response rate with minimal toxic side effects, comparable to international standards.
Not an isolated success story
The success of NexCAR19 is not an isolated story. Researchers at the Centre for Stem Cell Research (CSCR) — a unit of Bangalore based Institute for Stem Cell Science and Regenerative Medicine (BRIC-inStem) and Christian Medical College (CMC) Vellore has been conducting a clinical trial for lentiviral vector-based gene therapy for haemophilia A. The process involves gene therapy to modify patients’ haematopoietic stem cells to express Factor VIII, and promises to eliminate the need for lifelong protein replacement therapy. The trial results show a prolonged production of Factor VIII in peripheral blood of all five treated patients who have reported reduced or no bleeding episodes over a year, and with no major side effects. The annualised bleeding rate was observed as zero for all five patients over a cumulative follow-up of 81 months (median follow-up, 14 months; range, 9 to 27). While U.S. FDA approved Roctavian gene therapy for Haemophilia is priced at $2 million; the Indian innovation promises to be meaningfully affordable to masses.
For sickle cell disease, the CSIR- Institute of Genomics and Integrative Biology (CSIR-IGIB) in Delhi is leading a national flagship program to explore gene editing approaches using CRISPR-Cas9. This programme, while rooted in robust science is also tailored to factor-in the affordability, cultural context, and ability to deliver within the public health system.
One of the leading causes of vision loss in people over 60 years is age-related macular degeneration (AMD). A Bangalore based start-up, Eyestem Research demonstrated preclinical safety and efficacy of an induced pluripotent stem cell (iPSC)-derived retinal pigment epithelium (RPE) for treating AMD. It recently concluded Phase I clinical trial with their innovative product Eyecyte-RPETM with promising safety and efficacy outcomes. The trial has reported early signals of improved structure and function of retina in patients with dry AMD, a condition that currently has no approved treatment. In all three cohorts no serious adverse events were observed. Additionally, the first six patients had an average improvement of 14.9 letters in early treatment diabetic retinopathy study (ETDRS) standard vision tests within four-six months post-transplantation. This alignment of innovation with accessibility is the defining characteristic of India’s innovation model.
India’s quiet revolution in cell and gene therapy is also supported by timely investment in infrastructure and regulation. The Indian Council of Medical Research (ICMR) has published comprehensive guidelines for gene therapy product development and clinical trials, outlining stepwise processes from vector design to patient follow-up. The CSCR at Vellore has established a state-of-the-art Good Manufacturing Practice (GMP) facility, while the institutions such as BRIC-inStem, CSIR-Institute of Genomics & Integrative Biology (CSIR-IGIB), and the Indian Institute of Science (IISc) are increasingly acting as anchors for platform development by standardising vectors, sharing GMP facilities, and building scalable delivery systems. This integrated and holistic approach has immense potential to accelerate the development of Gene and Cell Therapy in India.
Treading cautiously
However, this promise is not without its challenges. Like any other advanced therapeutics, cell and gene therapies are not without risks. The potential side effects can include immune reactions, off-target genetic edits, or even mutations due to random insertion of genes that may activate cancer. While the technological advancements and stringent regulatory frameworks have drastically improved safety, there is a need for de-risking, long term monitoring, and careful trial design specially for paediatric and vulnerable populations. India needs to reduce its import dependency on critical reagents that drive up the baseline cost. The GMP grade manufacturing capacity has to be expanded further and advance clinical infrastructure is required to support long-duration trials especially in rural India. There is also a need for focused effort to community engagement to address ethical and societal challenges and apprehensions especially for paediatric and vulnerable populations. But these hurdles are not structural dead-ends. Acknowledging and addressing these challenges in a timely manner can further accelerate the pace of innovation.
Aiming to supply next-generation therapeutics
In this process, apart from making advanced therapies cheaper, India can also model a new way of approaching health care innovation in and for global south. If India can bring down the cost by several folds, it reframes what is possible for other low- and middle-income countries. The lessons learned from modular vector platforms to community centred consent processes can inform international standards and practices. Much like it did with generic antiretrovirals (ARVs) during the AIDS crisis, India can become a global supplier and system innovator for next-generation therapeutics.
The current focus on BioE3 policy and biomanufacturing will further help to accelerate the pace of innovation that has to be grounded in ethics, society, inclusivity, and affordability. India can shift the narrative from one where cutting-edge medicine is a boutique offering for the elite, to one where it is an ethical imperative, delivered through smart, scalable design.
To achieve this, India need not focus on catching up with the West. It has the potential to chart its own new course and demonstrate once again that affordability can drive innovation without compromising on science.
