India’s Cell & Gene Therapy Landscape: Promise, Progress, and Challenges
The CGT Revolution: A Paradigm Shift in Treatment
Cell and Gene Therapy (CGT) represents one of the most transformative developments in modern medicine, marking a shift from treating symptoms to addressing diseases at their genetic and cellular roots.
The field gained significant momentum with the 2017 FDA approval of Kymriah, the first CAR T-cell therapy, followed by Luxturna, a groundbreaking treatment for inherited retinal disease. Today, CGT has evolved into a robust therapeutic platform, with over 2,000 clinical trials worldwide and multiple approved treatments for conditions ranging from rare genetic disorders to certain types of cancer. In India, the field has emerged as a promising frontier, with research institutions and biotech companies actively developing indigenous CGT solutions while establishing international collaborations to advance this revolutionary approach to medicine.
Decoding the Basics of CGT: How does the technology work?
Gene Therapy
Gene therapy works by correcting faulty genes that cause diseases through two main approaches. In-vivo therapy involves directly introducing healthy genes into the patient’s body using viral vectors as delivery vehicles. The vectors carry the correct gene variant to target cells, replacing the faulty genes. In contrast, ex-vivo therapy involves removing the patient’s cells, modifying them with the correct gene in a laboratory using viral vectors, and then reinfusing these corrected cells back into the patient. Both methods ultimately aim to cure genetic conditions by ensuring cells have working copies of the previously defective genes.
Cell Therapy
Cell therapy involves using cells as medicine to treat diseases or repair damaged tissues. In this approach, specific cells are extracted from a patient with a medical condition. These cells are then modified in a laboratory setting through various processes including expansion (increasing cell numbers), activation (enhancing cell function), or differentiation (transforming cells into specific cell types). Once the cells have been modified and prepared, they are infused back into the patient for therapeutic benefits. This process has shown promise in treating conditions like certain cancers, autoimmune disorders, and degenerative diseases among others.
The Next Frontier in Cancer Treatment: CAR-T
Chimeric Antigen Receptor T-cell (CAR-T) therapy is a groundbreaking advancement in cancer immunotherapy, merging the fields of cell and gene therapy. This revolutionary treatment engineers a patient’s own immune cells (T-cells) to become precision cancer-fighting agents. By genetically modifying these cells to recognize and attack specific cancer markers, CAR-T therapy has achieved remarkable success rates, particularly in treating blood cancers like leukemia and lymphoma. Since the first FDA approval of Kymriah in 2017, CAR-T therapy has evolved from an experimental treatment to a life-saving option for patients who have exhausted conventional treatment methods for specific cancer types.
While this treatment is currently available for specific non-tumourous cancer types, the research is moving at a blazing pace for solid state tumourous cancers. Clinical trials targeting aggressive tumors like glioblastoma, pancreatic, and breast cancer are showing promise. Scientists are developing enhanced CAR-T cells that can better penetrate and survive in solid tumors, potentially expanding treatment options for millions of cancer patients worldwide. While there have been advancements in CAR-T for solid tumors, there’s no commercial therapy available in the market and it’s still early days with no clinical candidate yet in Phase-3 trials anywhere globally.
It’s worthwhile talking about another modality — CAR-NK which is a potential alternative to CAR-T cell therapy. Natural Killer (NK) cells can be engineered with chimeric antigen receptors and offer the advantage of being sourced from donors (allogeneic), potentially making manufacturing simpler and more cost-effective. However, clinical data for CAR-NK therapies has been less compelling compared to CAR-T’s proven success, and challenges remain in achieving consistent therapeutic responses.
India’s CGT Landscape: The Market, Early Movers & Emerging Champions
The market for such therapies is huge. For instance, let’s look at B-Cell Lymphoma for which there are 2 commercial cell therapies now available in India. Current standard treatments include Chemotherapy and Bone Marrow transplants. The number of transplants in India are estimated to be between 2800–3300 / year. CAR-T therapies will compete directly against this. Assuming a price of ~30l/ therapy makes this a market of ~850–1000 Cr for a single therapy for a single indication for a single market — India. This is without factoring in increased market size due to lack of donor limitations like in the case of BMTs. By any means, the market for such innovative therapies is huge and is slated to grow significantly over the next decade both in India and globally.
With Global Therapies focused largely on the USA, Europe and Chinese markets, there is a large and pressing need for homegrown companies to develop and commercialize cutting edge CAR-T & Cell Therapies.
However, the reality paints a grim picture — the # of clinical trials in India that are CGT focused have been <10 vs 150+ trials in the USA and 160+ trials in China. Due to the different regulatory path to market these products, India has seen only two approvals thus far. Currently, a small group of companies are active in this space — Immuneel Therapeutics launched in Jan 2025 , Dr. Reddy’s is running clinical trials for CD19 CAR-T, and ImmunoACT is seeing commercial sales. Other players like Intas, Cipla, and Natco are making initial investments, while research institutions like Tata Memorial Hospital are exploring CAR-NK therapy. The field faces significant challenges in manufacturing infrastructure, quality systems, and technical expertise, particularly in areas like vector biology where talent remains scarce.
1. The first successful foray in India into CAR-T came from Mumbai based ImmunoACT. Founded by Rahul Purwar in 2018, ImmunoACT has commercialized India’s first CAR-T cell therapy in April 2024. NexCAR 2019 is India’s first CAR-T cell therapy indicated for B-Cell Lymphoblastic Leukemia.
With a novel technology platform that’s used to design CAR and manufacturing capability to scale production of patient-scale CAR-T cells in a cGMP compliant environment, ImmunoACT is a pioneer in the space in India. Hyderabad based Generic Pharma major, Laurus Labs has invested a total of 120Cr+ in ImmunoACT across rounds with a ~34% stake. This is a rare investment from a company in generic pharma — a sector traditionally very risk averse and focused on cost optimization rather than on innovation. This investment is significant on many accounts — it’s helped develop and commercialize India’s first CAR-T therapy and potentially will bring multiple such novel therapies to market in India helping tackle a large unmet patient need in the market; this investment also sets precedent — Indian scientists are no less than their western counterparts and when backed by private risk capital, it’s possible to develop in India products of a global standard at a fraction of the cost.
2. Immuneel Therapeutics, founded by Kiran Mazumdar Shaw of Biocon is another Indian company focused on developing and commercializing Cutting-edge Cell & Gene Therapies. They are the second company in India after ImmunoACT to successfully launch Qartemi, a CAR-T therapy that is aimed at treating Non-Hodgkin’s Lymphoma. Immuneel has raised ~$27Mn across multiple rounds from Eight Roads Ventures, Khosla Ventures among other investors.
3. NKure Therapeutics, a Bangalore-based cell and gene therapy company, is developing affordable off-the-shelf Natural Killer (NK) cell-based immunotherapies for cancer and infectious diseases in India. While current autologous cell therapies face scalability challenges and high costs, and allogeneic alternatives often risk complications like GVHD, NKure aims to bridge this gap.
4. East Ocyon Bio is another company operating in the Cell and Gene Therapy Space. Founded by Dr. Dinesh Kundu and Dr. Renu Kundu, EOB is developing a pipeline of clinical assets that are cell therapies based on Natural Killer (NK) Cells, i.e. CAR-NK therapies. They have recently raised a $4.2Mn Seed round from Aeravti Ventures and generic pharma company, Micro Labs.
5. Eyestem represents India’s entry into ophthalmologic cell therapy, developing treatments for retinal degenerative diseases. The company’s lead product aims to address retinal pigment epithelium degeneration, a key factor in conditions like age-related macular degeneration (AMD). Traditional pharmaceutical companies BE, Alkem, and Natco have backed this initiative with a ₹51 crore investment. Unlike CAR-T therapies that dominate the cell therapy landscape, Eyestem’s approach focuses on replacing damaged retinal cells with lab-grown alternatives, demonstrating applications of cell therapy beyond oncology.
6. Aurigene Oncology, a Dr. Reddy’s subsidiary, has achieved significant milestones in developing India’s first BCMA-directed CAR-T cell therapy for multiple myeloma. In their Phase 1 SWASTH study, the therapy demonstrated promising results with all eight heavily pre-treated patients achieving clinical response, including 62.5% reaching stringent complete response. Notably, no high-grade Cytokine Release Syndrome or neurotoxicity events were reported, addressing key safety concerns. Following these results, DCGI has approved the progression to Phase 2 trials
Therapy Pricing and Clinical Acceptance in India
In the US, CGT therapies like CAR-T are priced high ranging from ($350,000 -$500,000) per treatment, while Indian players can price the therapies significantly lower owing to lower costs of development, production and commercialization. Both therapies commercially available in India today are priced ~INR 30L per treatment. While actual margins depend on level of control in the manufacturing, estimated peg a ~25%+ net margin levels for fully indigenous manufactured therapies.
Existing CGT manufacturers who are using imported machines (Ex — Miltenyi) for manufacturing typically are required to purchase reagents from the same supplier which are usually sold at high margins driving up costs and consequently final cost of therapy to patients. This can lead to an increase in costs pushing the final price of the therapy upwards by as high as ~80–100% in many cases. Experts in the know have commented that the launch of Qartemi was initially planned to be at a price point of ~75L/ therapy but the pricing was revised downward to ~30–35L/therapy and the launch was pushed by ~6 months and there are parallel R&D efforts ongoing to develop an indigenous process (Indigenous CAR) to drive prices down sustainably.
Given that most Cancer patients seeking to access CAR-T are terminal, there is expected resistance to newer therapies from the Clinical community but there’s also growing acceptance among the clinical community and CAR-T will become mainstream and will find its place in the clinical guidance which continues to evolve.
CGT Success in India : Key Steps to Build on to Enhance Capabilities
Technical Capabilities, Manufacturing & Talent
The development of CGT products is predominantly an engineering challenge, distinct from traditional small molecule drug discovery. The critical gap in India lies specifically in vector biology expertise — a specialized skill crucial for CAR design and development. This talent gap can’t be quickly addressed through conventional hiring or training, as the expertise requires years of specialized experience. To add to this, Gene Therapy manufacturing is CAPEX heavy — requiring ~$10–12Mn for establishing a facility as compared to <$0.5Mn for Cell Therapy. To bridge this gap, India needs to develop focused collaborations with international institutions that have strong vector biology programs, while simultaneously building long-term capability through specialized academic programs.
Quality Systems & Regulatory Framework
The regulatory environment is evolving positively, with DCGI’s team developing robust processes for CGT drug applications. There’s notable support from the PMO to improve access to rare disease therapies. While companies are seeking regulatory waivers to accelerate development timelines, the focus needs to be on establishing clear guidelines for CGT product development and approval pathways. The grapevine says that there are ongoing attempts to seek regulatory waivers to help expedite path to commercialization — this is a fine line that needs to be skirted by the DCGI not only because of the importance of patient safety, but also because it sets precedent.
Infrastructure & Ecosystem Development
The immediate priority should be the establishment of shared infrastructure models where institutes and startups can access pilot-scale facilities for initial development work. This could help optimize the high CAPEX requirements while building early-stage capabilities. Additionally, creating centralized quality control labs could help standardize testing and release procedures across different manufacturers, ensuring consistent product quality while sharing infrastructure costs. This is also something that would become highly relevant in cases where Tertiary Care Providers directly purchase machines for the manufacturing of Cell Therapies but don’t have in-house ability to Control Quality effectively.
India’s cell and gene therapy landscape stands at a critical juncture. While initial approvals and growing industry participation signal progress, key challenges remain in vector biology expertise and infrastructure development. Success in the coming years will depend on strategic collaborations, talent development, and a balanced regulatory approach. The goal remains clear: developing a robust CGT ecosystem that can deliver innovative, cost-effective therapies for India’s patient population.
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