The field of cell and gene therapy has experienced remarkable progress since the early 2000s. A pivotal milestone was the USFDA’s approval of Carticel by Genzyme in 1997, marking the first cell therapy approved in the U.S. for the treatment of cartilage defects in the knee. This groundbreaking achievement paved the way for the development of autologous cell therapies targeting multiple medical conditions. As of 2024, there are over 68 non-genetically modified cell therapies and 32 gene therapies approved for clinical use worldwide, with the US, EU, and Japan playing key roles in regulatory and commercial approvals. The industry has also seen substantial growth in the number of companies dedicated to cell and gene therapy development, with over 100 companies globally actively contributing to this transformative sector. Concurrently, 1,221 clinical trials for cell and gene therapies are ongoing worldwide, spanning in various stages, including 342 in Phase 1/1b, 525 in Phase 2/2b, and 254 in Phase 3, highlighting the sector’s robust development pipeline.
In India, the shift from clinical trials to commercial-scale production of cell and gene therapies has been led by companies like Regrow Biosciences, Stempeutics, ImmunoACT, Eyestem, and Aurigene Oncology. Regrow Biosciences has successfully launched three commercial autologous cell therapy products, targeting tissue damage in bone, cartilage and the urethra. Meanwhile, Stempeutics, focuses on the development of stem cellbased products, including Stempeucel, a therapy designed to treat critical limb ischemia and osteoarthritis. Similarly, Eyestem is advancing its development of Eyecyte-RPE, a novel cell therapy aimed at treating age-related macular degeneration. Aurigene Oncology is advancing its first-in-class autologous CAR-T therapy, Ribrecabtagene autoleucel (DRL-1801), for the treatment of multiple myeloma, reflecting the growing momentum within India’s clinical-stage cell and gene therapy pipeline.
The commercialisation of cell and gene therapies globally has seen substantial financial impact, with several therapies generating significant revenue. For instance, Yescarta (Gilead Sciences) is projected to achieve $966 million in US revenue and $321 million in Europe in 2024. Similarly, Carvykti (Ciltacabtagene Autoleucel) reported $286 million in sales in a single quarter, while Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne Muscular Dystrophy, is forecast to achieve over $1 billion in 2024, with peak sales expected to exceed $6 billion by 2027. On the autologous front, Vericel’s MACI, an autologous chondrocyte implantation (ACI) for knee cartilage repair, recorded $164.8 million in revenue in 2023, representing a 25 per cent year-on-year growth. Similarly, Epicel, a therapy for patients with severe burns, generated $31.6 million in revenue in the same year, underscoring the rising demand for autologous cellular therapies in regenerative medicine.
Reimbursement models in the US, EU and Japan
Reimbursement models for cell and gene therapies have evolved significantly in recent years to improve patient access and reduce financial strain on healthcare systems. Given the high cost of such therapies, regulatory authorities and health agencies have adopted innovative payment models to balance affordability with industry sustainability.
In the US, the Centers for Medicare & Medicaid Services (CMS) have implemented outcomes-based agreements to reimburse companies for therapies only when they achieve clinical efficacy. This has been adopted for products like Zolgensma (for spinal muscular atrophy) and Luxturna (for inherited retinal disease). Pay-for-performance models link payments to treatment outcomes, ensuring that reimbursement only occurs when the therapy delivers measurable benefits to the patient. Medicaid Best Price Exclusion is another mechanism that allows states to negotiate reimbursement terms directly with manufacturers for innovative therapies.
In the EU, reimbursement for cell and gene therapies is managed through Managed Entry Agreements (MEAs). This approach enables payers to mitigate the uncertainty around clinical outcomes. Countries like Germany, Italy, and the UK have used MEAs to allow for costsharing between public health systems and manufacturers. NHS England, for example, has implemented the outcomebased reimbursement for Kymriah (CAR-T therapy) and Zynteglo (gene therapy for beta-thalassemia). These agreements ensure that manufacturers share part of the financial risk if the therapy fails to meet its intended clinical targets.
Japan has taken a progressive approach with its SAKIGAKE Designation, a system similar to the US’s RMAT (Regenerative Medicine Advanced Therapy) designation. The SAKIGAKE framework provides conditional and time-limited approval for regenerative products with an option for post-marketing data collection. This allows therapies to enter the market faster, with subsequent data collected to support long-term efficacy and safety claims. Notably, the approval of Temcell for graft-versus-host disease (GvHD) and other therapies for rare diseases has been possible under this system. Outcome-based payments are also used in Japan, with hospitals being reimbursed only if patients demonstrate positive health outcomes.
Indian landscape: Ayushman Bharat and health insurance
Government support and health policy play an instrumental role in the broader adoption of cell and gene therapies. India’s Ayushman Bharat Pradhan Mantri Jan Arogya Yojana (AB PM-JAY), launched in 2018, aims to provide health coverage of up to Rs 5 lakh per family annually, offering financial protection to economically weaker sections of society. As of March 2023, over 21 crore Ayushman Bharat cards have been issued, facilitating over 6.2 crore hospital admissions worth approximately Rs 79,000 crore. In September 2024, the Union Cabinet expanded the scheme to cover all senior citizens aged 70 and above, regardless of income, potentially benefiting an additional six crore people. Inclusion of cell and gene therapies under this scheme could significantly improve patient access, particularly for low-income populations in need of transformative therapies.
In addition to government initiatives, India’s growing middle and upper classes are increasingly turning to private health insurance to access innovative treatments. As of 2023, approximately 51 per cent of India’s population had health insurance coverage, with 35 per cent under government schemes (like Ayushman Bharat), 12 per cent through private health insurance, and four per cent through employer-provided insurance. The Insurance Regulatory and Development Authority of India (IRDAI) has been instrumental in driving health insurance coverage, and the average sum insured rose from Rs 5 lakh in 2020 to Rs 7.5 lakh in 2023. If private insurance schemes begin to cover advanced therapies like cell and gene therapy, patients will have broader access to curative therapies that were previously unaffordable. Moreover, this move would incentivise the commercial sector to offer more affordable options, ultimately driving demand for innovative therapies within India’s growing health insurance landscape.
Hopeful future: Transformational therapies redefining lives and society
As we look toward the future, it is clear that cell and gene therapies are not just scientific marvels — they are lifelines of hope for millions of people suffering from chronic, debilitating, and life-threatening diseases. These therapies go beyond symptomatic relief, offering the potential for lasting cures that restore health, dignity, and independence. Patients who were once burdened with the daily struggles of genetic disorders, cancer, and tissue degeneration can now envision a future free from pain, dependence, and the constant uncertainty of disease progression. For the first time, children born with rare genetic mutations have a chance to lead normal lives.
The societal impact of these therapies extends far beyond individual patients. When a father who was once unable to walk can return to work and support his family, or when a child can see the world clearly after receiving gene therapy for vision loss, the benefits are felt by entire families, communities, and economies. These ripple effects reduce the long-term burden on healthcare systems, minimise hospitalisations, and reduce the need for chronic medical support. Countries that embrace these innovative therapies see a healthier, more productive population that can contribute meaningfully to society and the economy.
However, this journey is not without its challenges. Regulatory hurdles, lack of financial support, and limited insurance coverage have slowed the adoption of transformational therapies. Unlike conventional pharmaceuticals, the development of cell and gene therapies requires sophisticated infrastructure, longer development cycles, and stringent oversight to ensure safety and efficacy. Stronger regulatory support, targeted government incentives, and inclusion in national health programs like Ayushman Bharat can catalyse faster adoption.
As society steps into the future driven by personalised, regenerative medicine, it is essential for stakeholders — from regulators to healthcare providers — to recognise the profound potential of these therapies. Policymakers must view them not as experimental luxuries but as essential tools to improve population health. By bridging the gap between research, regulation, and access, governments can empower millions of patients to experience life-changing treatment.
Hope is a powerful thing, and cell and gene therapies embody that hope. They remind us that diseases we once feared as “incurable” can now be confronted head-on. With proper support, these therapies can create a future where people no longer live at the mercy of genetic fate, where healthcare becomes preventive rather than reactive, and where patients regain control of their lives.
This is a pivotal moment in the history of medicine. If governments, investors, regulators, and healthcare providers come together, they can create a future where transformational therapies are accessible to all. By doing so, we ensure that “no patient is left behind.” These therapies promise to be more than just medical breakthroughs; they are stories of resilience, courage, and a profound belief in the human spirit.