Cell and gene therapy (CGT) has emerged as a transformative frontier in medical science, offering potential cures for previously untreatable conditions, including various haematological and genetic disorders.
However, the journey from a promising scientific discovery to a commercially viable and clinically approved therapy is fraught with challenges. In India, where the CGT industry is still in its infancy, startups and research institutions face unique hurdles that can significantly delay progress. As a clinical researcher, I believe that addressing these bottlenecks requires a comprehensive approach that involves regulatory reforms, infrastructure development, and structured support for emerging biotech companies.
The paradox of start-ups: The need for early handholding
Startups in the CGT space begin with a visionary idea but often struggle with limited resources, making them vulnerable during the initial years. Ironically, when they achieve success and visibility, they attract attention from investors, regulators, and collaborators. However, it is during the early stages—when they are conducting pre-clinical research, seeking regulatory approvals, and setting up manufacturing—that they require the most support.
A single-window system is necessary, where startups can receive structured guidance on their shortcomings, regulatory appreciation, and fast-track reviews of their proposals. Currently, the fragmented nature of the regulatory process means that startups spend excessive time navigating approvals instead of focusing on research and development.
Strengthening preclinical research: The foundation for safe human trials
Preclinical research is a critical step in CGT development, assessing the safety and efficacy of a therapy in in-vitro (laboratory) and in-vivo (animal) models before progressing to human trials. However, one of the most significant challenges in India is the lack of standardized Good Manufacturing Practice (GMP)-compliant facilities for conducting preclinical studies. Many start-ups conduct their preclinical work in well-established research institutions, only to later discover that these facilities do not have a formal GMP certification, necessitating costly and time-consuming repetition of work.
To address this, it would be helpful if leading government-funded research facilities could be mandated to operate under GMP compliance with proper certifications.
Clinical trials: Regulatory support and streamlined processes
Once a therapy moves from preclinical research to human trials, regulatory scrutiny intensifies. While this is necessary for patient safety, India’s current clinical trial approval process is slow and complex, often taking 8-10 months to evaluate a facility for trial readiness. This is particularly detrimental for CGT startups working within limited financial constraints.
A robust regulatory framework with streamlined approval timelines is crucial. Clinical trials must be conducted at well-equipped facilities, with strict adherence to patient enrollment criteria. Any deviations at this stage can lead to ethical and scientific concerns, delaying the approval process further. The creation of dedicated clinical trial hubs for CGT, where startups can access pre-approved infrastructure and expertise, can significantly accelerate the clinical development process.
Manufacturing and GMP compliance
Manufacturing CGT products are significantly different from producing traditional pharmaceuticals. The personalised nature of these therapies, especially autologous cell therapies, necessitates highly controlled environments that comply with GMP regulations. In India, the number of GMP-compliant CMO’s specialising in CGT manufacturing is limited, forcing many startups to seek international collaborations, which adds to costs and delays.
The government should incentivise the development of GMP-accredited manufacturing hubs through public-private partnerships. These facilities should cater specifically to CGT startups, providing them with the infrastructure needed to produce clinical-grade ingredients (reagents, consumables etc) without excessive capital expenditure.
Long-term follow-up and post-market surveillance
One of the key differences between CGT and conventional drug development is the long-term monitoring required after therapy administration. Unlike small-molecule drugs or biologics, CGT interventions have the potential to cause off-target effects leading to long-term unwanted adverse effects, necessitating rigorous long-term follow-up to identify any late-emerging adverse effects.
While Phase 1 and Phase 2 trials in CGT typically involve only 40-45 patients, post-commercialisation surveillance is essential to detect rare complications that may arise years later. A robust mechanism for long-term follow-up of CGT patients, with mandated periodic health assessments, is critical to ensuring patient safety and building confidence in these therapies. We may look at establishing a national registry for CGT-treated patients.
India has the potential to become a global leader in CGT, but this requires proactive reforms in research support, regulatory frameworks, and manufacturing capabilities. Start-ups in this space need structured hand-holding from regulators, access to GMP-compliant preclinical and clinical trial facilities, and a streamlined approval process. By addressing these challenges, India can create an ecosystem where innovative CGT therapies move seamlessly from research to the bedside, ultimately benefiting patients who desperately need breakthrough treatments.
The writer is a paediatric haematologist and a bone marrow transplant specialist in New Delhi. He is also a researcher innovating cell therapy solutions.
The opinions expressed in this article are those of the author and do not purport to reflect the opinions or views of THE WEEK.
