Global Cell and Gene Therapy: The Next Wave of Biotechnology Innovation

Parijat Jain, PhD, MBA

In the United States and the United Kingdom (UK), cell and gene therapy (CGT) is reshaping treatment paradigms and setting the stage for the next wave of biotechnology innovation. Over the past decade, the US experienced a surge in CGT approvals, with 43 products cleared by the FDA, including 6 chimeric antigen receptor (CAR) T-cell therapies and a therapy based on clustered regularly interspaced short palindromic repeats. Meanwhile, the UK has become a global leader in advanced therapies, expanding CGT infrastructure and integrating CGTs into National Health Service (NHS) care while approving multiple advanced therapy medicinal products.

As CGT continues to evolve, innovation is set to expand the reach of these therapies, offering new solutions for treating a broader range of complex diseases and changing how we approach global patient care.

Overcoming Global CGT Challenges: US vs UK

Despite significant progress, both the US and the UK face challenges that directly affect treatment and care globally. Although the FDA’s accelerated approval pathways have expedited reviews, the fragmented health care system in the US creates inconsistent coverage and delays patient access. Similarly, in the UK, budget constraints within the NHS have delayed the adoption of high-cost therapies and reliance on international supply chains for manufacturing exacerbates access challenges.

Although CGT is expanding to treat diseases with larger patient populations, such as solid tumors and diabetes, both countries must find sustainable funding solutions. The high cost of therapies, ranging from $1 million to $3 million, places significant strain on health care systems, including Medicare/Medicaid in the US and the NHS in the UK. These financial challenges are further amplified globally, as low- and middle-income countries often lack the infrastructure and resources to provide access to CGT treatments.

Improving access to CGT requires innovative funding models such as outcome-based pricing, cross-border regulatory collaboration, alignment of global manufacturing strategies and payment systems, and strengthening health care infrastructure through public-private partnerships, especially in emerging markets, to ensure equitable access to these life-changing therapies. It also requires the use of emerging technologies and the willingness to adopt innovative solutions on a global scale.

The Role of Emerging Technologies in CGT Innovation

Emerging technologies such as mRNA, artificial intelligence (AI), and scalable manufacturing play vital roles in advancing CGT. With the global CGT market expected to grow substantially in the coming years, the journey ahead will require the industry to leverage these technologies to address challenges in manufacturing, cost, and access. mRNA offers flexibility, AI enhances precision, and scalable manufacturing improves accessibility, together driving faster development, lower costs, and more personalized therapies.

One of the most exciting applications of mRNA technology is its ability to reprogram immune cells, such as T-cells and natural killer cells, within the body. This innovation eliminates the need for costly and time-consuming ex vivo manipulation. For solid tumors, mRNA instructs immune cells to produce specific receptors or cytokines, enabling them to better adapt to hostile tumor environments. This innovative approach overcomes the difficulties associated with solid tumors without relying on traditional immunosuppressants.

By analyzing vast data sets, AI models identify novel therapeutic targets for complex diseases such as solid tumors and autoimmune disorders. These models improve CAR T-cell designs, refine gene-editing strategies, and fine-tune constructs to enhance their effectiveness. AI also speeds up patient selection, predicts therapeutic responses, and optimizes clinical trial design, shortening the traditionally lengthy development cycle.

The Heart of CGT’s Future

CGT holds the potential to offer curative or long-lasting solutions for treatment instead of relying on palliative care. Genetic disorders, autoimmune diseases, and cancers could all benefit from one-time therapies that significantly improve outcomes and quality of life. Although the initial costs and infrastructure demands will put a strain on health care systems, the long-term benefits of CGT will outweigh these challenges.

As CGT evolves, personalized medicine will take center stage. Tailored therapies that target unique genetic profiles will improve efficacy, safety, and patient outcomes. Both the US and the UK should streamline regulatory processes by embracing adaptive trial designs and creating frameworks for approving individualized treatments. By fostering clear guidance and stronger collaboration between regulators, industry, and health care providers, personalized care can be accelerated, and the results will far outweigh the challenges both regions face, ultimately transforming health care delivery worldwide.

Breakthroughs such as in vivo gene editing and mRNA-based immune system reprogramming will broaden CGT’s reach to more common chronic diseases such as diabetes, cardiovascular conditions, and autoimmune disorders. These innovations will enhance patient outcomes, increase treatment accessibility, and reduce long-term health care costs. To ensure global success, key actions include scaling manufacturing, aligning regulatory frameworks, and adopting innovative payment models.

The global impact of CGT cannot be overstated. This ability to deliver targeted, individualized therapies will not only improve patient outcomes but also reduce the overall burden on health care systems by providing long-term solutions that eliminate the need for ongoing treatments. By overcoming the challenges of manufacturing, cost, and access, CGT has the potential to reach underserved populations, particularly in low- and middle-income countries, where access to advanced treatments has historically been limited.

Bio

Parijat Jain, PhD, MBA, vice president, cell and gene therapy, BioIVT

Jain is an accomplished leader with over 20 years of experience in the pharmaceutical and life sciences industries. He is known for his extensive experience in both public and private equity companies, having held senior roles at Novartis, Teva, and Lonza, with responsibilities spanning from profit and loss management, strategic business development, and commercial to operations.

Currently serving as the vice president of cell and gene therapy (CGT) and normal human and animal business units at BioIVT, Jain plays a key role in advancing business growth, bringing expertise in driving growth, innovation, and market leadership.

Jain holds a PhD and MBA and is deeply invested in the future of CGT, including how artificial intelligence integration, personalized medicine, and regulatory harmonization efforts will accelerate global patient access to these transformative therapies. His experiences in the areas of CGT starting material and supplements, marketing strategy, and business management align with his vision for advancing biologics and gene therapies, particularly in rare diseases. His contributions to the field highlight his commitment to driving innovation and improving patient outcomes through advanced therapies.