Cell and Gene Therapy at a Turning Point

It is an exciting and dynamic time to be involved in the cell and gene therapy industry. Kalorama Information estimates the global cell and gene therapy market at approximately $16 billion in 2025, spanning key therapeutic areas such as oncology, neurology, musculoskeletal, dermatology, cardiology, and others. Kalorama projects double‑digit global market growth through 2030.

“Kalorama Information estimates the global cell and gene therapy market at $16 billion in 2025 and projects double‑digit growth through 2030.”

The science behind these therapies is advancing rapidly, and attention is now shifting toward how to manufacture, scale, and commercialize these innovations effectively. After years of breakthrough research, the field is moving into a phase that requires industrialization and standardization, particularly in how products are produced and delivered to patients. As a result, the industry is increasingly adopting standardized configurations and closed, automated manufacturing systems.

Kalorama Information estimates the global cell and gene therapy market at approximately $16 billion in 2025, spanning key therapeutic areas such as oncology, neurology, musculoskeletal, dermatology, cardiology, and others. Kalorama projects double‑digit global market growth through 2030.

Cell and gene therapies are already transforming the treatment of cancer and genetic diseases, and their influence continues to expand. What once felt highly specialized or experimental is now finding applications across a wide range of medical fields. Beyond oncology, cell and gene therapies are being developed for autoimmune diseases, cardiovascular conditions, musculoskeletal disorders, dermatological diseases, and more. This breadth highlights how foundational these technologies have become.

Progress, however, has not been uniform across therapeutic areas. Blood cancers and neurological disorders have advanced particularly quickly, while many other indications remain largely within clinical trials. Solid tumors represent one of the largest opportunities, yet they continue to be among the most challenging targets. Over the coming years, the field is expected to evolve rapidly as a broader range of cell and gene therapy products move toward approval. While the industry is increasingly global, regional differences in approvals and regulatory approaches continue to shape how therapies reach the market.

Despite the diversity of indications and technologies, several common trends are emerging worldwide. Technology continues to advance, therapeutic indications are expanding, and cell and gene therapies are reaching new regions. At the same time, companies are grappling with manufacturing and scaling challenges, while exploring new business models aimed at improving affordability and access.

Cell and gene therapies offer promise for both acquired diseases such as cancer, diabetes, and Parkinson’s disease, as well as inherited genetic conditions by correcting defective genetic material. While scientific progress has been rapid, manufacturing has become one of the biggest hurdles. High costs and the complexity of scaling production are challenges felt across the industry. In response, companies are increasingly turning to standardized production setups and closed, automated systems that can improve reliability and efficiency.

Although gene therapy and cell therapy concepts have been explored for decades, early efforts faced significant technical barriers. Over time, incremental advances and the introduction of enabling technologies such as CRISPR and next‑generation sequencing have reshaped the landscape. Today, cell and gene therapy is one of the most active areas in biomedicine, with many approved therapies demonstrating real‑world success. Lower barriers to entry have also fueled growth across the sector, with more than 2,000 companies now created or involved in cell and gene therapy development.

Financial investment remains a critical factor shaping the field. Through the first three quarters of 2025, cell and gene therapy companies attracted roughly $12 billion in investment, as reported by Kalorama Information in Cell and Gene Therapy Market Deals: Investments, M&As, and Partnerships, 2021-2025 (3rd Edition), published December 2025. That $12 billion investment figure reflects a broader downward trend over the past several years. Total annual funding peaked around $73 billion in 2021 before falling to roughly $40 billion per year between 2022 and 2024, and a projected $17 billion in 2025. The 2024 total was somewhat inflated by Novo Nordisk’s acquisition of Catalent for $16.5 billion. While the fourth quarter of 2024 saw a funding uptick, funding levels through 2025 have remained more modest.

“Through the first three quarters of 2025, cell and gene therapy companies attracted roughly $12 billion in investment, reports Kalorama Information.”

The tighter funding environment has influenced the pace and structure of deal‑making. IPOs and follow‑on public offerings have declined sharply, with only occasional signs of recovery. For cell and gene therapy startups in particular, reduced venture and private investment has become a reality, even though the number of deals per month has remained relatively steady. Mergers and acquisitions continue to account for the largest share of funding but have fluctuated significantly. In contrast, collaboration agreements, licensing deals, manufacturing partnerships, and distribution or co‑marketing arrangements have remained consistent or increased in frequency, suggesting an industry that is maturing even amid financial pressure.

Despite challenges, there is encouraging regulatory news. In January 2026, the U.S. Food and Drug Administration (FDA) announced a more flexible approach to overseeing chemistry, manufacturing, and control (CMC) requirements for cell and gene therapies, designed to support innovation and accelerate product development while still maintaining rigorous standards for safety, purity, and potency. This update clarifies how the FDA will apply regulatory flexibility throughout the development lifecycle, including clinical development, process validation, and preparation for Biologics License Application (BLA) submissions. Over the past decade, the FDA’s Center for Biologics Evaluation and Research (CBER) has approved close to 50 cell and gene therapy products, reflecting its growing experience in regulating these complex biologics and the rapid expansion of the field.

“In January 2026, the U.S. FDA announced a more flexible approach to overseeing chemistry, manufacturing, and control requirements for cell and gene therapies.”

FDA leadership has emphasized that flexibility is essential for therapies that are inherently complex and often individualized for patients. By clearly communicating regulatory approaches that were previously applied on a case‑by‑case basis, the agency aims to reduce uncertainty and help developers prepare more effectively for biologics license application submissions. As scientific capabilities continue to evolve, the FDA has signaled that removing unnecessary barriers while maintaining oversight is a high priority.

Taken together, these developments point to an industry at a critical inflection point. Cell and gene therapy is no longer defined solely by scientific possibility. The focus has shifted toward execution, manufacturing readiness, regulatory clarity, and long‑term sustainability. While challenges remain, the momentum behind these therapies continues to build, driven by both technological progress and a shared commitment to getting transformative treatments into the hands of patients who need them most.