The global cell and gene therapy (CGT) clinical trials market size was estimated at USD 10.75 billion in 2024 and is projected to reach USD 39.41 billion by 2033, growing at a CAGR of 15.58% from 2025 to 2033. This strong growth outlook reflects the rapid evolution of advanced therapeutic modalities aimed at addressing complex and previously untreatable diseases. The market is being driven by increased research and development funding, rising patient demand for innovative and potentially curative therapies, growing interest in the use of cell and gene therapies for cancer treatment, and the presence of a favorable regulatory environment. Together, these factors are accelerating the number of clinical trials globally and expanding the overall CGT development pipeline.

A major driver of the market is the accelerating pace of innovation in genetic medicine, supported by increasing regulatory backing for advanced and regenerative therapies. Significant advancements in genome-editing technologies, such as CRISPR and other next-generation gene-editing platforms, along with progress in both viral and non-viral vector delivery systems, have enabled researchers to design highly targeted therapeutic candidates. These therapies are being developed to treat rare genetic disorders, various cancers, and autoimmune diseases with greater precision and long-term efficacy. Regulatory authorities, including the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have introduced supportive frameworks such as accelerated approval pathways, orphan drug designations, and Regenerative Medicine Advanced Therapy (RMAT) designations. These regulatory incentives help reduce development timelines and financial burdens for companies, thereby encouraging the initiation and expansion of early-phase CGT clinical trials.

In addition, the influx of funding and the rise in strategic collaborations among biotechnology companies, large pharmaceutical firms, and academic research institutions are playing a crucial role in driving market growth. Increased financial backing from venture capital firms and public private partnerships has provided the resources needed to advance CGT programs from small-scale pilot studies to large, multi-center, and international clinical trials. Leading companies such as Bluebird Bio, CRISPR Therapeutics, and Beam Therapeutics are actively initiating numerous Phase I and Phase II trials across therapeutic areas, including sickle cell disease, chimeric antigen receptor T-cell (CAR-T) therapies, and gene-editing–based oncology pipelines. Furthermore, the need to thoroughly evaluate safety profiles, optimal dosing regimens, and long-term therapeutic efficacy has increased reliance on specialized contract research organizations (CROs) and trial infrastructure specifically designed to support the complex requirements of CGT studies.

Key Market Trends & Insights:

• North America held the largest share of the global cell and gene therapy clinical trials market, accounting for 50.90% of total revenue in 2024. This dominance is supported by a strong biotechnology ecosystem, high R&D investments, advanced clinical research infrastructure, and favorable regulatory policies. The U.S., in particular, is expected to witness significant growth over the forecast period due to the high concentration of CGT developers, academic research centers, and early adoption of innovative therapeutic technologies.
• The Asia Pacific region is expected to be the fastest-growing market for cell and gene therapy clinical trials during the forecast period. Growth in this region is driven by rapidly evolving regulatory frameworks, increasing healthcare and life sciences investments, and the expansion of local biotechnology and pharmaceutical ecosystems. Countries such as China, Japan, South Korea, and India are emerging as key hubs for CGT research, supported by government initiatives and improving clinical trial capabilities.
• By phase, the Phase III segment led the market with the largest revenue share of 54.3% in 2024. This reflects the growing number of late-stage trials aimed at validating safety and efficacy prior to regulatory approval. Meanwhile, the Phase I segment is anticipated to grow at the fastest CAGR over the forecast period. This rapid growth is driven by the expanding pipeline of early-stage investigational cell and gene therapy programs and the increasing number of novel candidates entering first-in-human studies.
• By indication, the oncology segment led the market with the largest revenue share in 2024, driven by the strong focus on CAR-T therapies, gene-edited immune cells, and other advanced oncology treatments. The cardiology segment is anticipated to grow at a lucrative CAGR over the forecast period, supported by the expanding application of regenerative cell and gene therapies for cardiovascular diseases that are often resistant to conventional medical or surgical interventions.

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Market Size & Forecast:

• 2024 Market Size: USD 10.75 Billion
• 2033 Projected Market Size: USD 39.41 Billion
• CAGR (2025-2033): 15.58%
• North America: Largest market in 2024
• Asia-Pacific: Fastest growing market

Key Players

• IQVIA
• ICON Plc
• LabCorp
• Charles River Laboratories
• PAREXEL International Corp.
• Syneos Health
• Medpace
• Thermo Fisher Scientific, Inc.
• Novotech
• Veristat, LLC

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Conclusion:

In conclusion, the global cell and gene therapy clinical trials market is witnessing rapid expansion, supported by strong growth in research funding and increasing demand for innovative, precision-based therapies. Advancements in genome editing technologies and delivery systems are enabling the development of highly targeted treatments for complex diseases, including cancer, rare genetic disorders, and autoimmune conditions. Favorable regulatory frameworks, such as accelerated approvals and special designations, are significantly reducing development timelines and encouraging early- and late-stage clinical trial activity. Strategic collaborations among biotech companies, pharmaceutical firms, and academic institutions are further strengthening the clinical pipeline and supporting large-scale, multi-center trials. Regional leadership by North America and accelerated growth in Asia Pacific highlight the global nature of CGT development. Overall, continued technological innovation, regulatory support, and investment momentum are expected to sustain long-term growth in the cell and gene therapy clinical trials market.