Cell and Gene Therapy Clinical Trials Market by Therapy Type (Gene Therapy, Cell Therapy, Combination Therapies), by Application (Oncology, Genetic Disorders, Cardiovascular Diseases, Neurological Disorders, Infectious Diseases), by Phase of Development (Preclinical Trials, Phase I Trials, Phase II Trials, Phase III Trials, Phase IV Trials), by Indication (Cancer, Hematological Disorders, Rare Diseases, Autoimmune Diseases, Cardiovascular Diseases), by End-User (Pharmaceutical and Biotechnology Companies, Academic and Research Institutes, CROs, Hospitals and Clinics), and by Region; Global Insights & Forecast (2024 – 2030)

As per Intent Market Research, the Cell and Gene Therapy Clinical Trials Market was valued at USD 10.2 billion in 2023 and will surpass USD 28.4 billion by 2030; growing at a CAGR of 15.8% during 2024 - 2030.

The cell and gene therapy clinical trials market is witnessing rapid growth due to breakthroughs in regenerative medicine, genetic engineering, and the increasing prevalence of diseases such as cancer, genetic disorders, and cardiovascular conditions. These therapies are transforming the treatment landscape, offering potential cures for previously untreatable diseases by targeting the root causes at the genetic level. The expansion of clinical trials is essential for the approval of these therapies, ensuring their safety, efficacy, and long-term benefits. As the market advances, new technologies like CRISPR gene editing and cell-based therapies are shaping the development of treatments that could revolutionize patient care across the globe.

The rapid pace of innovation is reflected across the market, driven by significant investments from pharmaceutical companies, academic research institutions, and contract research organizations (CROs). With these advancements, the scope for cell and gene therapies is expanding rapidly, providing new opportunities and challenges in terms of regulatory approvals, clinical trial designs, and safety protocols. The following content will highlight key subsegments within the cell and gene therapy clinical trials market, providing insights into the largest and fastest-growing areas within each segment.

Therapy Type Segment Is Largest Owing to Growth of Gene Therapy

The gene therapy segment is the largest within the cell and gene therapy clinical trials market, driven by its broad application across various therapeutic areas, including genetic disorders, cancer, and rare diseases. Gene therapy involves the direct modification of a patient’s genetic material to correct genetic defects or to introduce beneficial genes, providing a potential cure rather than symptomatic treatment. This innovative approach is gaining significant attention from both researchers and pharmaceutical companies, as it offers the potential to eradicate or modify diseases at their genetic origin. As a result, gene therapy has dominated clinical trial investments, especially in rare genetic disorders like sickle cell anemia and hemophilia.

The growing number of gene therapy clinical trials is propelled by advancements in gene-editing technologies such as CRISPR-Cas9, which offer unprecedented precision in editing the genome. Furthermore, gene therapy has shown substantial promise in oncology, with several cancer treatments entering advanced clinical trial phases. As clinical trial results continue to demonstrate the effectiveness of gene therapy in curing previously untreatable diseases, its adoption is expected to continue growing, solidifying its position as the largest subsegment within the therapy type category.

Application Segment in Oncology Is Largest Due to High Prevalence of Cancer

The oncology application segment dominates the cell and gene therapy clinical trials market due to the high global burden of cancer and the urgent need for novel treatment options. Cancer remains one of the leading causes of death worldwide, with conventional treatments such as chemotherapy and radiation often being ineffective for many patients. As a result, gene and cell therapies, especially those that employ CAR-T (Chimeric Antigen Receptor T-cell) technology, are attracting significant attention in oncology clinical trials. These therapies aim to enhance the body’s immune system to fight cancer more effectively, providing hope for patients with otherwise limited treatment options.

The oncology application is particularly attractive for clinical trials because of the diverse range of cancers that can be targeted, including leukemia, lymphoma, and solid tumors. The success of therapies like Kymriah and Yescarta in treating certain cancers has paved the way for more research and trials focused on oncology. The growing recognition of the effectiveness of gene and cell therapies in cancer treatment, along with increasing regulatory approvals, ensures that oncology will remain the largest application segment in the cell and gene therapy clinical trials market.

Phase of Development Segment Is Fastest Growing in Phase I Trials

The Phase I trials segment is the fastest growing in the cell and gene therapy clinical trials market, driven by the increasing number of new gene and cell therapies entering the first phase of clinical trials. Phase I trials are critical in determining the safety, dosage, and potential side effects of a new treatment, making it an essential step in the drug development process. As pharmaceutical companies and biotech firms focus on advancing novel therapies, the number of Phase I trials has increased, particularly in gene and cell-based therapies, which are often more complex than traditional drug treatments.

With advancements in gene editing technologies, including CRISPR, and the ongoing development of novel cell therapies like CAR-T and TCR (T-cell receptor) therapies, Phase I trials have become a primary focus of investment and clinical research. These trials play a vital role in gathering initial safety data, which is necessary for progressing to later stages. As a result, Phase I trials are seeing substantial growth, representing a significant portion of the market as companies work to push their gene and cell therapies into clinical application.

Indication Segment in Cancer Is Largest Due to Expanding Research and Treatment Demand

The cancer indication segment is the largest within the cell and gene therapy clinical trials market, driven by the increasing prevalence of cancer globally and the demand for more targeted and effective treatments. Traditional treatments like chemotherapy, radiation, and surgery are often ineffective in certain types of cancers, especially those in later stages. Gene and cell therapies offer new approaches, such as immunotherapies that harness the body’s immune system to target and destroy cancer cells. Technologies like CAR-T cell therapy are making significant strides in oncology, with numerous clinical trials targeting various cancers, including leukemia, lymphoma, and solid tumors.

Cancer therapies have the largest share of clinical trials within cell and gene therapy due to the urgent need for innovative treatments. Clinical trials targeting cancer not only focus on improving patient outcomes but also aim to reduce side effects and improve the quality of life for cancer patients. As the field of oncology continues to embrace gene and cell therapies, cancer remains the largest indication, with extensive investment and research focusing on developing cutting-edge therapies to treat this complex disease.

End-User Segment in Pharmaceutical and Biotechnology Companies Is Largest Due to Research Investment

Pharmaceutical and biotechnology companies represent the largest end-user category in the cell and gene therapy clinical trials market. These companies are leading the way in conducting clinical trials for gene and cell therapies, driven by the potential of these treatments to revolutionize the healthcare landscape. The pharmaceutical industry has significantly increased its investment in research and development for gene therapies, particularly for rare genetic disorders, cancer, and autoimmune diseases. Biotechnology companies, which specialize in cutting-edge therapies, are also at the forefront, developing innovative solutions using genetic engineering and stem cell technologies.

These companies are playing a pivotal role in advancing clinical trials, as they have the resources and expertise to conduct large-scale trials, navigate regulatory requirements, and bring new therapies to market. The involvement of major pharmaceutical and biotech players accelerates the development of gene and cell therapies, ensuring that they meet the rigorous standards required for clinical success. As a result, pharmaceutical and biotechnology companies remain the largest end-users in the market, continuing to drive research and clinical trials forward.

North America Region Is Largest Due to Advanced Healthcare Infrastructure and Investment

North America holds the largest share of the global cell and gene therapy clinical trials market, primarily due to its advanced healthcare infrastructure, significant investments in biotechnology research, and high prevalence of diseases such as cancer and genetic disorders. The United States, in particular, is a leader in clinical trial activities, with numerous pharmaceutical companies, research institutions, and hospitals conducting gene and cell therapy trials. The U.S. Food and Drug Administration (FDA) also plays a crucial role in accelerating the approval of novel therapies, further fostering innovation and clinical research in the region.

The market in North America is further bolstered by substantial funding from both public and private sectors, including government programs supporting research and development in gene therapy. With the presence of major pharmaceutical companies and biotechnology firms, North America continues to dominate the market and is expected to maintain its leadership position as clinical trials for cell and gene therapies advance.

Leading Companies and Competitive Landscape

The competitive landscape of the cell and gene therapy clinical trials market features key players such as Novartis, Gilead Sciences, Bristol-Myers Squibb, Bluebird Bio, CRISPR Therapeutics, and Sarepta Therapeutics, which are driving innovation and clinical trials in the field. These companies are involved in extensive research to develop new therapies for cancer, genetic disorders, and other diseases, utilizing cutting-edge technologies such as CRISPR gene editing, CAR-T cell therapy, and stem cell-based treatments.

As the market grows, collaboration between pharmaceutical companies, biotechnology firms, and academic institutions is increasingly common, enabling companies to leverage expertise, resources, and funding. Strategic mergers and acquisitions, along with investments in clinical trials, are key strategies employed by companies to stay competitive. With the growing interest in personalized medicine and the promise of gene and cell therapies, the competitive landscape will continue to evolve, with innovation and regulatory approvals being central to the success of companies in the market

Recent Developments:

• Novartis announced the FDA approval of Kymriah for adults with large B-cell lymphoma, expanding its gene and cell therapy offerings in oncology.
• Gilead Sciences acquired Pionyr Immunotherapeutics, a leader in immune-oncology therapies, to enhance its gene therapy and cancer treatment capabilities.
• Bluebird Bio launched a Phase III clinical trial to evaluate its gene therapy for sickle cell disease, marking a significant step in the treatment of genetic blood disorders.
• CRISPR Therapeutics partnered with Vertex Pharmaceuticals to conduct a clinical trial using CRISPR-Cas9 technology for the treatment of beta-thalassemia and sickle cell disease.
• Spark Therapeutics announced the expansion of its clinical trials for its gene therapy treatment for Hemophilia A, with promising early-stage results

List of Leading Companies:

• Novartis
• Gilead Sciences
• Kite Pharma (A Gilead Company)
• Bristol-Myers Squibb
• Bluebird Bio
• Regeneron Pharmaceuticals
• CRISPR Therapeutics
• Sarepta Therapeutics
• Editas Medicine
• Spark Therapeutics (A Roche Company)
• Gene Editing Institute
• Cell and Gene Therapy Catapult
• Orchard Therapeutics
• Cellectis
• Allogene Therapeutics

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