The global cell and gene therapy clinical trials market was evaluated at US$ 11.57 billion in 2023 and is expected to attain around US$ 40.98 billion by 2032, growing at a CAGR of 15.09% from 2023 to 2032. The growth of the cell and gene therapy clinical trials market is driven by their potential to treat and potentially cure a wide range of diseases, from genetic disorders to certain cancers and viral infections.

Market Overview

The cell and gene therapy clinical trials market is experiencing rapid growth, driven by the promising potential of these therapies to address previously untreatable diseases. Typically structured as phase I/II studies, these trials enroll small groups of participants to assess safety and efficacy. However, shortening this process presents challenges, such as finding the optimal dosage to avoid adverse effects while ensuring effectiveness. Additionally, thorough evaluation is necessary to determine if benefits outweigh risks before broader approval.

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Following market approval, phase four trials are essential for continued safety monitoring and outcome assessment over several years. As the CGT market moves from cutting-edge science to approved therapies, demand for best practices and improved tools will rise. Biotech and pharma companies face unique challenges in navigating regulatory pathways, patient recruitment, logistics, and manufacturing for advanced therapies.

Key Insights

• North America has dominated the market with 49% revenue share in 2022.
• By phase, the phase II segment has contributed more than 54% of the total revenue share in 2022.
• By indication, the oncology sector has captured 47.70% of the total revenue share in 2022.

Regional Stance

In North America, particularly the United States, the cell and gene therapy market boast the largest share of global revenue and is poised to maintain this dominance in the foreseeable future. The American Society of Gene & Cell Therapy (ASGCT) serves as a pivotal professional organization, uniting scientists, physicians, and advocates within the field. However, despite the region’s leadership in CGT innovation, challenges emerge in the development process, especially concerning patient access and recruitment during clinical trials.

Health Canada plays a crucial role in regulating gene and cell therapy products, categorizing them as biological drugs under the Canadian Food and Drugs Act. Additionally, specific criteria determine the regulatory pathway for cellular products, potentially subjecting them to a standards-based approach under the Safety of Human Cells, Tissues, and Organs for Transplantation Regulations.

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In March 2024, Orchard Therapeutics outlined its plans for launching Lenmeldy (atidarsagene autotemcel) in the U.S., marking the sole approved therapy for children with early-onset Metachromatic Leukodystrophy.

• In December 2022, Pfizer reported positive top-line results from a Phase 3 study of its gene therapy candidate for Hemophilia B.
• In April 2023, AmerisourceBergen introduced its Cell and Gene Therapy Integration Hub.
• In June 2023, American Gene Technologies launched Addimmune, a spinoff company dedicated to the development of an HIV cure.

In the Asia Pacific region, particularly in countries like India and China, the cell and gene therapy market is anticipated to witness the fastest compound annual growth rate (CAGR) during the forecast period. In India, the National Ethical Guidelines for Biomedical and Health Research Involving Human Participants govern all clinical trials involving human subjects, including those for gene therapy. These guidelines prioritize the safety, privacy, and voluntary participation of human participants, ensuring ethical standards are upheld.

China, on the other hand, has seen a significant increase in the number of cell and gene therapy clinical trials, with a notable focus on CAR-T therapies. Hematological cancers, such as leukemia and lymphoma, account for approximately 75% of active CAR-T trials, indicating substantial progress in this area. Moreover, CAR-T technology is expanding into solid tumor treatments, demonstrating its potential across different cancer types, including gastrointestinal, liver, lung, and breast cancers. As the Asia Pacific region emerges as a key player in the cell and gene therapy landscape, regulatory frameworks and clinical trial trends highlight the region’s growing importance and potential for innovation and market expansion in the coming years.

• In February 2024, India conducted its first human clinical trials for gene therapy targeting haemophilia A, as announced by the Science and Technology minister.
• In March 2023, the first cell therapy project in Korea, supported by WuXi Advanced Therapies, received Clinical Phase I/II IND Approval, marking a significant milestone in the field.

Report Highlights

In the cell and gene therapy market, clinical trials are categorized into phases I, II, III, and IV based on their progression stages. Currently, the majority of therapies are in phase II trials, making this phase the dominant segment globally. Phase II trials hold particular significance in advancing clinical oncology, with collaborative efforts playing a crucial role in driving medical breakthroughs. Organizations like the BMT CTN provide infrastructure for innovative multi-institutional phase II trials, especially relevant to bone marrow transplantation. Successful execution of phase II proposals requires careful consideration of various factors. These trials aim to evaluate the effectiveness and safety of new drugs or drug combinations for specific indications. As such, meticulous planning and adherence to regulatory standards are essential to ensure meaningful outcomes and pave the way for further advancements in cell and gene therapy.

By Indications

The oncology segment emerged as the dominant sector, commanding a significant revenue share globally. Within the Oncology segment, the focus is on the Oncology Cell and Gene Therapy program, which is dedicated to evaluating and expediting the development of transformative cancer therapies leveraging advanced technologies with curative potential. Situated within the Oncology Center of Excellence (OCE), this program prioritizes emerging oncology science to ensure excellence in medical product regulation.

By leveraging a strong scientific foundation, the Oncology Cell and Gene Therapy program collaborates with academia, industry stakeholders, patient advocacy groups, professional societies, and international regulators. This collaborative approach aims to accelerate the development and streamline the evaluation process of safe and effective products for cancer patients. The majority of gene therapy clinical trials conducted thus far, approximately 66%, focus on treating various cancers, including prostate, head and neck, kidneys, lungs, breast, and skin cancers.

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Market Dynamics

Surge in gene therapy clinical trials

The surge in gene therapies is revolutionizing healthcare and pharmaceuticals, propelling a significant increase in clinical trials globally. Despite limited market entries, the number of cell and gene therapy (CGT) trials is escalating rapidly. While clinical holds are also rising, they appear disproportionate to the overall trial growth. Early clinical successes and robust funding have sparked tremendous industry momentum, creating an opportune moment for Low- and Middle-Income Countries (LMICs) to enter the market. By addressing the needs of communities burdened by high disease prevalence, LMICs are driving the expansion of the cell and gene therapy clinical trials market, marking a pivotal moment in the industry’s evolution.

• In June 2023, Sarepta Therapeutics announced FDA approval for ELEVIDYS, the first gene therapy for Duchenne Muscular Dystrophy.
• In September 2023, the FDA initiated a pilot program aimed at accelerating the development of therapies for rare diseases.

Restraint

High costs and limited economic returns

The substantial costs associated with research, development, and production, coupled with the high price tags demanded by companies for these therapies, ranging from several hundred thousand to a few million euros per patient, pose significant challenges. This often leads to prolonged negotiations or outright rejection by National Health Systems (NHS) unwilling to bear the financial burden, particularly for rare diseases with a very low patient population. Companies may opt to discontinue approved Advanced Therapy Medicinal Products (ATMPs) even after approval and negotiation of reimbursement policies due to insufficient economic returns. Such economic constraints limit the growth potential of the cell and gene therapy clinical trials market.

Advancements in universal cell therapies

The rapid technological innovation in gene and cell therapy presents a significant opportunity for the clinical trials market. Current regulatory models, designed for traditional therapies, often hinder the progress of promising candidates tailored to address mutations found in a limited patient population. However, the emergence of universal cell therapies offers a promising solution. These therapies utilize gene editing to engineer “immune stealth” allogeneic donor cells, capable of evading host immune detection. This breakthrough has applications in both regenerative medicine and adoptive cell immunotherapy, opening doors to broader patient populations. With gene therapy standing at the forefront of biotechnology, recent advancements and future possibilities create a fertile ground for growth in the cell and gene therapy clinical trials market.

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Recent Developments

• In February 2024, Cencora’s FormularyDecisions launched a payer resource dedicated to cell and gene therapy products.
• In October 2022, Century Therapeutics and Bristol Myers Squibb entered into a strategic collaboration for the development of iPSC-derived allogeneic cell therapies.

Key Players in the Cell and Gene Therapy Clinical Trials Market

• IQVIA
• ICON Plc
• Laboratory Corporation of America Holdings
• Charles River Laboratories International, Inc.
• PAREXEL International Corp.
• Syneos Health
• Medpace Holdings, Inc.
• PPD Inc.
• Novotech
• Veristat, LLC

Market Segmentation

By Phase

• Phase I
• Phase II
• Phase III
• Phase IV

By Indication

• Oncology
• Cardiology
• CNS
• Musculoskeletal
• Infectious Diseases
• Dermatology
• Endocrine, Metabolic, Genetic
• Immunology & Inflammation
• Ophthalmology
• Hematology
• Gastroenterology
• Others

• North America
• Europe
• Asia Pacific
• Latin America
• Middle East & Africa (MEA)

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