Cell And Gene Therapy Clinical Trials Market Size, Share & Trends Analysis Report By Phase (Phase I, Phase II, Phase III, Phase IV), By Indication (Oncology, CNS, Cardiology), By Region, And Segment Forecasts, 2021 - 2028

Report Overview

The global cell and gene therapy clinical trials market size was valued at USD 9.2 billion in 2020 and is expected to expand at a compound annual growth rate (CAGR) of 22.3% from 2021 to 2028. The market is majorly driven by an increase in R&D funding, rising patient demand for innovative therapies, growing interest in cell and gene therapies for cancer treatment, and a favorable regulatory environment. Cell and gene therapy (CGT) represents a new frontier in the fight against many deadly diseases, including rare genetic disorders and cancers. It represents the new wave of innovation in the life sciences industry. Interest in the cell and gene therapy field has grown gradually since technological advancements in 2000. In the 20 years between 1997 and 2017, there were only about 20 FDA-approved biologics license applications (BLAs) for cell-based therapies. In 2019, there were more than 1,000 CGT trials, the majority of which were cell-based therapies. In addition, FDA is projected to approve 10 to 20 new BLAs for cell therapy products each year in the U.S. alone by 2025.

The next generation of cell and gene therapies holds a significant promise for patients as an alternative treatment option or personalized medicine. Besides, the success rate of these products is also higher as compared to those for small-molecule products. This is due to the fact that CGT tends to target precise disease drivers rather than the broad targets for small-molecule therapy. Between 2008 and 2018, the R&D success rate from Phase I to the introduction for CGT products was 11% as compared to small-molecule products representing 8.2%.

Substantial growth is happening in the size and sophistication of the companies entering the cell and gene therapy market. Many of the early movers in this market were small biotech startups. A significant interest was seen in the major biopharma industry. The majority of the large-sized pharmaceutical companies are now investing in cell and gene therapy. Novartis acquired AveXis, a commercial gene therapy company, for USD 8.7 billion in 2018. Other noteworthy acquisitions include that of Kite Pharma by Gilead; Spark Therapeutics by Roche; and Celgene by Bristol-Myers Squibb.

Besides, the first half of 2020 has been extraordinary for numerous reasons, the most noticeable of which is of course the COVID-19 pandemic and the disruption it has caused in all the areas of life and business, including cell and gene therapy sector. Even with delays in clinical trials, regulatory slowdowns, and challenges of running R&D enterprises from home, companies still have been able to attract investors and raise significant financing for their programs.

Phase Insights

Phase II led the market and accounted for more than 50.0% in 2020. The phase I segment followed the phase II segment in terms of revenue in 2020. This is mainly attributed to the fact that a large number of new entrants are working in the market.

By representing that modified genes introduced within the targeted cells are stable, early phase clinical trials are demonstrating proof‐of‐concept for gene therapy, even in situations when they failed to attain a therapeutic benefit. This comprises early CGT trials of products targeting hemophilia A & B, X‐linked adrenoleukodystrophy, primary immunodeficiency disorders, and other rare diseases. Many of these are exhibiting clinical benefits and are on an expedited path to approval.

Though the vast majority of cell and gene therapy clinical trials are in phase I and II, the proportion of trials now in the late phase including phase III and phase IV has been growing. As of February 2020, cell and gene therapy products account for approximately 12% of the pharmaceutical industry’s clinical pipeline.

Indication Insights

The oncology segment dominated the market with a share of 47.0% in 2020 and is anticipated to maintain its lead over the forecast period. As per the estimates, out of all the total number of CGT clinical trials, the oncology segment alone consists of 600 ongoing trials. The investment in oncology highlights the importance of developing innovative and faster ways to deliver CGT clinical trials and address the challenges of scaling to commercialization.

Besides, the development of CAR-T cell therapies, such as Kymriah and Yescarta, and their success in fighting hematological malignancies have paved the way for an increase in the investment in the CGTs in oncology. Both the biotech and pharmaceutical companies are investing globally in this approach to treat various types of cancer.

Regional Insights

North America accounted for the largest share of 48.3% in 2020 and is expected to maintain its lead over the forecast period. This is attributed to a favorable regulatory environment, especially in the U.S. The U.S. FDA has established a collaborative regulatory procedure for CGTs with early and steady engagement with the sponsor, along with special regulatory designations useful for many CGTs. Besides, the regulatory approval process in the U.S. is evolving and becoming favorable for vendors for developing cell and gene therapy products. The U.S. FDA is designating the orphan drug status, breakthrough designation, accelerated approvals, and RMAT designations for CGTs to expedite the approval process.

Asia Pacific is anticipated to expand at the fastest CAGR of 23.1% during the forecast period. A rising number of biotechnology firms in the Asia Pacific region are specializing in regenerative treatments. The market growth is boosted by the approval of the first genetic therapy agent by the U.S. FDA in 2017. Moreover, with the constant growth of medical tourism centers like Thailand, Singapore, and India, the region is projected to maintain its place as the epicenter of cell research and therapy.

Key Companies & Market Share Insights

The market is highly competitive. Market players are undertaking numerous strategic initiatives, such as acquisitions, collaborations, and partnerships, to gain a greater market share. For instance, in January 2020, Charles River Laboratory acquired HemaCare Corporation, a prominent player in the production of human-derived cellular products for the cell therapy market, for USD 380 million in cash.

Besides, the COVID-19 has forced many CGT companies to shift their strategy. They are mostly well-positioned to research and manufacture vaccines for COVID-19 as they generally employ viral vectors to get genes into cells. The pandemic has prompted many firms to highlight the potential of their programs for treating the disease. The CGT firms have taken a range of measures to manage the immediate effects of the pandemic. They have plans to improve their supply chain resilience and address bottlenecks in their current practices. Some prominent players in the global cell and gene therapy (CGT) clinical trials market include:

• IQVIA

• ICON Plc

• Covance

• Charles River Laboratory

• PAREXEL International Corporation

Cell And Gene Therapy Clinical Trials Market Report Scope

Segments Covered in the Report

This report forecasts revenue growth at the global, regional, and country levels and provides an analysis of the latest industry trends and opportunities in each of the sub-segments from 2016 to 2028. For the purpose of this study, Grand View Research has segmented the global cell and gene therapy clinical trials market report on the basis of phase, indication, and region:

• Phase Outlook (Revenue, USD Million, 2016 - 2028)

  • Phase I

  • Phase II

  • Phase III

  • Phase IV

• Indication Outlook (Revenue, USD Million, 2016 - 2028)

  • Oncology

  • Cardiology

  • CNS

  • Musculoskeletal

  • Infectious diseases

  • Dermatology

  • Endocrine, metabolic, genetic

  • Immunology & inflammation

  • Ophthalmology

  • Hematology

  • Gastroenterology

  • Others

• Regional Outlook (Revenue, USD Million, 2016 - 2028)

  • North America

    • U.S.

    • Canada

  • Europe

    • U.K.

    • Germany

    • France

    • Italy

    • Spain

  • Asia Pacific

    • India

    • Japan

    • China

    • Australia

    • South Korea

  • Latin America

    • Brazil

    • Mexico

    • Argentina

    • Colombia

  • Middle East & Africa

    • South Africa

    • Saudi Arabia

    • UAE