Cell and gene therapies are highly advanced biological products that can treat chronic diseases such as chronic wounds, diabetic foot ulcers, pressure ulcers, and venous ulcers. They also help to cure bone-related defects, such as cartilage defects of the knee and ankle, and several cancers, including those affecting the blood, skin, prostate, liver, and kidney. These products have helped resolve rare genetic diseases, ocular disorders, and cardiovascular diseases (CVDs). Many cell and gene therapy products, including chimeric antigen receptor (CAR) T-cell therapies, genetic modulation–based gene therapies, and autologous and allogenic cell-based therapies, are available for treating chronic, rare, and genetic diseases.
The market for cell and gene therapy is growing at an exceptional rate because it represents a way to counter the growing incidence of chronic diseases. As the total addressable market is huge and offers tremendous growth opportunities, many vendors are making it a priority to develop and launch innovative and breakthrough cell and gene therapy products. Global governing bodies are offering regulatory support and granting fast-track, breakthrough, and regenerative medicine advanced therapy (RMAT) designations to expedite product approvals.
Geography: The global cell and gene therapy market is segmented into five major regions: North America, Europe, Asia/Pacific (APAC), Latin America, and Middle East/Africa (MEA). In 2018, North America led the market with a share of 60%, followed by Europe, APAC, Latin America, and MEA.
Revenue: The United States is the largest contributor to both regional and global markets. In fact, North America has more than 400 companies that are actively engaged in research and product development of cell and gene therapy products. Europe is expected to be another prominent market due to the launch of new cell and gene therapy products. APAC is likely to emerge as a major market due to the high presence of chronic diseases in countries such as China and Japan.
Product type: The global cell and gene therapy market is segmented into gene therapy and cell therapy. Cell therapy was the dominant segment in 2018 and accounted for a share of around 76% of the global market. However, gene therapy products are likely to replace/outpace many cell therapy products by 2024 and to account for a market share of more than 50%, which is more than two times that of 2018. The launch of innovative and breakthrough products and the high adoption of CAR T-cell based therapies are the factors responsible for the growth of the cell therapy segment.
Factors influencing the market
Favorable patient demographics: The increased prevalence of several chronic diseases is contributing to growth of the cell and gene therapy market. Approximately one in three adults suffers from more than one chronic condition or multiple chronic conditions globally. Chronic diseases mainly include CVDs, cancer, chronic lung diseases, and diabetes.
Cell and gene therapy–based products have the capability to act as an effective and disease-modifying treatment, which could otherwise have been incurable through conventional therapies. The global cell and gene therapy market is witnessing double-digit growth due to the high demand for cell-based immunotherapies and gene therapy products to treat certain cancers such as prostate and hematological cancers.
The increasing use of cell therapy products for treating burns, venous leg ulcers, diabetic foot ulcers, injuries, and pressure ulcers in hospitalized patients is another factor contributing to market growth. According to the American College of Surgeons, approximately 1–2% of the global population experiences chronic wounds in their lifetime.
New product approvals/launches: Vendors are strategically focusing on the development and launch of single-use bioprocessing products to remain competitive and gain traction in the market. New product approvals and launches, coupled with R&D activities, help vendors expand their presence, enhance market growth, and sustain market position in the global cell and gene therapy market. Vendors are also actively launching innovative devices to penetrate and tap the huge growth potential of the market. Novartis AG, Gilead Sciences, Vericel, MolMed, Anterogen, Organogenesis, Amgen, Dendreon, Orchard Therapeutics, and Spark Therapeutics have significant market shares worldwide due to their continuous involvement in product innovations and launches.
Strategic acquisitions/investments: At present, the cell and gene therapy market is witnessing significant M&A activity. Vendors, especially global players, are increasingly focusing on pursuing inorganic growth strategies such as acquisitions and investments to expand presence, enhance product portfolio, and improve expertise in the market. Multinational players such as Novartis, F. Hoffmann-La Roche, Gilead Sciences, Bristol-Myers Squibb, Celgene, and Pfizer are targeting emerging market players that have strong product pipelines.
Favorable regulatory developments: Several cell and gene therapy products are receiving regulatory approvals faster than other products due to superior safety and efficacy profiles for treating a broad range of diseases. Companies are launching these products in the market more quickly as the approval process is becoming more streamlined. The regulatory approval process, particularly in the United States, is evolving and becoming favorable for vendors for developing cell and gene therapy products. The U.S. Food and Drug Administration is designating orphan drug status, breakthrough designation, accelerated approvals, and RMAT designations for cell and gene therapies to expediate the approval process.
Therefore, the favorable regulatory support in the form of priority review, RMAT designations, orphan drug designations, and breakthrough designations will positively influence the demand from end users and provide major impetus to the market.
Growing demand for CAR T-cell therapy products: CAR T-cell therapies have gained significant traction in recent years. They represent the single most rapidly growing product type in the market, and they are generating revenue at a phenomenal rate. At present, CAR T-cell therapy is the fastest advancing technology in the treatment of cancer and has the capability to replace several existing therapies. CAR T-cell therapy addresses current challenges in cancer care through superior efficacy, safety, and delivery mechanisms. CAR T-cell therapy has brought itself into focus due to highly personalized nature of this therapy and utilization of advanced genetic engineering technology.
CAR T-cell therapy has a few advantages that may not be available with other cancer-curing drugs. The most important advantage is remission that patients achieve in case of blood cancer. Once T cells are administered, they not only kill the tumor cells but also keep on growing and dividing. They provide long-lasting immunity against further tumor cell occurrence.
The landmark approvals of Yescarta and Kymriah have spurred extraordinary development in this field. The possibility of bringing a groundbreaking therapy to the market has sparked a wave of investment and innovation from pharma/biotech companies worldwide. Kymriah by Novartis contains tisagenlecleucel, and Gilead Sciences subsidiary Kite Pharma introduced Yescarta (axicabtagene ciloleucel). Kymriah is approved for both acute lymphocytic leukemia and diffuse large B-cell lymphoma, whereas Yescarta is approved for acute lymphoblastic leukemia and is currently undergoing a clinical trial for approval in diffusing large B-cell lymphoma. The potential for Kymriah and Yescarta to cure adults with aggressive B-cell lymphoma and selected pediatric and young adult patients with acute lymphoblastic leukemia is bringing new hope to those who previously tried other therapies.
Owing to the huge market potential of CAR-T cell therapy, many companies have entered the field. In 2018, more than 90 CAR T-cell therapies were under investigation in more than 100 clinical trials worldwide. Small pharma/biotech companies are developing new CAR T-cell therapies, offering opportunities for acquisitions and collaborations. For instance, CARsgen Therapeutics alone has over 11 CAR T-cell therapy products, out of which 5 are in clinical trials.
Increasing R&D funding: Many government organizations and private firms have started funding biotech start-ups and research institutes that are active in the R&D of cell and gene therapy products. Both public and private sectors are supporting cell and gene therapy developers. North America and Europe are at the forefront in terms of R&D activities and funding to develop and commercialize cell and gene therapy products.
For instance, Alliance for Cancer Gene Therapy awards both Young Investigator and Clinical Translation grants of $250,000 to $500,000 over a period of two to three years, inclusive of a maximum of 10% indirect costs. In May 2019, the California Institute for Regenerative Medicine and the National Heart, Lung, and Blood Institute entered a collaboration to co-fund and accelerate the development of cell and gene therapies to cure sickle-cell disease. This agreement was created under the National Institutes of Health’s Cure Sickle Cell initiative.
The Medical Research Council in the United Kingdom is also funding cell and gene therapy. For example, it is funding research projects to improve the understanding of fundamental stem cell biology and regenerative processes, as well as development projects that apply regenerative technologies to improve human health.
Barath Palada is an analyst at Arizton Advisory and Intelligence.