Cell and Gene Therapy Market is segmented By Therapy Type, By Application and By Region (North America, Latin America, Europe, Asia Pacific, Middle East and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2024-2031
Market Overview
The global cell and gene therapy market reached US$ XX billion in 2023 and is expected to reach US$ XX billion by 2031, growing at a CAGR of XX% during the forecast period 2024-2031.
Cell and gene therapy approaches can be combined to create gene-modified cell therapies. In this case, a patient's cells are genetically modified (gene therapy) and then reintroduced as a therapeutic agent (cell therapy). A prominent example is CAR-T cell therapy, where a patient’s T cells are genetically modified to target cancer cells and then reinfused to combat the disease. Both cell and gene therapies hold transformative potential for treating chronic, inherited and otherwise incurable diseases by addressing them at a fundamental level rather than managing symptoms.
The cell and gene therapy market demand is driven with several approvals and strong growth in all stages of clinical development and by the potential of these therapies to address unmet medical needs, especially for chronic and life-threatening diseases that lack effective treatment options. For instance, according to the American Society of Cell and Gene Therapy, the cell and gene therapy field has continued its impressive momentum in the first quarter of 2024 with several notable approvals and strong growth in all stages of clinical development. More than 4,000 gene, cell and RNA treatments are now in research, which is exciting. The number of medicines in the clinical pipeline increased significantly in all three stages of development, with Phase I projects seeing the largest gain (11 percent).
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Market Dynamics: Drivers & Restraints
Growing utilization of advanced delivery vectors or vehicles
Growing utilization of advanced delivery vectors or vehicles is significantly driving the cell and gene therapy market growth and is expected to drive over the forecast period. Introducing genetic material (DNA or RNA) into a patient's cells to treat an illness is known as cell and gene therapy. Delivery vectors are used in these treatments to carry the genetic material into the target cells. The efficacy of gene and cell therapy is hampered by the limits of current delivery vectors. To transfer genetic material into target cells more effectively, newer delivery vectors are being developed. Better treatment results and possibly a lower dosage requirement could arise with it.
Patients will occasionally experience an immunological reaction to the current delivery vectors. To reduce this immunological reaction, more sophisticated vectors are being developed. For instance, on May 16, 2023, AGC Biologics launched its BravoAAV and ProntoLVV viral vector platforms. AGC Biologics’ new platforms offer fast, efficient and reproducible clinical and commercial GMP production and release.
Additionally, on March 9, 2023, Charles River Laboratories International, Inc. launched its off-the-shelf helper offering, which is designed to secure supply and streamline adeno-associated virus (AAV)-based gene therapy programs from early discovery through commercial manufacturing.
High development costs and expensive manufacturing processes
High development costs and expensive manufacturing processes are expected to hamper the cell and gene therapy market. The production of cell and gene therapy is highly intricate, requiring specialized equipment, cleanroom facilities, and adherence to rigorous regulatory standards. Each therapy often needs to be customized to individual patients, as seen in autologous therapies like CAR-T, where cells are harvested from each patient, modified, and then reinfused. This level of customization drastically increases production costs and time.
For instance, the cost of goods for manufacturing CAR-T cell therapies can exceed $100,000 per patient. For therapies like Zolgensma, a one-time gene therapy treatment for spinal muscular atrophy, the cost is around $2.1 million per patient, making it one of the most expensive therapies worldwide.
Cell and gene therapy production relies on specialized raw materials, such as viral vectors for gene delivery and specific growth media for cell culture. These materials are costly and often sourced from limited suppliers, increasing expenses. Additionally, viral vector manufacturing, in particular, is complex, requiring significant time and resources.
For instance, according to the National Institute of Health (NIH), estimating the prevalence and incidence of their corresponding diseases, applying a model of the increase in quality-adjusted life years for each therapy and simulating the launch prices and expected spending of all available gene therapies annually. The results of NIH simulation suggest that annual spending on gene therapies will be approximately $20.4 billion, under conservative assumptions.
Market Segment Analysis
The global cell and gene therapy market is segmented based on therapy type, application and region.
Therapy Type:
The cell therapy segment is expected to dominate the global cell and gene therapy market share
The cell therapy segment holds a major portion of the cell and gene therapy market share and is expected to continue to hold a significant portion of the market share over the forecast period due to its broad applications, robust clinical success and technological advancements in regenerative medicine and oncology. Cell therapies, particularly CAR-T therapies and stem cell-based treatments, have shown impressive efficacy in treating certain cancers and regenerative conditions, which has accelerated their adoption and positioned them as key drivers of growth in the market.
For instance, in April 2024, India's first indigenously-developed CAR T-cell therapy launched for cancer treatment, hailing it as a major breakthrough that provides "new hope for humankind" in the battle against the disease. Developed by the Indian Institute of Technology (IIT) Bombay and Tata Memorial Centre, the therapy is being rolled out in India at about one-tenth of its price outside the country.
Significant funding from research institutes and governments has bolstered cell therapy R&D, particularly in cancer and regenerative medicine. Increased funding has facilitated clinical trials and advancements in technology and infrastructure, supporting cell therapy’s market leadership.
For instance, researchers at the University of California, San Francisco (UCSF) have begun enrollment for a clinical trial that uses a new, powerful version of CAR T as a potential therapy for glioblastoma, the most common and deadly adult brain cancer. The clinical trial is funded by an $11 million grant to UCSF by the California Institute for Regenerative Medicine (CIRM), which funds stem cell and gene therapy research.
The gene therapy segment is the fastest-growing segment in the cell and gene therapy market
The gene therapy segment is expected to be the fastest-growing segment for cell and gene therapy market over the forecast period due to its groundbreaking potential to treat genetic disorders at their source, increasing clinical successes, expanding approvals and advancements in gene-editing technologies like CRISPR. Unlike traditional treatments that manage symptoms, gene therapy aims to correct underlying genetic causes, offering the potential for long-term or permanent cures.
For instance, in December 2023, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. Additionally, one of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.
Gene therapy has a unique advantage in its ability to address the root cause of genetic disorders. By introducing, removing or repairing genetic material within cells, gene therapy can offer a permanent solution for conditions that previously only had symptom-management treatments. This potential for “one-and-done” treatments for genetic diseases has generated strong demand and investment in gene therapy.
For instance, Zolgensma is a one-time-only gene therapy treatment for children aged less than two years with spinal muscular atrophy (SMA) that targets the defective SMN1 gene, and it has become the world’s most expensive treatment at $2.1 million per dose. This one-time treatment has shown high efficacy, allowing infants to meet developmental milestones and greatly improving their quality of life.
Market Geographical Share
North America is expected to hold a significant position in the global cell and gene therapy market
North America region is expected to hold the largest share in cell and gene therapy market over the forecast period. North America, especially the United States, has a well-established ecosystem for medical research and innovation, with numerous top-tier academic and research institutions focused on biotechnology and life sciences. This R&D infrastructure supports groundbreaking research in cell and gene therapy, leading to rapid innovation and commercialization.
For instance, Broad Stem Cell Research Center researchers are also innovating approaches that combine gene and cell therapy techniques. Center member Donald Kohn, M.D., has been developing therapies of this kind to treat genetic blood and immune disorders such as sickle cell disease for over 30 years. While advancing several of these therapies through clinical trials, center collaborators are working to enhance and adapt these therapeutic approaches to treat a wider array of diseases.
The U.S. Food and Drug Administration (FDA) has implemented specific pathways, designations and guidance to support the development and approval of cell and gene therapies. Programs like the Regenerative Medicine Advanced Therapy (RMAT) designation expedite the review process for promising therapies, making the U.S. an attractive market for cell and gene therapy companies to launch their products.
For instance, in August 2024, Ocugen, Inc., a biotechnology company focused on discovering, developing and commercializing novel gene and cell therapies and vaccines, cleared that it has received notification from the FDA to begin its expanded access program (EAP) for the treatment of adult patients, aged 18 and older, with retinitis pigmentosa (RP) with OCU400—a modifier gene therapy product candidate.
Asia Pacific is growing at the fastest pace in the Cell and Gene Therapy market
The Asia Pacific region is significantly experiencing the fastest growth in cell and gene therapy market growth. The increasing prevalence of genetic disorders in the region is driving the demand for cell and gene therapy solutions. As the population ages and lifestyles change, the prevalence of rare diseases, inherited disorders, and cancers is on the rise. For instance, according to the Tata Institute of Genetics and Society, 5000-8000 rare genetic diseases have been identified globally, 450 of which have been reported in India. Mostly affecting children, they carry a huge socio-economic, emotional and physical burden on affected families.
This has led to increased investments in innovative therapies to address the root causes of these conditions. Increased awareness and advocacy for genetic research and personalized medicine have prompted biotechnology companies to focus on developing advanced gene editing technologies and therapies tailored to specific genetic disorders.
For instance, according to Sing Health, a study in Singapore has identified genetic variations that can lead to certain medical conditions and adverse drug responses among different ethnicities. The findings could help improve the preventive care strategy under the Republic's Healthier SG initiative, set to launch in 2023.
The study found that mutations leading to familial hypercholesterolemia were more common among Chinese people, with 1.05 percent of Chinese participants having these mutations. Additionally, genetic variants in some genes that predispose carriers to hereditary breast and ovarian cancer were found in one in 110 Malays, one in 160 Indians, and one in 160 Chinese.
Market Competitive Landscape
The major global players in the cell and gene therapy market include Novartis AG, Gilead Sciences, Inc., Bristol Myers Squibb company., Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc., CSL Behring LLC, Amgen, Inc., Orchard Therapeutics group, Krystal Biotech, Inc., bluebird bio, Inc. and among others.
Emerging Players
The emerging players in the cell and gene therapy market include Editas Medicine, Intellia Therapeutics, Rocket Pharmaceuticals, Regenxbio, Affinia Therapeutics and among others.
Market Scope
Key Developments
- In September 2024, CPC (Colder Products Company), part of Dover and a leading manufacturer of connection technologies used in biopharmaceutical processing, launched a new aseptic micro-connector that fits directly into the freeze cassettes used in cell and gene therapy (CGT) processing.
- In April 2024, Walgreens cleared that it will start to work directly with drugmakers to bring cell and gene therapy to U.S. patients as part of a broader expansion of its specialty pharmacy services. The company said it is launching a new business unit dedicated to its specialty pharmacy segment, which will include specialty pharmacy subsidiary AllianceRx.
- In May 2024, in partnership with Italy-based PBL, ProPharma, a US-headquartered provider of regulatory, clinical and compliance services for the life sciences industry and a portfolio company of Odyssey Investment Partners, launched the Cell Factory Box (CF Box), an enclosed, fully automated cell factory device. This device enables the decentralized manufacturing of all types of cell and gene therapy (CGT) in Class D (ISO8) or controlled not classified surrounding areas.
Why Purchase the Report?
- To visualize the global cell and gene therapy market segmentation based on therapy type, application and region and understand key commercial assets and players.
- Identify commercial opportunities by analyzing trends and co-development.
- Excel data sheet with numerous data points of the cell and gene therapy market with all segments.
- PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
- Product mapping is available in excel consisting of key products of all the major players.
The global cell and gene therapy market report would provide approximately 54 tables, 46 figures and 197 pages.
Target Audience 2024
- Manufacturers/ Buyers
- Industry Investors/Investment Bankers
- Research Professionals
- Emerging Companies
