Cell and Gene Therapy Market to Hit $187.44 Billion by 2034

Cell and Gene Therapy Market Overview, Developments and Growth

The global cell and gene therapy market size is calculated at USD 21.82 billion in 2024 and is expected to be worth USD 187.44 billion by 2034, expanding at a CAGR of 24% from 2024 to 2034, as a result of rising clinical trials and the increasing number of product approvals.

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Oncology Accounts for the Most Active Therapeutic Segment Through All the Stages of Development.

The cell and gene therapy market is a rapidly growing sector within the healthcare industry that involves the use of cellular and genetic materials to treat various diseases and medical conditions. Cell therapy involves the transplantation or administration of live cells into a patient's body to restore or improve cellular function, while gene therapy involves the introduction or alteration of genetic material to treat genetic disorders or modify the function of cells.

Cell and gene therapies have the potential to revolutionize the treatment of a wide range of diseases. They can be used for conditions such as cancer, genetic disorders, cardiovascular diseases, neurodegenerative diseases, and autoimmune disorders, among others. This broad applicability enhances the market's growth potential. 

Oncology continued to be the most active therapeutic area of research across all stages of development, including preclinical through preregistration. In the field of gene and cell therapy, there were more than 1,300 candidates reported in development specifically for oncology in 2021. This highlights the significant focus and investment in developing innovative therapies to address cancer and improve patient outcomes.

Cell and Gene Therapy Market Trends

• Rising Prevalence of Chronic Disorders: The rising incidences and prevalence of chronic disorders such as cancer and hematological, immunological, genetic, and neurological disorders potentiate the demand for cell and gene therapy.
• Increasing Investments: The growing demand for cell and gene therapy products raises investment from both the government and private organizations. In 2023, call and gene therapy developers raised $3.5 billion across 65 deals.
• Growing Biotech Sector: The Biotech sector is a rapidly evolving field due to the rising awareness and investments globally.
• Stringent Regulatory Policies: The increasing interest in cell and gene therapy products results in a rise in product launches. According to the IQVIA report, 76 cell and gene therapy products have been approved globally as of the end of 2023.

Global Investments in Advanced Therapies: What’s Next for Cell and Gene Treatments?

The biopharma sector is seeing a surge in investment and innovation, with both major global companies and emerging start-ups pouring resources into advanced therapies like cell, gene, and gene-modifying treatments. In 2018 alone, global investment in these advanced therapies reached about $13 billion. The following year, 19 major mergers and acquisitions in the cell and gene therapy (CGT) space were completed, totaling over $156 billion.

With more than 900 companies worldwide dedicated to CGTs and over 1,000 clinical trials in progress, the industry is poised for significant growth. Starting in 2025, we could see 10 to 20 new advanced therapies approved each year. Europe is a key player in this field, with 33% of these clinical trials taking place there.

Next-generation cell and gene therapies are making groundbreaking strides, offering potential cures for patients with few other treatment options, especially for rare and ultra-rare diseases. The primary focus areas for these therapies are cancer, musculoskeletal conditions, and eye diseases. Numerous products have already been approved and launched globally, and the number of clinical trials continues to increase.

In Europe, these therapies are classified as Advanced Therapeutic Medicinal Products (ATMPs). They are driven by a range of innovative scientific advancements, including CAR-T and TCR-T therapies, stem cells, siRNA, oligonucleotides, gene editing technologies (such as CRISPR, Zinc Fingers, and TALENs), and viral transfection methods.

Collaborations, Acquisition and Investments in Cell and Gene Therapy are Paving the Market Growth

Collaborations, acquisitions, and investments play a crucial role in driving advancements and commercialization in the field of cell and gene therapy. The complex nature of these therapies requires multidisciplinary expertise and substantial financial resources, making strategic partnerships and investments essential for success. Here, we will explore the significance of collaborations, acquisitions, and investments in accelerating progress in the cell and gene therapy sector.

For instance,

• In May 2021, Biogen and Ginkgo Bioworks formed a collaboration in the field of gene therapy. Their goal was to revolutionize the manufacturing process of recombinant adeno-associated virus (AAV)-based vectors, aiming to set a new industry standard.

Collaborations among pharmaceutical companies, biotech firms, academic institutions, and research organizations are common in the cell and gene therapy space. These collaborations bring together complementary expertise, resources, and technologies to tackle the challenges associated with the development, manufacturing, and commercialization of cell and gene therapies.

Strategic acquisitions are another means by which companies expand their capabilities and enhance their position in the cell and gene therapy market. Established pharmaceutical companies often acquire smaller biotech firms with promising pipelines or novel technologies. These acquisitions provide access to innovative therapies and specialized expertise, allowing larger companies to strengthen their product portfolio and accelerate the development and commercialization of new treatments.

Acquisitions in Cell & Gene Therapies in Q2 2021

In recent years, there has been a surge in partnerships, acquisitions, and investments in the cell and gene therapy space. Major pharmaceutical companies have been actively engaging in collaborations with biotech firms, academic institutions, and research organizations to gain access to promising technologies and therapies.

Role of Clinical Trials in Cell and Gene Therapy Market

Clinical trials play a vital role in the development and commercialization of cell and gene therapies. These trials are essential for assessing the safety and efficacy of these innovative treatments, gathering evidence for regulatory approvals, and establishing their value in clinical practice. Here, we will explore the significant role of clinical trials in the cell and gene therapy market. In the comparison between 2020 and 2021, there was a notable rise in the number of therapies in preclinical development. In 2020, there were 911 therapies in preclinical studies, while in 2021, the number increased to 1,223 therapies. This indicates significant growth and focus on preclinical research and development in the field.

Clinical trials are designed to evaluate the safety profile of cell and gene therapies in human subjects. These trials follow rigorous protocols and monitoring procedures to identify any potential adverse effects or complications associated with the treatment. Safety data collected from clinical trials help researchers and regulatory authorities make informed decisions about the risks and benefits of these therapies. Clinical trials also assess the effectiveness of cell and gene therapies in treating specific diseases or conditions. As of May 2021, there were around 1,745 therapies in various stages of development, ranging from preclinical to preregistration. These trials measure various endpoints, such as improvements in patient outcomes, reduction in symptoms, or disease progression. Efficacy data generated from well-designed and controlled clinical trials are crucial for demonstrating the therapeutic benefits of these treatments.

Clinical trials focus on addressing unmet medical needs and providing innovative solutions for patients who have limited treatment options. By demonstrating the efficacy of cell and gene therapies in challenging diseases or conditions, clinical trials generate interest and demand for these treatments. This drives market growth by creating new opportunities to meet the needs of patients who were previously underserved. Clinical trials are instrumental in driving market growth in the field of cell and gene therapy. They provide evidence of safety, efficacy, and economic value, support regulatory approvals, and facilitate market access and reimbursement. Clinical trials also foster collaboration, expand the indications for these therapies, and address unmet medical needs. With the continued advancement of clinical trial programs, the cell and gene therapy market is expected to grow significantly in the coming years.

FDA Approved Cell and Gene Therapies as of December 2022

Segmental Insights

By therapy type, the cell therapy segment registered its dominance over the global cell and gene therapy market in 2023. Cell therapy is the transplantation of human cells to replace or repair damaged tissues. The therapy is used to prevent or treat several chronic disorders. Stem cell-based therapies are one of the most common types of cell therapy used clinically. The segment’s growth is attributed to the growing research and development activities, technological advancements, innovative products, and the increasing number of clinical trials. As of November 2024, 21,746 cell therapy-based clinical trials have been registered in the clinicaltrials.gov.

By therapeutic class, the infectious disease segment dominated the cell and gene therapy market in 2023. The rising incidences of infectious diseases and the COVID-19 pandemic increase the demand for cell and gene therapy for infectious diseases. The rising demand for genome editing methods like CRISPR/Cas for infectious diseases augments the market.

By therapeutic class, the oncology segment is anticipated to grow with the highest CAGR in the market during the studied years. The rising prevalence of cancer, the latest innovations, and favorable government initiatives to effectively treat cancer drive the segment’s growth.

By delivery method, the in vivo therapy segment is expected to grow exponentially during the forecast period. In vivo, therapy involves the incorporation of cell and gene therapy products directly into the patient’s body instead of growing in a test tube. It is a simple and cost-effective method, eliminating the need to isolate cells from the patient, manipulate them, and insert them into the patient.

By end-use, the hospitals segment held the largest share of the market in 2023. Hospitals provide all the necessary resources, including trained professionals, to provide appropriate cell and gene therapies.

Geographical Landscape of the Cell and Gene Therapy Market

The North America region holds a prominent position in the global cell and gene therapy market. With its advanced healthcare infrastructure, strong regulatory framework, and significant investments in research and development, North America has become a hub for innovation and commercialization in this field.

The cell and gene therapy market in North America is experiencing significant growth due to the high prevalence of chronic diseases such as cancer, cardiovascular diseases, and genetic disorders. These conditions have created a demand for personalized and effective treatment options. Cell and gene therapies have emerged as promising solutions, addressing the root causes of these diseases and offering new possibilities for treatment and management.

The presence of leading pharmaceutical and biotechnology companies in North America further strengthens the market. These companies have been at the forefront of research and development in cell and gene therapies, investing heavily in clinical trials and infrastructure. Their expertise, combined with collaborations with academic institutions and research organizations, has accelerated the translation of scientific discoveries into commercial products. The regulatory landscape in North America has also been supportive of the cell and gene therapy market. The U.S. Food and Drug Administration (FDA) has established expedited regulatory pathways, such as the Regenerative Medicine Advanced Therapy (RMAT) designation and the Fast Track designation, to facilitate the development and approval of promising therapies.

The United States, in particular, dominates the cell and gene therapy market in North America. The country has a well-established biotechnology sector, a favorable regulatory environment, and a large patient population, making it an attractive market for cell and gene therapy companies. Several key players in the field are headquartered in the United States, driving innovation and market expansion. In January 2024, the Biden-Harris Administration announced that sickle cell disease would be the primary focus of the Cell and Gene Therapy (CGT) Access Model, owing to more than 100,000 cases in America. The main goal is to increase access to high-quality, affordable healthcare and lower healthcare costs.

The Asia Pacific region is emerging as a promising market for cell and gene therapy. With its large and diverse population, increasing prevalence of chronic diseases, and growing investments in healthcare infrastructure, the region offers significant opportunities for the development and commercialization of cell and gene therapies.

Several countries in the Asia Pacific region have shown strong commitment to advancing cell and gene therapies through supportive regulatory frameworks and government initiatives. For example, Japan has implemented the Act on the Safety of Regenerative Medicine, which aims to accelerate the approval process for regenerative medicine products. China has also made significant investments in the field, launching the Made in China 2025 initiative to promote the development of innovative therapies.

SWOT Analysis of Cell and Gene Therapy Market

Strengths:

• Many market players have extensive expertise in cell and gene therapy research and development, allowing them to innovate and develop novel therapies.
• Market players with experience in conducting clinical trials have an advantage in generating valuable data and advancing their therapies through the regulatory process.
• Collaborations with academic institutions, research organizations, and other industry players enable access to expertise, resources, and patient populations, fostering innovation and growth.

Weaknesses:

• Developing and commercializing cell and gene therapies is a costly endeavor, requiring significant investment in research, clinical trials, manufacturing, and infrastructure. This can pose financial challenges for smaller companies.
• The production of cell and gene therapies often involves complex manufacturing processes, which can be time-consuming and expensive. Scaling up production to meet the growing demand can be a challenge for some market players.
• The cell and gene therapy field is subject to stringent regulatory requirements to ensure safety and efficacy. Navigating the regulatory landscape and obtaining approvals can be a time-consuming and complex process, delaying market entry.

Opportunities:

• The increasing prevalence of chronic diseases and genetic disorders creates a significant market opportunity for cell and gene therapies.
• Market players can explore the potential of cell and gene therapies in addressing a broader range of diseases and conditions, expanding their market reach.
• Advances in gene editing technologies, manufacturing processes, and delivery systems provide opportunities for innovation and improved therapeutic outcomes.
• Expanding into emerging markets with unmet medical needs can open up new growth opportunities for market players.

Threats:

• The cell and gene therapy market is becoming increasingly competitive, with the entry of new players and established pharmaceutical companies diversifying their portfolios.
• Meeting regulatory requirements and securing reimbursement can be challenging, especially in different regions with varying regulations and healthcare systems.
• Any adverse events or safety issues related to cell and gene therapies can have a significant impact on market perception and regulatory scrutiny.
• The use of genetic modification and the potential long-term effects of cell and gene therapies raise ethical and social concerns that may impact public acceptance and market adoption.

Role of Innovative Therapies in the Cell and Gene Therapy Market

Innovative therapies play a crucial role in the growth of the cell and gene therapy market. These therapies offer novel approaches to treating diseases and conditions by harnessing the power of cells and genes.

Innovative cell and gene therapies have the potential to address unmet medical needs for a wide range of diseases and conditions. They provide new treatment options for patients who have limited or no effective therapies available. By targeting diseases at the cellular and genetic levels, these therapies offer the potential for improved outcomes and quality of life for patients.

Cell and gene therapies have the ability to deliver personalized medicine approaches. They can be tailored to individual patients based on their genetic profile, disease characteristics, or other specific factors. Personalized therapies have the potential to be more targeted and effective, leading to better treatment outcomes. This shift towards personalized medicine is a significant driver for market growth. Innovative cell and gene therapies have shown promising results in the treatment of various types of cancers. For example, CAR T cell therapies have demonstrated remarkable success in certain forms of leukemia and lymphoma. These therapies involve modifying a patient's own immune cells to recognize and target cancer cells. Their potential to achieve long-lasting remission and even cure in some cases has revolutionized cancer treatment and contributed to market growth.

Innovations in gene editing technologies, such as CRISPR-Cas9, have opened up new possibilities for the treatment of genetic disorders. These technologies allow precise modification of genes, offering potential solutions for inherited diseases with a genetic component. Gene editing can correct genetic defects, restore normal gene function, or enhance therapeutic outcomes. The development and application of gene editing technologies have propelled the growth of the cell and gene therapy market. Regulatory agencies have recognized the potential of cell and gene therapies and have implemented expedited pathways to facilitate their development and approval. This regulatory support has encouraged investment in research and development, leading to the emergence of innovative therapies. Regulatory frameworks that prioritize patient safety while expediting the approval process have contributed to the growth of the market.

Latest Announcements by Industry Leaders

Leonard Schleifer, CEO of Regeneron, in a recent interview, suggested that the big thing for the biotech sector is gene therapy or genetics. He said that associating genes with disease is going to drive the pharmaceutical industry by emphasizing innovative treatments to repair certain genes and silence others.

Recent Developments in the Cell and Gene Therapy Market

• In November 2024, iotaSciences announced the launch of its newly developed Single-Cell Cloning Platform XT, a fluid shaping technology, at the Cell 2024 in London. The instrument was developed to facilitate stem cell handling and gene editing.
• In July 2024, Miltenyi Biotec announced a collaboration with the Translational Health Science and Technology Institute (THSTI) to address the growing demand for innovative treatments for cancer and sickle cell disease by developing cell and gene therapies.
• In October 2024, researchers from the University of California developed a novel technology that can easily create a gateway into the nucleus. The technique could open new possibilities in gene therapy, drug delivery, and other forms of personalized medicine.

Competitive Landscape

The cell and gene therapy market has experienced remarkable growth and progress in recent years, holding immense potential for transforming healthcare with personalized and targeted therapies. Several key players dominate the competitive landscape in this field. To gain a larger share of the market, players are using strategies like investments, alliances, acquisitions, and mergers.

Some of the major market companies include Alnylam Pharmaceuticals Inc., Amgen Inc., Biogen Inc., CORESTEM Inc., Dendreon Pharmaceuticals LLC., Helixmith Co. Ltd., JCR Pharmaceuticals Co. Ltd., Kolon TissueGene Inc., Novartis AG, and Pfizer Inc.

Segments Covered in the Report

By Therapy Type

• Cell Therapy
  
  • Stem Cells
  • T Cells
  • Dendritic Cells
  • NK Cells
  • Tumor Cells
• Gene Therapy

By Therapeutic Class

• Cardiovascular Disease
• Cancer
• Genetic Disorder
• Rare Diseases
• Oncology
• Hematology
• Ophthalmology
• Infectious Disease
• Neurological Disorders

By Delivery Method

• In Vivo
• Ex vivo

By End-Users

• Hospitals
• Cancer Care Centers
• Wound Care Centers
• Others

By Region

• North America
• Europe
• Asia Pacific
• Latin America
• MEA