Global Cell and Gene Therapy Market 2025 – 2034

Reports Description

As per the Cell And Gene Therapy Market analysis conducted by the CMI Team, the global Cell and Gene Therapy Market is expected to record a CAGR of 18.79% from 2025 to 2034. In 2025, the market size is projected to reach a valuation of USD 25.20 Billion. By 2034, the valuation is anticipated to reach USD USD118.63 Billion.

Overview

The Cell and Gene Therapy (CGT) market is rapidly evolving as a transformative segment within the broader biotechnology and pharmaceutical industries. It focuses on treating, preventing, or potentially curing diseases by modifying or replacing cells or genetic material. With applications ranging from cancer and rare genetic disorders to neurodegenerative and autoimmune diseases, CGT represents a shift toward highly personalized and targeted treatment approaches.

Innovations in gene editing, viral vector delivery systems, and manufacturing technologies are accelerating the development pipeline, while regulatory bodies are increasingly adapting frameworks to support safe and timely approvals. As scientific understanding deepens and clinical successes grow, the CGT market is poised to become a cornerstone of modern medicine.

Key Trends & Drivers                                                                                                  

The Cell and Gene Therapy Market Trends presents significant growth opportunities due to several factors:

• Rising Prevalence of Chronic and Rare Diseases: The evolving nature of cancer, rare genetic disorders, and chronic illnesses acts as the primary impetus fueling the CGT market. These are conditions for which set therapies have no longer proven to be efficacious in the longer term, thus directing the need toward curative scale treatments such as gene therapy and CAR-T cell therapies. With the conditions genetically transmitted across the generations and cancer being generated with age, the urgency has mounted for developing and advancing novel and targeted treatments.

• Advancements in Gene Editing and Cell Engineering: The founding advents of gene editing technologies, like CRISPR-Cas9, TALENs, and zinc finger nucleases, have ushered in a paradigm shift in CGTs. Such tools allow extremely precise editing of aberrant genes and are, as a result, increasingly being used in clinical development. On the other hand, more advanced cell engineering methods enable immune cells to be reprogrammed in ways that more successfully target diseases. With all the advances in science, such therapies have being further reduced in complexity, risk, and development time.

• Increasing Demand for Personalized Medicine: Healthcare is shifting toward a personalized model where treatments are tailored according to individual genetic and molecular profiles. CGT fits very well into this model, providing therapies designed very specifically for the unique biology of an individual patient. For instance, the CAR-T therapy has modified the patient’s own immune cells for fighting their cancer. The personalized approaches get the treatments closer to the patients along with reducing side effects and having a better chance for lasting efficacy.

• Supportive Regulatory Environment: The regulatory authorities like FDA and EMA are increasingly putting their frameworks in place to support accelerated approval of CGT products. Programs including the FDA Breakthrough Therapy designation and the EMA PRIME scheme allow for the expedient review of therapies showing promise. These pathways are of particular importance in getting new therapies to market, especially where it concerns life-threatening and rare diseases. Regulators are also amending guidelines to better evaluate the fairly unique risks and benefits of CGTs.

• Increase in Public and Private Investments: From this scene, the cell and gene therapy industry is gathering substantial financial support from government agencies and private investors. Government bodies provide funds for CGT research with an attempt to foster innovation and treat the rare diseases, while the venture capitalists and the pharmaceutical giants invest in startups and in collaboration with academia. With these investments, growth of clinical trials, manufacturing infrastructure, and commercial readiness can be enhanced. Funding also lets companies take chances with new therapeutic modalities.

• Improved Manufacturing and Delivery Technologies: The manufacturing of CGT is complex and expensive, and historically, these have been barriers to it. The technology is working to eliminate these barriers. The advancements in the closed-loop automated systems and scalable viral vector production are making the CGT production more efficient. Furthermore, the improvements in the delivery technologies like lipid nanoparticles and viral vectors are facilitating the safe and efficacious gene delivery.

Significant Threats

The Cell and Gene Therapy Market faces several significant threats that could impact its growth and profitability in the future. Some of these threats include:

• Complexity in Manufacturing and Supply Chain: The handling of the live cells along with maintaining the sterile conditions and fulfilling the stringent regulatory requirements. Many of such therapies are patient-specific therefore interfering with their ability to scale up. Any delays, contaminations, or quality control failures throughout the chain may interrupt supply and the delivery of treatment. Further complicating matters is the need to maintain cold-chain logistics with near-perfect timing between production and clinical sites. A mishap anywhere along this chain may compromise product quality and jeopardize patient safety.

• Safety, Efficacy, and Ethical Concerns: While there is promising preliminary evidence of therapeutic effects of CGTs, they are still quite new and faced with questions about their long-term safety and efficacy. Some therapies can lead to life-threatening side effects like cytokine release syndrome or insertional mutagenesis. Sometimes, there is an apprehension about some distant unintended genetic consequences of gene editing. Such issues may cause public hesitation, legal challenges, or the stoppage of trials, thereby undermining confidence in the technology.

Opportunities

• Expansion Beyond Oncology into Other Therapeutic Areas: While oncology remains a core focus there has been great CGT consideration for the treatment in the non-cancerous conditions such as hemophilia, spinal muscular atrophy, Parkinsonism, and cardiovascular disorders. Further discoveries and delivery technology developments continue to expand the therapeutic horizon for CGT. One strong clinical and commercial value bond thus comes from treating chronic and rare diseases with an often-one-dose, perhaps-curative therapy. This diversification also reduces markets for cancer-related ones along with broadening the target population.

• Integration of AI and Digital Tools: AI, machine learning, and big data analytics are creating new efficiencies for CGT development. AI can identify genetic targets, predict patient responses, and optimize clinical trial design to reduce time and cost. Also, digital twins and predictive models can be used in manufacturing to improve quality control and scale-up. AI-driven diagnostic tools identify candidates for personalized therapies.

Category Wise Insights

By Therapy Type

• Cell Therapy: The importance of the cell therapy is administering the live cells into a patient’s body for treating or curing the illness. These cells may be obtained from the patient themselves or from a donor and are often manipulated or cultured for improving the therapeutic efficacy. Therapeutic synthesis is cell therapy, which is widely employed in cancer treatment; however, it is still developing in regenerative medicine, where it is intended for the repair of tissues and organs. It has very targeted means of action on cells, carcinogenic or otherwise, so it is able to address complex biomedical conditions, maybe such as autoimmune disorders and neurodegenerative diseases.

• Gene Therapy: The gene therapy refers to the alteration or replacement of the defective genes within the patient’s cells to correct genetic defects or improve the cellular function. They generally do this using the vectors often viruses are the carriers that shuttle the therapeutic genes into the affected cells. Gene therapy holds strong prospects for treating inherited disorders, such as spinal muscular atrophy, hemophilia, and some forms of blindness, as well as acquired disorders such as cancer. Gene therapy is thus becoming increasingly safer and precise with the development of gene-editing tools such as CRISPR-Cas9.

By Therapeutic Class

• Cardiovascular Disease: Cell and gene therapies could be of enormous value in treating cardiovascular diseases by repairing damaged heart tissue, restoring blood flow, and addressing genetic causes of heart failure or arrhythmias. Gene therapy would attempt to induce angiogenesis, reduce inflammation, or rectify inherited cardiac defects, whereas stem-cell-based cell therapy would focus on regenerating myocardium and ameliorating cardiac function after injury. Despite being in the early development stage, both preclinical and clinical trials are providing evidence of safety and potential efficacy.

• Cancer: With cancer as one of the important cells and gene therapy markets, treatments such as CAR-T cells, TCR therapies, and oncolytic viruses have been greatly transforming treatment paradigms. These therapies intervene with a patient’s immune system to modify it in such a way that it can then identify and destroy malignant cells, most often with great specificity and persistent effects. Significant success has been achieved with hematologic malignancies like leukaemia and lymphoma, and efforts are now underway to extend the success to solid tumours.

• Genetic Disorders: Gene therapy is the treatment of genetic disorders by correcting or replacing faulty genes at their very source. From spinal muscular atrophy to cystic fibrosis, Duchenne muscular dystrophy to selected metabolic disorders, solutions are being sought with promising results. These therapies are mostly administered as a single dose, thus giving the patient benefits that last a long time or, indeed, a cure. Advances in gene editing tools and vector design substantially increased the precision of the treatment with a reduced number of adverse effects.

• Rare diseases: Rare diseases, often brought about by specific gene mutations, are one of the focal areas of development of cell and gene therapies. Most such conditions do not have any approved therapies, thereby rendering CGT a solution for these unmet medical needs. The personalized treatment paradigm fits well with the very small and defined patient populations that are seen in the rare diseases. For instance, the gene replacement therapies have worked for the disorders like adrenoleukodystrophy and spinal muscular atrophy.

• Oncology: In oncology, cell and gene therapies have changed the entire paradigm in cancer treatment, notably in immunotherapy applications such as CAR-T and gene-modified T cells. These cells are directed to recognize the tumor antigens and generate a potent and specific immune response. CGTs are being developed for solid tumors as well to counteract the barriers created by the tumor microenvironment, which limit the trafficking and function of modified T cells, thus affecting their persistence. The genetic approaches also include the tumor suppressor gene replacement and modulation of the oncogenic pathways.

• Hematology: In hematology, cell and gene therapies find applications in blood disorders like sickle cell disease, beta-thalassemia, and hemophilia. Gene-editing practices are being used to correct the underlying genetic mutations for a potentially one-time curative intervention. Autologous stem cell-based therapies and in vivo gene delivery pathways have shown a lot of promise with some therapies being approved or nearing late-stage development.

• Ophthalmology: The gene therapy has opened new ways in ophthalmology, especially for inherited retinal disorders such as Leber congenital amaurosis and retinitis pigmentosa. These disorders generally cause progressive loss of sight or complete blindness, with the currently available therapeutic options being limited. Through gene therapy, one can actually try to restore or replace the defective gene in retinal cells to save or enhance vision. Since the eye is considered an immune-privileged organ accessible to restricted local delivery, it would be ideal as a target for gene therapy.

• Infectious Disease: While CGT traditionally has been applied to genetic and chronic diseases, its use for infectious diseases has lately been gaining attention. Gene therapies are being proposed for either boosting immune response or delivering antiviral compounds, mainly for persistent infections such as HIV. Cell therapies for infected cells may also be tested in scarring T cells and dendritic cells to improve antiviral responses or vaccine efficacy. Meanwhile, gene editing may provide functional cures by targeting viral DNA integrated into host genomes.

• Neurological Disorders: The neurological disorders like Parkinson’s disease, ALS, Huntington’s disease and epilepsy have intricate treatment challenges that can be addressed by the CGT. The gene therapy therefore provides the potential for the replacement of the defective gene or alteration of the neural pathways for restoring the brain function. The other cell therapies of interest, in particular stem cells, are being investigated for their capacity to regenerate or rescue damaged neurons. The ultimate goal of the approach is to slow or reverse progression of the disease, a goal that is largely unattainable with the current drugs.

• Others: Apart from the principal therapeutic areas, CGT is branching into various other fields, including musculoskeletal, autoimmune, dermatological, and metabolic disorders. In musculoskeletal conditions, stem cells might be tested for cartilage regeneration and orthopedic repair. Gene therapies are targeting autoimmune diseases such as lupus and rheumatoid arthritis on an immune-modulatory level. Gene therapy may also correct some inherited skin conditions like epidermolysis bullosa within dermatology.

By Delivery Method

• In Vivo: In vivo delivery refers to the method of introducing genetic materials or therapeutic agents directly into a patient in such a way that the modification of the target cells occurs within the organism. This delivery method is mostly employed in gene therapy by viral vectors or lipid nanoparticles or other delivery systems for conditions involving the liver, muscles, or the central nervous system. The in vivo delivery methods have the advantage of being less complex, less invasive and capable of treating distal or inaccessible tissues in a more focused manner. With advancements in vector technology achieving higher levels of targeting specificity and safety, in vivo gene therapy has sustained increasing interest, especially for monogenic disorders and certain systemic diseases.

• Ex Vivo: An ex vivo method entails isolation of a patient’s treatment cell or cells, genetic modification of the cells in a controlled laboratory environment, and then reinfusion into the patient. This methodology applies widely in cell therapy, e.g., CAR-T treatment for cancer, where T cells are engineered to recognize tumor antigens. The ex vivo approach permits between-gene-editing selection and quality assurance of cell types required for administration to the patient, thereby enhancing the efficacy and safety of the therapy. More challenging and demanding than the in vivo methods, however, cell modification ex vivo can be ideal for those cases that require individualized treatment and precise cellular modifications. Its efficacy remains a driving factor in further development within the CGT space.

By End-User

• Hospitals: The hospital is one of the major end-users in the CGT market which includes a macrocybernetic setup for the provision, surveillance, and management of the complex therapies. Such infrastructure promotes the carrying out of high-end procedures like leukapheresis and cell processing with post-treatment receiving therapies such as CAG-T and stem cell transplants. Multidisciplinary teams of oncologists, hematologists, geneticists, and special nurses working in hospitals together ensure the best course of treatment and support for patients.

• Cancer Care Centers: Cancer care centers are specialized treatment centers set up for the diagnosis and treatment of various types of malignancies, making them the key end users of cell and gene therapy. The centers hold these vital functions as administering immunotherapy treatments like CAR-T cell therapy would require careful handling and meticulous monitoring of patients. With a concerted effort in oncology management and protocols, cancer care centers can provide treatments cost-effectively tailored to the winding road toward recovery.

• Others: The other segment includes the academic research institutions, biotechnology clinics and specialty centers offering the CGT services. The academic institutions generally act as pioneers in the CGT innovation, conducting the early-stage research and clinical trials. Specialty clinics may focus on specific conditions, such as rare genetic disorders or ophthalmologic diseases, and offer tailored therapy protocols. Some biotech companies are establishing in-house treatment centers to directly administer their CGT products.

Report Scope

Regional Analysis

The Cell and Gene Therapy Market is segmented into various regions, including North America, Europe, Asia-Pacific, and LAMEA. Here is a brief overview of each region:

• North America: North America dominates cell and gene therapy (CGT) markets due to strong health infrastructure, substantial R&D investments, and a supportive regulatory landscape. Leading biotech entities present and contributed to the creation and growth of excellent clinical trial networks that stimulate innovation and commercialization. The U.S. is a pivotal market for early adoption of therapies like CAR-T. Strong governmental support, acceptance by the patient populace, and insurance reimbursements act as factors that augment market growth. Also, the availability of treatments years before commercialization is allowed via the FDA’s accelerated approval pathway and breakthrough therapy designations.

• Europe: Cell and gene therapy market in Europe is witnessing rapid growth with increasing investments in biotechnology and favorable regulatory initiatives such as the PRIME scheme by the European Medicines Agency. Countries such as Germany, the UK, and France are flower centers of research and clinical trials augmented by strong collaborative partnerships between public and private organizations. Increasing the incidence of chronic diseases and rare diseases is further creating market demand. However, heterogeneous healthcare systems and reimbursement procedures act as hurdles in market access. Several initiatives have been set to harmonize the regulatory framework and set up an efficient infrastructure for manufacturing.

• Asia-Pacific: Considered an emerging and fast-growing market for cell and gene therapies, this region sees rising healthcare expenditure, development of biotechnology sectors, and an increasing prevalence of various diseases as high growth factors. Besides China, Japan has invested heavily in CGT research, clinical trials, and manufacturing setups; South Korea, too, is gradually forging ahead. There has been an increasing adaptation in the regulatory bodies towards the faster approvals along with the increasing awareness among the clinicians and patients in boosting the market. Although infrastructural and reimbursement constraints exist in some pockets, expanding government schemes and international collaborations further bolster the market growth.

• LAMEA: The LAMEA cell and gene therapy market is at an early stage but is expected to grow at a significant rate owing to the increasing burden of diseases along with the improving healthcare infrastructure. Latin America, the Middle East, and Africa have witnessed heightened activity in biotechnology and clinical research but remain limited in market penetration due to regulatory hitches, absence of specialized facilities, and reimbursement issues. The increasing partnership with the international biotech players along with the increasing initiatives from the government for better healthcare accessibility is expected to propel the market growth. Improvements in awareness and infrastructure will put LAMEA on the map as a promising region for CGT, especially targeting unmet medical needs in rare and genetic diseases.

News Key Developments

In recent years, the Cell and Gene Therapy Market has experienced a number of crucial changes as the players in the market strive to grow their geographical footprint and improve their product line and profits by using synergies.

• In November 2024, iota Sciences has launched the new single-cell cloning platform XT which is a fluid shaping technology, at cell London. This was made for facilitating gene editing and stem cell handling.

These important changes facilitated the companies ability to widen their portfolios, to bolster their competitiveness and to exploit the possibilities for growth available in the Cell and Gene Therapy Market. This phenomenon is likely to persist since most companies are struggling to outperform their rivals in the market.

Leading Players

The Cell and Gene Therapy Market is highly competitive, with a large number of service providers globally. Some of the key players in the market include:

• Amgen Inc.
• Biogen Inc.
• Biomarin Pharmaceutical Inc.
• Bluebird Bio Inc. (Celgene Corporation)
• Bristol-Myers Squibb Company
• Freeline Therapeutics Ltd.
• Gene Biotherapeutics
• Gilead Sciences Inc.
• Horama S.A.
• JCR Pharmaceuticals Co. Ltd.
• Kolon TissueGene Inc.
• MeiraGTx Limited
• Novartis International AG
• Orchard Therapeutics Plc.
• Organogenesis Inc.
• Others

These companies implement a series of techniques in order to penetrate into the market, such as innovations, mergers and acquisitions and collaboration.

Emerging players in the gene therapy market are the forces behind innovation, working on new delivery technologies, expanding treatment targets, and methods for cost-effective production processes. Many such startups have advanced platform technologies into gene editing via CRISPR, non-viral vectors, and synthetic biology, all toward enhancing safety, precision, and efficacy.

Such companies generally target the rare genetic disorders or niche therapeutic areas that are being neglected by the major players. Existing pharmaceutical companies and academic institutions enter into strategic partnerships with them to fast-track clinical development and regulatory approvals. Emerging players are driving competitive advancements in expanding the gene therapy market by meeting unmet medical needs and creating accessibility.

The Cell and Gene Therapy Market is segmented as follows:

By Therapy Type

• Cell Theray
• Gene Therapy

By Therapeutic Class

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

By Delivery Method

• In Vivo
• Ex Vivo

By End-User

• Hospitals
• Cancer Care Centers
• Others

Regional Coverage:

North America

• U.S.
• Canada
• Mexico
• Rest of North America

Europe

• Germany
• France
• U.K.
• Russia
• Italy
• Spain
• Netherlands
• Rest of Europe

Asia Pacific

• China
• Japan
• India
• New Zealand
• Australia
• South Korea
• Taiwan
• Rest of Asia Pacific

The Middle East & Africa

• Saudi Arabia
• UAE
• Egypt
• Kuwait
• South Africa
• Rest of the Middle East & Africa

Latin America

• Brazil
• Argentina
• Rest of Latin America