U.S. Cell and Gene Therapy Market Size, Share, Growth, Report 2025 to 2034

U.S. Cell and Gene Therapy Market Size and Growth 2025 to 2034

The U.S. cell and gene therapy market size was valued at USD 5.72 billion in 2024 and is expected to be worth around USD 43.03 billion by 2034, growing at a compound annual growth rate (CAGR) of 23% over the forecast period 2025 to 2034.

The U.S. has seen a rapid rise in cell and gene therapy adoption due to increasing investments, technological innovations, and government support. Growth in the cell and gene therapy market would mainly be fueled by advancements in biotechnology, increased incidences of genetic disorders, and a rise in personalized medicine. Major drivers involve the technological advancement of tools used in gene editing, improved mechanisms of delivery, and increasing regulatory support. The soaring number of clinical trials, increasing investment in both the public and private sectors and exciting results from early-stage therapies further support market growth with new treatments that could potentially cure previously untreatable conditions.

The FDA has approved several breakthrough therapies, accelerating their market expansion. As clinical trials continue to grow, pharmaceutical and biotech companies are investing heavily in R&D, creating new opportunities. With an aging population and rising chronic disease cases, demand for these advanced treatments is increasing.

What is a cell and gene therapy?

Cell and gene therapy are advanced medical treatments designed to treat, prevent, or cure diseases at a genetic or cellular level. Cell therapy involves modifying or replacing diseased or damaged cells with healthy ones, often derived from a patient’s own body or a donor. This is commonly used in regenerative medicine, such as stem cell therapy for blood disorders or tissue repair. Gene therapy, on the other hand, works by introducing, removing, or altering genetic material within a patient’s cells to correct genetic disorders. It is being used to treat conditions like cancer, hemophilia, and genetic blindness.

Key Insights Beneficial to the U.S. Cell and Gene Therapy Market

• The FDA is expected to approve 10-20 new gene therapies per year in the coming decade.
• The number of clinical trials for gene therapy has doubled in the past five years.
• CAR-T cell therapy market saw over 100% year-on-year growth due to high treatment success rates.
• Investment in biotech firms specializing in gene therapy has surged by 300% in the last five years.
• Over 50% of healthcare startups in the U.S. are now focused on regenerative medicine.

U.S. Cell and Gene Therapy Market Growth Factors

• Rising Prevalence of Genetic Disorders: With the increasing prevalence of genetic diseases like cystic fibrosis, hemophilia, and muscular dystrophy, the demand for sophisticated therapies, including gene and cell therapies, is rising. Generally, genetic disorders have fewer treatment options, and gene therapy can act as a potential cure for such diseases because it aims to treat the basic cause of the disease at the genetic level. There's a large patient population following his recommendation, promoting innovative gene therapies, large advancement, and innovation in the class of drugs.
• Regulatory Approvals: This development is facilitated by a relatively user-friendly regulatory environment, including efforts of the U.S. FDA toward less complicated approval processes for gene and cell therapies. For instance, in a bid to spur treatments for rare diseases, the U.S. FDA offers market exclusivity and tax benefits to companies under the Orphan Drug Act. Some expedited approval pathways are through the breakthrough therapy designation, or the RMAT designation under regenerative medicine advanced therapy. It supports growth in the industry due to fast time-to-market.
• Cooperation and Collaborations: The market of cell and gene therapy is propelled by strategic partnerships of biopharma companies with research institutions and academia. Such collaboration would help share experience, resources, and technology in accelerating the development and commercialization of gene therapies. Secondly, collaborations create opportunities for the conduct of joint clinical trials and have easier access to funding, a necessity in overcoming high costs and technical challenges associated with the research and development of gene therapies.

U.S. Cell and Gene Therapy Market Trends

• Expansion of Gene Editing Technologies: Gene editing technologies, more specifically CRISPR technology, is revolutionizing the cell and gene therapy marketplace. The technology enables modifications directly at the DNA level, raising the possibility of the exact treatment of genetic disorders heretofore impossible. Advanced research on gene-editing tools will continue to advance to make the tools even better, more efficient, and safer so that they can be used even more broadly in various therapeutic domains, including inherited diseases, cancer, and other complex situations.
• Growing Demand for Personalized Medicine: Personalized medicine is the trend in gene therapy where treatments are designed according to an individual's genetic makeup. This increases the efficacy of therapies and decreases side effects by targeting specific genetic markers. Advances in genomic sequencing and precision medicine are allowing more patients to receive treatments designed to meet their specific genetic needs, driving the adoption of gene therapies in clinical settings.
• Advancements in Delivery Systems: Probably among the biggest challenges in gene therapy has been the efficient transfer of therapeutic genes to target cells. New tendencies of concern have been mechanisms towards improved performance, such as with viruses, nanoparticles, among advanced carriers, towards accuracy and efficiency and safety, even from gene therapies. Innovated gene delivery will have guaranteed reliability and reduced negative adverse reactions associated with it, giving good outcomes possibilities, making way for further growth of market growth.

Report Scope

U.S. Cell and Gene Therapy Market Dynamics

Drivers

• The increasing incidence of genetic disorders: Genetic diseases, in particular the rare and complex diseases have a highly motivated increasing incidence hence the demand for novel therapies. Sickle cell anemia and Duchenne muscular dystrophy together with inherited types of blindness are just a few such disease conditions with poor treatment options hence creating a high market place for gene therapies targeting source at the genetic level. Growing demand for effective solutions in this manner has fueled the adoption and investment in gene therapies.
• Supportive Government Initiatives: Government initiatives and funding programs can be considered as the key drivers behind the growth of the market for cell and gene therapies. In the U.S., agencies such as FDA, NIH, and more provide regulatory pathways, grants, and tax incentives on the development of gene therapy, especially for rare diseases and orphan diseases. Innovation is accelerated, financial risks are reduced, and product development timelines are shortened that makes it easier for gene therapies to come into markets faster.
• Breakthrough Clinical Results: Optimistic clinical trials have fueled greater investment and interest in gene therapies on the part of the medical and biotech communities. With every successful outcome regarding the treatment of previously uncurable diseases, new gene therapies gain FDA approvals, thereby fueling higher patient demand and confidence that feed back into each other. This has created an excellent base for the industry to expand upon and resulted in more funding and studies being pursued in the subject area.

Restraints

• Extremely High treatment costs: One of the major obstacles to the gene therapy market is the extremely high cost associated with development and treatment. The complexity in designing, manufacturing, and administering gene therapies makes it unaffordable for many patients. These very high costs, at the same time, raise concern in healthcare systems and insurers, hence, even though the treatment can cure the disease, access remains limited. Models for pricing and reimbursement policies will be required which will make this treatment accessible to a larger number.
• Regulatory Obstacles: While gene therapies are supported by the regulations, the approval process remains long and complicated. Though supportive, the U.S. FDA requires significant clinical data to be assured of both safety and efficacy before issuing approval. Novel gene therapies may take years and millions of dollars in investment, which might limit the pace of development and availability. This regulatory uncertainty creates financial and operational challenges for the companies.
• Limited Reimbursement Models: Major deterrence: There is not yet any well-defined and standardized model for reimbursement related to gene therapies. Gene therapies are largely one-time treatments, which are very cost-intensive. Thus, the alignment with traditional models of care-based reimbursement is not so easy. This, therefore, creates a barrier in terms of access to treatment for patients and companies trying to secure long-term financial sustainability. New innovative reimbursement models need to be established to address these challenges.

Opportunities

• Expansion into Rare Disease Treatments: Gene therapy is a promising avenue to treat rare diseases for which there are few or no therapeutic alternatives. The market of orphan drugs is rapidly increasing because for most patients with rare genetic diseases, there is little other option than experimental therapy. Gene therapy appears as an alternative to these underserved patient populations. In turn, a rapid investment with accompanying incentives on a regulatory level in rare disease treatments will mean massive growth potential for companies with gene therapy.
• Gene Therapy for Age-related Diseases: As the population of the world ages, Alzheimer's, Parkinson's, and macular degeneration among others are increasingly prevalent diseases. Gene therapies are one of the potential treatment modalities for age-related diseases. These are focused on correcting genetic factors, which lead to diseases. Researching specific gene therapies that specifically target the geriatric market would provide significant opportunity as the medical sector increasingly turns its focus towards novel approaches addressing chronic diseases, primarily gerontogenic ones.
• Emerging opportunity Combination Therapies: Combination therapies would be an emerging opportunity in the gene therapy market wherein gene therapy is combined with another mode of treatment such as immunotherapies or conventional drug therapies. This multi-mode approach improves efficacy, reduces side effects, and targets several pathways simultaneously. This kind of multi-pronged approach may well yield better outcomes, particularly in complex diseases like cancer.

Challenges

• High Development and Production Costs: This highly sophisticated and resource-intensive manufacturing of gene therapies involves complex technologies and facilities. Research, development, clinical trials, and manufacturing come with significant costs. This makes gene therapies some of the most expensive treatments in the market. The high development cost also limits the ability of production to scale up for companies to meet global demand and for patients to receive the treatment.
• Regulatory Approval Delay: Although there is support for gene therapies from the regulation, it may be a big issue if it takes time to get approval. The clinical trial process is long, and the most stringent safety and efficacy standards have to be met before the gene therapies in the market. This delay in the approval increases the cost for companies and decreases the access of patients to the new treatments. In addition to this, the regulatory landscape remains in transition, and policies vary region by region.
• Patient and Physician Resistance: Gene therapies are considered quite promising, yet a fraction of the medical world, that is to say, both patients and practitioners refuse to use gene therapies. There are serious reservations about long-term safety profiles the risks unknown regarding these gene therapies and the ethical implications surrounding genetic manipulation, leading both patients and physicians to remain skeptical. This has significantly retarded the use of these therapies, which cannot gain popularity if there is established alternative therapy.

U.S. Cell and Gene Therapy Market Segmental Analysis

The U.S. cell and gene therapy market is segmented into therapy type, therapeutic class, delivery method and end users. Based on therapy type, the market is classified into cell therapy, and gene therapy. Based on therapeutic class, the market is classified into cardiovascular disease, cancer, genetic disorder, rare diseases, oncology, hematology, ophthalmology, infectious disease, neurological disorders and others. Based on delivery method, the market is classified into in vivo and ex vivo. Based on end-users, the market is classified into hospitals, cancer care centers, wound care centers and others.

Therapy Type Analysis

Cell Therapy: Cell therapy is a form of medical treatment involving living cells used to prevent or treat disease. Cells are autologous (the patient's cells) or allogeneic (a donor's cells) and may be engineered in the lab before administration to the patient. Cell therapies are mainly used in regenerative medicine to repair or replace damaged tissues and cells, including conditions such as cancer, cardiovascular disease, and neurological disorders. This approach has attracted much attention because it can offer more personalized and targeted treatments.

Gene Therapy: Gene therapy is the introduction or alteration of genetic material within a patient's cells to treat or prevent disease. This can be done by replacing bad genes, correcting mutations, or adding new genes that can help in combating the disease. Gene therapy has proven to be promising for treating genetic disorders, cancers, and other complex diseases based on their etiologic causes. Gene therapy is becoming a new hallmark of personalized medicine due to its potential to specifically address genetic abnormalities. It aims to cure conditions based on research, and such techniques are being developed for other medical conditions as well.

Therapeutic Class Analysis

Cardiovascular Disease: Cardiovascular disease refers to a range of conditions affecting the heart and blood vessels, including coronary artery disease, heart failure, and arrhythmias. Cell and gene therapies have shown promise in addressing cardiovascular conditions by promoting tissue regeneration, repairing damaged blood vessels, or correcting genetic mutations that predispose individuals to heart disease. These therapies are indeed exciting, ongoing areas of medical research aiming to target the underlying causes at a cellular and genetic level, which enhance heart function, reduce symptoms, and hopefully even reverse some elements of cardiovascular disease.

Cancer: Cancer involves many different diseases of uncontrolled cell growth and spread. Cell and gene therapies in oncology attempt to target and treat cancer cells by modifying the patient's immune system or tumor cells. Gene therapies can introduce genes that make cancer cells more susceptible to treatment, whereas cell therapies may consist of a re-engineering process for the use of engineered immune cells, such as T-cells, that identify and destroy cancer. These innovative treatments are paving the way for more precise, individualized therapies that have the potential to improve survival rates and reduce side effects compared to traditional treatments.

Genetic Disorder: Genetic disorders are diseases caused by abnormalities in an individual's DNA, often inherited from parents. Cell and gene therapies hold immense potential in treating genetic disorders by targeting the genetic root causes. Gene therapy can be used for the replacement of defective genes, repair of mutations, and introduction of new genetic material to correct the disease. Such therapies are being developed in the treatment of inherited diseases such as cystic fibrosis, muscular dystrophy, and sickle cell disease. These are the hopes of long-term and even permanent cures to the disorder by addressing the genetic mutation that causes it, changing the treatment scenarios of genetic diseases.

Rarity Diseases: Rare diseases are, by definition, conditions that affect a small percentage of the population. Such diseases pose challenges because they have limited treatment options and less research conducted on them. Most rare diseases have a genetic origin; hence, cell and gene therapies are promising in such cases. The correction or replacement of genetic mutations causing rare diseases is what gene therapy promises to make possible in the treatment process. In some instances, cell therapy can also restore or even replace damaged tissues. The challenges associated with high prices and bureaucratic red tape exist, but research continues to reveal potential treatments for rare diseases, which heretofore could not be treated effectively.

Oncology: Oncothery is an area that deals with the diagnosis and treatment of cancer through the discovery of new theories in cellular and gene therapeutics. Gene therapy can manipulate the genetic content of the cells within cancers, so these are also used, making cancerous cells much more susceptible to treatments involving chemotherapy or radiation. Most applications of cell therapies in oncology involve altering a patient's immune cells, most typically their T-cells, for better identification of the cancers. These therapies form part of the very new, growing immuno-oncology arena, thereby bringing hope for patients to combat cancers resistant to traditional therapy, as well as widen possibilities for the personalizing of treatment.

Hematology: Hematology is medicine focused on disorders involving the blood and bone marrow. Hematology ranges from simple bleeding disorders or clotting to complex cancerous conditions. Cell and gene therapies in hematology are directed at the underlying causes of these diseases, which in most cases involve modification of blood cells or genes to correct a defect. For instance, gene therapy can be applied in the treatment of genetic blood disorders such as sickle cell disease and thalassemia by correcting defective genes that cause abnormal hemoglobin production. Cell therapies, including stem cell transplants, play an important role in restoring the blood supply in the body after treatments for blood cancers. These developments are a major stride toward individualized treatments in hematology.

Ophthalmology: Ophthalmology is a specialty concerned with the diagnosis and treatment of diseases in the eye. In ophthalmology, cell and gene therapies are being developed for a wide range of disorders, such as macular degeneration, retinitis pigmentosa, and other inherited retinal disorders. Gene therapies can be given to deliver healthy copies of defective genes to restore or improve vision. Cell therapies may be done through the transplantation of retinal cells or stem cells to replace damaged tissues that offer hope for restoring a person's sight who suffers from previously untreatable conditions. These new approaches may help stop or reverse vision loss, changing the course for people with eye diseases.

Infectious Disease: Infectious diseases caused by bacteria, viruses, or fungi continue to be a global health problem. Cell and gene therapies are emerging as potential treatments for some infectious diseases, particularly those caused by viruses. For example, gene therapy can modify the genetic makeup of immune cells to increase their effectiveness in combating infections, while cell therapies may give patients immune cells that can target infectious agents. More gene-editing technologies, including CRISPR, which can target and modify the DNA of pathogens directly, open new avenues in the fight against infectious diseases, including antibiotic-resistant infections.

Neurological Disorders: Neurological disorders include Alzheimer's disease, Parkinson's disease, and multiple sclerosis, which result from the degeneration of the nervous system with limited treatments available. Cell and gene therapies may hold the promise of breakthroughs in treating such conditions. Gene therapy involves introducing therapeutic genes into the brain to correct or replace faulty genes responsible for neurological dysfunction. Cell therapy might involve stem cell transplantation or other cell types that can replace damaged neurons or tissues. These are emerging therapies but seem promising for treating complex and debilitating neurological conditions.

Delivery Method Analysis

In Vivo: In vivo delivery can be described as the introduction of therapeutic cells or gene therapies straight into a patient's body. In this method, therapeutic cells or genetic materials are introduced into the body to target disease sites from inside the patient's body. For gene therapy, methods employed in vivo include delivering genes via vectors, mostly viruses, directly into the cells of the patient. For cell therapy, usually, cells are injected or infused into the bloodstream, hence traveling to the area to be treated. In vivo, therapies offer the benefits of targeting a particular tissue or organ, so they are accurate and very effective in most diseases.

Ex Vivo: Ex vivo delivery involves cell manipulation or engineering outside the patient's body for later introduction into the body to affect treatment. This involves harvesting cells from the patient, known as autologous, or from a donor, which is allogeneic, modifying or treating the cells in a laboratory, and then transplanting or infusing them back into the patient. Gene therapy might include editing the genes of those cells to correct mutations, while cell therapy could be expanding or reprogramming stem cells for therapeutic use. Ex vivo therapies highlighted the future possibilities of stem cell treatments and immunotherapies that create highly customized and target-directed treatments for different diseases.

End User Analysis

Hospitals: Hospitals are the primary delivery sites of advanced cell and gene therapies. Hospitals are centers of medical expertise and infrastructure where all the equipment, well-trained medical staff, and monitoring systems to support these complicated therapies are available. Gene or cell therapies usually require high-intensity procedures, from pre-treatment screening to preparation, administration, and post-treatment follow-up care. Hospitals are well-stocked for these high-risk treatments, especially in specialized units like oncology, hematology, and cardiology. Cell and gene therapies are also very dynamic; hence, hospitals are fundamental for clinical trials and are providing the latest treatments possible to patients.

Cancer Care Centers: Cancer care centers are specialized facilities committed to diagnosing, treating, and managing cancer. They are important for delivering targeted cancer therapies, such as cutting-edge cell and gene therapies. Specialized treatments, such as CAR-T cell therapy, where a patient's T-cells are genetically modified to fight cancer, are offered in cancer care centers. Gene therapy is also being pursued in cancer care centers for the administration of targeted therapies that might affect cancer cells or boost immune responses. Such centers play a very crucial role in offering tailored cancer care, as often access to the latest therapies under clinical trials and established treatments tailored to individual patient needs is possible.

U.S. Cell and Gene Therapy Market Top Companies

• Bluebird Bio
• Sangamo Therapeutics
• Editas Medicine
• AveXis (a Novartis company)
• Gilead Sciences (Kite Pharma)
• Fate Therapeutics
• Bellicum Pharmaceuticals
• Legend Biotech
• Allogene Therapeutics
• Poseida Therapeutics
• Rocket Pharmaceuticals

Newcomers in the cell and gene therapy industry focus their attention on innovative, next-generation therapies that seek to bridge unmet medical needs with diverse diseases. These companies aim at innovative methods, such as CRISPR-Cas9 techniques, which correct genetic mutations in an even more precise level; as well as pioneering cell-based therapies that are aimed to regenerate damaged tissues or even enhance immune responses. New entrants are looking at the accessibility, scalability, and affordability of therapies. They are working with big biopharma companies as well as academic institutions to speed up their clinical development and commercialization. The innovation flood stimulates competition and leads to rapid breakthroughs in the field.

CEO Statements

Andrew Obenshain, CEO of Bluebird Bio:

• "Cell and gene therapies have the potential to transform the treatment landscape for patients with serious genetic diseases. At Bluebird Bio, we are committed to developing life-changing treatments that harness the power of genetic science to bring long-term solutions to those in need."

Gilmore O'Neill, CEO of Editas Medicine:

• "At Editas Medicine, we are committed to advancing the field of gene editing to offer transformative therapies that can address some of the most challenging diseases. We believe that with our innovative CRISPR-based technologies, we have the potential to change the course of medicine and improve lives through precision therapies."

Bob Valamehr, CEO of Fate Therapeutics:

• "At Fate Therapeutics, we are committed to harnessing the transformative potential of cell and gene therapies to address some of the most pressing unmet needs in medicine. Our pioneering approach is designed to offer new hope for patients with serious and life-threatening diseases by delivering innovative, personalized treatments that have the potential to change lives."

Recent Developments

• In May 2024, Bluebird Bio carried out its first cell collection to support the gene therapy called LYFGENIA, which is being developed to treat sickle cell disease. The cell collection event was conducted at a Qualified Treatment Center and marks an important step in the commercialization process of this therapy. Designed to add a functional β-globin gene to a patient's hematopoietic stem cells, LYFGENIA may offer both an increase in hemoglobin and a reduction in vaso-occlusive events. This development underlines Bluebird Bio's commitment to gene therapy solutions for patients who suffer from severe genetic disorders, especially those with sickle cell disease.
• In October 2024, Sangamo Therapeutics agreed with the FDA to accept accelerated approval for its gene therapy ST-920 to treat Fabry disease. It submitted a Biologics License Application in the latter part of next year after the FDA grants a Type B interaction. As an intermediate endpoint, it utilized one-year post-treatment estimated glomerular filtration rate slope data from its ongoing Phase 1/2 STAAR trial. That pathway placed the market availability a few years ahead of current approaches, say three years, which should thereby fill the significant unmet medical needs of Fabry patients.
• In October 2024, Legend Biotech launched a completely new research and development center in Philadelphia in the year 2025. The new facility was established for cell therapies innovation, thus taking one step further than what the company already offered through its CAR-T therapy development. It was an interim location site for cell therapy innovation collaborations with academic institutions as well as biotech companies surrounding the site. Legend is expanding because it wants to push innovative treatments for patients to achieve better oncology outcomes. An investment is but one step in a more significant strategy set up to strengthen its presence in the U.S. biopharma market.

Market Segmentation

By Therapy Type

• Cell Therapy
  
  • Stem Cell Therapy
    
    • Hematopoietic Stem Cell Therapy
    • Mesenchymal Stem Cell Therapy
    • Other
  • Non-Stem Cell Therapy
    
    • T-Cell Therapy
    • Dendritic Cell Therapy
    • NK Cell Therapy
• Gene Therapy
  
  • In vivo Gene Therapy
    
    • Viral Vectors
    • Adenovirus
    • Lentivirus
    • Retrovirus
    • Others
  • Non-viral Vectors
    
    • Naked DNA
    • Oligonucleotides
    • Others
  • Ex vivo 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-Users

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