Cell and Gene Therapy Clinical Trials Market By Therapy Type (Cell Therapy {Autologous Cell Therapy, Allogeneic Cell Therapy, CAR-T Cell Therapy, Natural Killer Cell Therapy, Dendritic Cell Therapy, Stem Cell Therapy}, Gene Therapy {In-vivo Gene Therapy, Ex-vivo Gene Therapy}), By Phase (Phase I, Phase II, Phase III, Phase IV), By Disease Type (Oncology, Genetic Disorders, Neurological Disorders, Cardiovascular Diseases, Autoimmune Diseases, Infectious Diseases, Others), By Trial Sponsor (Pharmaceutical Companies, Biotechnology Companies, Academic and Research Institutions, Government and Non-Profit Organizations, Contract Research Organizations), By Gene Therapy Type (Viral Vector-based Gene Therapy {Adeno-associated Virus, Lentivirus, Retrovirus, Adenovirus}, Non-viral Vector-based Gene Therapy, Others), By Route of Administration (Intravenous, Intramuscular, Intrathecal, Intraocular, Subcutaneous, Others), and By End-User (Hospitals and Clinics, Academic and Research Institutes, Biopharmaceutical Companies, Contract Research Organizations, Others), Global Market Size, Segmental Analysis, Regional Overview, Company Share Analysis, Leading Company Profiles, and Market Forecast, 2025 – 2035.

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What trends will shape the Cell and Gene Therapy Clinical Trials Market in the coming years?

The Cell and Gene Therapy Clinical Trials Market accounted for USD 11.02 Billion in 2024 and USD 12.75 Billion in 2025 is expected to reach USD 54.76 Billion by 2035, growing at a CAGR of around 15.69% between 2025 and 2035. The cell and gene therapy clinical trials market is a transformative market; this is attributed to the development of genomic technologies and the growing approval of new therapies, and there is also a growing number of rare and genetic diseases.

AI and machine learning are increasingly being used in trial design, patient recruitment, and the analysis of data so that efficiencies are improved and timelines are shortened. There is an increasing move to decentralized and adaptive models of the trial, which make flexible monitoring in real-time possible. Additionally, the increasing regulatory encouragement, increased public and private funding, and an upsurge of partnerships between research institutions and biotech companies are boosting innovation. With the establishment of individualized medicine in the healthcare setting, there will be a growing need for patient-specific targeted treatment, thus shaping the future scenario of this marketplace.

What do industry experts say about the Cell and Gene Therapy Clinical Trials market trends?

“As trial complexity increases, the future of cell and gene therapy will depend on smarter trial designs, AI integration, and global site networks to reduce costs and accelerate development timelines.”

• Paul Evans, CEO & President of Velocity Clinical Research.

Which segments and geographies does the report analyze?

What are the key drivers and challenges shaping the Cell and Gene Therapy Clinical Trials market?

How does the increasing prevalence of rare genetic disorders stimulate global clinical research demand?

The Cell and Gene Therapy Clinical Trials Market is experiencing significant momentum due to the increase in rare genetic disorder cases, which are generating more worldwide demand for clinical research. Rare diseases are estimated to affect about 3.5-5.9% of the world population, and most of these diseases are genetic and are manifested at an early age. Such conditions are usually not cured with the help of therapeutic interventions, and scientists are trying to offer new kinds of therapies based on genes and cells. The rare disorders are likely to be genetically initiated (80%) and therefore the best candidates for innovative treatment methods. The necessity to apply disease-specific interventions is persuading governments and medical establishments to allocate funds to patient registries and rare disease networks.

These projects will help to find patients more easily and make the course of clinical trials much faster. There is an emerging interest in the research institutions in gene editing technologies to cure such complicated disorders. Highly burdened countries with inherited diseases are playing instrumental roles in the international clinical studies. The partnership between biotech companies, academia, and the pharma industry is growing desperate to compensate for this demand. In general, the high impact of rare genetic diseases is one of the primary factors that affect the development of this fast-growing clinical trial environment.

What impact do government incentives and funding have on advanced therapy development initiatives?

Government incentives and funding have a lot of impact on the cell and gene therapy clinical trials market, and they are instrumental in the promotion of research and the promotion of rapid clinical trials. Initiatives such as the Regenerative Medicine Advanced Therapy (RMAT) designation offered by the FDA have aided dozens of potentially novel therapies by defining a path to accelerated review and regulatory advice. In 2024 alone, the FDA received 59 requests for establishing RMAT, out of which 43 were granted, which goes to show how the agency is highly committed to expediting cell and gene therapies. On the same note, a combination of public programs like the Bespoke Gene Therapy Consortium run by the NIH has aided in normalizing and simplifying the development of gene therapy in rare diseases. Such initiatives cut down the time required to develop, decrease expenses, and stimulate the entry of private entrepreneurs.

The financial net companies have been able to jump into high-risk innovation spaces due to the existence of tax credits, the status of orphan drugs, and government-sponsored grants. Such incentives are especially helpful to startups and academic institutions so that they can advance their developments from the preclinical stage to clinical applications. The governments are also encouraging collaborations between the industry giants and the public research institutions to be able to have strong pipelines. Such strategic initiatives are speeding the time to therapy and expanding access for patients to new advanced therapies. Overall, popular goodwill is a critical momentum in determining prospects for the development of advanced therapies.

What challenges do high operational costs pose to small biotech trial scalability worldwide?

High costs of conducting operations are a major challenge to the cell and gene therapy clinical trials market, particularly to small and emerging biotech firms that would like to expand their trial operations on an international basis. Advanced therapy involves high costs in manufacturing vectors, building specialist facilities, and recruiting highly skilled people, which challenges limited budgets. In contrast to big drug companies, small biotechs do not always have funds in reserve to fund cross-border multiple-phase trials. Another cost and complexity factor is regulatory compliance with various countries, which tends to push the trial schedule back. Also, viral vectors and gene-editing tools are accepting clinical-level materials, which may be cost-prohibitive.

Contract research organizations (CROs) also require a lot of capital investment, and efficiency cannot be assured. These economic forces constrain innovation, reduce access to patients, and cause smaller companies to depend heavily on collaboration or being purchased by larger firms. Many potential treatments, therefore, find it hard to reach phase-two clinical trials. These cost concerns are major issues that need to be resolved to demonstrate fair and massively sustainable global growth in the cell and gene therapy sector.

How can AI-based trial matching systems enhance protocol efficiency and patient enrollment accuracy?

With cell and gene therapy, trial matching systems can be AI-based, and these systems will increase the accuracy of patient eligibility and more efficient protocols, much to the benefit of the Cell and Gene Therapy Clinical Trials Market. For example, AI systems have shown an accuracy of up to 87% in detecting eligible patients and greatly decreasing the screening time of a clinician. They are useful tools that automate the front-line tests, reducing manual errors and speeding up the hiring procedure. With a more accurate match of patients to inclusion requirements, AI facilitates a faster design of the protocol and consistent trial results. This is particularly important in cell and gene therapies in which patient selection can rarely exclude highly specific biomarkers and clinical parameters.

Administrative workloads, on the other hand, are minimized with AI, allowing scientists to concentrate on meaningful clinical processes. Besides, better matching enriches diversity and representation of enrollments, which further empowers the quality of data and confidence in regulations. AI is enabling trials to have smoother operations among several global sites, thanks to increased scalability and convenience at reduced operational costs. Finally, it improves the success rate and efficiency of high-cost and complicated trials in the market of cell and gene therapy clinical trials.

Why is decentralized trial design gaining momentum across global cell therapy development programs?

The decentralization of trials exhibited in the cell and gene therapy clinical trials market is the latest revolution being implemented due to its ability to resolve patient access and enrollment issues. The models enable the clinical processes through phone or local health service providers to take place outside the clinic or hospital, meaning patients do not have to travel miles to attend. As per data provided by the FDA, the average distance covered by clinical trial participants in the U.S. is 67 miles to sourcing study sites, which becomes a tall order for many.

The decentralized trials increase access to the trials, particularly for those living in rural and underserved areas, and participation and retention because of increased diversity. They are also able to simplify trial logistics and minimize operational interruptions, especially in cases where there are some global events such as pandemics. With the help of digital technologies and telehealth, the sponsor will be able to perform real-time monitoring and enhance the quality of data. Regulatory assistance is equally growing, and agencies are embracing flexible and patient-centric designs. This transition is particularly critical to complex cell and gene therapy protocols, with expanded coverage and scalability.

What are the key market segments in the Cell and Gene Therapy Clinical Trials industry?

Based on the product type, the Cell and Gene Therapy Clinical Trials Market is classified into Gene Therapy and Cell Therapy. The CAR-T cell therapy, which is becoming increasingly respected in the field due to its much-needed specificity and personalization, as stem cell therapies have been successfully used in regenerative medicine and neurologic disorders. Autologous treatments are less likely to be rejected by the immune system but are rather expensive, and allogeneic treatments enable scalability and are off-the-shelf.

The revolutionary gene therapies are proposed to transform medical treatment in the case of genetic and rare diseases, and both in vivo and ex vivo approaches are actively being developed. Specific types of viral vectors (e.g., AAV and lentivirus) are the dominant vectors of gene delivery, although there is an interest in non-viral technologies that might present safer and more scalable options. The recent backlog of new therapies and the rise of trial activity in both sectors prove that the market is ever-changing.

Based on the application, the Cell and Gene Therapy Clinical Trials Market is classified into viral vector-based and non-viral vector-based therapies. The viral vectors, in particular, adeno-associated virus (AAV), lentivirus, and retrovirus, are the most prominent delivery vectors owing to their high packaging efficiency and long-term expression capabilities. The AAV vectors find the most application in the treatment of retinal diseases and muscular diseases, along with neurological disorders, and lentiviral vectors become prominent in hematologic and immunologic therapies.

The non-viral vectors (such as plasmid DNA, liposomes, and nanoparticles) also come into focus because they can be less immunogenic, are easy to manufacture, and have the capacity to be dosed repeatedly. In vivo versus ex vivo mode of delivery also impacts the design of trials, as in vivo delivery is less cumbersome to administer, and ex vivo provides more control in the integration of genes. The diversification of gene therapy solutions is also realized with innovation in gene editing technologies such as CRISPR. These therapies are fast being transformed into clinical applications due to the growing regulatory approval and investment in the therapies.

Which regions are leading the Cell and Gene Therapy Clinical Trials market, and why?

The North America Cell and Gene Therapy Clinical Trials Market is leading due to the healthcare infrastructure, the presence of the largest biopharmaceutical enterprises, and a very conducive regulatory framework. The United States especially represents one of the centers of cell and gene therapy development promoted by government entities such as NIH and NIH and fast-tracked by the US FDA through programs, including RMAT and Fast Track.

The concentration of clinical trial sites, academic research, research institutions, and contract research organizations (CROs) also enhances the innovative and development potential of the region. There is also a high number of rare diseases and cancer patients in North America, which helps faster patient recruitment and trial performance. Biotech startups are partnering with large pharmaceutical firms to play a key role in expediting the development of therapies. North America is likely to stay on top ahead of the other regions in the world, as its future in the market is certainly attributed to the continued investment in genomic research and personalized medicine.

The Asia Pacific Cell and Gene Therapy Clinical Trials Market is growing due to the rising investments in the market of biotechnology, government, and a swiftly swelling medical care framework. China, India, Japan, and South Korea are the main countries contributing to this increase because they have a high number of patients, making recruitment quicker and different demographic populations easier to recruit. This has also seen the emerging region as a choice region to conduct clinical trials due to the reduction in operational costs and the increased number of qualified researchers and CROs.

Regulatory organs are slowly making approval processes less cumbersome so that trials can be started and undertaken more easily. There are more collaborations between local biotech firms and global pharmaceutical companies, speeding the innovation and transferring the technology. Asian countries have a strong chance of shaping the future development of cell and gene therapies owing to increased consideration of personalized medicine and the increased awareness of genetic disorders.

What does the competitive landscape of the Cell and Gene Therapy Clinical Trials market look like?

The Cell and Gene Therapy Clinical Trial Market is very competitive, and big companies such as CRISPR Therapeutics, Vertex Pharmaceuticals, Novartis, Gilead/Kite Pharma, Bluebird Bio, Spark Therapeutics, and Adaptimmune are developing strong pipelines in oncology, genetics, and rare diseases. Such firms are pouring millions of dollars into advanced CAR-T, TCR, and gene editing (such as CRISPR and base editing) technologies. The up-and-coming biotech companies like Sotio Biotech, Castle Creek Biosciences, Tessa Therapeutics, and Precision BioSciences are polishing off their niche markets with next-generation delivery programs and off-the-shelf allogeneic medicines. Joint strategic agreements, licensing, and fast-tracking of regulations are also ramping up the market action. Scaling the manufacturing process, vectoring optimization, and innovations in delivery routes continue to mark the difference in competition.

One of the latest examples is the acquisition of Verve Therapeutics by Eli Lilly, in which Lilly gained a CRISPR-based base-editing therapy to PCSK9 that, at early stages, demonstrated that it was able to lower LDL cholesterol by as much as 69%. The announcement has made Eli Lilly a success in the cardiovascular gene therapy field. Such therapies are faring well, and this is increasing the trust in in vivo gene editing platforms. Emerging rivalries in the industry are also influenced by the rising global investments and growing trial activity in the U.S., Europe, and Asia.

Cell and Gene Therapy Clinical Trials Market, Company Shares Analysis, 2024

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Which recent mergers, acquisitions, or product launches are shaping the Cell and Gene Therapy Clinical Trials industry?

• In July 2025, Eli Lilly completed the tender offer and will finalize the acquisition by July 25, 2025, adding Verve’s cardiovascular gene‑editing pipeline and bolstering Lilly’s presence in genetic medicine.
• In June 2025, Eli Lilly announced a definitive agreement to acquire Verve Therapeutics for up to $1.3 billion, integrating Verve’s CRISPR-based in vivo gene‑editing candidate VERVE‑102 (targeting PCSK9 for cholesterol/lipid reduction), which recently completed a Phase 1b trial with up to 69% LDL reduction.
• In March 2025, AstraZeneca agreed to acquire Belgian biotech EsoBiotec for up to $1 billion, securing next‑generation in vivo CAR‑T technology that enables injectable cell therapies for cancer and autoimmune diseases.

Report Coverage:

By Therapy Type

• Cell Therapy
  • Autologous Cell Therapy
  • Allogeneic Cell Therapy
  • CAR-T Cell Therapy
  • Natural Killer Cell Therapy
  • Dendritic Cell Therapy
  • Stem Cell Therapy
• Gene Therapy
  • In vivo Gene Therapy
  • Ex vivo Gene Therapy

By Phase

• Phase I
• Phase II
• Phase III
• Phase IV

By Disease Type

• Oncology
• Genetic Disorders
• Neurological Disorders
• Cardiovascular Diseases
• Autoimmune Diseases
• Infectious Diseases
• Others

By Trial Sponsor

• Pharmaceutical Companies
• Biotechnology Companies
• Academic and Research Institutions
• Government and Non-Profit Organizations
• Contract Research Organizations

By Gene Therapy Type

• Viral Vector-based Gene Therapy
  • Adeno-associated Virus
  • Lentivirus
  • Retrovirus
  • Adenovirus
• Non-viral Vector-based Gene Therapy
• Others

By Route of Administration

• Intravenous
• Intramuscular
• Intrathecal
• Intraocular
• Subcutaneous
• Others

By End-User

• Hospitals and Clinics
• Academic and Research Institutes
• Biopharmaceutical Companies
• Contract Research Organizations
• Others

By Region

North America

• U.S.
• Canada

Europe

• U.K.
• France
• Germany
• Italy
• Spain
• Switzerland
• Sweden
• Finland
• Netherland
• Poland
• Rest of Europe

Asia Pacific

• China
• Japan
• India
• Australia
• South Korea
• Singapore
• Indonesia
• Malaysia
• Philippines
• Rest of Asia Pacific

Latin America

• Brazil
• Argentina
• Mexico
• Rest of Latin America

Middle East & Africa

• GCC Countries
• South Africa
• Rest of the Middle East & Africa

List of Companies:

• CRISPR Therapeutics AG
• Vertex Pharmaceuticals Incorporated
• Gilead Sciences, Inc.
• Novartis AG
• bluebird bio, Inc.
• Sarepta Therapeutics, Inc.
• Spark Therapeutics, Inc.
• Editas Medicine, Inc.
• Intellia Therapeutics, Inc.
• Sana Biotechnology, Inc.
• Beam Therapeutics Inc.
• Verve Therapeutics, Inc.
• Precision BioSciences, Inc.
• Adaptimmune Therapeutics plc
• Regenxbio Inc.

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