Cell and Gene Therapy Market

Overview

The global Cell and Gene Therapy Market was valued at USD 12.4 billion in 2025 and is projected to reach USD 55.3 billion by 2034, growing at a CAGR of 18% during the forecast period (2026–2034). The growth in the market is driven by the rising number of regulatory approvals for one-time treatments, the expansion of CAR-T therapies into earlier lines of cancer treatment, and the entry of gene therapies into rare genetic diseases that had no disease-modifying options. Growing payer comfort with outcomes-based contracts and the build-out of qualified treatment centers are also widening patient access across oncology, rare blood disorders, neuromuscular conditions, and inherited eye and skin diseases. The market is shifting from conventional,  patient-specific, ex vivo manufacturing of cell therapies for late-line blood cancers toward earlier-line use, off-the-shelf formats, and in vivo approaches that program cells directly inside the body. Developers are testing CAR-T therapies in autoimmune disease and solid tumors, while gene editing is moving from sickle cell disease and beta thalassemia toward liver and cardiovascular targets. Government initiatives such as the US Food and Drug Administration's June 2025 decision to eliminate Risk Evaluation and Mitigation Strategies for approved BCMA- and CD19-directed autologous CAR-T therapies have removed special hospital certification and on-site tocilizumab requirements, easing the way for more centers to deliver treatment. The reauthorization of the Rare Pediatric Disease Priority Review Voucher program through the Mikaela Naylon Give Kids a Chance Act continues to reward developers of therapies for children with rare genetic diseases, while Regenerative Medicine Advanced Therapy designation and accelerated approval shorten development timelines. By Region, North America held the largest share of the market in 2025, led by the United States, where most commercial launches, treatment centers, and reimbursement pathways for one-time therapies are established. Asia-Pacific is expected to be the fastest-growing region during the forecast period, driven by domestic CAR-T approvals in China, insurance-backed access in Japan, and expanding hospital capability in India and South Korea.

Market Size & Share

Market Dynamics

KEY MARKET TREND

In Vivo Cell Engineering and Off-the-Shelf Platforms Emerging as a Transformational Trend

• Developers are shifting from patient-specific manufacturing toward in vivo programming, where a targeted lipid nanoparticle or viral vector instructs a patient's own T cells to become CAR-T cells inside the body. This route removes cell collection and lymphodepleting chemotherapy, and could cut the treatment timeline from several weeks to a few days.
• Allogeneic donor-derived products and automated closed-system manufacturing platforms are advancing in parallel, giving treatment centers ready-to-infuse doses and shorter waiting times. Standardized batches also improve cost control and quality consistency, which matters as payers press for lower prices on one-time therapies and as hospitals look for predictable scheduling and fewer manufacturing failures.
• Large pharmaceutical groups with established CAR-T franchises are building in vivo and next-generation capabilities, which raises competitive pressure on single-asset biotechnology companies. Control of vector supply, lipid delivery technology, and automated manufacturing is becoming a source of negotiating leverage, so platform licensing and co-development agreements are now the main route to scale.
• AbbVie completed its USD 2.1 billion acquisition of Capstan Therapeutics on 19 August 2025. The deal added an in vivo targeted lipid nanoparticle anti-CD19 CAR-T candidate for B cell-mediated autoimmune diseases, designed to generate CAR-T cells without lymphodepleting chemotherapy, along with a platform for delivering RNA payloads to specific cell types.

 

KEY MARKET DRIVER

Expanding Regulatory Approvals and Growing Payer Access to One-Time Therapies Driving Market Growth

• A steady flow of regulatory approvals is converting late-stage pipelines into marketed products, particularly in ultra-rare diseases where no disease-modifying option existed before. Each approval adds revenue and also trains treatment centers, payers, and caregivers, which shortens the path for the next launch and builds confidence among investors who fund the next wave of programs.
• The sector reached five products with more than USD 1 billion in annual sales in 2025, as Breyanzi, Carvykti, and Elevidys joined Zolgensma and Yescarta. This record shows that one-time therapies can sustain commercial scale, and it encourages large pharmaceutical groups to commit capital to new cell and gene therapy programs.
• Hospitals are building dedicated cell therapy units, and more transplant and oncology centers are qualifying to deliver treatment. Better manufacturing success rates and shorter turnaround from cell collection to infusion are lowering patient drop-out, which turns referrals into revenue and supports steady growth in treated patient numbers across major markets.
• The Centers for Medicare & Medicaid Services announced that 33 states, the District of Columbia, and Puerto Rico will take part in its Cell and Gene Therapy Access Model. Participating states represent about 84% of Medicaid beneficiaries with sickle cell disease and receive rebates when therapies fail to deliver promised outcomes.

 

KEY MARKET OPPORTUNITY

Expansion into Autoimmune Disease and Solid Tumors Creating New Revenue Pools

• CAR-T therapies are moving into B-cell-driven autoimmune diseases such as lupus, myasthenia gravis, and systemic sclerosis, where patients rely on lifelong immunosuppression. A single infusion that resets the immune system could replace years of chronic drug spending and hospital visits, which makes the commercial case attractive for both developers and payers.
• Solid tumors remain largely untapped by cell therapy. Tumor-infiltrating lymphocyte and T-cell receptor products are already marketed in melanoma and synovial sarcoma, and better target selection and armored CAR designs are improving tumor penetration. Success in common solid cancers would open patient pools far larger than today's blood cancer base.
• Emerging markets offer a large volume opportunity through local manufacturing. Domestic CAR-T developers in China and India are producing therapies at prices far below Western levels, and governments in these countries are supporting hospital capability and clinical research, which lowers the cost barrier and brings treatment within reach of a much wider patient group.
• Kyverna Therapeutics began a rolling Biologics License Application submission to the FDA in 2026 for miv-cel, a CD19 CAR-T therapy for stiff person syndrome. It is the first submission for an autoimmune CAR-T therapy, follows FDA alignment on a single-arm pivotal trial, and could support a US launch in 2027.

Cell and Gene Therapy Market Size, 2025-2034 (USD Billion)

Segmentation Analysis

Analysis by Therapy Modality

CAR-T cell therapy held the largest market share in 2025 because it is the most commercially mature class in the market, with multiple approved CD19- and BCMA-directed products established in lymphoma, leukemia, and multiple myeloma. Label expansions into earlier lines of therapy have increased the number of eligible patients, while hospitals have built repeatable workflows for cell collection, infusion, and monitoring. Strong response rates, a record of durable remissions, and growing physician familiarity continue to support prescribing. Manufacturers have also improved production success rates and turnaround times, which helps convert referrals into treated patients and keeps revenue concentrated in this class.

 

Gene-edited therapy is projected to grow at the fastest CAGR during the forecast period as the first approved CRISPR-based treatments move from initial launch toward broad commercial scale. Ex vivo editing for sickle cell disease and beta thalassemia is expanding across treatment centers, while in vivo editing programs aimed at liver and cardiovascular targets are advancing through clinical trials. Base editing and prime editing offer the potential to correct single-letter mutations with greater precision, which widens the range of diseases that can be addressed. Partnerships between editing platform developers and large pharmaceutical companies are also increasing funding and commercial reach for this modality.

 

Therapy Modality categories include

                    ·           CAR-T Cell Therapy (Dominating Segment)

                    ·           Gene-Edited Therapy (Highest CAGR Segment)

                    ·           Non-CAR-T Cell Therapy

                    ·           AAV Gene Therapy

                    ·           Lentiviral Stem Cell Gene Therapy

                    ·           Others

 

Analysis by Delivery Method

Ex vivo delivery held the largest market share in 2025 because the leading commercial products, including CAR-T therapies, stem cell gene therapies, and engineered tissue products, are manufactured outside the body and returned to the patient as a finished cell product. This route benefits from controlled editing conditions, quality testing before infusion, and years of clinical and regulatory experience. Treatment centers have established workflows for cell collection, shipping, and infusion, and payers are familiar with the reimbursement models that apply. These factors keep ex vivo products at the center of commercial revenue even as newer approaches advance.

 

In vivo delivery is projected to grow at the fastest CAGR during the forecast period as developers move away from complex cell manufacturing toward medicines delivered directly into the patient. Viral vectors and lipid nanoparticles can carry genetic instructions to target tissues in a single administration, which removes the need for cell collection and shortens treatment time. This approach also supports larger patient populations, including those treated in community settings, and offers lower cost of goods at scale. Rising investment in in vivo CAR-T, gene editing, and gene replacement programs is expected to convert a large share of the late-stage pipeline into approved products.

 

Delivery Method categories include

                    ·           Ex Vivo (Dominating Segment)

                    ·           In Vivo (Highest CAGR Segment)

 

Analysis by Indication

Oncology held the largest market share in 2025 because blood cancers were the first disease area to receive approved cell therapies, and prescribing has since expanded across lymphoma, leukemia, and multiple myeloma. Larger eligible patient pools than rare genetic diseases, and clear survival benefits in patients with few remaining options, sustain demand. Oncologists and hematologists have also built referral networks with transplant and cellular therapy centers, which supports a consistent flow of patients. Continued label expansion into earlier treatment lines and the arrival of tumor-infiltrating lymphocyte and T-cell receptor therapies for solid tumors add further depth to this segment.

 

Autoimmune disorders are projected to grow at the fastest CAGR during the forecast period as CAR-T cell therapy is tested in lupus, systemic sclerosis, myasthenia gravis, and other neuroimmunology conditions. Early clinical results show that deep B-cell depletion can reset immune activity and allow patients to stop long-term immunosuppressive drugs, which creates a strong value argument for payers. Because chronic autoimmune conditions affect far larger populations than most rare genetic diseases, even a small number of approvals could expand the market sharply. Both ex vivo and in vivo approaches are in development, which supports a deep pipeline.

 

Indication categories include

                    ·           Oncology (Dominating Segment)

                    ·           Autoimmune Disorders (Highest CAGR Segment)

                    ·           Neuromuscular Disorders

                    ·           Rare Blood Disorders

                    ·           Ophthalmic Disorders

                    ·           Metabolic Disorders

                    ·           Others

 

Analysis by End User

Hospitals held the largest market share in 2025 because cell and gene therapies need closely monitored care, specialized apheresis and cell processing units, intensive care backup, and trained multidisciplinary teams. Large academic and tertiary hospitals act as qualified treatment centers and handle the majority of CAR-T infusions and stem cell gene therapy procedures. Their access to transplant programs, cryogenic storage, and established relationships with manufacturers makes them the default site of care. Hospital finance teams are also best equipped to manage the high upfront cost of these therapies and the reimbursement process that follows.

 

Specialty clinics are projected to grow at the fastest CAGR during the forecast period as treatment protocols mature and monitoring requirements ease for selected products. Outpatient infusion of CAR-T therapies is becoming more practical, and infusion centers focused on hematology, oncology, and rare diseases are adding cell therapy capability. Clinics offer shorter waiting times, lower facility costs, and closer proximity to patients, which is valuable for follow-up that can extend over many years. In vivo products that need no cell collection will further favor delivery in outpatient settings, adding to the shift away from large hospitals.

 

End User categories include

                    ·           Hospitals (Dominating Segment)

                    ·           Specialty Clinics (Highest CAGR Segment)

                    ·           Research Institutes

                    ·           Others

By Region

Cell and Gene Therapy Market Regional Analysis

Regional Analysis

North America held the largest market share in 2025, with the United States generating most of the regional revenue. The region has the highest number of approved products, the largest network of qualified treatment centers, and established pathways such as accelerated approval and Regenerative Medicine Advanced Therapy designation. US payers are testing outcomes-based contracts, while manufacturers have built dedicated cell and viral vector production capacity close to major academic centers. Canada contributes through Health Canada approvals of leading CAR-T products and treatment at designated provincial centers, while Mexico is at an earlier stage of adoption, with demand centered on a limited number of specialized hospitals. High clinical trial activity and deep venture and pharmaceutical investment keep the region ahead of other markets.

 

Asia-Pacific is projected to grow at the fastest CAGR during the forecast period. Japan is the largest country market in the region, supported by national insurance coverage for approved CAR-T and gene therapies and a conditional approval pathway for regenerative medicine products. China is the fastest-growing country market, with several domestically developed CAR-T products on the market, priority review pathways, and large clinical trial capacity at lower cost. India is building affordable local CAR-T manufacturing, and South Korea is expanding hospital-based regenerative treatment under its advanced regenerative bio framework. Rising cancer incidence, growing hospital investment, and regional manufacturing partnerships continue to widen access across the region.

 

Countries and Regions Covered

Asia-Pacific (Fastest Growing Region)

o  Japan (Largest Country Market)

o  China (Fastest-Growing Country Market)

o  India

o  South Korea

o  Rest of Asia-Pacific

North America (Dominating Region)

o  United States (Largest Country Market)

o  Canada

o  Mexico

Europe

o  Germany (Largest Country Market)

o  France

o  United Kingdom

o  Italy

o  Rest of Europe

Latin America

o  Brazil (Largest Country Market)

o  Chile

o  Rest of Latin America

Middle East & Africa

o  Saudi Arabia (Largest Country Market)

o  United Arab Emirates (Fastest-Growing Country Market)

o  Rest of Middle East & Africa

Market Share

The Cell and Gene Therapy Market is consolidated, with a small group of large pharmaceutical companies such as Novartis, Gilead Sciences, Bristol Myers Squibb, Johnson & Johnson, and Vertex Pharmaceuticals holding the largest commercial franchises, alongside a growing set of specialized biotechnology companies that own single-product or rare disease portfolios. Competition centers on clinical durability, manufacturing reliability, turnaround time, and access to qualified treatment centers. Key success factors include control of viral vector and cell production capacity, strong regulatory execution, and payer contracting skills. Leading companies are prioritizing label expansion into earlier treatment lines, next-generation CAR constructs, in vivo delivery, and automation of manufacturing. Partnerships between product developers and commercial partners, together with acquisitions of platform and late-stage assets, are the main routes used to add capabilities and speed to market.

 

Key Players

                    ·           Novartis AG (Switzerland)

                    ·           Gilead Sciences, Inc. (US)

                    ·           Bristol Myers Squibb Company (US)

                    ·           Johnson & Johnson (US)

                    ·           Legend Biotech Corporation (US)

                    ·           Vertex Pharmaceuticals Incorporated (US)

                    ·           CRISPR Therapeutics AG (Switzerland)

                    ·           Sarepta Therapeutics, Inc. (US)

                    ·           Roche Holding AG (Switzerland)

                    ·           CSL Limited (Australia)

                    ·           Krystal Biotech, Inc. (US)

                    ·           Iovance Biotherapeutics, Inc. (US)

                    ·           Autolus Therapeutics plc (UK)

                    ·           Ferring Pharmaceuticals (Switzerland)

                    ·           PTC Therapeutics, Inc. (US)

                    ·           Ultragenyx Pharmaceutical Inc. (US)

                    ·           Mesoblast Limited (Australia)

                    ·           CARsgen Therapeutics Holdings Limited (China)

                    ·           Abeona Therapeutics Inc. (US)

 

Recent Market Developments

• In February 2026, Gilead Sciences announced a definitive agreement to acquire Arcellx for an implied equity value of about USD 7.8 billion, giving Kite full control of anitocabtagene autoleucel, a BCMA-directed CAR-T therapy for multiple myeloma. The FDA has accepted the Biologics License Application, with an action date of 23 December 2026, positioning Gilead for a second myeloma CAR-T launch.
• In April 2026, Regeneron received US FDA accelerated approval for Otarmeni (lunsotogene parvec-cwha), the first gene therapy for hearing loss caused by biallelic OTOF variants. Approved under the Commissioner's National Priority Voucher program, it opens a new organ system to AAV gene therapy and shows how faster review pathways can shorten time to market for ultra-rare diseases.
• In June 2026, CARsgen Therapeutics received approval from China's National Medical Products Administration for satricabtagene autoleucel, a Claudin18.2-directed CAR-T therapy for advanced gastric and gastroesophageal junction adenocarcinoma after at least two prior lines of therapy. It is the world's first CAR-T therapy approved for a solid tumor, and it validates solid tumors as a commercial target for cell therapy.
• In August 2026, Ultragenyx received US FDA accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), an AAV8 gene therapy for patients aged eight years and older with glycogen storage disease type Ia. It is the first treatment aimed at the underlying cause of the disease and is manufactured entirely at the company's own gene therapy facility in Massachusetts.

Frequently Asked Questions