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New Opportunities unfolding in the Cell and Gene Therapy Market

• The Dawn of In Vivo Gene Editing Therapies: 

It has been a transformative trend for in vivo gene editing, in which therapeutic edits to a patient's DNA occur directly in the body. This strategy makes it easier to handle the logistics of treatment and opens up the opportunity for treatment of a broader spectrum of genetic disorders. Companies are making great strides, e.g. Intellia Therapeutics, NTLA-2002, an in vivo Crispr based therapy for hereditary angioedema, is in late stage clinical trials in 2025. Editas Medicine also showed preclinical proof-of-concept for the company's in vivo gene editing approach at the end of 2024. The development of sophisticated delivery systems, such as lipid nanoparticles (LNPs), is key to this development and have the advantage of targeting tissues, such as the liver and hematopoietic stem cells, very precisely. This move towards single one-off curative treatments is an enormous opportunity to transform care of many genetic diseases.

• Automated and Decentralized Manufacturing Solutions:

In order to overcome the substantial logistic and cost challenges of the current conventional manufacturing paradigm, the cell and gene therapy market is increasingly concentrating on automation and decentralization. There are now 38 cell and gene therapies approved by the US Food and Drug Administration, and many of them were developed using manual processes that are hard to scale. The future is pointing towards purpose-built, automated systems capable of dealing with smaller and personalised batches in an efficient manner. This includes a trend towards decentralized manufacturing to bring the manufacturing process closer to clinical centers to decrease turnaround times and costs. In October 2024, Cellular Origins signed a deal with Fresenius Kabi to incorporate its robotics into a new manufacturing platform. Similarly, in April 2024, Multiply Labs and Stanford Medicine launched a collaboration to show the potential of automation in cell therapy production. These sorts of innovations are important to help make therapies more accessible and commercially viable.

Allogeneic "Off-the-Shelf" Therapies Indicate the New Era of Accessibility

The cell and gene therapy market is undergoing a pivotal change with the advent of allogeneic or "off-the-shelf" therapies. Derived from healthy donor cells, these types of treatments can be produced in large batches, stored and made available on-demand, for multiple patients. This is in contrast with autologous therapies, which are patient-specific and have a long and expensive manufacturing process. The movement towards allogeneic solutions holds the promise of democratizing access to treatment, reducing delays, and greatly lowering production costs. In 2025, Lonza was able to scale-up its commercial production of allogeneic cell therapies with closed system automation, an important step towards making the processes cost-effective.

The clinical pipeline of these universal therapies is progressing at a rapid pace. Poseida Therapeutics P-BCMA-ALLO1 which is an allogeneic CAR-T therapy for Multiple Myeloma is now in Phase 1 trials with encouraging early data. TC BioPharm's lead candidate, OmnImmune, is an allogeneic gamma-delta T cell therapy that has been modified in no way, and it is in Phase 2/3 trials for acute myeloid leukemia. In a notable 2024 study on 16 patients, an allogeneic CAR T-cells therapy CTX130 demonstrated a reassuring safety profile in patients with advanced kidney cancer. Furthermore, an 86% overall response rate was reported for a Phase 1 trial for relapsed multiple myeloma in 35 patients with multiple myeloma who received an allogeneic CAR T therapy. In another trial, seven patients have been treated at Houston Methodist with an allogeneic CAR T-cell therapy since April 2024 with three complete remissions. These developments highlight the potentiality of allogeneic platforms in the cell and gene therapy market at an enormous level.

Manufacturing/CdmOs Expansion Leaps to fill Unprecedented Market Demand

The tremendous growth of the cell and gene therapy market is driving a massive expansion of the manufacturing capabilities, as well as the supporting Contract Development and Manufacturing Organization (CDMO) ecosystem. As more and more therapies progress through clinical trials the need for GMP compliant specialized production facilities has become paramount. This has spurred a wave of investment in new infrastructure and technologies that would be able to deal with the special complexities of manufacturing viral vectors and cell-based medicines at scale. This surge is critical to eliminate production bottlenecks and ensure that a steady stream of these transformative treatments can reach patients.

Recent activity indicate the extent of this rise in the cell and gene therapy market. In 2025, specialized CDMO Matica Biotechnology has brought in more than 10 new manufacturing projects in the first half of the year alone. In June 2025, CDMO Vector BioMed signed an agreement with a research hospital in India to increase access to CAR T-cell therapy. Infrastructure investments are also major. Lonza opened a new facility for viral vector manufacturing in Massachusetts in 2024 to service late-stage trials. Thermo Fisher came out with Gibco CTS DynaCellect in 2024 for automated selection of T cells and is expected to introduce closed modular platforms for lentiviral production in 2025. Catalent improved its Maryland gene therapy facility in 2024 and expanded into turnkey viral vector services in 2025. Furthermore, Pharmaron is undertaking a PS151M expansion of its UK based gene therapy CDMO site, including 3,500sqm of future GMP commercial scale space and quadruples its process development capacity.

Segmental Analysis

Beyond cancer: Infectious diseases leading by therapeutic market share

The infectious disease segment is projected to have the largest market share in the cell and gene therapy market in 2024, due to the growing number of applications of these advanced treatments against a variety of infectious conditions. The use of RNA-based therapies in particular, is on the rise when it comes to treating infectious diseases. A significant event in 2025 was the announcement of a new collaboration with the aim of developing next-generation therapies against serious infectious diseases. The market is also witnessing a growth of new biotech startups that are focused on this therapeutic area.

The potential of cell and gene therapies to provide long-term or even curative solutions for chronic infections is a major contributing factor to the research and investment. For example, in August of 2024 antisense technology was awarded a "Sakigake Designation" in Japan for the development of a Hepatitis B treatment. In addition, the approval of an mRNA vaccine for the respiratory syncytial virus (RSV) was made in the second quarter of 2024. The development of gene editing technology such as the one proposed by the use of CAR-T cells to infecting diseases is strengthening, and some preclinical studies are evaluating the role of CAR-T cells to target viral reservoirs within the cell and gene therapy market.

In-Vivo Methods Deliver the Revenue Leadership in Unexcelled Therapeutic Efficiency

The in vivo delivery method took the largest revenue share in the cell and gene therapy market in 2024 - this is mainly because of its efficiency and its potential for wider applications. A major milestone has been the development and delivery of a bespoke in vivo CRISPR therapy to a patient in just six months in 2025 and therefore the possibilities for rapid personalised treatment. The in vivo segment is expected to be the fastest-growing segment in the gene therapy market, due to the ability to deliver systemic treatments, which can target various organs.

The cell and gene therapy market is experiencing a significant push for in vivo cell therapy applications by top biotech companies. Companies such as Capstan Therapeutics are bringing in vivo CAR-T candidates to clinical studies in 2024. The approval of the first in vivo gene therapy, Luxturna, in 2017 opened the floodgates of the current innovation. Investment in new in vivo delivery technologies, such as advanced viral vectors and non-viral technologies, are some prominent trends. There is also increasing interest in the development of redosable in vivo therapies, which could increase their utility. The development of in vivo gene editing is a large focus of a number of companies in 2025, as it is believed to be key to opening gene therapy up to more common diseases.

Why Cancer Care Centers Are Commanding the Major End-User Market Share

The cancer care centers segment generated the major market share during 2024 in the cell and gene therapy market, indicating the high concentration of cancer treatments in the cell and gene therapy market. The number of patients treated with such therapies by oncologists every year rose on average from 17 to 25 in 2025 - a sign of increasing experience and adoption. The pipeline for oncology is currently healthy with 178 new drug candidates advancing to late-stage development during the last year. The increasing number of people in the UK suffering from cancer, with the total number of people suffering in 2024 estimated to be over 3 million, and the number projected to increase to 3.5 million by 2025, means there is a clear need for the development of innovative treatments.

These specialised centers are a necessary part of the administration of complex therapies such as CAR-T. 7 such products have been approved by the FDA for administration. However, the number of qualified treatment centers in the US has stayed constant between 2024 and 2025, indicating difficulty with accessibility. Leading institutions such as OSUCCC - James were one of the first institutions to provide CAR T-cell therapy demonstrating the pioneer role of these institutions. The success of these therapies throughout the cell and gene therapy market depends heavily on the infrastructure and expertise in these centers and data regarding patient outcomes from these facilities are important for reimbursement.

Gene Therapy to Contribute Over 54% Revenue to Global Market

The gene therapy set to contribute the largest 54% revenue to the global cell and gene therapy market—is primarily driven by the classification of high-revenue gene-modified cell therapies (such as CAR-T) within this category, along with the successful commercialization of high-priced, “one-time cure” treatments. Unlike traditional chronic medications, therapies such as Zolgensma (USD 2.1 million) and Elevidys (USD 3.2 million) command premium pricing that generates substantial upfront revenue upon patient access. The most prominent therapy witnessing explosive demand in 2024 is Sarepta’s Elevidys (for Duchenne Muscular Dystrophy), which generated approximately USD 821 million in full-year sales following its FDA label expansion to treat a broader age range (ages 4 and above).

In oncology, Johnson & Johnson’s Carvykti has shown rapid growth, with Q3 2024 sales increasing 87% to reach USD 286 million, putting it on track to become a blockbuster alongside Gilead’s Yescarta, which leads the category with USD 1.6 billion in annual revenue. It has been found that the demand for gene therapy is heavily concentrated in Oncology (specifically relapsed/refractory blood cancers) and Rare Genetic Diseases (SMA, DMD, Sickle Cell). The approval of Casgevy—the first CRISPR-based therapy for Sickle Cell Disease—has opened a new multi‑billion‑dollar segment, though adoption remains gradual due to logistical complexities.

Geographically, cell and gene therapy market demand is overwhelmingly driven by North America, which holds a ~50–54% share. This regional dominance is supported by a favorable reimbursement infrastructure, a dense network of FDA-approved treatment centers (over 50 activated for Casgevy alone), and sustained R&D investments. For instance, Sarepta Therapeutics reported total product revenues of USD 1.79 billion in 2024, largely fueled by U.S. adoption of Elevidys—illustrating how a single region’s reimbursement policies can shape global revenue dynamics.

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Regional Analysis 

North America dominance defines the global cell and gene therapy market

North America has the command of the market by accounting for its dominant share of over 39.50%. The leadership of the region is led by a strong and unmatched innovation ecosystem. In 2024, the FDA in the US approved nine new cell and gene therapy products, highlighting a positive and proactive regulatory environment. This climate is creating an enormous research pipeline with more than 2,500 active Investigational New Drug (IND) applications for cell and gene therapies currently on file with this agency. There is a lot of money to put to work to support that progress; the US National Institutes of Health (NIH) has allocated more than $6 billion to cell and gene therapy research in 2024 alone.

The commercial infrastructure that is supporting the cell and gene therapy market is also growing rapidly. The North American cell therapy manufacturing automation market size was valued at a significant $710 million in 2024, indicating the high level of attention on a scalable form of production. Canada is also doing much, including one project that received an investment of more than $580 million to build the country's largest cell and gene therapy manufacturing facility, which opened in October 2024. The Canadian government further pledged to a research centre through an almost $140 million investment to expedite drug development. The U.S. has more than 400 enterprises that are actively developing these therapies with a specialized automation market with a worth of $490 million by 2024.

Europe's Growing Infrastructure Consolidates its Strong Position in the Market

Europe is getting cemented as an important market in the cell and gene therapy space owing to strategic investments in manufacturing and a growing research base. Germany is in the lead with 660 biotech companies, a large portion of which are dedicated to CGT development. The country has undertaken more than 50 clinical studies in the field of gene therapy, showing a deep commitment to the development of this area. The United Kingdom is also a major player with the Cell and Gene Therapy Catapult running ground-breaking pre-GMP sandbox environments to help developers scale up manufacturing. 

In a major infrastructure development, Bristol Myers Squibb is finishing a 200,000 square foot cell therapy facility in Leiden in The Netherlands, which was operational at the end of 2024. In another important expansion, as a new gene therapy manufacturing site, Ferring Pharmaceuticals set up a new facility in Finland in October 2024 to strengthen the supply of the company's bladder cancer therapy.

Asia Pacific to Prove to be a Dynamic and Rapidly Accelerating Market

The Asia Pacific region is the fastest growing cell and gene therapy market, driven by an explosion of clinical trial activity, as well as robust government support. There are now more than 650 cell and gene therapy clinical trials in progress throughout the APAC region. China is in the lead, carrying out more than 2,000 trials with cell therapies, one-third of all the trials in the world. As of August 2024, there are 2,519 investigator-initiated trials in China. 

In a major policy switch, China had started to allow foreign investment in cell and gene therapy in four major free trade zones in China in September 2024. South Korea is also putting serious money into it with the government allocating more than $1.2 billion to regenerative medicine projects. In early 2024, Korean biotech company GeneEdit raised $35 million during a Series B funding round. India is also another fast emerging player in the game; the value of the domestic gene therapy market in India was worth $472.24 million in 2024.

Strategic Investments and Acquisitions Ensuring Consolidation and Growth in the Cell and Gene Therapy Market

• BioNTech Acquires Pionyr Immunotherapeutics (January 2025): BioNTech completes its acquisition of Pionyr and its pipeline of novel myeloid-targeting antibody therapies, strengthening its oncology portfolio.
• Astellas Acquires Propella Therapeutics (February 2025): Astellas acquisitions Propella for around $175 million Astellas to get hold of its steroid receptor modulator, PRL-02, for treating prostate cancer.
• Gilead Sciences Acquires CymaBay Therapeutics (March 2024): In a significant $4.3 billion acquisition, Gilead acquired CymaBay in order to add its primary biliary cholangitis drug candidate seladelpar to its portfolio of liver disease products.
• AstraZeneca Acquires Amolyt Pharma (March 2024): AstraZeneca used up to $1.05 billion to acquire Amolyt, a rare endocrine diseases specialist, to build its rare disease pipeline.
• Bristol Myers Squibb Acquires Karuna Therapeutics (March 2024): BMS has finished its $14 billion acquisition of Karuna, obtaining its promising schizophrenia treatment KarXT and adding to the company's neuroscience portfolio.
• AbbVie Acquires ImmunoGen (February 2024): AbbVie completed its $10.1 billion acquisition of ImmunoGen, which gave it access to its centerpiece antibody-drug conjugate (ADC) ELAHERE for ovarian cancer.
• Novartis Acquires MorphoSys (February 2024): Novartis announced that they are looking to purchase MorphoSys for EUR2.7 billion in order to acquire a promising late-stage oncology asset.
• Johnson & Johnson Acquires Ambrx (January 2024): Johnson & Johnson announced it will purchase Ambrx Biopharma for $2 billion to access its portfolio of ADC candidates for a range of cancers.

Top Companies in the Cell and Gene Therapy Market

• ALLOGENE THERAPEUTICS
• Amgen Inc.
• Astellas Pharma Inc.
• Atara Biotherapeutics, Inc.
• Bayer AG
• Biomarin
• Bluebird Bio, Inc.
• Bristol-Myers Squibb Company
• Celgene Corporation
• Cellectis
• Gilead Sciences, Inc.
• Hoffmann-La Roche Ltd
• ImmunoACT
• Johnson & Johnson
• Krystal Biotech, Inc
• Novartis AG
• Orchard Therapeutics plc
• Sana Biotechnology
• uniQure NV.
• Vericel Corporation
• Other Prominent Players

Market Segmentation Overview

By Therapy Type

• Cell Therapy
  • Stem Cell Therapy
  • T-Cells
  • Dendritic Cells
  • NK Cells
• Gene Therapy
  • In-vivGene Therapy
  • Ex-vivGene Therapy
  • Gene Editing Therapies
  • RNA-Based Therapies
  • Others

By Indication / Therapeutic Area

• Oncology
• Solid Tumors
• Genetic Disorders
• Neurological Disorders
• Cardiovascular Diseases
• Ophthalmology
• Musculoskeletal / Orthopedic Disorders
• Infectious Diseases
• Metabolic Disorders
• Others

By Vector Type (Gene Delivery Method)

• Viral Vectors
  • Adeno-Associated Virus (AAV)
  • Lentivirus
  • Retrovirus
  • Herpes Simplex Virus (HSV)
  • Adenovirus
  • Others
• Non-Viral Vectors
  • Lipid Nanoparticles (LNPs)
  • Naked DNA/RNA Plasmids
  • Electroporation
  • Gene gun / microinjection
  • CRISPR-Cas Delivery Systems (non-viral)
  • Others

By Manufacturing Type

• In-house
• Contract Development & Manufacturing Organizations (CDMOs)
• Hybrid Models (e.g., early stage in-house, scale-up outsourced)
• Others

By End User

• Hospitals and Specialty Clinics
• Academic and Research Institutes
• Biopharma and Biotech Companies
• CDMOs and CROS
• Government/Public Health Bodies
• Others

By Region

• North America
  • The US
  • Canada
  • Mexico
• Europe
  • Western Europe
    • The UK
    • Germany
    • France
    • Italy
    • Spain
    • Rest of Western Europe
  • Eastern Europe
    • Poland
    • Russia
    • Rest of Eastern Europe
• Asia Pacific
  • China
  • India
  • Japan
  • Australia and New Zealand
  • South Korea
  • ASEAN
  • Rest of Asia Pacific
• Middle East and Africa
  • Saudi Arabia
  • South Africa
  • UAE
  • Rest of MEA
• South America
  • Argentina
  • Brazil
  • Rest of South America