Gene Therapy Platform Market Size & Trends
Estimated to be USD 2.14 billion in 2024, the global market for gene therapy platforms is expected to rise at a CAGR of 15.2% from 2025 to 2030. Several important factors are driving the expansionrapid technology improvements, rising frequency of hereditary and chronic diseases, expanding biopharmaceutical research funding and investment, and favorable regulatory systems allowing faster approvals.
Key Highlights
- North America dominated the global market for gene therapy platforms and accounted for the largest share of 35.96% in 2024.
- The gene therapy platform market in the U.S. is experiencing rapid advancements in gene therapy
- By platform type, the viral vector platforms segment dominated the market with the largest revenue share of 58.12% in 2024
- By application, the oncology segment held the largest revenue share in 2024
- By delivery method, the in vivo gene therapy segment held the largest revenue share in the 2024 market
Innovations in viral vector technologies, gene editing tools including CRISPR, and the integration of AI and automation in manufacturing are enhancing therapy precision and production efficiency. For instance, in March 2023, Catalent is enhancing its UpTempo AAV platform to accelerate the development of gene therapies.
The increasing number of FDA approvals for gene therapies targeting rare and genetic diseases has validated the clinical potential of these platforms, encouraging investment and R&D. For instance, in December 2023, CRISPR-based therapies, like Exagamglogene autotemcel (Casgevy), received FDA approval for sickle cell disease and beta-thalassemia, which represents an important turning point in gene therapy. These breakthroughs are pushing the field forward, enabling more efficient technology for various genetic disorders.
The rising prevalence of genetic disorders and chronic diseases worldwide is fueling demand for gene therapies that target disease at the molecular level. Conditions such as cystic fibrosis, hemophilia, spinal muscular atrophy, and various cancers are increasingly treated with gene therapy approaches that offer potentially curative outcomes. The World Health Organization's (WHO) projected a sharp increase in cancer cases globally in 2024, with over 35 million new cases expected by 2050— up 77% from 2022. This growing patient pool creates urgent demand for advanced treatments, driving investment and innovation in gene therapy technologies to address unmet medical needs.
Moreover, regulatory support for innovative therapies is driving market growth. Regulatory agencies, including the FDA and the European Medicines Agency (EMA), have streamlined approval processes for gene therapies, recognizing their transformative potential. In addition, regulatory incentives such as fast-track designations and orphan drug status further support the development of gene therapies. These measures help reduce development timelines and encourage the introduction of new treatments to address unmet medical needs.
The COVID-19 pandemic significantly impacted the market, disrupting clinical trials, manufacturing, and supply chains. The increased demand for raw materials for COVID-19 vaccine production caused shortages, affecting the availability of essential components for gene therapy manufacturing. Despite these challenges, the pandemic also spurred innovation, with some gene therapy developers adapting their efforts toward COVID-19-related therapeutics and vaccines, highlighting the resilience and potential of the gene therapy sector.
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Market Concentration & Characteristics
The degree of innovation in the market is high, as the field is witnessing rapid advancements in gene editing technologies and viral vectors. For instance, in November 2024, iotaSciences will debut its Single-Cell Cloning Platform XT at Cell 2024 in London, enhancing the original platform with advanced fluid-shaping technology. It targets cell biology and gene therapy researchers, focusing on stem cell handling and gene editing. This upgrade helps create more precise and effective gene therapies, speeds up the search for new treatments, and improves the trustworthiness of gene-editing methods, resulting in quicker progress in clinical trials and better treatment results.
The market's M&A activities are medium. Companies are acquiring smaller biotech firms to expand their gene therapy pipelines and enhance their technological capabilities. For instance, in January 2024, Kyowa Kirin finalized its acquisition of Orchard Therapeutics, a global provider of gene therapy for rare diseases. This acquisition enhanced Kyowa Kirin’s capabilities in developing advanced gene therapies for rare genetic disorders and increased its access to Orchard's innovative gene-editing technologies.
Regulations significantly shape the market by ensuring safety, efficacy, and ethical standards. The FDA's draft guidance on the Platform Technology Designation Program, released in May 2024, greatly affects the market for gene therapy platforms by creating a way for companies with an approved gene therapy platform, like a viral vector or gene editing system, to use existing data for new products that use the same platform. This set of rules is designed to make the development and production of gene therapies easier by cutting down on unnecessary studies and speeding up FDA reviews, which helps get new gene therapies to market faster when they use established platforms.
Product substitutes for the market include traditional pharmaceuticals, such as small-molecule drugs and biologics, which manage symptoms or disease progression without altering genetic material. RNA-based therapies such as siRNA, mRNA, and antisense oligonucleotides provide alternatives by modulating gene expression. Protein replacement therapies, genome editing technologies, base and prime editing, and CRISPR-based tools such as Retron Library Recombineering (RLR) Platform, Cas-CLOVER Nucleases are also used outside of viral vector platforms as substitutes, providing less invasive or more targeted alternatives to gene therapy, depending on the treated condition.
Companies are increasingly investing in scalable gene therapy platforms that enable the efficient development and manufacturing of personalized treatments. For instance, in April 2024, AGC Biologics introduced a new division specifically for Cell and Gene Technologies. This division will improve AGC Biologics' abilities and help developers by offering more resources, scientific knowledge, and skilled workers in cell and gene contract development and manufacturing (CDMO). These platforms combine advanced gene editing, vector development, and cell engineering tools to create tailored therapies for specific genetic conditions. As demand rises, the expansion focuses on enhancing cost-efficiency, regulatory adherence, and treating a wider range of diseases, including cancers.
Platform Type Insights
The viral vector platforms segment dominated the market with the largest revenue share of 58.12% in 2024 due to their high transduction efficiency, ability to deliver genetic material to specific cells, and capacity for long-term expression. Technological advancements drive the widespread adoption of viral vectors in clinical applications, including oncology, genetic disorders, and infectious diseases. For instance, in May 2023, AGC Biologics launched its BravoAAV and ProntoLVV viral vector platforms, combining three decades of expertise in AAV and LVV development. These platforms enable fast, efficient GMP production and release, reducing development timelines to nine months for clinical and commercial use.
“The launch of these two platforms is the latest in a series of enhancements aimed at deliberately growing our cell and gene therapy services to support developers from early phases through clinical and commercial stage manufacturing. By offering a new templated approach for viral vector production, BravoAAV and ProntoLVV will support companies bringing these life-changing treatments by helping to accelerate project timelines, reach clinical and commercial milestones, and deliver effective therapies to patients as quickly as possible.”
The gene editing platforms segment is anticipated to grow at the fastest CAGR during the forecast period, driven by advancements in precision technologies, including CRISPR/Cas9, TALENs, and prime editing. For instance, in November 2022, Ionis partnered with Metagenomi to incorporate gene editing into its extensive technology platform, further advancing these tools for targeted genetic modifications. These innovations offer potential cures for genetic disorders, cancers, and infectious diseases, with CRISPR-based therapies, such as those for sickle cell disease, already gaining regulatory approvals.
Application Insights
The oncology segment held the largest revenue share in 2024 due to the increasing demand for targeted, personalized cancer treatments. Key drivers include advancements in CAR-T cell therapy, CRISPR gene editing, and other gene modification technologies that allow for more effective treatments. In addition, the segment continues to expand with innovations in gene therapy. For instance, in September 2024, Vironexis Biotherapeutics received FDA clearance for the first clinical trial of an AAV-delivered cancer immunotherapy, which represents a major leap forward in targeted, in vivo cancer treatment.
The hematological disorders segment is expected to grow at the fastest CAGR for the forecast period, driven by significant advancements in gene editing technologies and increasing clinical approvals. For instance, in June 2023, BioMarin's Roctavian, a gene therapy for severe hemophilia A, received FDA approval. This therapy offers patients the potential for long-term clotting factor production with a single infusion. This success demonstrates the future potential of gene therapy platforms to address complex blood disorders, including hemophilia, driving investment in platform technologies such as gene editing, viral vector delivery systems, and personalized medicine approaches.
Delivery Method Insights
The in vivo gene therapy segment held the largest revenue share in the 2024 market due to its ability to deliver genetic material directly into a patient's body, enabling targeted treatment of complex diseases. This method reduces the need for cell extraction and reinfusion, streamlining the therapeutic process. Key drivers include advancements in viral vector technologies, such as AAV and lentiviral vectors, and growing success in treating inherited disorders such as spinal muscular atrophy and hemophilia. For instance, Stylus Medicine established itself in May 2025 with the aim of advancing precision in vivo genetic therapies and maximizing the potential of CAR-T treatments. Its platform aims to improve the accuracy and scalability of gene integration, which speeds up the creation of lasting cures and enhances the effectiveness of in vivo gene therapy technologies.
The ex vivo gene therapy area is predicted to grow the quickest during the forecast period because it can accurately change cells outside the body before putting them back in, allowing for controlled and targeted treatment. Key drivers include advancements in gene-editing technologies such as CRISPR, increasing demand for personalized therapies, and improved cell culture and expansion techniques. This approach particularly effectively treats blood disorders, cancers, and immune-related diseases. In addition, according to an article published in January 2025, CAR-T cell therapy involves removing T-cells from the patient, genetically altering them to target cancer antigens, expanding them outside the body, and then reinfusing the modified cells into the patient. This process follows the ex vivo gene therapy approach, where cells are changed and expanded outside the body before being returned to the patient for treatment.
End Use Insights
The pharmaceutical and biotechnology companies segment held the largest revenue share in 2024, due to their significant investments in R&D, advanced manufacturing capabilities, and vital clinical pipelines. Pharmaceutical and biotech companies are investing heavily in advanced vector engineering technologies to enhance gene therapies' efficacy, specificity, and safety. For instance, in October 2024, Roche will use Dyno Therapeutics' engineered AAV capsid platform to develop advanced gene therapies for unspecified neurological diseases. This collaboration could generate over $1 billion in revenue for Dyno.
The Contract Research Organizations (CROs) and Contract Development and Manufacturing Organizations (CDMOs) segment is expected to grow at the fastest CAGR in the market for gene therapy platforms, driven by several key factors. The increasing number of gene therapy candidates progressing through clinical trials significantly raised the demand for specialized services offered by CROs and CDMOs. These organizations provide essential expertise in process development, regulatory compliance, and scalable manufacturing, enabling biotech firms to navigate the complexities of gene therapy development efficiently. In addition, integrating AI in manufacturing processes optimizes production efficiency and scalability, driving market growth.
Regional Insights
The North American gene therapy platform market dominated the global gene therapy platform market and accounted for the largest share of 35.96% in 2024. The region continues to dominate global gene therapy advancements, with a flourishing biotech ecosystem driving the development of advanced technologies such as viral vectors and CRISPR gene editing. For example, in May 2025, Andelyn Biosciences quickly created and provided a viral vector gene therapy for a baby with a very rare condition called NEDAMSS, finishing the production in only 10 months, showing how gene therapy is becoming a more available treatment choice.
U.S. Gene Therapy Platform Market Trends
The gene therapy market in the U.S. is experiencing rapid advancements, driven by the increasing prevalence of chronic genetic diseases and a growing pipeline of therapies. Government agencies such as the FDA continue to support the field by fast-tracking approvals for gene therapies, facilitating an environment conducive to rapid innovation. In addition, advanced gene-editing technologies, including CRISPR, enable precise targeting of genetic abnormalities, while regulatory incentives from the FDA encourage innovation and faster commercialization.
Asia Pacific Gene Therapy Platform Market Trends
The gene therapy platform market in Asia Pacific is experiencing rapid growth driven by increasing investments from countries such as China, India, and Japan in gene therapy research and clinical trials. Governments and private sectors prioritize rare genetic disorders and precision medicine, driving demand for personalized gene therapies tailored to diverse genetic profiles. Rules and guidelines are changing to allow quicker approvals and sales, while the area is becoming a center for high-tech manufacturing methods, like making viral vectors and using CRISPR for editing, which help lower treatment costs.
The gene therapy platform market in China is experiencing rapid growth, driven by advancements in biotechnology infrastructure, regulatory support, and clinical trial activity. In November 2023, Chinese startups leverage AI to advance adeno-associated virus (AAV) gene therapy development. Porton Advanced, a biotechnology company, partnered with BioMap, an AI firm specializing in life sciences, to enhance AAV vector design. BioMap's xTrimo platform, a large protein-centric language model, will be utilized to develop models that improve AAV assembly efficiency and distribution. This collaboration aims to overcome challenges in creating tissue-specific AAV capsids, a critical aspect of targeted gene therapies. By integrating AI with AAV technology, the partnership seeks to accelerate the development of gene therapies and expand their clinical applications.
Europe Gene Therapy Platform Market Trends
The gene therapy platform market in Europe is driven by innovative research and the region's expanding focus on personalized medicine and genetically based therapies. European governments and institutions are investing in biotech, particularly in the UK, Germany, and France, where technologies for delivering gene therapies are integrated into research programs targeting diseases such as cancer, cardiovascular disorders, and rare genetic conditions. Regulatory support from the European Medicines Agency (EMA) has enabled the commercialization of gene therapies.
The UK gene therapy platform market is characterized by rapid growth driven by substantial venture capital investment and a robust biotech ecosystem. The UK captures over 55% of European venture capital funding in life sciences, underscoring its leadership in cell and gene therapy (CGT) innovation. Key trends include increased focus on advanced therapies such as CAR-T, genome editing tools like CRISPR, and strategic collaborations and manufacturing capacity expansions. The sector also benefits from a strong pipeline of platform technologies to optimize scalability and automation in cell and gene therapy manufacturing. For instance, in May 2024, Ori Biotech unveiled its IRO Platform at the Annual International Society for Cell & Gene Therapy Conference, which represents an important step forward in manufacturing innovation.
Latin America Gene Therapy Platform Market Trends
The gene therapy platform market in Latin America is driven by increasing healthcare demands, especially in Brazil and Argentina. Brazil, in particular, is showing potential in gene therapy due to its large population base and improving healthcare infrastructure. Regional governments recognized the potential of gene therapy and are supporting its development. For instance, in March 2024, Fiocruz and Caring Cross partnered to produce CAR-T cell and stem cell gene therapies locally in Brazil, aimed at treating infectious, oncological, and genetic diseases, including leukemia, lymphoma, and HIV. The collaboration emphasizes affordable treatments, fostering local manufacturing, and using platform technologies for CAR-T production.
“By sharing our knowledge and expertise, this collaboration will establish local manufacturing capabilities for our innovative CAR-T cell manufacturing platform. When integrated with local point-of-care manufacturing, this approach enables us to drastically cut production expenditures and enable access to these therapies at a fraction of the cost compared to the U.S. and Europe. We are hopeful this collaboration will make these treatments more affordable and serve as a global model for improving access to advanced medical therapies.”
Middle East & Africa Gene Therapy Platform Market Trends
The gene therapy platform market in the Middle East and Africa (MEA) is experiencing notable growth, particularly in regions such as the UAE and South Africa, where healthcare systems are rapidly improving. Regional factors such as local biotech investments, regulatory frameworks, and disease prevalence influence the growth, which is driven by collaborations and developments within or targeting that region. For instance, in June 2024, the Department of Health - Abu Dhabi (DoH) signed a Memorandum of Understanding with Opus Genetics to accelerate the development of gene therapies for rare inherited retinal diseases (IRD), focusing on patient-centered treatments in the UAE. This partnership stimulates growth in the region's market for gene therapy platforms by encouraging the integration of advanced gene therapy techniques, such as viral vectors and CRISPR technologies, into clinical practice.
Key Gene Therapy Platform Company Insights
Major companies are focusing on M&A, strategic partnerships, expansions with technology providers, and product development. For instance, in October 2024, Terumo Blood and Cell Technologies (Terumo BCT) expanded its presence in Latin America to assist cell and gene therapy companies in Colombia, Brazil, and Mexico by providing automated manufacturing solutions. This expansion helps local developers and manufacturers simplify complicated processes for cell and gene therapy, which is important for making these treatments more available to patients in a region that depends a lot on government healthcare systems.
Key Gene Therapy Platform Companies
The following are the leading companies in the market for gene therapy platforms. These companies collectively hold the largest market share and dictate industry trends.
- Lonza
- BioIT
- Autonomous Ltd.
- Hypertrust Patient Data Care (Part of Accenture)
- IBM
- L7 Informatics, Inc.
- TrakCel
- IDBS
- Novartis
- SAP SE
- DEEP GENOMICS
- ElevateBio
- Sarepta Therapeutics, Inc.
- CRISPR Therapeutics
- Precision BioSciences
- AstraZeneca
- Andelyn Biosciences
- Renova Therapeutics
Gene Therapy Platform Market Report Scope
|
Report Attribute |
Details |
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Market size value in 2025 |
USD 2.43 billion |
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Revenue forecast in 2030 |
USD 4.93 billion |
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Growth rate |
CAGR of 15.2% from 2025 to 2030 |
|
Actual data |
2018-2024 |
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Forecast period |
2025–2030 |
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Quantitative units |
Revenue in USD million/billion and CAGR from 2025 to 2030 |
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Report Coverage |
Revenue forecast, company ranking, competitive landscape, growth factors, and trends |
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Segments Covered |
Platform type, application, delivery method, end use, region |
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Regional scope |
North America; Europe; Asia Pacific; Latin America; MEA |
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Country scope |
U.S.; Canada; Mexico; UK; Germany; France; Italy; Spain; Sweden; Denmark; Norway; Japan; China; India; Australia; South Korea; Singapore; Brazil; Argentina; South Africa; Saudi Arabia; UAE; Kuwait |
|
Key companies profiled |
Lonza; BioIT; Autolomous Ltd; Hypertrust Patient Data Care (Part of Accenture); IBM; L7 Informatics Inc.; TrakCel; IDBS; Novartis; SAP SE; DEEP GENOMICS; ElevateBio; Sarepta Therapeutics Inc.; CRISPR Therapeutics; Precision BioSciences; AstraZeneca; Andelyn Biosciences; Renova Therapeutics |
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Customization scope |
Free report customization (equivalent to up to 8 analysts' working days) with purchase. Changes or additions to the scope of the country, region, and segment are possible. |
|
Pricing and purchase options |
Avail yourself of customized purchase options to meet your exact research needs. Explore purchase options |
Global Gene Therapy Platform Market Report Segmentation
This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2018 to 2030. For this study, MIR has segmented the global market report for gene therapy platforms based on platform type, application, delivery method, end use, and region.
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Platform Type Outlook (Revenue, USD Million, 2018-2030)
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Viral Vector Platforms
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Adeno-associated Virus (AAV)
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Lentivirus
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Retrovirus
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Adenovirus
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Herpes Simplex Virus (HSV)
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Non-Viral Vector Platforms
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Lipid Nanoparticles (LNPs)
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Electroporation & Microinjection Platforms
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Polymer-based Delivery Systems
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Naked DNA/RNA Delivery
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Gene Editing Platforms
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CRISPR-Cas Systems
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TALEN
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ZFNs
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Application Outlook (Revenue, USD Million, 2018 - 2030)
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Oncology
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Rare Genetic Disorders
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Cardiovascular Diseases
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Neurological Disorders
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Ophthalmic Diseases
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Hematological Disorders (e.g., Hemophilia, Sickle Cell)
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Musculoskeletal Disorders
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Infectious Diseases (e.g., HIV, COVID-19 adjunct therapies)
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Delivery Mode Outlook (Revenue, USD Million, 2018-2030)
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In Vivo Gene Therapy
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Ex Vivo Gene Therapy
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Autologous Cell-Based Gene Therapy
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Allogeneic Cell-Based Gene Therapy
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Others (In-situ Gene therapy)
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End Use Outlook (Revenue, USD Million, 2018 - 2030)
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Pharmaceutical & Biotechnology Companies
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Academic & Research Institutions
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Contract Development & Manufacturing Organizations (CDMOs)
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Hospitals & Gene Therapy Centers
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Regional Outlook (Revenue, USD Million, 2018-2030)
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North America
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U.S.
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Canada
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Mexico
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Europe
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UK
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Germany
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France
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Italy
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Spain
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Sweden
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Denmark
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Norway
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Asia Pacific
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Japan
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China
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India
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Australia
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South Korea
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Thailand
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Latin America
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Brazil
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Argentina
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Middle East and Africa
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Saudi Arabia
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South Africa
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UAE
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Kuwait
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