Global Gene Therapy Platform Market Size Was valued at USD 2.2 Bn in 2024 and is predicted to reach USD 9.8 Bn by 2034 at a 16.5% CAGR during the forecast period for 2025-2034.
Gene therapy's rapid ascent has been one of the most groundbreaking advances in modern medicine. By modifying, replacing, or suppressing faulty genes that lead to disease development, gene therapy offers long-term therapeutic alternatives, particularly for rare or previously incurable genetic disorders. Gene therapy platforms encompass the underlying technology and delivery mechanisms that enable these medications, including viral and non-viral vectors, gene editing systems, and others.
Healthcare professionals, biopharmaceutical companies, and research institutes are increasingly interested in gene therapy as the prevalence of hereditary and chronic diseases worldwide rises. Key markers of market maturity and expansion include the rise in FDA approvals, the development of personalized medicine, and an increase in clinical studies employing innovative gene therapy platforms. The market is expanding due to the growing use of customized medicine, which is fueled by better genetic diagnoses and patient-specific therapies. Additionally, partnerships and collaborations among pharmaceutical corporations, academic institutions, and biotech companies are fostering innovation and resource sharing, enabling the creation of more accessible and effective gene therapy platforms.
Furthermore, the effectiveness and safety of gene therapies have been significantly enhanced by innovations such as viral vectors, non-viral delivery systems, and genome editing tools like CRISPR-Cas9. The industry is expanding due to increasing investments by pharmaceutical companies, favourable regulatory environments, and growing patient demand for personalised treatment.
Competitive Landscape
Some of the Major Key Players in the Gene Therapy Platform Market are:
- Lonza
- BioIT
- L7 Informatics, Inc.
- TrakCel
- IDBS
- Sarepta Therapeutics, Inc.
- CRISPR Therapeutics
- Precision BioSciences
- AstraZeneca
- Andelyn Biosciences
- Novartis
- SAP SE
- Autolomous Ltd
- DEEP GENOMICS
- ElevateBio
- Renova Therapeutics
- Hypertrust Patient Data Care (Part of Accenture)
- IBM
- Others
Market Segmentation:
The gene therapy platform market is segmented based on platform, delivery mode, application, and end-user. Based on platform, the market is segmented into viral vector platforms (adenovirus, adeno-associated virus (AAV), retrovirus, lentivirus, herpes simplex virus (HSV)), non-viral vector platforms (electroporation & microinjection platforms, polymer-based delivery systems, lipid nanoparticles (LNPs), naked DNA/RNA delivery), and gene editing platforms (TALENs, CRISPR-Cas Systems, ZFNs). By delivery mode, the market is segmented into In vivo gene therapy, ex vivo gene therapy (allogeneic cell-based gene therapy, autologous cell-based gene therapy), and in-situ gene therapy. By application, the market is segmented into cardiovascular diseases, oncology, rare genetic disorders, neurological disorders, ophthalmic diseases, hematological disorders, musculoskeletal disorders, and infectious diseases. By end-user, the market is segmented into hospitals & gene therapy centers, academic & research institutions, pharmaceutical & biotechnology companies, and contract development & manufacturing organizations (CDMOs).
Based On The Platform, The Viral Vector Platforms Segment Is Accounted As A Major Contributor To The Gene Therapy Platform Market
The viral vector platforms segment is expected to hold a major global market share in 2024. Since viral vector platforms are so effective in delivering genetic material into target cells, they have long been the mainstay of gene delivery systems. By leveraging the inherent propensity of viruses to infect cells, these platforms use modified viruses, including lentiviruses, retroviruses, adenoviruses, and adeno-associated viruses (AAV), to deliver therapeutic genes. Their extensive use is motivated by continual advancements in vector design that improve safety profiles and reduce immunological responses, as well as their shown effectiveness in clinical settings.
Oncology Segment To Witness Growth At A Rapid Rate
Due to the increasing demand for personalised and targeted cancer treatments, the oncology segment accounted for the largest revenue share in 2024. Oncolytic viruses, immune-modulating genes, and suicide genes are some of the methods being researched to target cancer cells specifically while preserving healthy tissue. The use of gene-modified immune cells, such as CAR-T treatments, to activate the patient's immune system has revolutionised cancer treatment and improved outcomes for patients who would otherwise have had limited alternatives. In the oncology sector of the gene therapy industry, significant investment and innovation are being driven by this emphasis on tailored and targeted treatments.
In The Region, The North American Gene Therapy Platform Market Holds A Significant Revenue Share
The North American gene therapy platform market is expected to register the highest market share in revenue in the near future. With a thriving biotech industry propelling the development of cutting-edge technologies like viral vectors and CRISPR gene editing, the region continues to dominate global gene therapy advancements. Additionally, the FDA and other government organizations continue to assist the sector by expediting gene therapy approvals, creating an atmosphere that encourages quick innovation. Furthermore, precision targeting of genetic defects is made possible by cutting-edge gene editing technologies like CRISPR, and FDA regulatory incentives promote innovation and quicker commercialization.
In addition, Europe is projected to grow rapidly in the global gene therapy platform market driven by cutting-edge research and the growing emphasis on genetically based medicines and customized therapy in the area. With gene therapy delivery methods included in research programs aimed at diseases including cancer, cardiovascular ailments, and rare genetic conditions, European governments and institutions are investing in biotech, especially in the UK, Germany, and France. The European Medicines Agency's (EMA) regulatory assistance has made it possible for gene treatments to be commercialized.
Recent Development:
- February 2025: Andelyn Biosciences expands its AAV Curator platform to further innovate gene therapy. Under the terms of the agreement, Andelyn is authorized to conduct research and development services using Stanton Lab CNS capsids for its clients who are creating gene treatments.
- December 2024: MRC created its first two Centres of Research Excellence with the goal of developing cutting-edge treatments for diseases that are now incurable. The MRC CoREs bring together leading academic and industry expertise to speed up clinical application and translational research of advanced therapeutics. This promotes innovation, infrastructure development, and regulatory advancements that directly benefit and grow the gene therapy platform industry.
- May 2023: The US-based software business Form Bio, which specializes in life sciences, introduced FORMsightAI, a new suite of AI-based solutions for forecasting and maximizing the production results of cell and gene therapy structures. The full-service solution combines state-of-the-art, flexible AI models with Form Bio's foundational platform and a proficient group of computational life sciences professionals with expertise in data science, bioinformatics, and biology. Common problems that cell and gene therapy companies encounter include build truncations and manufacturing contaminations, which are significant contributors to unacceptable low manufacturing yields, protracted time to market, and issues with therapeutic safety. FORMsightAI solves these issues.
Gene Therapy Platform Market Report Scope:
|
Report Attribute |
Specifications |
|
Market Size Value In 2024 |
USD 2.2 Bn |
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Revenue Forecast In 2034 |
USD 9.8 Bn |
|
Growth Rate CAGR |
CAGR of 16.5% from 2025 to 2034 |
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Quantitative Units |
Representation of revenue in US$ Bn and CAGR from 2025 to 2034 |
|
Historic Year |
2021 to 2024 |
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Forecast Year |
2025-2034 |
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Report Coverage |
The forecast of revenue, the position of the company, the competitive market structure, growth prospects, and trends |
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Segments Covered |
By Platform, By Delivery Mode, By Application, And By End-User |
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Regional Scope |
North America; Europe; Asia Pacific; Latin America; Middle East & Africa |
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Country Scope |
U.S.; Canada; U.K.; Germany; China; India; Japan; Brazil; Mexico; France; Italy; Spain; South East Asia; South Korea |
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Competitive Landscape |
Lonza, BioIT, L7 Informatics, Inc., TrakCel, IDBS, Sarepta Therapeutics, Inc., CRISPR Therapeutics, Precision BioSciences, AstraZeneca, Andelyn Biosciences, Novartis, SAP SE, Autolomous Ltd, DEEP GENOMICS, ElevateBio, Renova Therapeutics, Hypertrust Patient Data Care (part of Accenture), IBM, and others. |
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Customization Scope |
Free customization report with the procurement of the report and modifications to the regional and segment scope. Particular Geographic competitive landscape. |
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Pricing And Available Payment Methods |
Explore pricing alternatives that are customized to your particular study requirements. |
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