The global gene therapy market size was valued at USD 5.8 Billion in 2024. Looking forward, the market is forecast to reach USD 17.9 Billion by 2033, exhibiting a CAGR of 13.3% from 2025-2033. North America currently dominates the market, holding a market share of over 56% in 2024. The North American market is driven by robust healthcare infrastructure, rising investments in research operations, advanced regulatory frameworks, and the presence of leading biotechnology firms fostering innovation.
Technological Advancements: Developments in viral vector technology are enhancing the efficacy and safety of gene therapy delivery. Moreover, CRISPR-Cas9 technology improves gene editing capabilities, which further accelerates gene therapy market statistics.
Industry Applications: Gene therapy is used to treat a variety of ailments, including cancer, genetic problems, and autoimmune conditions. Ex vivo gene therapy, in which cells are manipulated outside the body, is gaining popularity for treating specific genetic illnesses.
Geographical Trends: As per the gene therapy market statistics, North America dominates the market, owing to its strong healthcare infrastructure and developed biotechnology sector. However, Asia Pacific is emerging as a rapidly expanding market, propelled by a big patient population.
Competitive Landscape: Leading players are conducting comprehensive clinical studies to develop and get regulatory clearance for novel gene treatments aimed at a variety of ailments, including rare genetic abnormalities and cancer. Some of the key players in the market include Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., Bluebird Bio Inc., Mustang Bio Inc. (Fortress Biotech Inc.), Novartis AG, Orchard Therapeutics plc, UniQure N.V., Voyager Therapeutics Inc., etc.
Challenges and Opportunities: While the industry confronts constraints like regulatory limitations and high research costs, it also expects possibilities for increasing uses beyond rare disorders and investigating novel delivery modalities.
Future Outlook: The gene therapy market outlook is expected to be positive, with rising demand for novel therapies for genetic abnormalities and uncommon illnesses. Furthermore, individualized gene therapy tailored to unique patient characteristics is projected to drive market expansion.
Antigen genes are a critical segment in gene therapy, especially in the context of cancer immunotherapy. These genes code for tumor-specific antigens or antigens associated with infectious agents. By introducing antigen genes into the cells of the patients, the immune system can be activated to recognize and target cancer cells or pathogens. They also enable personalized cancer treatments, where patient-specific antigens can be targeted for a more effective and targeted immune response. For instance, Imugene reported a new clinical trial collaboration and supply agreement with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ, USA, to evaluate the safety and efficacy of Imugene's HER-Vaxx, a B-cell activating immunotherapy, in combination with MSD's anti-PD-1 therapy, pembrolizumab (KEYTRUDA), in patients with HER-2 positive gastric cancer.
A detailed breakup and analysis of the market based on the vector type have also been provided in the report. This includes viral vector (adenoviruses, lentiviruses, retroviruses, adeno-associated virus, herpes simplex virus, poxvirus, vaccinia virus, and others) and non-viral techniques (naked and plasmid vectors, gene gun, electroporation, lipofection, and others). According to the report, viral vector accounted for the largest market share.
Viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses, are gaining traction due to their efficient delivery of therapeutic genes into target cells. AAVs are favored for their safety profile and ability to integrate genetic material into the host genome. They are widely used for a variety of gene therapy applications, including treating genetic disorders, rare diseases, and certain types of cancer. Ongoing research and advancements in viral vector technology are driving the growth of this segment, with numerous clinical trials and approved therapies utilizing viral vectors. For instance, the U.S. FDA granted approval at an accelerated pace for SKYSONA- a lentiviral vector gene therapy shown to slow the progression of neurodegenerative dysfunction, or active cerebral adrenoleukodystrophy in boys with the age range of 4-17.
In-vivo gene therapy involves the direct delivery of therapeutic genes or genetic material into the body of the patient. This approach aims to modify or correct genetic defects within the tissues or organs of patients in their natural physiological environment. In-vivo gene therapy methods typically utilize viral vectors, such as adeno-associated viruses (AAVs) or lentiviruses, to transport the therapeutic genes. This segment of gene therapy is often used for diseases that require systemic treatment, such as certain types of inherited disorders or conditions where the target tissue is easily accessible within the body. For instance, recent advancements in gene supplementation therapy are expanding the options for the treatment of neurological disorders. Among the available delivery vehicles, adeno-associated virus (AAV) is often the favored vector.
The report has provided a detailed breakup and analysis of the market based on the application. This includes oncological disorders, rare diseases, cardiovascular diseases, neurological disorders, infectious diseases, and others. According to the report, oncological disorders represented the largest segment.
Gene therapy is effective in the treatment of various cancers, including solid tumors and hematological malignancies. Approaches range from introducing tumor-suppressing genes to activating the immune system against cancer cells. CAR-T cell therapy, a type of gene therapy, is gaining traction for its success in treating certain types of leukemia and lymphoma. The ongoing research and development (R&D) activities of gene therapies for oncological disorders are impelling the market growth. For instance, the U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to or whose disease has returned after at least four last lines (different types) of treatment. Abecma is the first cell-based gene therapy approved by the FDA for treating multiple myeloma.
The market research report has also provided a comprehensive analysis of all the major regional markets, which include North America (the United States and Canada); Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, and others); Europe (Germany, France, the United Kingdom, Italy, Spain, Russia, and others); Latin America (Brazil, Mexico, and others); and the Middle East and Africa. According to the report, North America accounted for the largest market share.
North America has a robust healthcare infrastructure, a well-established biotechnology industry, and significant investment in gene therapy research and development (R&D). Favorable regulatory policies, including fast-track designations and orphan drug status, are accelerating the approval and commercialization of gene therapies. Key players in the gene therapy market are headquartered in North America, contributing to the leadership of the region in gene therapy innovations. For instance, as per an update by the National Institutes of Health (NIH), the NIH, USFDA, ten pharmaceutical companies, and five non-profit organizations have partnered to accelerate the development of gene therapies for the 30 million Americans who suffer from a rare disease. Such initiatives are anticipated to increase the demand for gene therapy, thereby contributing to market growth.
The market research report has provided a comprehensive analysis of the competitive landscape. Detailed profiles of all major companies have also been provided. Some of the key players in the market include:
Abeona Therapeutics Inc.
Amgen Inc.
Astellas Pharma Inc.
Biogen Inc.
Bluebird Bio Inc.
Mustang Bio Inc. (Fortress Biotech Inc.)
Voyager Therapeutics Inc.
(Please note that this is only a partial list of the key players, and the complete list is provided in the report.)
January 2023: Voyager Therapeutics and Neurocrine Biosciences entered into a strategic collaboration for the commercialization & development of Voyager’s GBA1 program and other next-generation gene therapies for neurological diseases.
June 2023: The U.S. FDA granted approval to Sarepta for ELEVIDYS gene therapy to treat DMD in children of age 4-5 years.
May 2023: Krystal Biotech was granted approval for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment.
December 2023: Swiss Agency for Therapeutic Products granted approval to Libmeldy for the treatment of early-onset metachromatic leukodystrophy.
January 2024: Biogen and Ginkgo Bioworks announced that they have completed the gene therapy collaboration involving AAV-based vectors. This is expected to fuel the demand for gene therapies in the coming years.
2. How big is the global gene therapy market?
3. What is the expected growth rate of the global gene therapy market during 2025-2033?
4. What are the key factors driving the global gene therapy market?
5. What is the leading segment of the global gene therapy market based on gene type?
6. What is the leading segment of the global gene therapy market based on vector type?
7. What is the leading segment of the global gene therapy market based on delivery method?
8. What is the leading segment of the global gene therapy market based on application?
9. What are the key regions in the global gene therapy market?
10. Who are the key players/companies in the global gene therapy market?
Gene Therapy Market Analysis:
Market Growth and Size: The industry is expanding rapidly, fueled by the rising prevalence of genetic abnormalities and rare diseases. Additionally, pharmaceutical businesses are increasingly investing in research and development (R&D) activities, which are positively impacting the market.Technological Advancements: Developments in viral vector technology are enhancing the efficacy and safety of gene therapy delivery. Moreover, CRISPR-Cas9 technology improves gene editing capabilities, which further accelerates gene therapy market statistics.
Industry Applications: Gene therapy is used to treat a variety of ailments, including cancer, genetic problems, and autoimmune conditions. Ex vivo gene therapy, in which cells are manipulated outside the body, is gaining popularity for treating specific genetic illnesses.
Geographical Trends: As per the gene therapy market statistics, North America dominates the market, owing to its strong healthcare infrastructure and developed biotechnology sector. However, Asia Pacific is emerging as a rapidly expanding market, propelled by a big patient population.
Competitive Landscape: Leading players are conducting comprehensive clinical studies to develop and get regulatory clearance for novel gene treatments aimed at a variety of ailments, including rare genetic abnormalities and cancer. Some of the key players in the market include Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., Bluebird Bio Inc., Mustang Bio Inc. (Fortress Biotech Inc.), Novartis AG, Orchard Therapeutics plc, UniQure N.V., Voyager Therapeutics Inc., etc.
Challenges and Opportunities: While the industry confronts constraints like regulatory limitations and high research costs, it also expects possibilities for increasing uses beyond rare disorders and investigating novel delivery modalities.
Future Outlook: The gene therapy market outlook is expected to be positive, with rising demand for novel therapies for genetic abnormalities and uncommon illnesses. Furthermore, individualized gene therapy tailored to unique patient characteristics is projected to drive market expansion.
Gene Therapy Market Trends:
Advancements in Viral Vector Technology
Continuous progress and innovation in viral vector technology are important factors driving market growth. Viral vectors are used as delivery vehicles to introduce therapeutic genes into the cells of patients. Recent advances in this technique have greatly improved the safety and efficacy of gene therapy therapies. These developments include the creation of more specific and focused viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses, with AAVs gaining popularity due to their ability to transport genetic material with minimal immune reaction and damage. Furthermore, as per the gene therapy market overview, researchers are focusing on developing viral vectors that may carry bigger genetic payloads, broadening the range of curable disorders. These technical innovations not only increase the efficacy of gene treatments but also minimize the likelihood of adverse effects, making them safer for patients. As a result, the advancement of viral vector technology is accelerating the acceptance and efficacy of gene therapy treatments for a broader range of medical problems. For instance, MERCK KGaA, the VirusExpress 293 Adeno-Associated Virus (AAV) Production Platform, offers full viral vector manufacturing, including AAV and lentiviral vectors. Additionally, Catalent Inc. launched UpTempo Virtuoso platform process for the development and manufacturing of adeno-associated viral (AAV) vectors.Investment And Funding in Research and Development
The growing investments and funding for gene therapy research and development (R&D) efforts are driving market expansion. Pharmaceutical businesses, biotechnology firms, and government agencies are putting major resources into developing gene therapy technologies and therapeutics. Gene treatments have a strong potential for addressing unmet medical needs and providing long-term benefits, which is drawing significant funding. Major pharmaceutical corporations are either purchasing gene therapy startups or building their own gene therapy sections to speed up research, which is positively impacting the gene therapy market outlook. Furthermore, government subsidies and incentives motivate academic institutions and research firms to conduct gene therapy experiments. The Orphan Drug Act in the United States, as well as comparable legislation in other countries, gives incentives for the development of rare illness medicines. For instance, the Cystic Fibrosis Foundation reported investing in SalioGen Therapeutics to support the company's preclinical research into novel gene therapy for cystic fibrosis. Salonen's Gene Coding approach is designed to turn on, turn off, or modify the function of any gene in the genome.Rising Prevalence of Genetic Disorders and Rare Diseases
The rising prevalence of genetic abnormalities and rare illnesses is propelling the market growth. Many of these diseases have little or no effective treatment choices, making gene therapy an intriguing alternative. As our understanding of genetics advances, more people are diagnosed, and the demand for novel medicines grows. Moreover, gene therapy has the ability to treat the underlying causes of many disorders by repairing or replacing defective genes. This comprises illnesses including muscular dystrophy, cystic fibrosis, and other hereditary metabolic disorders. Pharmaceutical and biotechnology businesses are investing in gene therapy research to fulfill this expanding medical demand, resulting in market expansion and improvements in the area. For instance, as per the United States Center for Disease Control and Prevention article on spinal muscular atrophy (SMA), SMA is a genetic disorder that affects around 1 in every 10,000 people. Therefore, it is one of the most common rare diseases.Supportive Regulatory Environment
Regulatory organizations in several countries, especially the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), recognize the potential of gene therapy to address essential medical needs. Consequently, they have created structures and accelerated the research and approval of gene treatments. Additionally, fast-track designations, breakthrough treatment designations, and orphan drug status are among the regulatory measures available to gene therapy candidates. These designations ease the clinical trial procedure, shorten development schedules, and encourage corporations to participate in gene therapy research. For instance, the U.S. FDA approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. Such approvals are expected to drive market growth due to the rise in the development of gene therapies.Gene Therapy Market Segmentation:
This report provides an analysis of the key trends in each segment of the global gene therapy market, along with forecast at the global, regional, and country levels from 2025-2033. The market has been categorized based on gene type, vector type, delivery method, and application.Breakup by Gene Type:
Antigen
Cytokine
Tumor Suppressor
Suicide Gene
Deficiency
Growth Factors
Receptors
Others
The report has provided a detailed breakup and analysis of the market based on the gene type. This includes antigen, cytokine, tumor suppressor, suicide gene, deficiency, growth factors, receptors, and others.Antigen genes are a critical segment in gene therapy, especially in the context of cancer immunotherapy. These genes code for tumor-specific antigens or antigens associated with infectious agents. By introducing antigen genes into the cells of the patients, the immune system can be activated to recognize and target cancer cells or pathogens. They also enable personalized cancer treatments, where patient-specific antigens can be targeted for a more effective and targeted immune response. For instance, Imugene reported a new clinical trial collaboration and supply agreement with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ, USA, to evaluate the safety and efficacy of Imugene's HER-Vaxx, a B-cell activating immunotherapy, in combination with MSD's anti-PD-1 therapy, pembrolizumab (KEYTRUDA), in patients with HER-2 positive gastric cancer.
Breakup by Vector Type:
Viral Vector
Adenoviruses
Lentiviruses
Retroviruses
Adeno-Associated Virus
Herpes Simplex Virus
Poxvirus
Vaccinia Virus
Others
Non-Viral Techniques
Naked and Plasmid Vectors
Gene Gun
Electroporation
Lipofection
Others
Viral vector holds the largest share in the industry.A detailed breakup and analysis of the market based on the vector type have also been provided in the report. This includes viral vector (adenoviruses, lentiviruses, retroviruses, adeno-associated virus, herpes simplex virus, poxvirus, vaccinia virus, and others) and non-viral techniques (naked and plasmid vectors, gene gun, electroporation, lipofection, and others). According to the report, viral vector accounted for the largest market share.
Viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses, are gaining traction due to their efficient delivery of therapeutic genes into target cells. AAVs are favored for their safety profile and ability to integrate genetic material into the host genome. They are widely used for a variety of gene therapy applications, including treating genetic disorders, rare diseases, and certain types of cancer. Ongoing research and advancements in viral vector technology are driving the growth of this segment, with numerous clinical trials and approved therapies utilizing viral vectors. For instance, the U.S. FDA granted approval at an accelerated pace for SKYSONA- a lentiviral vector gene therapy shown to slow the progression of neurodegenerative dysfunction, or active cerebral adrenoleukodystrophy in boys with the age range of 4-17.
Breakup by Delivery Method:
In-Vivo Gene Therapy
Ex-Vivo Gene Therapy
The report has provided a detailed breakup and analysis of the market based on the delivery method. This includes in-vivo gene therapy and ex-vivo gene therapy.In-vivo gene therapy involves the direct delivery of therapeutic genes or genetic material into the body of the patient. This approach aims to modify or correct genetic defects within the tissues or organs of patients in their natural physiological environment. In-vivo gene therapy methods typically utilize viral vectors, such as adeno-associated viruses (AAVs) or lentiviruses, to transport the therapeutic genes. This segment of gene therapy is often used for diseases that require systemic treatment, such as certain types of inherited disorders or conditions where the target tissue is easily accessible within the body. For instance, recent advancements in gene supplementation therapy are expanding the options for the treatment of neurological disorders. Among the available delivery vehicles, adeno-associated virus (AAV) is often the favored vector.
Breakup by Application:
Oncological Disorders
Rare Diseases
Cardiovascular Diseases
Neurological Disorders
Infectious Disease
Others
Oncological disorders account for the majority of the market share.The report has provided a detailed breakup and analysis of the market based on the application. This includes oncological disorders, rare diseases, cardiovascular diseases, neurological disorders, infectious diseases, and others. According to the report, oncological disorders represented the largest segment.
Gene therapy is effective in the treatment of various cancers, including solid tumors and hematological malignancies. Approaches range from introducing tumor-suppressing genes to activating the immune system against cancer cells. CAR-T cell therapy, a type of gene therapy, is gaining traction for its success in treating certain types of leukemia and lymphoma. The ongoing research and development (R&D) activities of gene therapies for oncological disorders are impelling the market growth. For instance, the U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to or whose disease has returned after at least four last lines (different types) of treatment. Abecma is the first cell-based gene therapy approved by the FDA for treating multiple myeloma.
Breakup by Region:
North America
United States
Canada
Asia-Pacific
China
Japan
India
South Korea
Australia
Indonesia
Others
Europe
Germany
France
United Kingdom
Italy
Spain
Russia
Others
Latin America
Brazil
Mexico
Others
Middle East and Africa
North America leads the market, accounting for the largest gene therapy market share.The market research report has also provided a comprehensive analysis of all the major regional markets, which include North America (the United States and Canada); Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, and others); Europe (Germany, France, the United Kingdom, Italy, Spain, Russia, and others); Latin America (Brazil, Mexico, and others); and the Middle East and Africa. According to the report, North America accounted for the largest market share.
North America has a robust healthcare infrastructure, a well-established biotechnology industry, and significant investment in gene therapy research and development (R&D). Favorable regulatory policies, including fast-track designations and orphan drug status, are accelerating the approval and commercialization of gene therapies. Key players in the gene therapy market are headquartered in North America, contributing to the leadership of the region in gene therapy innovations. For instance, as per an update by the National Institutes of Health (NIH), the NIH, USFDA, ten pharmaceutical companies, and five non-profit organizations have partnered to accelerate the development of gene therapies for the 30 million Americans who suffer from a rare disease. Such initiatives are anticipated to increase the demand for gene therapy, thereby contributing to market growth.
Competitive Landscape:
Key industry competitors are currently involved in a number of strategic initiatives. These firms are constantly growing their gene therapy portfolios via research, acquisitions, and collaborations. They are undertaking comprehensive clinical studies to develop and get regulatory clearance for novel gene treatments that will treat a variety of ailments, including rare genetic abnormalities and cancer. In addition, they are investing in manufacturing facilities to increase output and ensure gene treatments are available to a larger patient population. Furthermore, these industry heavyweights are pursuing collaborations with academic institutions and smaller biotech businesses to promote innovation and improve gene therapy technology.The market research report has provided a comprehensive analysis of the competitive landscape. Detailed profiles of all major companies have also been provided. Some of the key players in the market include:
Abeona Therapeutics Inc.
Adaptimmune Therapeutics plc
Adverum Biotechnologies Inc.Amgen Inc.
Astellas Pharma Inc.
Biogen Inc.
Bluebird Bio Inc.
Mustang Bio Inc. (Fortress Biotech Inc.)
Novartis AG
Orchard Therapeutics plc
UniQure N.V.Voyager Therapeutics Inc.
(Please note that this is only a partial list of the key players, and the complete list is provided in the report.)
Gene Therapy Market News:
January 2023: Spark Therapeutics and Neurochase established a strategic collaboration to develop Neurochase’s unique delivery technology for use with selected gene treatments for rare disorders in the CNS. In this agreement, Neurochase will contribute its extensive knowledge in direct drug delivery technology to Spark’s premier AAV platform.January 2023: Voyager Therapeutics and Neurocrine Biosciences entered into a strategic collaboration for the commercialization & development of Voyager’s GBA1 program and other next-generation gene therapies for neurological diseases.
June 2023: The U.S. FDA granted approval to Sarepta for ELEVIDYS gene therapy to treat DMD in children of age 4-5 years.
May 2023: Krystal Biotech was granted approval for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment.
December 2023: Swiss Agency for Therapeutic Products granted approval to Libmeldy for the treatment of early-onset metachromatic leukodystrophy.
January 2024: Biogen and Ginkgo Bioworks announced that they have completed the gene therapy collaboration involving AAV-based vectors. This is expected to fuel the demand for gene therapies in the coming years.
Key Questions Answered in This Report
1. What is gene therapy?2. How big is the global gene therapy market?
3. What is the expected growth rate of the global gene therapy market during 2025-2033?
4. What are the key factors driving the global gene therapy market?
5. What is the leading segment of the global gene therapy market based on gene type?
6. What is the leading segment of the global gene therapy market based on vector type?
7. What is the leading segment of the global gene therapy market based on delivery method?
8. What is the leading segment of the global gene therapy market based on application?
9. What are the key regions in the global gene therapy market?
10. Who are the key players/companies in the global gene therapy market?
Table of Contents
1 Preface3 Executive Summary12 Value Chain Analysis14 Price Analysis
2 Scope and Methodology
4 Introduction
5 Global Gene Therapy Market
6 Market Breakup by Gene Type
7 Market Breakup by Vector Type
8 Market Breakup by Delivery Method
9 Market Breakup by Application
10 Market Breakup by Region
11 SWOT Analysis
13 Porters Five Forces Analysis
15 Competitive Landscape
List of Figures
List of Tables
Companies Mentioned
- Abeona Therapeutics Inc.
- Adaptimmune Therapeutics plc
- Adverum Biotechnologies Inc.
- Amgen Inc.
- Astellas Pharma Inc.
- Biogen Inc.
- Bluebird Bio Inc.
- Mustang Bio Inc. (Fortress Biotech Inc.)
- Novartis AG
- Orchard Therapeutics plc
- UniQure N.V.
- Voyager Therapeutics Inc.
Methodology
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Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 140 |
| Published | May 2025 |
| Forecast Period | 2024 - 2033 |
| Estimated Market Value ( USD | $ 5.8 Billion |
| Forecasted Market Value ( USD | $ 17.9 Billion |
| Compound Annual Growth Rate | 13.3% |
| Regions Covered | Global |
| No. of Companies Mentioned | 12 |
