Gene Therapy Market Size & Share Analysis - Growth Trends & Forecasts (2025 - 2030)

The Report Covers Global Cell and Gene Therapy Market and Competitive Analysis. The Market is Segmented by Indication (Cancer, Metabolic Disorders, Eye Disorders, Spinal Muscular Atrophy, and Other Indications), Technology (Adeno Virus Vector, Adeno-associated Virus Vector, Lentiviral Vector, Retroviral Vector, Herpes Virus Vector, and Other Technologies), and Geography (North America, Europe, Asia-Pacific, Middle East and Africa, and South America). The report offers the value (in USD million) for the above segments.

Gene Therapy Market Size & Share Analysis - Growth Trends & Forecasts (2025 - 2030)

Gene Therapy Market Size

Gene Therapy Market Summary
Study Period 2019 - 2030
Base Year For Estimation 2024
Market Size (2025) USD 9.19 Billion
Market Size (2030) USD 31.58 Billion
CAGR (2025 - 2030) 28.00 %
Fastest Growing Market Asia Pacific
Largest Market North America

Major Players

Gene Therapy Market Major Players

*Disclaimer: Major Players sorted in no particular order

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Gene Therapy Market Analysis

The Gene Therapy Market size is estimated at USD 9.19 billion in 2025, and is expected to reach USD 31.58 billion by 2030, at a CAGR of 28% during the forecast period (2025-2030).

The gene therapy market landscape has witnessed significant evolution in regulatory frameworks and approval pathways, reflecting the maturing nature of this therapeutic approach. A notable milestone was achieved in August 2022 when the US FDA approved Zynteglo (betibeglogene autotemcel), marking a breakthrough as the first cell-based genetic therapy for treating beta-thalassemia in both adult and pediatric patients. This approval has set important precedents for the evaluation and authorization of future gene therapy industry products, particularly in rare genetic disorders. The regulatory environment continues to adapt to accommodate innovative therapeutic approaches while maintaining rigorous safety standards and efficacy requirements.


The industry has experienced substantial consolidation through strategic partnerships and collaborations, aimed at combining complementary capabilities and accelerating therapeutic development. This trend is exemplified by significant investment activities, such as the Cystic Fibrosis Foundation's strategic investment in SalioGen Therapeutics in January 2022, supporting novel genetic therapy approaches for cystic fibrosis. These partnerships are increasingly focusing on developing platform technologies that can be applied across multiple therapeutic areas, rather than single-product developments. The collaboration landscape has evolved to include more risk-sharing arrangements and milestone-based partnerships.


Therapeutic innovation in the gene therapy market has expanded beyond traditional approaches to include novel delivery systems and editing technologies. Companies are increasingly focusing on developing scalable manufacturing processes and improving vector design to enhance therapeutic efficacy. This is evidenced by ElevateBio's substantial funding raise of USD 525 million to advance cell and growth of the gene therapy market technologies. The field has seen particular advancement in the development of non-viral delivery systems and the optimization of viral vectors for improved targeting and reduced immunogenicity.


Market access strategies have become increasingly sophisticated, with companies developing innovative pricing models and risk-sharing agreements to address the high costs associated with gene therapies. Healthcare systems are adapting their reimbursement frameworks to accommodate these transformative but expensive treatments. Companies are also investing in building specialized manufacturing facilities and distribution networks to ensure reliable supply chains for these complex biologics. The focus has shifted towards developing commercially viable manufacturing processes that can support larger patient populations while maintaining product quality and consistency. The trends in the gene therapy market are indicative of a maturing market that is poised for significant growth.

Segment Analysis: By Indication

Cancer Segment in Gene Therapy Market

The cancer gene therapy market dominates the global gene therapy market, accounting for approximately 57% of the total market share in 2024. This significant market position is driven by the increasing prevalence of various types of cancers worldwide and the growing adoption of gene therapy as a targeted treatment approach. Gene therapy's ability to specifically target cancer cells while minimizing damage to healthy cells has made it an increasingly attractive treatment option. The segment's growth is further supported by extensive research and development activities focused on developing new gene therapy solutions for different types of cancers. Major pharmaceutical companies and biotechnology firms are investing heavily in cancer-focused gene therapy research, with several promising candidates in late-stage clinical trials. Additionally, the successful commercialization of several cancer gene therapy products and increasing regulatory approvals have strengthened this segment's market position.

Market Analysis of Gene Therapy Market: Chart for By Indication

Remaining Segments in Gene Therapy Market

The gene therapy market encompasses several other significant segments, including spinal muscular atrophy (SMA), metabolic disorders, eye disorders, and other therapeutic areas. The SMA segment has emerged as a particularly important area, representing the second-largest share of the market, driven by successful therapeutic outcomes and increasing adoption rates. Eye disorders represent another crucial segment, with gene therapy showing promising results in treating various inherited retinal diseases and age-related eye conditions. The metabolic disorders segment continues to expand as new gene therapy approaches are developed for rare genetic metabolic conditions. These segments are characterized by ongoing clinical trials, increasing investment in research and development, and growing interest from both pharmaceutical companies and healthcare providers in expanding treatment options for patients with previously untreatable conditions.

Segment Analysis: By Technology

Adeno-associated Virus Vector Segment in Gene Therapy Market

The Adeno-associated Virus (AAV) Vector segment has emerged as the dominant technology in the gene therapy medicine market, commanding approximately 35% market share in 2024. This segment's leadership position is attributed to several key advantages of AAV vectors, including their ability to provide long-term DNA persistence in target cells, minimal inflammatory response from the host, and high efficiency in gene delivery. The segment's prominence is further strengthened by its extensive application in treating various genetic disorders, particularly in areas like spinal muscular atrophy and inherited retinal diseases. AAV vectors have demonstrated remarkable success in clinical trials and commercialized therapies, with their ability to effectively target specific tissues and cells while maintaining a strong safety profile making them the preferred choice for many gene therapy applications.

Remaining Segments in Gene Therapy Market Technology

The gene therapy based drug market encompasses several other significant vector technologies, each serving unique therapeutic applications. Lentiviral vectors have established themselves as crucial tools for ex vivo gene therapy applications, particularly in treating blood disorders and immunodeficiencies. Retroviral vectors continue to play an important role in specific therapeutic applications, leveraging their ability to integrate genetic material into dividing cells. Adeno virus vectors are valued for their large packaging capacity and high transduction efficiency, while Herpes virus vectors have found specialized applications in neurological conditions due to their natural neurotropism. Each of these vector technologies contributes distinctly to the advancement of gene therapy, offering different advantages in terms of payload capacity, target cell specificity, and therapeutic applications.

Gene Therapy Market Geography Segment Analysis

Gene Therapy Market in North America

North America represents the most established gene therapy market, characterized by advanced healthcare infrastructure, significant research and development activities, and favorable regulatory frameworks. The region encompasses the United States, Canada, and Mexico, with each country contributing uniquely to the market landscape. The presence of major pharmaceutical companies, research institutions, and a robust pipeline of gene therapy products has positioned North America as a global leader in this field. The region benefits from substantial healthcare spending, increased investment in biotechnology, and growing awareness about genetic disorders.

Market Analysis of Gene Therapy Market: Forecasted Growth Rate by Region

Gene Therapy Market in the United States

The United States dominates the North American gene therapy market, driven by its extensive research capabilities, advanced healthcare system, and strong regulatory framework. The country hosts numerous clinical trials and research initiatives, supported by organizations like the FDA and NIH. The presence of leading pharmaceutical companies and research institutions has created a robust ecosystem for gene therapy development. The Department of Health and Human Services oversees gene therapy clinical trials, ensuring proper regulatory oversight. In terms of market presence, the United States accounts for approximately 89% of the North American gene therapy market in 2024, reflecting its dominant position in the region.

Gene Therapy Market Growth Trajectory in the United States

The United States continues to demonstrate strong gene therapy market growth potential, with a projected CAGR of approximately 25% from 2024 to 2029. This growth is supported by increasing investments in R&D, rising prevalence of genetic disorders, and an expanding pipeline of gene therapy products. The country's commitment to advancing gene therapy is evident through its robust clinical trial infrastructure and supportive regulatory environment. The FDA's continued efforts to streamline approval processes for gene therapies, coupled with increasing private sector investments, position the United States as a key driver of innovation in this field.

Gene Therapy Market in Europe

Europe represents a significant global gene therapy market, with a well-established healthcare infrastructure and strong research capabilities across its key markets, including Germany, the United Kingdom, France, Italy, and Spain. The region benefits from collaborative research initiatives, supportive regulatory frameworks, and increasing investments in biotechnology. The European Medicines Agency's progressive approach to gene therapy regulation has created a favorable environment for development and commercialization. The presence of numerous research institutions and biotechnology companies contributes to the region's strong position in gene therapy innovation.

Gene Therapy Market in Germany

Germany leads the European gene therapy market size, supported by its robust healthcare system and significant investments in research and development. The country's leadership is driven by its strong pharmaceutical industry, advanced research facilities, and supportive regulatory environment. German research institutions are actively involved in developing innovative gene therapy solutions, particularly in areas such as cancer and rare genetic disorders. The country holds approximately 23% of the European gene therapy market share in 2024, establishing itself as the largest market in the region.

Gene Therapy Market Growth Trajectory in France

France demonstrates remarkable gene therapy market growth potential, driven by its strong focus on biotechnology innovation and research excellence. The country has established itself as a hub for gene therapy development, with multiple world-leading biotech and research clusters. French research institutions and companies are actively advancing various gene therapy programs, particularly in rare diseases and genetic disorders. The market is projected to grow at approximately 28% CAGR from 2024 to 2029, supported by government initiatives and increasing private sector investments.

Gene Therapy Market in Asia-Pacific

The Asia-Pacific region represents a rapidly evolving global gene therapy market, encompassing diverse markets including China, Japan, India, Australia, and South Korea. Each country contributes unique strengths to the regional market, from China's large patient population and research capabilities to Japan's advanced healthcare infrastructure. The region is witnessing increased investment in biotechnology and healthcare infrastructure, creating new opportunities for gene therapy development and commercialization. The Asia-Pacific gene editing market by country is also gaining momentum, driven by advancements in genetic technologies.

Gene Therapy Market in China

China leads the Asia-Pacific gene therapy market size, leveraging its large population base, growing healthcare expenditure, and significant investments in biotechnology research. The country has made substantial progress in developing its gene therapy capabilities, supported by government initiatives and increasing private sector participation. Chinese research institutions and biotech companies are actively advancing various gene therapy programs, particularly in areas such as cancer treatment and rare genetic disorders.

Gene Therapy Market Growth Trajectory in India

India is emerging as a rapidly growing market for gene therapy in the Asia-Pacific region, driven by its improving regulatory framework and increasing focus on biotechnology research. The country's large patient population and growing healthcare infrastructure create significant opportunities for gene therapy development. Indian research institutions and biotech companies are increasingly participating in gene therapy research and development, particularly in areas such as cancer treatment and genetic disorders.

Gene Therapy Market in the Middle East and Africa

The Middle East and Africa region presents an emerging market for gene therapy, with GCC countries and South Africa leading the development. The region is characterized by increasing healthcare expenditure, growing awareness of genetic disorders, and improving healthcare infrastructure. While the market is still developing, GCC countries emerge as the largest market, while South Africa shows promising growth potential, driven by its research capabilities and improving healthcare system.

Gene Therapy Market in South America

South America's gene therapy market is gradually evolving, with Brazil and Argentina as key contributors to regional growth. The market is characterized by increasing research activities, improving regulatory frameworks, and growing awareness about genetic disorders. Brazil emerges as both the largest and fastest-growing market in the region, supported by its large population base and increasing investments in healthcare infrastructure. The region shows significant potential for future growth as healthcare systems continue to develop and access to advanced therapies improves.

Gene Therapy Industry Overview

Top Companies in Gene Therapy Market

The gene therapy market features established pharmaceutical giants like Novartis, Roche, and Gilead Sciences alongside specialized players such as Bluebird Bio, UniQure, and Spark Therapeutics. Gene therapy companies are heavily investing in research and development to advance their gene therapy platforms, with a particular focus on viral vectors and CRISPR-based technologies. Strategic collaborations between pharmaceutical companies and research institutions have become increasingly common to accelerate product development and commercialization. Manufacturing capabilities and scalability remain key differentiators, with companies like Amgen and Biogen establishing dedicated gene therapy manufacturing facilities. The industry has witnessed significant investment in expanding geographic presence, particularly in emerging markets, while maintaining strong intellectual property portfolios through patents and licensing agreements.

Market Dominated by Strategic Partnerships and Innovation

The gene therapy industry exhibits a moderate level of consolidation, characterized by a mix of global pharmaceutical conglomerates and specialized biotech firms. Large pharmaceutical companies have strengthened their position through strategic acquisitions of smaller, innovative gene therapy companies, as exemplified by Roche's acquisition of Spark Therapeutics and Novartis's expansion through strategic partnerships. The market structure encourages collaboration between companies with complementary capabilities, where larger firms provide manufacturing and commercialization expertise while smaller biotechs contribute innovative technologies and research capabilities.


The competitive landscape is further shaped by regional clusters of expertise, particularly in the United States and Europe, where established research infrastructure and regulatory frameworks support industry growth. Market participants are increasingly focusing on vertical integration, developing in-house capabilities across the value chain from research and development to manufacturing and commercialization. The industry has witnessed a trend toward consolidation through mergers and acquisitions, particularly in specialized therapeutic areas where companies seek to build comprehensive gene therapy platforms.

Innovation and Scale Drive Market Success

Success in the gene therapy market increasingly depends on companies' ability to balance innovation with operational efficiency. Established players must continue investing in novel delivery technologies and expanding their therapeutic applications while optimizing manufacturing processes to reduce costs and improve scalability. Companies need to develop robust intellectual property strategies, maintain strong relationships with healthcare providers and payers, and navigate complex regulatory requirements across different regions. Building comprehensive patient support programs and establishing reliable supply chains have become critical factors for market success.


New entrants can gain a competitive advantage by focusing on underserved therapeutic areas or developing innovative delivery platforms that address current limitations in gene therapy. The market presents significant opportunities for companies that can demonstrate superior safety profiles and long-term efficacy of their therapies. Success factors include establishing strategic partnerships for manufacturing and commercialization, securing sustainable funding sources, and developing cost-effective production methods. Companies must also consider potential regulatory changes, particularly around safety monitoring and pricing, while building relationships with patient advocacy groups and healthcare providers to ensure market acceptance of their therapies.

Gene Therapy Market Leaders

  1. Gilead Sciences, Inc.

  2. Novartis

  3. Amgen Inc.

  4. Bluebird Bio

  5. Biogen Inc

  6. *Disclaimer: Major Players sorted in no particular order
Gene Therapy Market Concentration
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Gene Therapy Market News

  • In October 2022, Sarepta Therapeutics applied to the US FDA to grant accelerated approval to the gene therapy S (delandistrogene moxeparvovec) for the treatment of Duchenne Muscular Dystrophy (DMD).
  • In August 2022, the US FDA approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

Gene Therapy Market Report - Table of Contents

1. INTRODUCTION

  • 1.1 Study Assumptions and Market Definition
  • 1.2 Scope of the Study

2. RESEARCH METHODOLOGY

3. EXECUTIVE SUMMARY

4. MARKET DYNAMICS

  • 4.1 Market Overview
  • 4.2 Market Drivers
    • 4.2.1 Rising Investments in Research and Development
    • 4.2.2 Technological Advancements
    • 4.2.3 Growing Prevalence of Target Diseases like Cancer
  • 4.3 Market Restraints
    • 4.3.1 Lack of Standard Regulations
    • 4.3.2 High Price of Products
  • 4.4 Porter's Five Forces Analysis
    • 4.4.1 Threat of New Entrants
    • 4.4.2 Bargaining Power of Buyers/Consumers
    • 4.4.3 Bargaining Power of Suppliers
    • 4.4.4 Threat of Substitute Products
    • 4.4.5 Intensity of Competitive Rivalry

5. MARKET SEGMENTATION (Market Size by Value- USD million)

  • 5.1 By Indication
    • 5.1.1 Cancer
    • 5.1.2 Metabolic Disorders
    • 5.1.3 Eye Disorders
    • 5.1.4 Spinal Muscular Atrophy
    • 5.1.5 Other Indications
  • 5.2 By Technology
    • 5.2.1 Adeno Virus Vector
    • 5.2.2 Adeno-associated Virus Vector
    • 5.2.3 Lentiviral Vector
    • 5.2.4 Retroviral Vector
    • 5.2.5 Herpes Virus Vector
    • 5.2.6 Other Technologies
  • 5.3 Geography
    • 5.3.1 North America
    • 5.3.1.1 United States
    • 5.3.1.2 Canada
    • 5.3.1.3 Mexico
    • 5.3.2 Europe
    • 5.3.2.1 Germany
    • 5.3.2.2 United Kingdom
    • 5.3.2.3 France
    • 5.3.2.4 Italy
    • 5.3.2.5 Spain
    • 5.3.2.6 Rest of Europe
    • 5.3.3 Asia-Pacific
    • 5.3.3.1 China
    • 5.3.3.2 Japan
    • 5.3.3.3 India
    • 5.3.3.4 Australia
    • 5.3.3.5 South Korea
    • 5.3.3.6 Rest of Asia-Pacific
    • 5.3.4 Middle-East and Africa
    • 5.3.4.1 GCC
    • 5.3.4.2 South Africa
    • 5.3.4.3 Rest of Middle East and Africa
    • 5.3.5 South America
    • 5.3.5.1 Brazil
    • 5.3.5.2 Argentina
    • 5.3.5.3 Rest of South America

6. COMPETITIVE LANDSCAPE

  • 6.1 Company Profiles
    • 6.1.1 Amgen Inc.
    • 6.1.2 Biogen Inc.
    • 6.1.3 Bluebird Bio Inc.
    • 6.1.4 Gilead Sciences Inc. (Kite Pharma)
    • 6.1.5 Novartis AG
    • 6.1.6 F. Hoffmann-La Roche Ltd (Spark Therapeutics)
    • 6.1.7 UniQure NV
    • 6.1.8 Abeona Therapeutics Inc.
    • 6.1.9 Generation Bio
    • 6.1.10 Poseida Therapeutics
    • 6.1.11 Astellas Pharma
    • 6.1.12 Voyager Therapeutics Inc.
  • *List Not Exhaustive

7. MARKET OPPORTUNITIES AND FUTURE TRENDS

**Subject to Availability
**Competitive Landscape covers- Business Overview, Financials, Products and Strategies and Recent Developments
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Gene Therapy Industry Segmentation

As per the scope of this report, gene therapy is an advanced medical treatment that involves the transfer of a standard or healthy gene to replace a defective gene in a cell. It can cure various chronic and genetic disorders in human beings, for which no final cure has been developed. The market is segmented by Indication (Cancer, Metabolic Disorders, Eye Disorders, Spinal Muscular Atrophy, and Other Indications), Technology (Adeno Virus Vector, Adeno-associated Virus Vector, Lentiviral Vector, Retroviral Vector, Herpes Virus Vector, and Other Technologies), and Geography (North America, Europe, Asia-Pacific, Middle-East and Africa, and South America). The market report also covers the estimated market sizes and trends for 17 countries across major regions globally. The report offers the value (in USD million) for the above segments.

By Indication Cancer
Metabolic Disorders
Eye Disorders
Spinal Muscular Atrophy
Other Indications
By Technology Adeno Virus Vector
Adeno-associated Virus Vector
Lentiviral Vector
Retroviral Vector
Herpes Virus Vector
Other Technologies
Geography North America United States
Canada
Mexico
Europe Germany
United Kingdom
France
Italy
Spain
Rest of Europe
Asia-Pacific China
Japan
India
Australia
South Korea
Rest of Asia-Pacific
Middle-East and Africa GCC
South Africa
Rest of Middle East and Africa
South America Brazil
Argentina
Rest of South America
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Gene Therapy Market Research FAQs

How big is the Gene Therapy Market?

The Gene Therapy Market size is expected to reach USD 9.19 billion in 2025 and grow at a CAGR of 28% to reach USD 31.58 billion by 2030.

What is the current Gene Therapy Market size?

In 2025, the Gene Therapy Market size is expected to reach USD 9.19 billion.

Who are the key players in Gene Therapy Market?

Gilead Sciences, Inc., Novartis, Amgen Inc., Bluebird Bio and Biogen Inc are the major companies operating in the Gene Therapy Market.

Which is the fastest growing region in Gene Therapy Market?

Asia Pacific is estimated to grow at the highest CAGR over the forecast period (2025-2030).

Which region has the biggest share in Gene Therapy Market?

In 2025, the North America accounts for the largest market share in Gene Therapy Market.

What years does this Gene Therapy Market cover, and what was the market size in 2024?

In 2024, the Gene Therapy Market size was estimated at USD 6.62 billion. The report covers the Gene Therapy Market historical market size for years: 2019, 2020, 2021, 2022, 2023 and 2024. The report also forecasts the Gene Therapy Market size for years: 2025, 2026, 2027, 2028, 2029 and 2030.

Gene Therapy Market Research

Mordor Intelligence provides a comprehensive analysis of the gene therapy market. We leverage our extensive experience in tracking cell therapy technologies and emerging therapeutic approaches. Our research covers the rapidly expanding global gene therapy landscape. It includes detailed information on the cell and gene therapy market size and technological developments. The report offers an in-depth analysis of gene therapy companies and their innovations across various therapeutic areas, such as cancer gene therapy and genetic disorders. This information is available in an easy-to-download report PDF format.

Our detailed market analysis gives stakeholders crucial insights into gene therapy market growth trends and gene therapy market forecast data. We place particular emphasis on cell and gene therapy growth opportunities. The report examines the therapy market size across regions, including specialized coverage of the Asia-Pacific gene editing market by technology. Stakeholders benefit from comprehensive profiles of the largest gene therapy companies, detailed technological assessments, and strategic recommendations for market entry and expansion. The analysis includes emerging gene therapy trends and regulatory frameworks, providing a robust foundation for informed decision-making in this dynamic industry.