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市場調查報告書
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1987110

基因治療市場規模、佔有率、趨勢和預測：按基因類型、載體類型、遞送方法、應用和地區分類，2026-2034年

Gene Therapy Market Size, Share, Trends and Forecast by Gene Type, Vector Type, Delivery Method, Application, and Region, 2026-2034
出版日期: | 出版商: IMARC | 英文 148 Pages | 商品交期: 2-3個工作天內

價格

2025年全球基因治療市場規模為66億美元。展望未來，IMARC Group預測，該市場將以12.27%的複合年成長率從2026年成長至2034年，到2034年達到193億美元。目前，北美是該市場的主要驅動力，預計2025年將佔據超過56%的市場。北美市場受益於其完善的醫療保健基礎設施、不斷成長的研發投入、先進的法規結構以及眾多推動創新的領先生物技術公司。

囊腫纖維化、血友病和鐮狀貧血等遺傳性疾病的日益普遍，推動了對基因療法等創新治療方法的需求。此外，癌症和心血管疾病等慢性疾病中潛在的遺傳因素，也促使人們對標靶基因層面病因的治療方法產生了濃厚的興趣。同時，CRISPR（Cas9）、病毒和非病毒載體以及次世代定序等技術的快速發展，正在提高基因療法的效率和安全性。叢集創新顯著縮短了研究週期，並提高了臨床試驗的成功率。此外，許多政府機構和監管機構透過簡化核准程序和獎勵來支持基因療法。針對罕見疾病和癌症的新治療方法的核准，正在增強市場信心，並刺激基因療法的進一步發展。

由於美國遺傳性疾病（如血友病和脊髓性肌肉萎縮症）以及癌症和心血管疾病等慢性疾病的高發，美國正逐漸成為重要的市場領域。此外，針對罕見疾病和複雜疾病的基因療法的研發也推動了市場成長。這些治療方法針對疾病的根本遺傳病因，可望透過單次給藥來實現治癒。 2024年，Orchard Therapeutics宣布計劃在美國推出Lenmeldy™（atidalsagen autotemcell），這是首個獲得FDA核准的用於治療早發性異染性腦白質營養不良（MLD）的基因療法。雖然每次治療費用高達425萬美元，但該治療方法透過解決MLD的遺傳病因，具有突破性的潛力。該療法的推廣包括五個專業治療中心和一個創新的基於價值的報銷模式。

基因治療市場的發展趨勢：

病毒載體技術的進步

病毒載體技術的持續進步和創新是推動市場發展的關鍵因素。治療性基因透過病毒載體直接導入患者細胞。近年來，基因療法在安全性和有效性方面取得了顯著進步，其中許多進展包括開發出更具特異性和靶向性的病毒載體，例如腺結合病毒（AAV）和慢病毒。特別是AAV，由於其獨特的避免觸發免疫反應和最大限度減少組織損傷的能力，得到了廣泛應用。此外，根據基因療法市場概覽，研究人員正在致力於開發能夠攜帶更多遺傳物質的病毒載體，從而擴大可治療疾病的範圍。這些技術創新不僅提高了基因治療的效率，而且透過最大限度地降低副作用風險，使患者能夠獲得更安全的治療。因此，病毒載體技術的進步正在加速基因治療在更廣泛疾病領域的應用和療效。根據美國疾病管制與預防中心（CDC）的數據，2021年美國報告了1,777,566例新增癌症病例。例如，默克集團（Merck KGaA）的「VirusExpress 293腺結合病毒（AAV）生產平台」提供整體的病毒載體生產服務，包括AAV和慢病毒載體。此外，Catalent公司推出了腺結合病毒（AAV）載體開發和生產的「UpTempo Virtuoso」平台製程。

研發方面的投資與資金籌措

基因治療研發領域的投資和資金籌措不斷成長，推動了市場發展。基因治療創新基金（GTIF）的年度基金規模為610萬美元。基金委員會每年審核申請並做出兩次資助決定。該基金為致力於新型基因療法研發的學術研究人員提供津貼，用於支持其研究計畫所需的符合GMP（或GMP合規）標準的病毒載體材料。製藥公司、生技公司和政府機構正在投入大量資源開發基因治療技術和療法。基因治療具有滿足未被滿足的醫療需求並帶來長期益處的潛力，這推動了其獲得大量資金籌措。大型製藥企業正在收購基因治療Start-Ups或建立內部基因治療部門，以加速其研究，這對基因治療市場前景產生了積極影響。

遺傳性和罕見病患疾病率不斷上升

遺傳性疾病和罕見疾病的日益增加正在推動市場成長。基因療法之所以備受關注，是因為許多此類疾病缺乏有效的治療方法。隨著我們對遺傳學的理解不斷加深，確診患者人數不斷增加，對新藥的需求也隨之增加。此外，基因療法能夠透過修復或替換缺陷基因來治療許多疾病的根本原因，例如肌肉萎縮症、囊腫纖維化和其他遺傳代謝紊亂。製藥和生物技術公司正在加大對基因療法研究的投入，以滿足日益成長的醫療需求，從而推動市場擴張和該領域的進步。根據美國疾病管制與預防中心（CDC）統計，脊髓性肌肉萎縮症（SMA）是一種遺傳性疾病，影響約萬分之一的人口，因此是最常見的罕見疾病之一。

目錄

第1章：序言

第2章：調查方法

  • 調查目的
  • 相關利益者
  • 數據來源
    • 主要訊息
    • 二手資訊
  • 市場估值
    • 自下而上的方法
    • 自上而下的方法
  • 調查方法

第3章執行摘要

第4章：引言

第5章：全球基因治療市場

  • 市場概覽
  • 市場表現
  • 新冠疫情的影響
  • 市場預測

第6章 市場區隔：依基因類型分類

  • 抗原
  • 細胞激素
  • 腫瘤抑制基因
  • 自殺基因
  • 遺失的
  • 生長因子
  • 受體
  • 其他

第7章 市場細分：依向量類型

  • 病毒載體
    • 主要部分
      • 腺病毒
      • 慢病毒
      • 逆轉錄病毒
      • 腺結合病毒
      • 單純皰疹病毒
      • 痘病毒
      • 牛痘病毒
      • 其他
  • 非病毒技術
    • 主要部分
      • 裸載體和質體載體
      • 吉內根
      • 電穿孔
      • 脂質體
      • 其他

第8章 市場區隔：依管理方法

  • 體內基因治療
  • Exovivo基因療法

第9章 市場區隔：依應用領域分類

  • 腫瘤性疾病
  • 罕見疾病
  • 心血管疾病
  • 神經系統疾病
  • 感染疾病
  • 其他

第10章 市場區隔：依地區分類

  • 北美洲
    • 美國
    • 加拿大
  • 亞太地區
    • 中國
    • 日本
    • 印度
    • 韓國
    • 澳洲
    • 印尼
    • 其他
  • 歐洲
    • 德國
    • 法國
    • 英國
    • 義大利
    • 西班牙
    • 俄羅斯
    • 其他
  • 拉丁美洲
    • 巴西
    • 墨西哥
    • 其他
  • 中東和非洲

第11章 SWOT 分析

第12章：價值鏈分析

第13章：波特五力分析

第14章：價格分析

第15章 競爭格局

  • 市場結構
  • 主要企業
  • 主要企業簡介
    • Abeona Therapeutics Inc.
    • Adverum Biotechnologies, Inc.
    • Amgen Inc.
    • Astellas Pharma Inc.
    • BioMarin Pharmaceutical Inc.
    • bluebird bio, Inc.
    • Gilead Sciences, Inc.
    • Mustang Bio
    • Novartis AG
    • Orchard Therapeutics plc
    • Roche Holding AG
    • Sangamo Therapeutics
    • uniQure NV
Product Code: SR112026A5026

The global gene therapy market size was valued at USD 6.6 Billion in 2025. Looking forward, IMARC Group estimates the market to reach USD 19.3 Billion by 2034, exhibiting a CAGR of 12.27% from 2026-2034. North America currently dominates the market, holding a market share of over 56% in 2025. 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.

The growing incidence of genetic disorders, such as cystic fibrosis, hemophilia, and sickle cell anemia, is driving the demand for innovative treatments like gene therapy. Additionally, chronic diseases like cancer and cardiovascular conditions, which may have underlying genetic components, are increasing interest in therapies that target the root cause at the genetic level. Besides this, rapid advancements in technologies, including clustered regularly interspaced short palindromic repeats (CRISPR) associated protein 9 (Cas9), viral and non-viral vectors, and next-generation sequencing, are enhancing the efficiency and safety of gene therapy approaches. These innovations are significantly reducing research timelines and improving the success rates of clinical trials. Moreover, many governmental bodies and regulatory agencies are backing gene therapy through simplified approval procedures and incentives. Approval of new treatments for rare illnesses and cancer is boosting market confidence and promoting further development.

The United States has emerged as a crucial segment in the market attributed to high incidences of genetic disorders like hemophilia and spinal muscular atrophy, coupled with chronic conditions, such as cancer and cardiovascular diseases. Apart from this, the development of genetic treatments targeting rare and complex diseases is bolstering the market growth. These therapies address the root genetic causes of conditions, offering potentially curative solutions with a single administration. In 2024, Orchard Therapeutics announced the US launch plans for Lenmeldy(TM) (atidarsagene autotemcel), the first FDA-approved gene therapy for early-onset metachromatic leukodystrophy (MLD). With a one-time treatment cost of $4.25 million, the therapy offers transformative potential by addressing the genetic cause of MLD. The rollout includes five specialized treatment centers and innovative value-based reimbursement models.

GENE THERAPY MARKET TRENDS:

Advancements in Viral Vector Technology

Continuous progress and innovation in viral vector technology are important factors driving the market. Therapeutic genes are introduced directly into the cells of the patient through viral vectors. Gene therapy treatments have, in recent years, experienced enhanced safety and efficacy, among the many developments that include the development of more specific and targeted viral vectors such as adeno-associated viruses (AAVs) and lentiviruses, with AAVs enjoying widespread application because of their characteristic ability to carry genetic material in an immune reaction and damage-minimum approach. Moreover, according to the gene therapy market overview, researchers are working on developing viral vectors that may carry bigger genetic payloads, thus expanding the scope of curable disorders. These technical innovations not only enhance the efficiency of gene treatments but also minimize the chances of adverse effects, thus making them safer for patients. As a result, the development of viral vector technology is speeding up the acceptance and efficacy of gene therapy treatments for a wider range of medical conditions. According to the Centers for Disease Control and Prevention, in the United States, in 2021, 1,777,566 new cancer cases were reported. For example, MERCK KGaA, the VirusExpress 293 Adeno-Associated Virus (AAV) Production Platform, provides full viral vector manufacturing, including AAV and lentiviral vectors. Additionally, Catalent Inc. launched the 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 supporting the market growth. According to reports, the Gene Therapy Innovation Fund (GTIF) is a USD 6.1 Million annual fund. The funding panel reviews full applications and makes funding decisions twice a year. The fund provides grants to academic researchers working to advance new gene therapies and who require viral vector material manufactured to GMP (or GMP-like) grade in order to support the program. 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.

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. According to the United States Center for Disease Control and Prevention, SMA is a genetic disorder that affects around 1 in every 10,000 people. Therefore, it is one of the most common rare diseases.

GENE THERAPY INDUSTRY SEGMENTATION:

Analysis by Gene Type:

  • Antigen
  • Cytokine
  • Tumor Suppressor
  • Suicide Gene
  • Deficiency
  • Growth Factors
  • Receptors
  • Others

Antigens are recognized as central to advancing immunotherapy, particularly in oncology, by enhancing the immune system's ability to identify and eliminate cancer cells. These molecules, often specific to tumor cells, are the foundation for innovative treatments such as cancer vaccines and chimeric antigen receptor (CAR)-T cell therapies. Cancer vaccines harness tumor-associated antigens to activate the immune system, prompting a targeted attack on malignant cells. CAR-T therapies take this further by engineering patient T cells to recognize and destroy cancer cells expressing specific antigens, achieving remarkable success in treating aggressive and resistant cancers. Research is now focused on discovering novel antigens and refining delivery systems to boost effectiveness and reduce side effects. Personalized medicine is also leveraging antigen-based therapies to match treatments with individual tumor profiles, enhancing outcomes. As antigen research progresses, it is reshaping oncology, offering transformative potential for treating complex and refractory cancers.

Analysis 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 biggest market share, accounting for 55.5% in 2025. Viral vector (adenoviruses, lentiviruses, retroviruses, adeno-associated virus, herpes simplex virus, poxvirus, vaccinia virus, and others) dominates the gene therapy market due to its superior efficiency in delivering genetic material into host cells. Viral vectors are widely used for their ability to achieve high transduction rates and stable gene expression. Among these, AAV has gained significant traction for its safety profile and reduced immunogenicity, making it ideal for treating rare genetic disorders and chronic diseases. Lentiviruses and retroviruses are extensively used in ex vivo therapies like CAR-T cell treatments for cancer, further bolstering the segment's growth. Advances in vector engineering have addressed earlier concerns related to safety and toxicity, expanding their application scope across various therapeutic areas. Regulatory approvals for viral vector-based therapies and increased funding for research and development are further driving this segment. Additionally, collaborations between pharmaceutical companies and academic institutions are accelerating the commercialization of viral vector-based treatments, solidifying their dominance in the gene therapy market.

Analysis by Delivery Method:

  • In-Vivo Gene Therapy
  • Ex-Vivo Gene Therapy

In-vivo gene therapy market accounted for the largest share in 2025, holding 71.5% of the market. In-vivo gene therapy involves delivering genetic material directly into a patient's body using vectors such as viruses or non-viral techniques. It is most useful for treating diseases in which the affected cells are not easily accessible; hence, it is used to treat neurological disorders, liver diseases, and other genetic disorders. Advances in targeted delivery systems, building pipelines for rare and chronic diseases, and an increasing approval rate of innovative treatments also drive the market. Notable recent breakthroughs like addressing previously untreatable spinal muscular atrophy and even hemophilia have underpinned the potential of in-vivo gene therapy. The growing investment in research operations, coupled with favorable regulatory frameworks, is driving adoption, and this segment has become a core part of the gene therapy market.

Analysis by Application:

  • Oncological Disorders
  • Rare Diseases
  • Cardiovascular Diseases
  • Neurological Disorders
  • Infectious Disease

Oncological disorders hold the largest market share, attributed to the increasing incidence of cancer and the need for new therapy. Gene therapies against cancers are directed at altering genes to augment immune responses, inhibit tumor growth, or enhance the effectiveness of any existing therapy. CAR-T cell therapy, oncolytic viruses, and the delivery of tumor suppressor genes have shown promising effects in the treatment of both hematological malignancies and solid tumors. Advances in gene-editing technologies, such as CRISPR, and viral vectors, including lentiviruses and AAVs, further optimize the precision and safety of these therapies. Regulatory approvals of breakthrough therapies, such as CAR-T treatments for leukemia and lymphoma, have set a suitable foundation for further innovation in this segment. Furthermore, the increasing adoption of combination therapies that integrate gene-based approaches with traditional treatments, like chemotherapy and immunotherapy, enhances therapeutic outcomes.

Regional Analysis:

  • 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 dominates the market, holding 56% in 2025, due to its advanced health infrastructure, strong research and development ecosystem, and the initial adoption of cutting-edge medical technologies. The well-regulated frameworks provide streamlined pathways to expedite the development and approval of gene therapies. High prevalence rates of genetic disorders, cancers, and chronic diseases in this region are driving the demand for such innovative treatments. North America hosts some of the world's major biotech firms, top universities, and specialized treatment centers, so there's always an excellent opportunity to collaborate and advance commercialization activities. Value-based care models and well-entrenched reimbursement systems are also key contributors to this access to high-priced treatments for patients. In 2024, OmniaBio Inc. opened the largest CGT manufacturing facility in Hamilton, Ontario, Canada. This advanced site utilizes AI and automation to enhance production efficiency and serves North American and global biotherapy clients. Its first commercial customer is MEDIPOST, who will manufacture the stem cell therapy CARTISTEM for patients with osteoarthritis.t

KEY REGIONAL TAKEAWAYS:

UNITED STATES GENE THERAPY MARKET ANALYSIS

The gene therapy market in the United States is currently expanding due to several critical factors. Researchers are increasingly focusing on developing personalized treatments for genetic disorders, with advanced CRISPR technologies enabling precise gene editing and enhancing the efficacy of therapies. The US government is actively supporting gene therapy innovation through funding initiatives and regulatory incentives, such as accelerated approval pathways from the FDA, which is encouraging biotech firms to push the boundaries of genetic medicine. Pharmaceutical companies are forging strategic collaborations with academic institutions and startups to expedite the development of gene therapies for rare and chronic diseases. According to American Diabetes Association, 38.4 million people have diabetes in the United States which is 11.6% of the US population. Additionally, ongoing clinical trials are demonstrating promising results for conditions like sickle cell anemia, muscular dystrophy, and certain cancers, increasing investor confidence in gene therapy's potential. Insurance providers are also gradually expanding coverage for gene therapy treatments, further promoting patient access. Advances in viral vector technology and delivery mechanisms are overcoming past challenges related to the safe and effective delivery of genetic material, fuelling market growth. With an aging population and rising demand for innovative treatments, gene therapy is becoming a focal point in the U.S. healthcare system, positioning it for sustained market growth in the coming years.

EUROPE GENE THERAPY MARKET ANALYSIS

The gene therapy market in Europe is experiencing significant growth as advanced technologies are increasingly enabling the development of targeted treatments for rare and complex diseases. Researchers are making rapid strides in overcoming delivery challenges, with companies currently focusing on improving viral and non-viral vector systems to enhance the efficiency and safety of gene transfer. Regulatory authorities like the European Medicines Agency (EMA) are adapting their frameworks to accelerate the approval process for gene therapies, providing a favorable environment for innovation. Pharmaceutical companies are intensifying collaborations with biotech firms and academic institutions to expand their gene therapy pipelines, particularly in areas like oncology, genetic disorders, and cardiovascular diseases. According to reports, there are more than 385,000 new cancer cases in the UK every year, that's more than 1,000 every day. Healthcare providers are becoming more receptive to gene therapies as clinical trials demonstrate substantial efficacy in treating previously untreatable conditions, creating demand for these cutting-edge solutions. Moreover, the increasing prevalence of inherited diseases, coupled with a rising patient pool, is driving investments in gene-based treatments. The growing awareness and education among healthcare professionals about the potential of gene therapies are also contributing to market expansion. At the same time, advancements in CRISPR and other gene-editing technologies are continually improving the precision and effectiveness of therapies, positioning Europe as a key hub for gene therapy innovation.

ASIA PACIFIC GENE THERAPY MARKET ANALYSIS

The Asia Pacific gene therapy market is rapidly expanding as countries like China, India, and Japan are increasingly investing in gene therapy research and clinical trials. Healthcare systems are prioritizing the treatment of genetic disorders, particularly rare diseases, which are seeing rising prevalence in the region due to genetic factors and lifestyle changes. Governments and private organizations are actively funding gene therapy development, leading to a surge in innovation. The region is also witnessing an increase in the adoption of precision medicine, which is further accelerating the demand for gene therapies tailored to individual genetic profiles. Regulatory bodies in Asia Pacific are continuously updating their frameworks to support the commercialization and faster approval of gene therapies. Additionally, the region is becoming a hub for advanced manufacturing technologies, including viral vector production and CRISPR-based gene editing, which are reducing treatment costs and increasing the availability of therapies. Public awareness campaigns and collaborations between biotechnology firms and hospitals are contributing to the growing acceptance and utilization of gene therapies. The healthcare infrastructure is rapidly modernizing, enabling better access to these innovative treatments, especially in urban areas, while overcoming challenges such as affordability and distribution in rural locations. According to Press Information Bureau, the number of hospitals operating across India are 37,725.

LATIN AMERICA GENE THERAPY MARKET ANALYSIS

The gene therapy market in Latin America is experiencing rapid growth due to several key drivers. Health systems across the region are increasingly adopting advanced biotechnology, enabling greater access to gene therapy treatments. According to the Brazilian Federation of Hospitals (FBH) and the National Confederation of Health (CNSaude), of Brazil's 7,191 hospitals, 62% are private. Governments and healthcare providers are allocating more resources towards rare diseases, with ongoing collaborations between local and global biopharmaceutical companies to make gene therapies more affordable and accessible. Research and development investments are escalating, as companies are actively exploring new treatment modalities for genetic disorders prevalent in the region, including sickle cell anemia, hemophilia, and muscular dystrophy. Moreover, local regulatory bodies are continuously working to streamline approval processes, enhancing market accessibility and reducing the time to market for gene therapies. Healthcare professionals are expanding their knowledge and expertise in genetic treatments, driving awareness and acceptance of these therapies among patients. Public-private partnerships are also playing a crucial role in facilitating the introduction of cutting-edge treatments by leveraging funding, infrastructure, and regulatory expertise. Furthermore, the increasing prevalence of chronic genetic conditions in Latin America is further fuelling demand for innovative solutions, while local patient advocacy groups are pushing for more targeted and personalized treatments.

MIDDLE EAST AND AFRICA GENE THERAPY MARKET ANALYSIS

The gene therapy market in the Middle East and Africa (MEA) is experiencing rapid growth as healthcare providers are increasingly focusing on addressing rare genetic disorders through advanced treatments. With numerous government-backed initiatives, countries in the region are investing heavily in research and development to improve access to gene therapies. Several pharmaceutical companies are actively expanding their presence in the MEA region, forming strategic partnerships with local healthcare institutions and academic research bodies. As the region's healthcare infrastructure modernizes, hospitals are adopting advanced genetic testing and screening technologies, enabling earlier detection and personalized treatment approaches. According to the Ministry of Health and Prevention, in 2020, in the UAE there are 157 hospitals and 5369 health centers. Additionally, the country is divided into 53 public hospitals and 104 private hospitals. The increasing prevalence of genetic disorders, such as sickle cell anemia and hemophilia, is driving the demand for targeted therapies, especially in regions with high genetic disease burdens like the Gulf Cooperation Council (GCC) countries. Additionally, the adoption of innovative biotechnological methods in local manufacturing is reducing treatment costs, making gene therapies more accessible. Regulatory frameworks are also evolving to fast-track approvals for novel gene-based treatments, further accelerating market growth. With rising healthcare awareness and improved insurance coverage, patients are showing greater willingness to opt for gene therapies, contributing to the region's growing adoption of these cutting-edge treatments.

COMPETITIVE LANDSCAPE:

Key players in the market are focusing on advancing research operations to improve the safety, efficacy, and accessibility of gene-based treatments. They are actively investing in clinical trials, scaling production capacities, and forming strategic partnerships to accelerate innovation. Emphasis is placed on improving vector technologies and streamlining regulatory processes to bring therapies to market faster. Companies are also expanding their therapeutic pipelines to target a broader range of diseases, including rare genetic disorders and cancers. Efforts to reduce costs and enhance delivery methods are aimed at making gene therapy more accessible globally. In 2024, Precigen announced a strategic focus on commercializing its PRGN-2012 AdenoVerse(R) gene therapy for recurrent respiratory papillomatosis (RRP), with plans for a rolling biologics license application (BLA) submission under an accelerated approval pathway later in 2024. The company is reallocating resources, pausing non-priority programs, and preparing for a potential 2025 launch.

The report provides a comprehensive analysis of the competitive landscape in the gene therapy market with detailed profiles of all major companies, including:

  • Abeona Therapeutics Inc.
  • Adverum Biotechnologies, Inc.
  • Amgen Inc.
  • Astellas Pharma Inc.
  • BioMarin Pharmaceutical Inc.
  • bluebird bio, Inc.
  • Gilead Sciences, Inc.
  • Mustang Bio
  • Novartis AG
  • Orchard Therapeutics plc
  • Roche Holding AG
  • Sangamo Therapeutics
  • uniQure NV

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 2026-2034?

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 Preface

2 Scope and Methodology

  • 2.1 Objectives of the Study
  • 2.2 Stakeholders
  • 2.3 Data Sources
    • 2.3.1 Primary Sources
    • 2.3.2 Secondary Sources
  • 2.4 Market Estimation
    • 2.4.1 Bottom-Up Approach
    • 2.4.2 Top-Down Approach
  • 2.5 Forecasting Methodology

3 Executive Summary

4 Introduction

  • 4.1 Overview
  • 4.2 Key Industry Trends

5 Global Gene Therapy Market

  • 5.1 Market Overview
  • 5.2 Market Performance
  • 5.3 Impact of COVID-19
  • 5.4 Market Forecast

6 Market Breakup by Gene Type

  • 6.1 Antigen
    • 6.1.1 Market Trends
    • 6.1.2 Market Forecast
  • 6.2 Cytokine
    • 6.2.1 Market Trends
    • 6.2.2 Market Forecast
  • 6.3 Tumor Suppressor
    • 6.3.1 Market Trends
    • 6.3.2 Market Forecast
  • 6.4 Suicide Gene
    • 6.4.1 Market Trends
    • 6.4.2 Market Forecast
  • 6.5 Deficiency
    • 6.5.1 Market Trends
    • 6.5.2 Market Forecast
  • 6.6 Growth Factors
    • 6.6.1 Market Trends
    • 6.6.2 Market Forecast
  • 6.7 Receptors
    • 6.7.1 Market Trends
    • 6.7.2 Market Forecast
  • 6.8 Others
    • 6.8.1 Market Trends
    • 6.8.2 Market Forecast

7 Market Breakup by Vector Type

  • 7.1 Viral Vector
    • 7.1.1 Market Trends
    • 7.1.2 Key Segments
      • 7.1.2.1 Adenoviruses
      • 7.1.2.2 Lentiviruses
      • 7.1.2.3 Retroviruses
      • 7.1.2.4 Adeno-Associated Virus
      • 7.1.2.5 Herpes Simplex Virus
      • 7.1.2.6 Poxvirus
      • 7.1.2.7 Vaccinia Virus
      • 7.1.2.8 Others
    • 7.1.3 Market Forecast
  • 7.2 Non-Viral Techniques
    • 7.2.1 Market Trends
    • 7.2.2 Key Segments
      • 7.2.2.1 Naked and Plasmid Vectors
      • 7.2.2.2 Gene Gun
      • 7.2.2.3 Electroporation
      • 7.2.2.4 Lipofection
      • 7.2.2.5 Others
    • 7.2.3 Market Forecast

8 Market Breakup by Delivery Method

  • 8.1 In-Vivo Gene Therapy
    • 8.1.1 Market Trends
    • 8.1.2 Market Forecast
  • 8.2 Ex-Vivo Gene Therapy
    • 8.2.1 Market Trends
    • 8.2.2 Market Forecast

9 Market Breakup by Application

  • 9.1 Oncological Disorders
    • 9.1.1 Market Trends
    • 9.1.2 Market Forecast
  • 9.2 Rare Diseases
    • 9.2.1 Market Trends
    • 9.2.2 Market Forecast
  • 9.3 Cardiovascular Diseases
    • 9.3.1 Market Trends
    • 9.3.2 Market Forecast
  • 9.4 Neurological Disorders
    • 9.4.1 Market Trends
    • 9.4.2 Market Forecast
  • 9.5 Infectious Disease
    • 9.5.1 Market Trends
    • 9.5.2 Market Forecast
  • 9.6 Others
    • 9.6.1 Market Trends
    • 9.6.2 Market Forecast

10 Market Breakup by Region

  • 10.1 North America
    • 10.1.1 United States
      • 10.1.1.1 Market Trends
      • 10.1.1.2 Market Forecast
    • 10.1.2 Canada
      • 10.1.2.1 Market Trends
      • 10.1.2.2 Market Forecast
  • 10.2 Asia-Pacific
    • 10.2.1 China
      • 10.2.1.1 Market Trends
      • 10.2.1.2 Market Forecast
    • 10.2.2 Japan
      • 10.2.2.1 Market Trends
      • 10.2.2.2 Market Forecast
    • 10.2.3 India
      • 10.2.3.1 Market Trends
      • 10.2.3.2 Market Forecast
    • 10.2.4 South Korea
      • 10.2.4.1 Market Trends
      • 10.2.4.2 Market Forecast
    • 10.2.5 Australia
      • 10.2.5.1 Market Trends
      • 10.2.5.2 Market Forecast
    • 10.2.6 Indonesia
      • 10.2.6.1 Market Trends
      • 10.2.6.2 Market Forecast
    • 10.2.7 Others
      • 10.2.7.1 Market Trends
      • 10.2.7.2 Market Forecast
  • 10.3 Europe
    • 10.3.1 Germany
      • 10.3.1.1 Market Trends
      • 10.3.1.2 Market Forecast
    • 10.3.2 France
      • 10.3.2.1 Market Trends
      • 10.3.2.2 Market Forecast
    • 10.3.3 United Kingdom
      • 10.3.3.1 Market Trends
      • 10.3.3.2 Market Forecast
    • 10.3.4 Italy
      • 10.3.4.1 Market Trends
      • 10.3.4.2 Market Forecast
    • 10.3.5 Spain
      • 10.3.5.1 Market Trends
      • 10.3.5.2 Market Forecast
    • 10.3.6 Russia
      • 10.3.6.1 Market Trends
      • 10.3.6.2 Market Forecast
    • 10.3.7 Others
      • 10.3.7.1 Market Trends
      • 10.3.7.2 Market Forecast
  • 10.4 Latin America
    • 10.4.1 Brazil
      • 10.4.1.1 Market Trends
      • 10.4.1.2 Market Forecast
    • 10.4.2 Mexico
      • 10.4.2.1 Market Trends
      • 10.4.2.2 Market Forecast
    • 10.4.3 Others
      • 10.4.3.1 Market Trends
      • 10.4.3.2 Market Forecast
  • 10.5 Middle East and Africa
    • 10.5.1 Market Trends
    • 10.5.2 Market Breakup by Country
    • 10.5.3 Market Forecast

11 SWOT Analysis

  • 11.1 Overview
  • 11.2 Strengths
  • 11.3 Weaknesses
  • 11.4 Opportunities
  • 11.5 Threats

12 Value Chain Analysis

13 Porters Five Forces Analysis

  • 13.1 Overview
  • 13.2 Bargaining Power of Buyers
  • 13.3 Bargaining Power of Suppliers
  • 13.4 Degree of Competition
  • 13.5 Threat of New Entrants
  • 13.6 Threat of Substitutes

14 Price Analysis

15 Competitive Landscape

  • 15.1 Market Structure
  • 15.2 Key Players
  • 15.3 Profiles of Key Players
    • 15.3.1 Abeona Therapeutics Inc.
      • 15.3.1.1 Company Overview
      • 15.3.1.2 Product Portfolio
    • 15.3.2 Adverum Biotechnologies, Inc.
      • 15.3.2.1 Company Overview
      • 15.3.2.2 Product Portfolio
      • 15.3.2.3 Financials
    • 15.3.3 Amgen Inc.
      • 15.3.3.1 Company Overview
      • 15.3.3.2 Product Portfolio
      • 15.3.3.3 Financials
      • 15.3.3.4 SWOT Analysis
    • 15.3.4 Astellas Pharma Inc.
      • 15.3.4.1 Company Overview
      • 15.3.4.2 Product Portfolio
      • 15.3.4.3 Financials
      • 15.3.4.4 SWOT Analysis
    • 15.3.5 BioMarin Pharmaceutical Inc.
      • 15.3.5.1 Company Overview
      • 15.3.5.2 Product Portfolio
      • 15.3.5.3 Financials
      • 15.3.5.4 SWOT Analysis
    • 15.3.6 bluebird bio, Inc.
      • 15.3.6.1 Company Overview
      • 15.3.6.2 Product Portfolio
      • 15.3.6.3 Financials
      • 15.3.6.4 SWOT Analysis
    • 15.3.7 Gilead Sciences, Inc.
      • 15.3.7.1 Company Overview
      • 15.3.7.2 Product Portfolio
      • 15.3.7.3 Financials
      • 15.3.7.4 SWOT Analysis
    • 15.3.8 Mustang Bio
      • 15.3.8.1 Company Overview
      • 15.3.8.2 Product Portfolio
    • 15.3.9 Novartis AG
      • 15.3.9.1 Company Overview
      • 15.3.9.2 Product Portfolio
      • 15.3.9.3 Financials
      • 15.3.9.4 SWOT Analysis
    • 15.3.10 Orchard Therapeutics plc
      • 15.3.10.1 Company Overview
      • 15.3.10.2 Product Portfolio
      • 15.3.10.3 Financials
    • 15.3.11 Roche Holding AG
      • 15.3.11.1 Company Overview
      • 15.3.11.2 Product Portfolio
      • 15.3.11.3 Financials
      • 15.3.11.4 SWOT Analysis
    • 15.3.12 Sangamo Therapeutics
      • 15.3.12.1 Company Overview
      • 15.3.12.2 Product Portfolio
      • 15.3.12.3 Financials
      • 15.3.12.4 SWOT Analysis
    • 15.3.13 uniQure NV
      • 15.3.13.1 Company Overview
      • 15.3.13.2 Product Portfolio
      • 15.3.13.3 Financials

List of Figures

  • Figure 1: Global: Gene Therapy Market: Major Drivers and Challenges
  • Figure 2: Global: Gene Therapy Market: Sales Value (in Billion USD), 2020-2025
  • Figure 3: Global: Gene Therapy Market Forecast: Sales Value (in Billion USD), 2026-2034
  • Figure 4: Global: Gene Therapy Market: Breakup by Gene Type (in %), 2025
  • Figure 5: Global: Gene Therapy Market: Breakup by Vector Type (in %), 2025
  • Figure 6: Global: Gene Therapy Market: Breakup by Delivery Method (in %), 2025
  • Figure 7: Global: Gene Therapy Market: Breakup by Application (in %), 2025
  • Figure 8: Global: Gene Therapy Market: Breakup by Region (in %), 2025
  • Figure 9: Global: Gene Therapy (Antigen) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 10: Global: Gene Therapy (Antigen) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 11: Global: Gene Therapy (Cytokine) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 12: Global: Gene Therapy (Cytokine) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 13: Global: Gene Therapy (Tumor Suppressor) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 14: Global: Gene Therapy (Tumor Suppressor) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 15: Global: Gene Therapy (Suicide Gene) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 16: Global: Gene Therapy (Suicide Gene) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 17: Global: Gene Therapy (Deficiency) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 18: Global: Gene Therapy (Deficiency) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 19: Global: Gene Therapy (Growth Factors) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 20: Global: Gene Therapy (Growth Factors) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 21: Global: Gene Therapy (Receptors) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 22: Global: Gene Therapy (Receptors) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 23: Global: Gene Therapy (Other Gene Types) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 24: Global: Gene Therapy (Other Gene Types) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 25: Global: Gene Therapy (Viral Vector) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 26: Global: Gene Therapy (Viral Vector) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 27: Global: Gene Therapy (Non-Viral Techniques) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 28: Global: Gene Therapy (Non-Viral Techniques) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 29: Global: Gene Therapy (In-Vivo Gene Therapy) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 30: Global: Gene Therapy (In-Vivo Gene Therapy) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 31: Global: Gene Therapy (Ex-Vivo Gene Therapy) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 32: Global: Gene Therapy (Ex-Vivo Gene Therapy) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 33: Global: Gene Therapy (Oncological Disorders) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 34: Global: Gene Therapy (Oncological Disorders) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 35: Global: Gene Therapy (Rare Diseases) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 36: Global: Gene Therapy (Rare Diseases) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 37: Global: Gene Therapy (Cardiovascular Diseases) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 38: Global: Gene Therapy (Cardiovascular Diseases) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 39: Global: Gene Therapy (Neurological Disorders) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 40: Global: Gene Therapy (Neurological Disorders) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 41: Global: Gene Therapy (Infectious Disease) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 42: Global: Gene Therapy (Infectious Disease) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 43: Global: Gene Therapy (Other Applications) Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 44: Global: Gene Therapy (Other Applications) Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 45: North America: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 46: North America: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 47: United States: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 48: United States: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 49: Canada: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 50: Canada: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 51: Asia-Pacific: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 52: Asia-Pacific: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 53: China: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 54: China: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 55: Japan: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 56: Japan: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 57: India: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 58: India: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 59: South Korea: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 60: South Korea: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 61: Australia: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 62: Australia: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 63: Indonesia: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 64: Indonesia: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 65: Others: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 66: Others: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 67: Europe: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 68: Europe: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 69: Germany: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 70: Germany: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 71: France: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 72: France: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 73: United Kingdom: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 74: United Kingdom: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 75: Italy: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 76: Italy: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 77: Spain: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 78: Spain: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 79: Russia: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 80: Russia: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 81: Others: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 82: Others: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 83: Latin America: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 84: Latin America: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 85: Brazil: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 86: Brazil: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 87: Mexico: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 88: Mexico: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 89: Others: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 90: Others: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 91: Middle East and Africa: Gene Therapy Market: Sales Value (in Million USD), 2020 & 2025
  • Figure 92: Middle East and Africa: Gene Therapy Market: Breakup by Country (in %), 2025
  • Figure 93: Middle East and Africa: Gene Therapy Market Forecast: Sales Value (in Million USD), 2026-2034
  • Figure 94: Global: Gene Therapy Industry: SWOT Analysis
  • Figure 95: Global: Gene Therapy Industry: Value Chain Analysis
  • Figure 96: Global: Gene Therapy Industry: Porter's Five Forces Analysis

List of Tables

  • Table 1: Global: Gene Therapy Market: Key Industry Highlights, 2025 and 2034
  • Table 2: Global: Gene Therapy Market Forecast: Breakup by Gene Type (in Million USD), 2026-2034
  • Table 3: Global: Gene Therapy Market Forecast: Breakup by Vector Type (in Million USD), 2026-2034
  • Table 4: Global: Gene Therapy Market Forecast: Breakup by Delivery Method (in Million USD), 2026-2034
  • Table 5: Global: Gene Therapy Market Forecast: Breakup by Application (in Million USD), 2026-2034
  • Table 6: Global: Gene Therapy Market Forecast: Breakup by Region (in Million USD), 2026-2034
  • Table 7: Global: Gene Therapy Market: Competitive Structure
  • Table 8: Global: Gene Therapy Market: Key Players