基因治療市場機會、成長促進因素、產業趨勢分析及預測（2024-2032年）

2024 年全球基因治療市場價值為 90 億美元，預計到 2034 年將以 19.4% 的複合年成長率成長至 513 億美元。

基因遞送系統的進步、基因治療研發投資的增加、基因治療靶向疾病（如癌症和罕見遺傳疾病）的日益普遍以及監管產品核准數量的不斷成長，共同推動了基因治療市場的顯著成長。基因和細胞療法合約生產的普及以及遞送技術的策略性發展，進一步提升了市場前景。監管機構正在簡化有前景的基因療法的核准流程，各國政府也在加強對創新基因藥物平台的投入。隨著醫療保健系統日益關注個人化和標靶治療，基因療法因其在分子層面解決複雜疾病的潛力而備受關注。市場參與者也在利用數位化平台共享資料、加速臨床試驗並最佳化藥物研發流程。同時，精準醫療和生物資訊學的更廣泛融合，正幫助基因療法提高療效、減少副作用並帶來長期的治療益處。這些趨勢正將基因療法推向現代醫學下一階段的顛覆性發展階段。

2024年，病毒載體市佔率高達85.1%，凸顯了其作為最有效遞送載體的持續主導地位。這些載體，包括腺相關病毒和慢病毒，因其能夠精準遞送遺傳物質並長期維持穩定的基因表現而被廣泛應用。其廣泛的臨床應用以及在已獲批准療法中取得的成功，進一步鞏固了其商業可行性，使其成為基因療法研發的基石。穩健的生產流程和可擴展的生產能力也為其在各種治療產品線中的廣泛應用提供了支持。

由於其能夠系統性地治療複雜的多器官疾病，體內基因治療市場預計在2024年將達到58億美元。體內療法能夠將基因直接導入體內，無需進行細胞萃取和回輸。這種方法有助於擴大治療範圍，並降低手術複雜性。非病毒和混合遞送系統（例如奈米顆粒和脂質製劑）的進步正在進一步提高標靶精準度和治療效果。

預計到2024年，北美基因治療市佔率將達到51.2%，成為基因治療創新和商業化的主導地區。這一領先地位得益於穩健的投資環境、有利的監管框架和完善的醫療基礎設施。憑藉領先的生物技術公司、傑出的學術機構和高水準的研發活動，該地區，尤其是美國，正不斷拓展基因治療科學的邊界。強大的公私合作和豐富的人才儲備正在加速前沿療法的臨床開發和商業化應用。

全球基因治療市場的主要參與者包括Sangamo Therapeutics、安進（Amgen）、Spark Therapeutics（羅氏旗下）、Beam Therapeutics、Krystal Biotech、Sarepta Therapeutics、AGTC、Orchard Therapeutics、bluebird bio、百時美施貴寶（Bristol-Myers Sbbqui Company（Gile） Sciences）、諾華（Novartis）、Helixmith、輝瑞（Pfizer）、Intellia Therapeutics、拜耳（Bayer）、UniQure NV、BioMarin Pharmaceutical、CRISPR Therapeutics和Audentes Therapeutics（安斯泰來製藥旗下）。為了鞏固市場地位，基因治療領域的領導者正優先考慮策略性併購，以增強其技術平台並擴大產品線。許多公司正與學術機構和小型生物技術創新者建立研發合作關係，以獲取新型療法並加速臨床試驗進程。增加對內部生產能力和全球供應鏈基礎設施的投資，可確保更快的規模生產和合規性。各公司也正加強爭取快速通道、孤兒藥和突破性療法認定，以縮短產品上市時間。

目錄

第1章：方法論與範圍

第2章：執行概要

第3章：行業洞察

• 產業生態系分析
• 產業影響因素
  • 成長促進因素
    • 基因編輯技術的進步
    • 罕見遺傳性疾病和先天性疾病的盛行率不斷上升
    • 投資和合作不斷增加
    • 人們對基因診斷的認知不斷提高，基因診斷技術也日益進步。
  • 產業陷阱與挑戰
    • 高昂的研發和製造成本
    • 基因治療遞送的複雜性與潛在副作用
  • 市場機遇
    • 開發個人化和精準基因療法
    • 隨著醫療基礎設施的不斷發展，新興市場將迎來擴張。
• 成長潛力分析
• 監管環境
  • 北美洲
    • 美國
    • 加拿大
  • 歐洲
  • 亞太地區
  • 拉丁美洲
  • 中東和非洲
• 技術格局
  • 當前趨勢
  • 新興技術
• 管道分析
• 未來市場趨勢
• 波特的分析
• PESTEL 分析

第4章：競爭格局

• 介紹
• 公司市佔率分析
  • 全球的
  • 北美洲
  • 歐洲
  • 亞太地區
• 公司矩陣分析
• 主要市場參與者的競爭分析
• 競爭定位矩陣
• 關鍵進展
  • 併購
  • 夥伴關係與合作
  • 新產品發布
  • 擴張計劃

第5章：市場估算與預測：基於Vector的數據，2021-2034年

• 主要趨勢
• 病毒載體
  • 逆轉錄病毒載體
  • 腺相關病毒載體
  • 慢病毒載體
  • 其他病毒載體
• 非病毒載體
  • 質粒DNA
  • 脂質奈米顆粒（LNPs）
  • 寡核苷酸
  • 其他非病毒載體

第6章：市場估算與預測：以交付方式分類，2021-2034年

• 主要趨勢
• 體內基因治療
• 體外基因治療

第7章：市場估計與預測：依基因類型分類，2021-2034年

• 主要趨勢
• 抗原編碼基因
• 細胞激素編碼基因
• 腫瘤抑制基因
• 自殺基因
• 基因替代（針對缺陷）
• 生長因子基因
• 受體編碼基因
• 其他基因類型

第8章：市場估算與預測：依指示劑分類，2021-2034年

• 主要趨勢
• 腫瘤學
  • 急性淋巴母細胞白血病（ALL）
  • 大B細胞淋巴瘤
  • 頭頸部鱗狀細胞癌
  • 黑色素瘤
• 神經系統疾病
  • 杜氏肌肉營養不良症（DMD）
  • 脊髓性肌肉萎縮症（SMA）
• 血友病A和B
• 肝病
• 遺傳性視網膜疾病
• 周邊動脈疾病
• 其他跡象

第9章：市場估算與預測：依最終用途分類，2021-2034年

• 主要趨勢
• 醫院
• 專科診所和基因治療中心
• 學術和研究機構
• 其他用途

第10章：市場估計與預測：依地區分類，2021-2034年

• 主要趨勢
• 北美洲
  • 美國
  • 加拿大
• 歐洲
  • 德國
  • 英國
  • 法國
  • 西班牙
  • 義大利
  • 荷蘭
• 亞太地區
  • 中國
  • 印度
  • 日本
  • 澳洲
  • 韓國
• 拉丁美洲
  • 巴西
  • 墨西哥
  • 阿根廷
• 中東和非洲
  • 南非
  • 沙烏地阿拉伯
  • 阿拉伯聯合大公國

第11章：公司簡介

• Amgen
• Applied Genetic Technologies Corporation (AGTC)
• Audentes Therapeutics, Inc. (Astellas Pharma)
• Bayer
• Beam Therapeutics
• BioMarin Pharmaceutical
• bluebird bio
• Bristol-Myers Squibb Company
• CRISPR Therapeutics
• Gilead Sciences
• Helixmith
• Intellia Therapeutics
• Krystal Biotech
• Novartis
• Orchard Therapeutics
• Pfizer
• Sangamo Therapeutics
• Sarepta Therapeutics
• Spark Therapeutics (Roche)
• UniQure NV

The Global Gene Therapy Market was valued at USD 9 billion in 2024 and is estimated to grow at a CAGR of 19.4% to reach USD 51.3 billion by 2034.

This significant growth trajectory is being fueled by advances in gene delivery systems, rising investments in gene therapy development, the growing prevalence of diseases targeted by gene therapy, such as cancer and rare genetic disorders, and an increasing number of regulatory product approvals. Expanding access to contract manufacturing for gene and cell therapies, along with strategic developments in delivery technology, are further enhancing the market outlook. Regulatory agencies are streamlining approval processes for promising gene therapies, and governments are boosting funding toward innovative genetic medicine platforms. As healthcare systems focus on personalized and targeted treatments, gene therapy is gaining traction for its potential to address complex diseases at the molecular level. Market players are also leveraging digital platforms to share data, accelerate clinical trials, and optimize drug discovery pipelines. Meanwhile, broader integration of precision medicine and bioinformatics is helping gene therapies achieve better treatment efficacy, reduced side effects, and long-term therapeutic benefits. These trends are positioning gene therapy as a disruptive force in the next phase of modern medicine.

In 2024, the viral vectors segment held an 85.1% share, underscoring their continued dominance as the most effective delivery vehicles. These vectors, including adeno-associated viruses and lentiviruses, are widely used due to their superior ability to deliver genetic material accurately and maintain consistent gene expression over time. Their broad clinical application and proven success in approved treatments have reinforced their commercial viability, making them a cornerstone of gene therapy development. Robust manufacturing protocols and scalable production also support their widespread deployment across therapeutic pipelines.

The in vivo gene therapy segment accounted for USD 5.8 billion in 2024, owing to its systemic approach to treating complex and multi-organ diseases. In vivo methods enable direct gene delivery into the body, eliminating the need for cell extraction and reinfusion. This method supports broader therapeutic access and reduces procedural complexity. Advances in non-viral and hybrid delivery systems-such as nanoparticles and lipid-based formulations are further enhancing targeting precision and treatment outcomes.

North America Gene Therapy Market held a 51.2% share in 2024, making it the dominant region for gene therapy innovation and commercialization. This leadership is supported by a robust investment environment, favorable regulatory frameworks, and a sophisticated healthcare infrastructure. With leading biotech firms, prominent academic institutions, and high R&D activity, the region, particularly the U.S., continues to push the boundaries of gene therapy science. Strong public-private collaborations and a deep talent pool are accelerating clinical development and commercial adoption of cutting-edge therapies.

Prominent players in the Global Gene Therapy Market include Sangamo Therapeutics, Amgen, Spark Therapeutics (Roche), Beam Therapeutics, Krystal Biotech, Sarepta Therapeutics, AGTC, Orchard Therapeutics, bluebird bio, Bristol-Myers Squibb Company, Gilead Sciences, Novartis, Helixmith, Pfizer, Intellia Therapeutics, Bayer, UniQure N.V., BioMarin Pharmaceutical, CRISPR Therapeutics, and Audentes Therapeutics (Astellas Pharma). To strengthen their foothold, leading companies in the gene therapy sector are prioritizing strategic mergers and acquisitions to enhance their technology platforms and expand product pipelines. Many firms are entering collaborative R&D partnerships with academic institutions and smaller biotech innovators to access novel therapies and accelerate clinical timelines. Increasing investment in in-house manufacturing capabilities and global supply chain infrastructure ensures faster scaling and regulatory compliance. Companies are also intensifying their focus on securing fast-track, orphan drug, and breakthrough therapy designations to reduce time-to-market.

Table of Contents

Chapter 1 Methodology and Scope

• 1.1 Market scope and definition
• 1.2 Research design
  • 1.2.1 Research approach
  • 1.2.2 Data collection methods
• 1.3 Data mining sources
  • 1.3.1 Global
  • 1.3.2 Regional/Country
• 1.4 Base estimates and calculations
  • 1.4.1 Base year calculation
  • 1.4.2 Key trends for market estimation
• 1.5 Primary research and validation
  • 1.5.1 Primary sources
• 1.6 Forecast model
• 1.7 Research assumptions and limitations

Chapter 2 Executive Summary

• 2.1 Industry 360° synopsis
• 2.2 Key market trends
  • 2.2.1 Regional trends
  • 2.2.2 Vector trends
  • 2.2.3 Delivery method trends
  • 2.2.4 Gene type trends
  • 2.2.5 Indication trends
  • 2.2.6 End Use trends
• 2.3 CXO perspectives: Strategic imperatives
  • 2.3.1 Key decision points for industry executives
  • 2.3.2 Critical success factors for market players
• 2.4 Future outlook and strategic recommendations

Chapter 3 Industry Insights

• 3.1 Industry ecosystem analysis
• 3.2 Industry impact forces
  • 3.2.1 Growth drivers
    • 3.2.1.1 Advancements in gene editing technologies
    • 3.2.1.2 Increasing prevalence of rare genetic and inherited diseases
    • 3.2.1.3 Rising investments and collaborations
    • 3.2.1.4 Growing awareness and improved genetic diagnostics
  • 3.2.2 Industry pitfalls and challenges
    • 3.2.2.1 High development and manufacturing costs
    • 3.2.2.2 Complexity of gene therapy delivery and potential side effects
  • 3.2.3 Market opportunities
    • 3.2.3.1 Development of personalized and precision gene therapies
    • 3.2.3.2 Expansion in emerging markets with growing healthcare infrastructure
• 3.3 Growth potential analysis
• 3.4 Regulatory landscape
  • 3.4.1 North America
    • 3.4.1.1 U.S.
    • 3.4.1.2 Canada
  • 3.4.2 Europe
  • 3.4.3 Asia Pacific
  • 3.4.4 Latin America
  • 3.4.5 Middle East and Africa
• 3.5 Technology landscape
  • 3.5.1 Current trends
  • 3.5.2 Emerging technologies
• 3.6 Pipeline analysis
• 3.7 Future market trends
• 3.8 Porter's analysis
• 3.9 PESTEL analysis

Chapter 4 Competitive Landscape, 2024

• 4.1 Introduction
• 4.2 Company market share analysis
  • 4.2.1 Global
  • 4.2.2 North America
  • 4.2.3 Europe
  • 4.2.4 Asia Pacific
• 4.3 Company matrix analysis
• 4.4 Competitive analysis of major market players
• 4.5 Competitive positioning matrix
• 4.6 Key developments
  • 4.6.1 Merger and acquisition
  • 4.6.2 Partnership and collaboration
  • 4.6.3 New product launches
  • 4.6.4 Expansion plans

Chapter 5 Market Estimates and Forecast, By Vector, 2021 - 2034 ($ Mn)

• 5.1 Key trends
• 5.2 Viral vectors
  • 5.2.1 Retro viral vectors
  • 5.2.2 Adeno-associated virus vectors
  • 5.2.3 Lentiviral vectors
  • 5.2.4 Other viral vectors
• 5.3 Non-viral vectors
  • 5.3.1 Plasmid DNA
  • 5.3.2 Lipid nanoparticles (LNPs)
  • 5.3.3 Oligonucleotides
  • 5.3.4 Other non-viral vectors

Chapter 6 Market Estimates and Forecast, By Delivery Method, 2021 - 2034 ($ Mn)

• 6.1 Key trends
• 6.2 In vivo gene therapy
• 6.3 Ex vivo gene therapy

Chapter 7 Market Estimates and Forecast, By Gene Type, 2021 - 2034 ($ Mn)

• 7.1 Key trends
• 7.2 Antigen-encoding genes
• 7.3 Cytokine-encoding genes
• 7.4 Tumor suppressor genes
• 7.5 Suicide genes
• 7.6 Gene replacement (for deficiency)
• 7.7 Growth factor genes
• 7.8 Receptor-encoding genes
• 7.9 Other gene types

Chapter 8 Market Estimates and Forecast, By Indication, 2021 - 2034 ($ Mn)

• 8.1 Key trends
• 8.2 Oncology
  • 8.2.1 Acute Lymphoblastic Leukemia (ALL)
  • 8.2.2 Large B-cell lymphoma
  • 8.2.3 Head & neck squamous cell carcinoma
  • 8.2.4 Melanoma
• 8.3 Neurological disorders
  • 8.3.1 Duchenne muscular dystrophy (DMD)
  • 8.3.2 Spinal muscular atrophy (SMA)
• 8.4 Hemophilia A & B
• 8.5 Hepatological diseases
• 8.6 Inherited retinal disease
• 8.7 Peripheral arterial disease
• 8.8 Other indications

Chapter 9 Market Estimates and Forecast, By End Use, 2021 - 2034 ($ Mn)

• 9.1 Key trends
• 9.2 Hospitals
• 9.3 Specialty clinics and gene therapy centers
• 9.4 Academic and research institutions
• 9.5 Other End uses

Chapter 10 Market Estimates and Forecast, By Region, 2021 - 2034 ($ Mn)

• 10.1 Key trends
• 10.2 North America
  • 10.2.1 U.S.
  • 10.2.2 Canada
• 10.3 Europe
  • 10.3.1 Germany
  • 10.3.2 UK
  • 10.3.3 France
  • 10.3.4 Spain
  • 10.3.5 Italy
  • 10.3.6 Netherlands
• 10.4 Asia Pacific
  • 10.4.1 China
  • 10.4.2 India
  • 10.4.3 Japan
  • 10.4.4 Australia
  • 10.4.5 South Korea
• 10.5 Latin America
  • 10.5.1 Brazil
  • 10.5.2 Mexico
  • 10.5.3 Argentina
• 10.6 Middle East and Africa
  • 10.6.1 South Africa
  • 10.6.2 Saudi Arabia
  • 10.6.3 UAE

Chapter 11 Company Profiles

• 11.1 Amgen
• 11.2 Applied Genetic Technologies Corporation (AGTC)
• 11.3 Audentes Therapeutics, Inc. (Astellas Pharma)
• 11.4 Bayer
• 11.5 Beam Therapeutics
• 11.6 BioMarin Pharmaceutical
• 11.7 bluebird bio
• 11.8 Bristol-Myers Squibb Company
• 11.9 CRISPR Therapeutics
• 11.10 Gilead Sciences
• 11.11 Helixmith
• 11.12 Intellia Therapeutics
• 11.13 Krystal Biotech
• 11.14 Novartis
• 11.15 Orchard Therapeutics
• 11.16 Pfizer
• 11.17 Sangamo Therapeutics
• 11.18 Sarepta Therapeutics
• 11.19 Spark Therapeutics (Roche)
• 11.20 UniQure N.V.