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Report
Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing
Market Research Report I 2025-12-30 I 166 Pages I BCC Research
Description
Report Scope
The report provides an overview of genetic modification therapies clinical applications: gene therapies, genetically modified cell therapies, RNA therapies and gene editing market and analyzes market trends. It includes global revenue ($ million) for the base year 2024, estimated data for 2025 and forecast data from 2026 through 2030. In this report, the market is segmented based on platform technology, application and region.
Based on platform technology, the market is segmented into gene editing, gene therapy, gene-modified cell therapy and RNA therapy platforms. The application segment covers oncology, rare diseases, cardiovascular diseases, musculoskeletal conditions, hematological disorders and other applications. The gene therapy segment is segmented based on the delivery systems into adeno-associated virus (AAV), herpes simplex virus (HSV)-1, lentivirus and others. The RNA therapy segment is segmented into antisense oligonucleotides, small interfering RNAs and others based on type.
The report also focuses on the regional segmentation of the market. The regions covered in this study include North America, Europe, Asia-Pacific, and the Rest of the World, with a focus on major countries in these regions.
The report also includes major driving trends and restraints that affect the market and vendor landscape. It analyzes environmental, social and corporate governance (ESG) developments, discussing pipeline activities and emerging technologies related to the market. The report concludes with an analysis of the competitive landscape and industry structure. It also has a dedicated section of company profiles that covers details of leading companies. The report does not include mRNA vaccines.
Report Includes
- 103 data tables and 63 additional tables
- Overview and an analysis of the global market for clinical applications of genetic modification therapies
- In-depth analysis of global market trends, featuring historical revenue data for 2022-2024, estimated figures for 2025, as well as forecasts for 2030. This analysis includes projections of compound annual growth rates (CAGRs) through 2030
- Evaluation of the current market size and revenue growth prospects specific to the genetic modification therapies, accompanied by a market share analysis by platform technology, application and region
- Analysis of current and future demand in the genetic modification therapies, along with a detailed analysis of the competitive environment, market regulations and reimbursement practices
- Review of gene editing, gene therapy, CAR T-cell therapy, RNA therapy and lentiviral production, and discussion of how they are playing a key role in genetic modification therapies
- Analysis of drivers, challenges and opportunities affecting market growth
- Coverage of evolving technologies, the current and future market potential, R&D activities, growth strategies, new product pipeline, regulatory framework and reimbursement scenarios, and ESG trends of the market
- Market share analysis of the key market participants of the industry, along with their research priorities, product portfolios, global rankings and competitive landscape
- Profiles of the leading companies, including Gilead Sciences Inc., Alnylam Pharmaceuticals Inc., Novartis AG, Bristol-Myers Squibb Co., and Sarepta Therapeutics Inc.
Executive Summary
Summary:
The global market for genetic modification therapies is estimated to grow from $14.8 billion in 2025 to reach $32.1 billion by the end of 2030, at a compound annual growth rate (CAGR) of 16.8% from 2025 through 2030.
Genetic modification therapies are next-generation biologic therapies developed on the basis of the alteration of genetic material. Each platform technology in gene modification therapy, including gene therapy or RNA therapy, has significant potential in the coming years. These therapies are developed for debilitating conditions with limited treatment options, making them specialized. Products based on gene therapy are often developed as one-time treatments for rare, inherited conditions. The industry has a robust clinical pipeline, and many biopharmaceutical companies are entering the field. Currently, these therapies are available for rare diseases, cancer and hematological conditions. However, the industry may witness the approval of products for other application areas, such as musculoskeletal, cardiovascular and neurological conditions.
Table of Contents
Chapter 1 Executive Summary
Market Outlook
Scope of Report
Market Summary
Market Dynamics and Growth Factors
Emerging Technologies
Segmental Analysis
Regional Analysis
Conclusion
Chapter 2 Market Overview
Overview
Porter's Five Forces Analysis
Threat of New Entrants (Low)
Bargaining Power of Suppliers (Moderate)
Bargaining Power of Buyers (Moderate)
Threat of Substitute (Low)
Industry Rivalry (High)
Macroeconomic Factors Analysis
Impact of the U.S. Tariffs
Geopolitical Factors
Chapter 3 Genetic Modification Therapies: Technology and Applications
Genetic Modification Therapies
Gene Therapy Technologies
RNA Therapy Technologies
Gene Editing Technologies
Different Types of Genome Editing Tools
Delivery Methods of Genome Editing
Gene-Modified Cell Therapy Technologies
Delivery Technologies
Clinical Applications
Chapter 4 Market Dynamics
Market Dynamics
Market Drivers
Increasing Prevalence of Rare Genetic Disorders
Growing Focus on Precision Medicine
Market Restraints
Regulatory Requirements on Adverse Event Tracking and Long-Term Monitoring
Ethical Concerns on Genome Editing Techniques
High Cost of Genetic Modification Therapies
Market Opportunities
Expanding Application Areas of Genetic Modification Therapies
Off-the-Shelf Products in Gene-Modified Cell Therapies
Chapter 5 Emerging Technologies and Pipeline Analysis
Key Takeaways
Emerging Technologies
In Vivo Delivery of CRISPR
Improved Cas Variants
Advances in RNAi Delivery Systems
RNA Editing
Pipeline Analysis
RNA Therapies
Gene Editing Therapies
Gene Therapies and Gene-Modified Cell Therapies
Chapter 6 Market Segmentation Analysis
Segmentation Breakdown
Market Analysis by Platform Technology
Key Takeaways
RNA Therapy
Gene-Modified Cell Therapy
Gene Therapy
Gene Editing
Market Analysis by Application
Key Takeaways
Oncology
Rare Diseases
Cardiovascular Diseases
Musculoskeletal Conditions
Hematological Disorders
Others
Geographic Breakdown
Market Analysis by Region
Key Takeaways
North America
Europe
Asia-Pacific
Rest of the World
Chapter 7 Competitive Intelligence
Key Takeaways
Industry Structure
Company Share Analysis of the RNA Therapies Market
Company Share Analysis of the Gene Therapies Market
Company Share Analysis of the Gene-Modified Cell Therapies Market
Strategic Analysis
Chapter 8 Sustainability in the Genetic Modification Therapies Market: An ESG Perspective
Introduction to ESG
ESG Risk Ratings
Conclusion
Chapter 9 Appendix
Methodology
Sources
Abbreviations
Company Profiles
ALNYLAM PHARMACEUTICALS INC.
AMGEN INC.
BIOGEN
BRISTOL-MYERS SQUIBB CO.
F. HOFFMANN-LA ROCHE LTD.
FERRING
GENETIX BIOTHERAPEUTICS INC.
GILEAD SCIENCES INC.
JOHNSON & JOHNSON
LILLY USA LLC.
NOVARTIS AG
NOVO NORDISK A/S
SANOFI
SAREPTA THERAPEUTICS INC.
VERTEX PHARMACEUTICALS INC.
Emerging Startups/Market Disruptors
List of Tables
Summary Table : Global Market for Genetic Modification Therapies, by Region, Through 2030
Table 1 : Genetic Modification Platforms
Table 2 : Comparison of Traditional Pharmaceuticals and Genetic Modification Therapies
Table 3 : Distinct Features of the Genetic Modification Approaches
Table 4 : Comparison of Genome Editing Tools
Table 5 : Gene-Modified Cell Therapy Technologies
Table 6 : Applications of Genetic Modification Therapies
Table 7 : List of Selected Pipeline Molecules
Table 8 : List of Gene Editing Therapeutic Trials in Phase I
Table 9 : List of Gene Editing Therapeutic Trials in Phase I/II, II and III
Table 10 : List of Selected Pipeline Molecules
Table 11 : Global Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 12 : Global Market for RNA Therapy, by Region, Through 2030
Table 13 : Global Market for RNA Therapy, by RNA Type, Through 2030
Table 14 : Global Market for Antisense Oligonucleotides, by Region, Through 2030
Table 15 : RNAi Approved Drugs List
Table 16 : Global Market for Small Interfering RNAs, by Region, Through 2030
Table 17 : Global Market for Other RNA-Based Therapeutics, by Region, Through 2030
Table 18 : Approved CAR T-Cell Therapies
Table 19 : Global Market for Gene-Modified Cell Therapy, by Region, Through 2030
Table 20 : Global Market for Gene Therapy, by Region, Through 2030
Table 21 : Global Market for Gene Therapy, by Delivery System, Through 2030
Table 22 : Global Market for AAV Delivery System in Gene Therapy, by Region, Through 2030
Table 23 : Global Market for HSV-1 Delivery System in Gene Therapy, by Region, Through 2030
Table 24 : Global Market for Lentivirus Delivery System in Gene Therapy, by Region, Through 2030
Table 25 : Global Market for Other Delivery Systems in Gene Therapy, by Region, Through 2030
Table 26 : Global Market for Gene Editing Therapy, by Region, Through 2030
Table 27 : Global Market for Genetic Modification Therapies, by Application, Through 2030
Table 28 : Global Market for Genetic Modification Therapies for Oncology, by Region, Through 2030
Table 29 : Global Market for Genetic Modification Therapies for Rare Diseases, by Region, Through 2030
Table 30 : Global Market for Genetic Modification Therapies for Cardiovascular Diseases, by Region, Through 2030
Table 31 : Global Market for Genetic Modification Therapies for Musculoskeletal Conditions, by Region, Through 2030
Table 32 : Global Market for Genetic Modification Therapies for Hematological Disorders, by Region, Through 2030
Table 33 : Global Market for Genetic Modification Therapies for Other Applications, by Region, Through 2030
Table 34 : Global Market for Genetic Modification Therapies, by Region, Through 2030
Table 35 : North American Market for Genetic Modification Therapies, by Country, Through 2030
Table 36 : North American Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 37 : North American Market for Gene Therapy, by Delivery System, Through 2030
Table 38 : North American Market for RNA Therapy, by RNA Type, Through 2030
Table 39 : North American Market for Genetic Modification Therapies, by Application, Through 2030
Table 40 : U.S. Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 41 : U.S. Market for Gene Therapy, by Delivery System, Through 2030
Table 42 : U.S. Market for Genetic Modification Therapies, by Application, Through 2030
Table 43 : Canadian Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 44 : Canadian Market for Gene Therapy, by Delivery System, Through 2030
Table 45 : Canadian Market for Genetic Modification Therapies, by Application, Through 2030
Table 46 : Mexican Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 47 : Mexican Market for Gene Therapy, by Delivery System, Through 2030
Table 48 : Mexican Market for Genetic Modification Therapies, by Application, Through 2030
Table 49 : European Market for Genetic Modification Therapies, by Country, Through 2030
Table 50 : European Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 51 : European Market for Gene Therapy, by Delivery System, Through 2030
Table 52 : European Market for RNA Therapy, by RNA Type, Through 2030
Table 53 : European Market for Genetic Modification Therapies, by Application, Through 2030
Table 54 : German Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 55 : German Market for Gene Therapy, by Delivery System, Through 2030
Table 56 : German Market for Genetic Modification Therapies, by Application, Through 2030
Table 57 : U.K. Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 58 : U.K. Market for Gene Therapy, by Delivery System, Through 2030
Table 59 : U.K. Market for Genetic Modification Therapies, by Application, Through 2030
Table 60 : French Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 61 : French Market for Gene Therapy, by Delivery System, Through 2030
Table 62 : French Market for Genetic Modification Therapies, by Application, Through 2030
Table 63 : Italian Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 64 : Italian Market for Gene Therapy, by Delivery System, Through 2030
Table 65 : Italian Market for Genetic Modification Therapies, by Application, Through 2030
Table 66 : Spanish Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 67 : Spanish Market for Gene Therapy, by Delivery System, Through 2030
Table 68 : Spanish Market for Genetic Modification Therapies, by Application, Through 2030
Table 69 : Rest of Europe Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 70 : Rest of Europe Market for Gene Therapy, by Delivery System, Through 2030
Table 71 : Rest of Europe Market for Genetic Modification Therapies, by Application, Through 2030
Table 72 : Asia-Pacific Market for Genetic Modification Therapies, by Country, Through 2030
Table 73 : Asia-Pacific Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 74 : Asia-Pacific Market for Gene Therapy, by Delivery System, Through 2030
Table 75 : Asia-Pacific Market for RNA Therapy, by RNA Type, Through 2030
Table 76 : Asia-Pacific Market for Genetic Modification Therapies, by Application, Through 2030
Table 77 : Chinese Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 78 : Chinese Market for Gene Therapy, by Delivery System, Through 2030
Table 79 : Chinese Market for Genetic Modification Therapies, by Application, Through 2030
Table 80 : Japanese Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 81 : Japanese Market for Gene Therapy, by Delivery System, Through 2030
Table 82 : Japanese Market for Genetic Modification Therapies, by Application, Through 2030
Table 83 : South Korean Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 84 : South Korean Market for Gene Therapy, by Delivery System, Through 2030
Table 85 : South Korean Market for Genetic Modification Therapies, by Application, Through 2030
Table 86 : Indian Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 87 : Indian Market for Gene Therapy, by Delivery System, Through 2030
Table 88 : Indian Market for Genetic Modification Therapies, by Application, Through 2030
Table 89 : Australian Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 90 : Australian Market for Gene Therapy, by Delivery System, Through 2030
Table 91 : Australian Market for Genetic Modification Therapies, by Application, Through 2030
Table 92 : Rest of Asia-Pacific Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 93 : Rest of Asia-Pacific Market for Gene Therapy, by Delivery System, Through 2030
Table 94 : Rest of Asia-Pacific Market for Genetic Modification Therapies, by Application, Through 2030
Table 95 : RoW Market for Genetic Modification Therapies, by Subregion, Through 2030
Table 96 : RoW Market for Genetic Modification Therapies, by Platform Technology, Through 2030
Table 97 : RoW Market for Gene Therapy, by Delivery System, Through 2030
Table 98 : RoW Market for RNA Therapy, by RNA Type, Through 2030
Table 99 : RoW Market for Genetic Modification Therapies, by Application, Through 2030
Table 100 : Recent Developments in the Genetic Modification Therapies Market, 20222025
Table 101 : Key Focus Areas in ESG Metrics
Table 102 : ESG Risk Rankings for Genetic Modification Therapies Companies, 2025
Table 103 : Report Information Sources
Table 104 : Abbreviations Used in the Global Genetic Modification Therapies Market
Table 105 : Alnylam Pharmaceuticals Inc.: Company Snapshot
Table 106 : Alnylam Pharmaceuticals Inc.: Financial Performance, FY 2023 and 2024
Table 107 : Alnylam Pharmaceuticals Inc.: Product Portfolio
Table 108 : Alnylam Pharmaceuticals Inc.: News/Key Developments, 20222025
Table 109 : Amgen Inc.: Company Snapshot
Table 110 : Amgen Inc.: Financial Performance, FY 2023 and 2024
Table 111 : Amgen Inc.: Product Portfolio
Table 112 : Amgen Inc.: News/Key Developments, 20222025
Table 113 : Biogen: Company Snapshot
Table 114 : Biogen: Financial Performance, FY 2023 and 2024
Table 115 : Biogen: Product Portfolio
Table 116 : Biogen: News/Recent Developments, 2022
Table 117 : Bristol-Myers Squibb Co.: Company Snapshot
Table 118 : Bristol-Myers Squibb Co.: Financial Performance, FY 2023 and 2024
Table 119 : Bristol-Myers Squibb Co.: Product Portfolio
Table 120 : Bristol-Myers Squibb Co.: News/Key Developments, 2025
Table 121 : F. Hoffmann-La Roche Ltd.: Company Snapshot
Table 122 : F. Hoffmann-La Roche Ltd.: Financial Performance, FY 2023 and 2024
Table 123 : F. Hoffmann-La Roche Ltd.: Product Portfolio
Table 124 : F. Hoffmann-La Roche Ltd.: News/Key Developments, 2023
Table 125 : Ferring: Company Snapshot
Table 126 : Ferring: Financial Performance, FY 2023 and 2024
Table 127 : Ferring: Product Portfolio
Table 128 : Ferring: News/Key Developments, 20232024
Table 129 : Genetix Biotherapeutics Inc.: Company Snapshot
Table 130 : Genetix Biotherapeutics Inc.: Financial Performance, FY 2023 and 2024
Table 131 : Genetix Biotherapeutics Inc.: Product Portfolio
Table 132 : Genetix Biotherapeutics Inc.: News/Key Developments, 2025
Table 133 : Gilead Sciences Inc.: Company Snapshot
Table 134 : Gilead Sciences Inc.: Financial Performance, FY 2023 and 2024
Table 135 : Gilead Sciences Inc.: Product Portfolio
Table 136 : Gilead Sciences Inc.: News/Key Developments, 20232024
Table 137 : Johnson & Johnson: Company Snapshot
Table 138 : Johnson & Johnson: Financial Performance, FY 2023 and 2024
Table 139 : Johnson & Johnson: Product Portfolio
Table 140 : Johnson & Johnson: News/Key Developments, 2024
Table 141 : Lilly USA LLC.: Company Snapshot
Table 142 : Lilly USA LLC.: Financial Performance, FY 2023 and 2024
Table 143 : Lilly USA LLC.: Product Portfolio
Table 144 : Lilly USA LLC.: News/Key Developments, 2025
Table 145 : Novartis AG: Company Snapshot
Table 146 : Novartis AG: Financial Performance, FY 2023 and 2024
Table 147 : Novartis AG: Product Portfolio
Table 148 : Novartis AG: News/Key Developments, 20232025
Table 149 : Novo Nordisk A/S: Company Snapshot
Table 150 : Novo Nordisk A/S: Financial Performance, FY 2023 and 2024
Table 151 : Novo Nordisk A/S: Product Portfolio
Table 152 : Novo Nordisk A/S: News/Key Developments, 20232025
Table 153 : Sanofi: Company Snapshot
Table 154 : Sanofi: Financial Performance, FY 2023 and 2024
Table 155 : Sanofi: Product Portfolio
Table 156 : Sanofi: News/Key Developments, 2024
Table 157 : Sarepta Therapeutics Inc.: Company Snapshot
Table 158 : Sarepta Therapeutics Inc.: Financial Performance, FY 2023 and 2024
Table 159 : Sarepta Therapeutics Inc.: Product Portfolio
Table 160 : Sarepta Therapeutics Inc.: News/Key Developments, 2024
Table 161 : Vertex Pharmaceuticals Inc.: Company Snapshot
Table 162 : Vertex Pharmaceuticals Inc.: Financial Performance, FY 2023 and 2024
Table 163 : Vertex Pharmaceuticals Inc.: Product Portfolio
Table 164 : Vertex Pharmaceuticals Inc.: News/Key Developments, 20232024
Table 165 : List of a Few Emerging Startups
List of Figures
Summary Figure : Global Market Shares for Genetic Modification Therapies, by Region, 2024
Figure 1 : Porter's Five Forces Analysis: Genetic Modification Therapies Market
Figure 2 : Delivery Systems for Gene Therapy and Gene-Modified Cell Therapy
Figure 3 : Global Market Dynamics of Genetic Modification Therapies
Figure 4 : Emerging Technologies in the Genetic Modification Therapies Market
Figure 5 : Market Shares for Genome Editing Clinical Trials, by Technology, 2009-2024
Figure 6 : Market Shares for Genome Editing Clinical Trials, by Diseases, 2009-2024
Figure 7 : Global Market Shares for Genetic Modification Therapies, by Platform Technology, 2024
Figure 8 : Global Market Shares for RNA Therapy, by RNA Type, 2024
Figure 9 : Global Market Shares for Gene Therapy, by Delivery System, 2024
Figure 10 : Global Market Shares for Genetic Modification Therapies, by Application, 2024
Figure 11 : Global Market Shares for Genetic Modification Therapies, by Region, 2024
Figure 12 : North American Market Shares for Genetic Modification Therapies, by Country, 2024
Figure 13 : European Market Shares for Genetic Modification Therapies, by Country, 2024
Figure 14 : Asia-Pacific Market Shares for Genetic Modification Therapies, by Country, 2024
Figure 15 : RoW Market Shares for Genetic Modification Therapies, by Subregion, 2024
Figure 16 : Global Market Shares for RNA Therapies, by Company, 2024
Figure 17 : Global Market Shares for Gene Therapies, by Company, 2024
Figure 18 : Global Market Shares for Gene-Modified Cell Therapies, by Company, 2024
Figure 19 : Alnylam Pharmaceuticals Inc.: Revenue Share, by Country/Region, FY 2024
Figure 20 : Amgen Inc.: Revenue Share, by Business Unit, FY 2024
Figure 21 : Amgen Inc.: Revenue Share, by Country/Region, FY 2024
Figure 22 : Biogen: Revenue Share, by Country/Region, FY 2024
Figure 23 : Bristol-Myers Squibb Co.: Revenue Share, by Business Unit, FY 2024
Figure 24 : Bristol-Myers Squibb Co.: Revenue Share, by Country/Region, FY 2024
Figure 25 : F. Hoffmann-La Roche Ltd.: Revenue Share, by Business Unit, FY 2024
Figure 26 : F. Hoffmann-La Roche Ltd.: Revenue Share, by Country/Region, FY 2024
Figure 27 : Ferring: Revenue Share, by Business Unit, FY 2024
Figure 28 : Ferring: Revenue Share, by Country/Region, FY 2024
Figure 29 : Genetix Biotherapeutics Inc.: Revenue Share, by Business Unit, FY 2024
Figure 30 : Gilead Sciences Inc.: Revenue Share, by Business Unit, FY 2024
Figure 31 : Gilead Sciences Inc.: Revenue Share, by Country/Region, FY 2024
Figure 32 : Johnson & Johnson: Revenue Share, by Business Unit, FY 2024
Figure 33 : Johnson & Johnson: Revenue Share, by Country/Region, FY 2024
Figure 34 : Lilly USA LLC.: Revenue Share, by Country/Region, FY 2024
Figure 35 : Novartis AG: Revenue Share, by Business Unit, FY 2024
Figure 36 : Novartis AG: Revenue Share, by Country/Region, FY 2024
Figure 37 : Novo Nordisk A/S: Revenue Share, by Business Unit, FY 2024
Figure 38 : Novo Nordisk A/S: Revenue Share, by Country/Region, FY 2024
Figure 39 : Sanofi: Revenue Share, by Country/Region, FY 2024
Figure 40 : Sarepta Therapeutics Inc.: Revenue Share, by Country/Region, FY 2024
Figure 41 : Vertex Pharmaceuticals Inc.: Revenue Share, by Business Unit, FY 2024
Figure 42 : Vertex Pharmaceuticals Inc.: Revenue Share, by Country/Region, FY 2024
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