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PUBLISHER: BCC Research | PRODUCT CODE: 1895164
PUBLISHER: BCC Research | PRODUCT CODE: 1895164
Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing
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PAGES: 166 Pages
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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.
The North American market for genetic modification therapies is estimated to grow from $7.3 billion in 2025 to reach $16.4 billion by the end of 2030, at a CAGR of 17.7% from 2025 through 2030.
The European market for genetic modification therapies is estimated to grow from $4.1 billion in 2025 to reach $8.6 billion by the end of 2030, at a CAGR of 16.3% from 2025 through 2030.
Report Scope
The report provides an overview of genetic modification therapies clinical applications: gene therapies, genetically modified cell therapies, RNA therapies and gene editing market and analyzes market trends. It includes global revenue ($ 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.
Product Code: BIO159B
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
Product Code: BIO159B
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, 2022-2025
- 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, 2022-2025
- 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, 2022-2025
- 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, 2023-2024
- 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, 2023-2024
- 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, 2023-2025
- 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, 2023-2025
- 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, 2023-2024
- 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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