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Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing

Publisher BCC Research
Published Dec 30, 2025
Length 166 Pages
SKU # WA20694389

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.


Companies Mentioned

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.

Table of Contents

166 Pages
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

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