Table of Content

1. Methodology and Scope
1.1. Research Methodology
1.2. Research Objective and Scope of the Report
2. Definition and Overview
3. Executive Summary
3.1. Snippet by Drug Type
3.2. Snippet by Indication
3.3. Snippet by Region
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Rising Prevalence of Genetic Disorders
4.1.1.2. Advancements in RNA-based therapeutics and drug delivery systems
4.1.2. Restraints
4.1.2.1. High Development Costs and Regulatory Hurdles
4.1.2.2. Limited awareness and adoption in developing regions
4.1.3. Opportunity
4.1.3.1. Increasing adoption of artificial intelligence and computational biology in drug discovery
4.1.4. Impact Analysis
5. Strategic Insights and Industry Outlook
5.1. Market Leaders and Pioneers
5.1.1. Emerging Pioneers and Prominent Players
5.1.2. Established leaders with largest selling Brand
5.1.3. Market leaders with established Product
5.2. CXO Perspectives
5.3. Latest Developments and Breakthroughs
5.4. Case Studies/Ongoing Research
5.5. Regulatory and Reimbursement Landscape
5.5.1. North America
5.5.2. Europe
5.5.3. Asia Pacific
5.5.4. Latin America
5.5.5. Middle East & Africa
5.6. Porter’s Five Force Analysis
5.7. Pipeline Analysis
5.8. Supply Chain Analysis
5.9. Patent Analysis
5.10. SWOT Analysis
5.11. Unmet Needs and Gaps
5.12. Recommended Strategies for Market Entry and Expansion
5.13. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
5.14. Pricing Analysis and Price Dynamics
5.15. Key Opinion Leaders
6. By Drug Type
6.1. Introduction
6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
6.1.2. Market Attractiveness Index, By Drug Type
6.2. Eteplirsen*
6.2.1. Introduction
6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
6.3. Fomivirsen
6.4. Mipomersen
6.5. Defibrotide
6.6. Volanesorsen
6.7. Inclisiran
6.8. Givosiran
6.9. Nusinersen
6.10. Lumasiran
6.11. Others
7. By Indication
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
7.1.2. Market Attractiveness Index, By Indication
7.2. Adult*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Pediatric
8. By Region
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
8.1.2. Market Attractiveness Index, By Region
8.2. North America
8.2.1. Introduction
8.2.2. Key Region-Specific Dynamics
8.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
8.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
8.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
8.2.5.1. U.S.
8.2.5.2. Canada
8.3. Europe
8.3.1. Introduction
8.3.2. Key Region-Specific Dynamics
8.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
8.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
8.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
8.3.5.1. Germany
8.3.5.2. U.K.
8.3.5.3. France
8.3.5.4. Spain
8.3.5.5. Italy
8.3.5.6. Rest of Europe
8.4. Latin America
8.4.1. Introduction
8.4.2. Key Region-Specific Dynamics
8.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
8.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
8.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
8.4.5.1. Mexico
8.4.5.2. Brazil
8.4.5.3. Argentina
8.4.5.4. Rest of South America
8.5. Asia-Pacific
8.5.1. Introduction
8.5.2. Key Region-Specific Dynamics
8.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
8.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
8.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
8.5.5.1. China
8.5.5.2. India
8.5.5.3. Japan
8.5.5.4. South Korea
8.5.5.5. Rest of Asia-Pacific
8.6. Middle East and Africa
8.6.1. Introduction
8.6.2. Key Region-Specific Dynamics
8.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
8.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
9. Competitive Landscape
9.1. Competitive Scenario
9.2. Market Positioning/Share Analysis
9.3. Mergers and Acquisitions Analysis
10. Company Profiles
Key Market Players
10.1. Ionis Pharmaceuticals, Inc*
10.1.1. Company Overview
10.1.2. Product Portfolio
10.1.2.1. Product Description
10.1.2.2. Product Key Performance Indicators (KPIs)
10.1.2.3. Historic and Forecasted Product Sales
10.1.2.4. Product Sales Volume
10.1.3. Financial Overview
10.1.3.1. Company Revenue’s
10.1.3.2. Geographical Revenue Shares
10.1.3.3. Revenue Forecasts
10.1.4. Key Developments
10.1.4.1. Mergers & Acquisitions
10.1.4.2. Key Product Development Activities
10.1.4.3. Regulatory Approvals etc.
10.1.5. SWOT Analysis
10.2. Sarepta Therapeutics
10.3. Alnylam Pharmaceuticals, Inc.
10.4. Percheron Therapeutics
10.5. Isarna Therapeutics GmbH.
10.6. Novartis AG
10.7. Jazz Pharmaceuticals
10.8. Akcea Pharma
10.9. Genzyme
10.10. Biogen
* Similar data will be provided for each market player.
Emerging Market Players
10.11. Wave Life Sciences Ltd*
10.11.1. Pipeline Products Description
10.11.2. Product Key Performance Indicators (KPIs)
10.11.3. Key Activities
10.11.4. Market Entry Timelines
10.11.5. Product Penetration Rate
10.11.6. Sales Estimation and Projections
10.12. Cenna Biosciences, Inc.
10.13. Amarna Therapeutics B.V.
10.14. Idera Pharmaceuticals, Inc
* Similar data will be provided for each market player.
LIST NOT EXHAUSTIVE
12. Appendix
12.1 About Us and Services
12.2 Contact Us