RNA interference — the cellular mechanism for silencing gene expression using short interfering RNA — has been translated into five approved commercial therapies by Alnylam Pharmaceuticals: inclisiran (Leqvio) for cardiovascular disease, patisiran (Onpattro) for ATTR amyloidosis, givosiran (Givlaari) for acute hepatic porphyria, lumasiran (Oxlumo) for primary hyperoxaluria, and vutrisiran (Amvuttra) for ATTR amyloidosis. Inclisiran's six-monthly dosing schedule — enabling cholesterol reduction with only two injections per year — represents a fundamental commercial advantage over daily oral statins and creates a new paradigm for chronic disease management. Alnylam's pipeline extends RNAi across metabolic, neurological, and cardiovascular disease.
Topics Covered
• RNAi Technology Mechanism
• Alnylam Approved Product Portfolio
• Inclisiran Cardiovascular Opportunity
• ATTR Amyloidosis Market
• RNAi Pipeline — Metabolic and Neurological
• Competitor RNAi Programs
• GalNAc Delivery Technology
• Partnering and Licensing Landscape
Table of Contents
1. Executive Summary
2. Market Overview
3. RNAi Technology Mechanism
4. Alnylam Approved Product Portfolio
5. Inclisiran Cardiovascular Opportunity
6. ATTR Amyloidosis Market
7. RNAi Pipeline — Metabolic and Neurological
8. Competitor RNAi Programs
9. GalNAc Delivery Technology
10. Partnering and Licensing Landscape
11. Competitive Landscape
12. Regional Market Analysis
13. Strategic Conclusions and Recommendations
14. Appendix
List of Tables
Table 1. Market Overview and Key Data 2025
Table 2. Competitive Landscape 2025
Table 3-8. Topic-Specific Analysis Tables
Table 3. Leading Companies — Portfolio and Strategy Assessment 2025
Table 4. M&A and Partnership Activity 2023-2025
Table 5. Pipeline Assessment 2025
Table 6. Key Risks and Mitigation Strategies
Companies Profiled
Amgen
Gilead Sciences
Regeneron
Biogen
Vertex
BioNTech
Moderna
Genentech
CSL Behring
