RNA 의약품 2.0 : mRNA, saRNA, circRNA, siRNA, ASO, RNA 편집 기술 및 전달 기술 혁신 - 파이프라인, 플랫폼 기술 상황 및 세계 시장 전망(2025-2040년)

Executive Summary / Description

RNA medicines have progressed from proof-of-concept vaccine platforms to programmable therapeutic operating systems for the human body.

This report analyzes the shift from first-generation mRNA vaccines to the RNA Medicine Stack - spanning saRNA, circRNA, self-replicating RNA, ASO/siRNA, ADAR-based RNA editing, tRNA modification platforms, programmable RNA degraders, and exosome-mediated RNA delivery.

RNA therapeutics now target:

• Cancer
• Genetic metabolic disorders
• Cardiovascular disease
• Fibrosis and immunology
• Regenerative & neurodegenerative medicine

Key forces driving a structural boom: Driver and it's Impact

• Manufacturing maturity & scale: Lower COGS, faster clinical PoC
• AI-assisted design of UTRs & codons: Improved translation, half-life, safety
• Organ-tropic LNPs & polymers: Liver -> CNS -> immune -> heart delivery
• Emergence of circRNA durability: Long-acting gene expression without integration
• RNA editing (ADAR): Redosable, reversible gene correction
• Exosome delivery: Low-immunogenic payload distribution
• N-of-1 & personalized RNA constructs: Oncology & rare disease revolution

RNA is evolving into software-like therapeutics capable of precise, reversible, tissue-specific programming of human biology.

Market Outlook

Global RNA therapeutics market expected to grow from ~USD 57B (2024) -> USD 260-300B+ by 2040

Growth vectors: oncology, cardiometabolic RNA vaccines, fibrosis, regenerative medicine, rare diseases, and N-of-1 personalized RNA therapeutics.

Long-duration circRNA and saRNA pipelines suggest new dosing commercial models (quarterly, semi-annual regenerative RNA treatments).

Scientific & Technology Landscape

RNA Modality Taxonomy: Category and Subtype

• Messenger RNA: Conventional mRNA, self-amplifying RNA (saRNA), circular RNA
• Gene Silencing: siRNA, ASO, RNA-targeted small molecules
• RNA Editing: ADAR-guided platforms
• RNA Stabilization/Degradation Control: tRNA repair, RNA-binding modulators
• RNA Delivery: LNPs, polymer nanoparticles, exosomes, viral-free vectors
• Organelle-Targeted RNA: Mitochondrial RNA delivery (emerging)

Key Technology Breakthroughs

• saRNA enabling 50-100x lower dosing
• circRNA providing 2-4 month durability
• UTR engineering libraries for immune-silencing and control
• Cationic LNP evolution -> organ-targeted delivery
• Exosome-RNA delivery for CNS + oncology penetration
• AI-designed codon & secondary structure folding

RNA Manufacturing Evolution

• High-yield cell-free IVT
• Template engineering to minimize dsRNA contamination
• High-capacity modular RNA plants (mRNA foundries)

Table of Content

1. Introduction

• 1.1 Scope & methodology
• 1.2 RNA modality classification
• 1.3 Technology maturity curve

2. Biology & Mechanisms

• 2.1 RNA structure & translation
• 2.2 Innate immune activation & dampening strategies
• 2.3 Codon optimization, UTR logic & RNA half-life control
• 2.4 ADAR editing vs CRISPR gene editing
• 2.5 saRNA vs mRNA vs circRNA

3. RNA Drug Platforms

• 3.1 mRNA vaccines and therapeutics
• 3.2 Self-amplifying RNA (saRNA) tech
• 3.3 Circular RNA (circRNA) therapeutics
• 3.4 siRNA & ASO interference technologies
• 3.5 RNA editing platforms
• 3.6 tRNA repair tech
• 3.7 RNA degraders

4. Delivery Technologies

• 4.1 LNP evolution (cationic -> ionizable -> targeted)
• 4.2 Polymer nanoparticles
• 4.3 Exosome RNA systems
• 4.4 Organ-specific delivery (liver, muscle, CNS, heart)
• 4.5 Mitochondrial RNA delivery

5. Clinical Landscape

• 5.1 Pipeline by stage & mechanism
• 5.2 Key indications
• 5.3 Regulatory paradigm for RNA medicines
• 5.4 Safety learnings (reactogenicity, immune tolerance)

6. Digital, AI & Personalization

• 6.1 AI-optimized RNA design tools
• 6.2 Personalized oncology RNA vaccines
• 6.3 N-of-1 RNA therapy programs
• 6.4 Digital twin dosing algorithms

7. Market Intelligence

• 7.1 Market size forecast 2025-2040
• 7.2 Pricing benchmarks
• 7.3 Revenue acceleration triggers

8. Deals & Investment

• 8.1 Top deals: Moderna, BioNTech, GSK, Arcturus
• 8.2 Strategic partnerships & M&A tracking

9. Strategic Outlook

• 9.1 next-gen winners
• 9.2 bottlenecks & risks
• 9.3 future of "RNA operating systems"

10. Appendix