摘要 小核酸药物生产成本中，单体占比约 30%-40%，载体占比约 5%，单 体和载体的用量比例约为每克药物需要 40 克单体和 2 克载体。成本控 制的关键在于单体，但商业化阶段对单体纯度要求极高（99%以上）， 与研发阶段（95%-98%）有显著差异。 商业化小核酸药物生产需通过严格的 FDA 质量体系认证，包括质量、生 产、设施设备、实验室检测、仓储及包装六大系统，对硬件（检测设备， 数据完整性）和软件（人员培训，管理体系）均有极高要求，新进入者 需多年准备以符合 GMP 标准。 进入跨国药企（MNC）供应链需通过 FDA 审核及 MNC 的全球质量、 EHS（环保、健康、安全）审计，审计标准由 PSCI 制定，涵盖健康安 全、环境管理、工作条件和商业道德，近年来碳足迹和 eCOVadis 成为 新关注点。 跨国药企对原材料价格敏感度低，更关注产品质量和稳定供应，稳定供 应建立在严格的 EHS 审计基础上。一旦选定供应商，除非发生重大事故， 否则不会轻易更换，因为他们更关注创新而非成本。 小核酸产业链行业大咖分享 20251123 Q&A 小核酸药物生产端整个产业链的价值分布情况如何？ 小核酸药物作为一 ...

Summary of Key Points from the Conference Call Industry Overview - The small nucleic acid drug market has significant potential, with an expected market size of $40 billion by 2035, creating over $10 billion in investment opportunities across the industry chain [1][2] - Major multinational companies like Novartis are expected to play a crucial role in the commercialization of small nucleic acid drugs, while local Chinese suppliers are relatively scarce [1][2] Unique Advantages of Small Nucleic Acid Drugs - Small nucleic acid drugs offer several unique advantages: 1. **Precise Gene Targeting**: They can directly intervene in gene transcription or translation, preventing the production of pathogenic proteins [3] 2. **Broad Target Range**: They can target traditionally undruggable targets, including non-coding RNA and mutated genes, providing new treatment avenues for various diseases [3] 3. **Long-lasting Treatment**: Chemical modifications enhance stability and prolong half-life, making them suitable for chronic disease management [3] 4. **Short Development Cycle**: Rational design allows for rapid drug development, significantly shortening early-stage development time [3] 5. **Strong Platform Capability**: Technologies like NLP and GenAI can adapt to different targets, reducing development costs and increasing success rates [3] 6. **Potential for Combination and Personalized Therapies**: They are positioned to become the third major class of drugs after small molecules and antibodies [3] Current Market Status - As of now, 22 small nucleic acid drugs have been approved, primarily for rare diseases such as SMA, DMD, and ATTR. The market is expanding into chronic and common diseases like hyperlipidemia and hepatitis B, with the overall market size nearing $6 billion [4] Manufacturing Process and China's Role - Small nucleic acids are typically produced via solid-phase synthesis, with China playing a vital role in providing key intermediates at lower costs and faster speeds. China's supply chain management and environmental standards are well-established, making it difficult for global manufacturing to detach from Chinese suppliers [5][6] Investment Opportunities - The small nucleic acid industry chain presents high-value investment opportunities, particularly in CDMO companies that maintain high gross margins due to limited throughput and production capacity. Notable companies include WuXi AppTec, Kelun Pharmaceutical, and others [9] Key Players and Market Attention - Major players in the small nucleic acid market include Novartis, Pfizer, Eli Lilly, Merck, and Novo Nordisk. The year 2025 is particularly significant due to the expected release of critical data and advancements in treating previously untreatable diseases like Alzheimer's and Parkinson's [8][9] Conclusion - The small nucleic acid drug market is poised for substantial growth, driven by unique advantages, expanding applications, and significant investment opportunities, particularly in the context of rising interest from major pharmaceutical companies and advancements in treatment options for various diseases [1][2][9]