【siRNA药物行业深度：黎明已至，国产争先】 siRNA药物机制优势突出，递送为核心技术 相较于传统小分子和生物药，siRNA药物为代表的核酸疗法可精准沉默致病蛋白，高效降解且作用时间长，目前已 成为新药开发热门方向。 技术层面主要包括修饰和递送，其中递送为核心技术，肝外递送是目前的突破重心，技术路径包括LNP、抗体、 肽类以及VLP递送，各有优劣，其中AOC发展稍快。 【siRNA药物行业深度：黎明已至，国产争先】 siRNA药物机制优势突出，递送为核心技术 相较于传统小分子和生物药，siRNA药物为代表的核酸疗法可精准沉默致病蛋白，高效降解且作用时间长，目前已 成为新药开发热门方向。 技术层面主要包括修饰和递送，其中递送为核心技术，肝外递送是目前的突破重心，技术路径包括LNP、抗体、 肽类以及VLP递送，各有优劣，其中AOC发展稍快。 siRNA行业先驱Alnylam开发的GalNAc肝内递送凭借递送效率极高、特异性强以及作用持久等优势，已成为肝递送 的"标准答案"，其他海外巨头如Arrowhead和Dicerna等针对PCSK9、AGT、Lp(a)以及ApoC3等心血管常见靶点的 siRNA药物已进入 ...

siRNA行业先驱Alnylam开发的GalNAc肝内递送凭借递送效率极高、特异性强以及作用持久等优势，已 成为肝递送的"标准答案"，其他海外巨头如Arrowhead和Dicerna等针对PCSK9、AGT、Lp(a)以及ApoC3 等心血管常见靶点的siRNA药物已进入临床后期或商业化的收获期，而针对肌肉、神经系统以及眼部等 肝外器官目前仍处于研发早期阶段，目前Alnylam的C16递送平台已在CNS领域、Arrowhead的TRiM平 台在脂肪组织领域初步展现潜力，未来新靶点的发现与肝外递送平台将成为竞争的核心要点。 智通财经APP获悉，中邮证券发布研报称，siRNA药物可精准沉默致病蛋白，优势显著，递送系统为核 心技术。海外巨头基于GalNAc肝递送平台，针对心血管靶点的药物已进入收获期。国内企业百花齐 放，成为全球重要参与者，与MNC合作频发。 中邮证券主要观点如下： siRNA药物机制优势突出，递送为核心技术 相较于传统小分子和生物药，siRNA药物为代表的核酸疗法可精准沉默致病蛋白，高效降解且作用时间 长，目前已成为新药开发热门方向。技术层面主要包括修饰和递送，其中递送为核心技术，肝外递送是 目前 ...

Summary of siRNA Industry Insights Industry Overview - The siRNA drug market is projected to reach $50.3 billion by 2040, indicating a significant growth opportunity in the nucleic acid therapy sector, which is gaining traction as a new drug development direction [1][1][1] Key Insights - siRNA drugs offer high efficiency, low toxicity, and long-lasting effects compared to traditional small molecules and biologics, making them a popular choice in new drug development [1][1][1] - The GalNAc liver delivery platform has matured, showing high delivery efficiency and specificity, becoming the standard for liver-targeted siRNA drugs. Notable targets include PCSK9, AGT, Lp(a), and ApoC3, which have demonstrated therapeutic potential [1][1][1] - There is potential for breakthroughs in extrahepatic delivery systems, with early-stage developments in areas such as muscle, CNS, and ocular applications. Alnylam's C16 delivery platform is showing promise in Alzheimer's disease, while Arrowhead and Wave Life Sciences are exploring fat tissue targets [1][1][1] Market Dynamics - Domestic siRNA companies are positioned to capitalize on the demand for new technologies as traditional chronic disease drugs approach the end of their sales lifecycle. Collaborations with multinational corporations (MNCs) are expected to increase, particularly in liver delivery systems [2][2][2] - The industry outlook is optimistic, with domestic siRNA drugs likely to secure partnerships in popular targets like PCSK9 and Lp(a). New demands in weight loss and CNS delivery are further opening up the market as international giants make breakthroughs [2][2][2] Relevant Companies - A-share listed companies: Yuyuan Pharmaceutical, Frontier Biotech, Fuyuan Pharmaceutical, Bibetech, Sunshine Nuohuo, Chengdu Xian Dao - Hong Kong listed companies: Reebio, and upcoming IPOs such as Jingyin Biotech and Bowe Pharmaceutical - Upstream industry chain participants include WuXi AppTec, Lianhua Technology, Aorite, Lanxiao Technology, Nawei Microbiology, and Jiuzhou Pharmaceutical [2][2][2]