2025 年核酸药物品牌推荐 技术迭代浪潮下的创新领航者 | 一、市场背景 | 2 | | --- | --- | | 1.1 摘要 | 2 | | 1.2 核酸药物定义 | 2 | | 1.3 市场演变 | 2 | | 二、市场现状 | 3 | | 2.1 市场规模 | 3 | | 2.2 市场供需 | 3 | | 三、市场竞争 | 4 | | 3.1 市场评估维度 | 4 | | 3.2 市场竞争格局 | 5 | | 3.3 十大品牌推荐 | 5 | | 四、发展趋势 | 7 | | 4.1 技术突破驱动：肝外递送与 AI 设计引领创新浪潮 | 7 | | 4.2 商业化加速：License-out 与医保支付共促市场扩容 | 7 | | 4.3 政策与资本双轮驱动：全链条支持构建产业生态 | 7 | | 4.4 适应症拓展：从罕见病向慢性病与肿瘤领域渗透 | 8 | 2025 年核酸药物品牌推荐 一、市场背景 1.1 摘要 2018 年，全球第一款 siRNA 药物 Patisiran 获批，推动了整个行业向广角度治疗领域扩 张。仅在 2018 至 2020 年间，就有 4 款 siRNA 药物和 3 ...

Core Insights - The report from Dongfang Securities highlights the significant commercial value of extrahepatic delivery technology, driven by MNCs' increasing interest and the rapid development of domestic companies in multi-target drugs and extrahepatic delivery platforms [1][2][3] Group 1: Market Dynamics - Frequent transactions in small nucleic acids indicate rising global recognition of domestic drugs, exemplified by recent strategic collaborations involving Shengyin Biotech and major pharmaceutical companies like Eli Lilly and Roche, which include upfront payments and milestone payments totaling up to $12 billion and $15 billion respectively [1] - MNCs are shifting focus from liver-targeted delivery technologies to extrahepatic delivery systems, targeting organs such as fat, muscle, central nervous system, heart, and kidneys, which could address significant unmet clinical needs [2] Group 2: Domestic Innovation - Domestic companies have accumulated substantial experience in chemical modifications and liver-targeted delivery technologies for small nucleic acids, covering high-value areas such as hyperlipidemia, hypertension, hepatitis B, and weight loss [3] - The proactive development of extrahepatic delivery platforms and multi-target small nucleic acid drug platforms positions domestic firms at the forefront globally, attracting MNCs' interest [3] Group 3: Investment Opportunities - Potential investment targets include companies such as Yuyuan Pharmaceutical, Frontier Biotech, Fuyuan Pharmaceutical, Chengdu Xian Dao, and others, which are positioned to benefit from the growing market for small nucleic acid drugs [4]

Investment Rating - The industry investment rating is "Positive" (maintained) [6] Core Insights - The report highlights the significant commercial value of extrahepatic delivery technology for small nucleic acid drugs, which is expanding from liver diseases to multiple disease areas. Domestic companies are rapidly developing multi-target drugs and extrahepatic delivery platforms, which are expected to accelerate the release of potential clinical value [3][8] - The report emphasizes that multinational corporations (MNCs) are intensifying their focus on next-generation delivery platforms, moving from "liver validation" to "multi-organ expansion." This shift is crucial as it addresses unmet clinical needs in various tissues such as fat, muscle, central nervous system, heart, and kidneys [8] - The report indicates that 2026 is a pivotal year for small nucleic acid therapies, with domestic innovative drugs expected to become core assets for transactions. Domestic companies have accumulated rich experience in chemical modifications and liver-targeted delivery technologies, covering high-value areas such as hyperlipidemia, hypertension, hepatitis B, and weight loss [8] Summary by Sections Industry Overview - The report focuses on the pharmaceutical and biotechnology industry in China, specifically on small nucleic acid drugs and their evolving delivery technologies [5][6] Investment Recommendations and Targets - The report lists several investment targets, including: - YK Pharma (688658, Buy) - Hengrui Medicine (600276, Buy) - Xinda Biopharma (01801, Not Rated) - Other companies such as Chengdu XianDao (688222, Not Rated), Reborn Biotech (06938, Not Rated), and others [3]

Group 1 - The core viewpoint is that small nucleic acid drugs are expected to become the third major category of drugs, following small molecules and antibody drugs, due to their advantages such as targeting traditionally non-druggable targets and long-lasting effects [1] - The breakthrough of GalNAc technology in 2014 established a successful paradigm for liver-targeted delivery, significantly activating the development of the small nucleic acid drug industry [1] - The global small nucleic acid drug industry is currently achieving commercial profitability, breakthrough in indications, advancements in extrahepatic delivery, and dual-target breakthroughs [1] Group 2 - From a commercialization perspective, drugs like Amvuttra and Leqvio show significant potential, with global leader Alnylam expected to achieve profitability this year [2] - The years 2025-2026 are critical for indication breakthroughs, with weight loss indications expected to reach proof of concept (POC) and CNS indications likely to begin exploration [2] - Technological advancements in extrahepatic delivery are emerging, with TRiM and AOC delivery methods showing promise, and Avidity's AOC drug Del-zota expected to be commercially available by 2026 [2] Group 3 - Chinese small nucleic acid drug companies are demonstrating global competitiveness, with an accelerated growth in research pipelines [3] - Companies like Bewang and Ruibo have established business development (BD) transactions with multinational corporations (MNCs), showcasing the global competitiveness of Chinese small nucleic acid drugs [3] - The focus of Chinese small nucleic acid drug pipelines is expanding from cardiovascular diseases and hepatitis B to include weight loss and CNS indications, with potential for first-in-class (FIC) and best-in-class (BIC) drugs [3]