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中信建投:全球小核酸药物产业实现多维突破 中国创新药企展现全球竞争力
智通财经网·2025-10-31 02:06

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]