国泰海通｜医药：小核酸，大时代，靶向治疗新纪元

Core Insights - The small nucleic acid drug industry is entering a new era with a focus on siRNA drugs for common diseases, driven by technological advancements and expanded indications [1][2] - siRNA drugs have transitioned from rare diseases to common diseases, showcasing strong commercial potential in areas like cardiovascular diseases, chronic hepatitis B, weight loss, anticoagulation, and autoimmune diseases [1][3] Group 1: Industry Trends - The small nucleic acid drugs represent the third wave of therapeutics, following small molecules and antibody drugs, characterized by strong target expansion capabilities, robust research extensibility, long-lasting effects, and low resistance [1][2] - The industry is witnessing a surge in business development (BD) transactions, particularly involving multinational corporations (MNCs) leveraging their understanding of indications to acquire pipelines or collaborate on technology platforms [2][3] Group 2: Market Opportunities - The maturation of the liver delivery system and the proven commercial value of small nucleic acid drugs are driving ongoing licensing transactions, as many pharmaceutical companies face patent cliffs and seek new products to mitigate pipeline risks [2][3] - Chinese companies are expected to play a significant role in this space, capitalizing on their engineering advantages and rapid iteration capabilities in chemical synthesis to differentiate their products [2][3] Group 3: Technological Advancements - The current industrialization of siRNA drugs focuses on commercializing common diseases with clear biological mechanisms, where early movers can secure better commercial returns and collaboration opportunities [3] - The efficiency and specificity of the GalNac delivery system present significant opportunities for breakthroughs in new disease areas, particularly those previously challenging to target [3]