Core Viewpoint - The article discusses the revolutionary shift in small nucleic acid drugs, highlighting their potential to transition from symptomatic treatment to addressing root causes, marking a significant advancement in the pharmaceutical industry [4]. Group 1: Small Nucleic Acid Drug Technology - Small nucleic acid drugs (siRNA, ASO, etc.) are considered the "third wave" of drug development, following small molecules and antibody drugs [4]. - These drugs are not limited by their molecular structure and can target a broader range of disease pathways, significantly increasing the probability of successful drug development [4]. - Current advancements in delivery technologies and chemical modifications have led to extended half-lives for these drugs, improving patient compliance in chronic disease management [4]. Group 2: Market Growth and Commercialization - The global small nucleic acid drug market is projected to reach $20.6 billion by 2029 and $54.9 billion by 2034, with a compound annual growth rate (CAGR) of 21.6% [5]. - Major players like Alnylam, Ionis, and Arrowhead are leading the market, with multinational corporations (MNCs) such as Novartis and Roche investing heavily in this sector [5]. - Alnylam's core product, Amvuttra, is expected to exceed $2 billion in sales by 2025, indicating strong market performance [6]. Group 3: Technological Breakthroughs - Arrowhead has developed RNAi therapies targeting various diseases through its TRiM platform, expanding the application of small nucleic acid drugs beyond liver diseases [7]. - Avidity Biosciences has made significant progress in muscle tissue delivery using antibody-oligonucleotide conjugates (AOC) technology, indicating a broader therapeutic potential [7]. Group 4: China's Small Nucleic Acid Drug Industry - The Chinese small nucleic acid drug industry is experiencing a qualitative leap, overcoming delivery patent barriers and demonstrating global competitiveness in target selection and molecular design [8]. - A complete industrial chain has emerged in China, with domestic raw materials achieving localization, thus reducing R&D costs [8]. - Significant business development (BD) transactions have occurred, including a record $5 billion deal between Wobang Pharmaceutical and Novartis, showcasing the global recognition of Chinese innovation [8].

Investment Rating - The report maintains an "Overweight" rating for the small nucleic acid drug industry [1] Core Insights - The small nucleic acid drug sector is experiencing a paradigm shift from "symptomatic treatment" to "root cause treatment," marking it as the "third wave of pharmaceutical innovation" following small molecules and antibody drugs. This innovation is driven by the ability of RNA drugs to overcome traditional drug development limitations, significantly expanding target ranges and improving drug development efficiency [4][5] - The global small nucleic acid drug market is projected to reach USD 20.6 billion by 2029 and USD 54.9 billion by 2034, with a compound annual growth rate (CAGR) of 21.6%, indicating high growth potential [5] - The industry is entering a commercialization phase, with key players like Alnylam, Ionis, and Arrowhead leading the market, supported by significant investments from multinational corporations (MNCs) [5] Summary by Sections Market Growth and Trends - The small nucleic acid drug market is expected to see rapid expansion, particularly in the fields of rare diseases and major diseases such as cardiovascular and metabolic disorders [5] - Alnylam's core product, Amvuttra, is projected to exceed USD 2 billion in sales by 2025, contributing to the company's profitability [5] - Domestic companies in China are overcoming delivery technology barriers and demonstrating global competitiveness in target selection and molecular design [7] Technological Advancements - Breakthroughs in delivery technologies and chemical modifications are enhancing the efficacy and half-life of small nucleic acid drugs, improving patient compliance in chronic disease management [4][5] - Arrowhead's TRiM platform has successfully developed RNAi therapies for various diseases, expanding the application of small nucleic acid drugs beyond liver diseases [5] Industry Ecosystem - The domestic small nucleic acid drug industry has established a complete industrial chain, with upstream raw materials achieving localization, thus reducing research and development costs [9] - Significant business development (BD) transactions have occurred, with notable collaborations between Chinese companies and global pharmaceutical giants, marking a historic high for the industry [9]