小核酸行业：MNC加大BD，慢病+肝外领域潜力无限

Investment Rating - The report suggests a positive investment opportunity in the small nucleic acid drug industry, highlighting its unique advantages and potential for growth [2]. Core Insights - Small nucleic acid drugs possess unique advantages such as broad target accessibility, strong specificity, high development efficiency, and prolonged dosing intervals, which significantly enhance patient compliance and open up potential clinical demand [2]. - Recent technological breakthroughs in small nucleic acid drug design have addressed challenges such as vascular degradation, immune activation, and delivery difficulties, leading to successful market approvals and showcasing excellent efficacy and safety [2]. - The global market for RNAi therapies is projected to grow from $12 million in 2018 to $25 billion by 2030, with a CAGR of 449.2%, indicating a rapid expansion phase for small nucleic acid drugs [2]. - Major multinational corporations (MNCs) are increasingly investing in the small nucleic acid field, with significant collaborations and development projects emerging, particularly in China [2]. - The report emphasizes the limited number of approved small nucleic acid products, suggesting that Chinese companies have the potential to capture a significant share of the global market [2]. Summary by Sections 1. Introduction and Core Technology of Small Nucleic Acid Drugs - Small nucleic acid drugs are defined as short-chain nucleic acids composed of dozens of nucleotides, targeting RNA to regulate protein expression [10]. - The report outlines the unique characteristics of small nucleic acid drugs, including their ability to target a wide range of disease-related genes and their potential to become a third major class of therapeutics after small molecules and antibody drugs [19]. 2. Market Overview of Small Nucleic Acid Drugs - The small nucleic acid drug market is experiencing rapid growth, with the global market size expected to reach $52.47 billion in 2024, reflecting an 18% year-on-year increase [50]. - The report forecasts that the RNAi therapy market in China will grow from approximately $4 million in 2022 to over $300 million by 2025, with a CAGR exceeding 300% [50]. 3. Disease Areas for Small Nucleic Acid Drugs - The report identifies genetic rare diseases as the most common indication for approved small nucleic acid drugs, with 16 out of 22 approved products targeting this area [51][52]. 4. Leading Overseas Companies in Small Nucleic Acid - The report highlights several leading companies in the small nucleic acid space, including Alnylam, Ionis, and Arrowhead, which are at the forefront of innovation and development [2]. 5. Recommended Companies to Watch - The report suggests monitoring companies with proprietary technology platforms and innovative pipeline layouts, such as Yuyuan Pharmaceutical, Rejuve Biotech, and others, as potential investment opportunities [2].