小核酸:技术突破,打开千亿市场
2025-11-18 01:15

Summary of Key Points from the Conference Call on Small Nucleic Acid Drugs Industry Overview - The global small nucleic acid drug market has reached $6.2 billion and is expected to exceed $40 billion by 2033, driven by new drug approvals, commercialization, and breakthroughs in non-liver-targeted therapies [1][9][14] - Major multinational pharmaceutical companies like Novartis and Roche are actively investing in this sector through business development (BD) and acquisitions, with over 20 small nucleic acid drugs already on the market and more than 300 in clinical stages [1][4][9] Core Insights and Arguments - Key technological breakthroughs include GalNAc delivery technology and chemical modification techniques, which significantly enhance drug stability and efficacy, facilitating successful clinical trials [1][5][8] - ASO (Antisense Oligonucleotides) and siRNA (small interfering RNA) are the two main types of small nucleic acids, with siRNA showing rapid development post-delivery technology breakthroughs [1][6][7] - The small nucleic acid drug development process faces challenges in delivering drugs to non-liver tissues, necessitating the development of new delivery systems like AOC (Antibody-Oligonucleotide Conjugates) [1][8] Market Dynamics - The small nucleic acid target landscape is concentrated on metabolic diseases, hyperlipidemia, and hepatitis B, with significant competition in targets like PCSK9 [3][16] - Since 2018, the total BD transaction value in the small nucleic acid field has reached $45.2 billion, indicating strong interest from multinational corporations [3][17] - Domestic companies in China are rapidly emerging, transitioning from follower strategies to gaining leading advantages in certain targets and technologies [3][20] Company-Specific Developments - Novartis: Acquired Avidity for $12 billion to obtain AOC delivery technology, indicating a strong commitment to the small nucleic acid sector [3][9][15] - Domestic Companies: Companies like Shiyao Group, BoWang Pharmaceutical, and Jingyin Biotechnology are actively pursuing relevant targets and have made significant progress in clinical stages [3][20][21] - Yuekang Pharmaceutical: Leading in the domestic A-share market with a revenue of approximately $383.8 billion in 2024 and a focus on small nucleic acid pipelines [22] - Frontier Biotech: Concentrating on IGA nephropathy with multiple candidates showing first-in-class potential [24] - Fuyuan Pharmaceutical: Focused on generics but also advancing in small nucleic acid innovation, with over 20 patented targets [25] - Shiyao Group: Notable for its rapid progress in the SRA field, with three drugs in clinical trials [26][27] - Rebio: Recently submitted a listing application and has established significant partnerships, indicating strong growth potential [31] Future Trends - The small nucleic acid field is expected to see a surge in new drug approvals and clinical data readouts in the coming years, with a focus on expanding indications beyond liver-targeted therapies [14] - The market is anticipated to grow significantly, with projections indicating a rise from $6.2 billion to over $40 billion by 2033, driven by new approvals and commercialization efforts [9][14] - Companies are increasingly focusing on innovative delivery systems and expanding their target indications to include CNS and other areas [8][14][30] Additional Important Insights - The sales of existing small nucleic acid products are growing rapidly, with drugs like Inclisiran expected to see sales increase from $754 million in 2024 to over $1.2 billion in 2025 [12][13] - The competitive landscape is intensifying, with domestic companies showing strong potential to emerge as leaders in the small nucleic acid market [10][30]