Summary of Expert Call on Small Nucleic Acid Drugs in China Industry Overview - **Industry Focus**: Small nucleic acid drugs in China, specifically RNA drugs, including siRNA and ASO [1][6] - **Market Potential**: Significant opportunities in chronic diseases and liver-related conditions due to lower technological barriers and rapid adoption of siRNA therapies [2][8] Key Insights Market Potential and Applications - **siRNA Advantages**: Demonstrates clear efficacy in metabolic diseases, particularly hyperlipidemia and hepatitis B, compared to existing treatments [2][9] - **ASO Preference**: Generally preferred for cancer, CNS disorders, and genetic diseases where siRNA efficacy is limited [2][7] - **Emerging Opportunities**: Rare diseases present significant opportunities in China due to the large population base, with many companies exploring these indications [8] Delivery Systems - **GalNAc Dominance**: The most popular delivery system in China is liver-targeted GalNAc, originally developed by Alnylam, with domestic companies working on structural modifications for proprietary IP [3][10] - **Extrahepatic Delivery**: Arrowhead leads globally in extrahepatic delivery, while Chinese firms are still adapting existing designs [3][10] Safety and Efficacy - **Safety Profiles**: siRNA drugs generally show a favorable safety profile, but chemical modifications for stability may introduce renal toxicity [2][13] - **Technological Modifications**: ASO drugs require higher dosages and nucleic entry, raising risks of side effects, necessitating further technological advancements [2][13] Manufacturing Challenges - **Cost of Production**: Manufacturing small nucleic acid drugs is more expensive than small molecule drugs but less costly than antibodies, with a reliance on chemical synthesis [14] Oncology Potential - **Limitations in Oncology**: siRNA faces challenges in oncology due to the need for penetration of the tumor microenvironment, while ASO may offer greater potential by targeting telomerase [15] Risks and Challenges - **Industry Risks**: Potential risks include price cuts from GPO programs, intensified competition, lower-than-expected innovative drug prices, slower consumption recovery, stricter regulations, and geopolitical tensions impacting operations [17] Conclusion - The small nucleic acid drug market in China is poised for growth, particularly in chronic diseases and rare conditions, with ongoing advancements in delivery systems and safety profiles. However, challenges in manufacturing, safety, and competition remain critical considerations for investors and companies in this sector.