According to Zhishi Consulting's predictions, by 2030, RNAi therapy in common diseases and oncology will account for 54% of the total market size. The Chinese RNAi therapy market is expected to grow from approximately $4 million in 2022 to over $300 million in 2025, with a CAGR exceeding 300%, and is projected to reach approximately $3 billion by 2030.
Recently, small nucleic acid drugs have gained renewed momentum globally, entering a peak period of business development deals. Novartis, AstraZeneca, Biogen, and others are betting heavily on the small nucleic acid field. Chinese pharmaceutical companies including Jecho Biopharmaceuticals and Suzhou Ribo Life Science have also made transaction progress. With relatively few small nucleic acid products currently on the market, Chinese companies are positioned to capture the global blue ocean market.
**Small Nucleic Acid Drugs Possess Unique Advantages**
Small nucleic acid drugs target RNA and regulate protein expression by promoting or inhibiting translation, including ASO (antisense oligonucleotides) and siRNA (small interfering RNA). These drugs offer key advantages including broad target accessibility, strong targeting specificity, high development efficiency, and extended dosing intervals. They enable precise targeted drug delivery while significantly improving chronic disease patient compliance, unlocking potential clinical demand.
**Technical Breakthroughs and Development Wave Create Excellent Investment Opportunities**
Small nucleic acid drug design requires consideration of three major aspects: sequence design, chemical modification, and delivery methods. Recent technical breakthroughs in these areas have successfully addressed challenges including intravascular degradation and clearance, innate immune activation, transmembrane difficulties, endosomal escape, and off-target effects.
Multiple small nucleic acid drugs have successfully obtained market approval, demonstrating good druggability across multiple targets with excellent efficacy and safety profiles. Nusinersen achieved $1.6 billion in sales in 2024, marking it as the world's first spinal muscular atrophy treatment. Vutrisiran generated $970 million in 2024 sales (up 73% year-over-year), having only received approval in 2022. Inclisiran recorded $750 million in 2024 sales (up 112% year-over-year), with H1 2025 revenue reaching $555 million (up 66% year-over-year), maintaining consistently high growth rates.
Future technical breakthroughs in extrahepatic delivery and chronic disease applications will exponentially expand market potential, creating excellent investment opportunities in the small nucleic acid field.
**Multiple New Small Nucleic Acid Drugs Approved, Market Entering Rapid Growth Phase**
According to Zhishi Consulting predictions, the global RNAi therapy market grew from $12 million in 2018 to $362 million in 2020, with a CAGR of 449.2%, and is expected to reach $25 billion by 2030. By 2030, RNAi therapy in common diseases and oncology will represent 54% of total market size.
The Chinese RNAi therapy market is projected to grow from approximately $4 million in 2022 to over $300 million in 2025, with a CAGR exceeding 300%, reaching approximately $3 billion by 2030.
**MNCs Betting on Small Nucleic Acid Field, Chinese R&D Capabilities Highlighted**
Small nucleic acid drugs are experiencing renewed global momentum, entering a business development peak with Novartis, AstraZeneca, Biogen, Eli Lilly, Pfizer, Roche, GSK, and Sanofi all investing heavily in the field.
Transactions involving Chinese pharmaceutical companies include Jecho Biopharmaceuticals' nearly $900 million joint development project with CRISPR, Suzhou Ribo's collaboration with Boehringer Ingelheim totaling over $2 billion, Sanofi's $130 million upfront payment for Greater China rights to an Arrowhead subsidiary Viatris pipeline asset, and Boatpharma's collaboration with Novartis exceeding $5.2 billion.
**Limited Current Small Nucleic Acid Products, Chinese Companies Positioned to Capture Global Blue Ocean**
As of mid-2025, 22 products have received global approval, including 13 ASO drugs, 7 siRNA drugs, and 2 nucleic acid aptamer drugs. Genetic rare diseases represent the most approved indication category, with 16 small nucleic acid drugs targeting genetic rare diseases, 3 targeting ophthalmologic diseases, and 1 targeting cardiovascular disease.
Future small nucleic acid drugs will demonstrate explosive potential across more fields, including long-acting chronic disease medications in metabolism and cardiovascular areas, functional cure combinations for rare diseases especially genetic conditions, and breakthrough therapies for extrahepatic delivery and neurological diseases.
Multiple innovative targets from Chinese companies have entered clinical phases and will continue generating substantial overseas collaboration deals.
**Investment Recommendations**
Focus on companies with proprietary technology platforms, novel pipeline layouts, or overseas expansion prospects. Recommended companies include Yikang Pharmaceutical (688658.SH), Hotgen Biotech (688068.SH), CSPC PHARMA (01093), Cardionovum (002294.SZ), Sinocelltech (688117.SH), BRII - B (02137), ChemPartner (688222.SH), Boatpharma, and Ribo Life Science.
Leading overseas companies include Alnylam (ALNY.US), Ionis (IONS.US), Arrowhead (ARWR.US), and Avidity (RNA).
**Risk Factors**
Innovative drug efficacy below expectations, new drug R&D and approval progress below expectations, competitive landscape deterioration, medical insurance negotiation prices below expectations, drug sales below expectations, policy uncertainties, and global business-related risks.