Shenwan Hongyuan Group Co., Ltd. released a research report stating that from a mechanism perspective, small nucleic acid drugs directly target the mRNA level, enabling them to address "undruggable" targets that traditional medications cannot reach, offering innovative therapeutic approaches for many refractory diseases. Driven by continuous technological advancement, increasing regulatory approvals, and growing clinical validation, the global small nucleic acid drug market is projected to experience accelerated growth, reaching $46.7 billion by 2033 with a compound annual growth rate of 26.1% from 2023. Currently, multiple domestically developed products are in Phase I/II clinical trials, and attention should be paid to upcoming clinical data readouts and potential overseas licensing opportunities.

Key insights from Shenwan Hongyuan Group Co., Ltd.:

**Small Nucleic Acid Drug Technology Matures with Significant Advantages** Compared to traditional small molecule drugs and antibody therapeutics, small nucleic acid drugs demonstrate notable advantages including shorter development cycles, abundant targets, sustained efficacy, and higher development success rates. Mechanistically, these drugs operate directly at the mRNA level, targeting previously "undruggable" sites that conventional therapies cannot address, providing novel treatment paradigms for numerous intractable conditions.

**From Rare Diseases to Common Disorders: Small Nucleic Acid Drugs Enter Rapid Development Phase** Currently, 22 small nucleic acid drugs have received global approval (with 3 withdrawn from market), primarily concentrated in rare disease areas such as spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and transthyretin amyloidosis (ATTR). In recent years, as clinical data emerges for applications in hyperlipidemia, hepatitis B, and hypertension, small nucleic acid drugs are demonstrating a clear transition from rare diseases to chronic and common conditions, with market expansion expected to accelerate.

According to Sullivan data, the global small nucleic acid drug market grew from $2.7 billion in 2019 to $4.6 billion in 2023, representing a compound annual growth rate of 14.3%. With ongoing technological progress, increasing approvals, and expanding clinical validation, the global market is anticipated to accelerate growth, achieving a 26.1% CAGR from 2023 to reach $46.7 billion by 2033.

**Domestic Companies Accelerate Blue Ocean Capture, Focus on Data Readouts and Business Development Progress** Following overseas product commercialization, domestic innovative pharmaceutical companies have accelerated their small nucleic acid drug development in recent years. Unlike overseas giants Alnylam and Ionis, which followed the classic progression from rare to common diseases, domestic small nucleic acid research has chosen to directly enter chronic disease markets, with indications concentrated in major disease categories including cardiovascular conditions, hepatitis B, and oncology.

Multiple domestically developed products are currently in Phase I/II clinical stages. Attention should be focused on subsequent clinical data readouts and potential overseas licensing opportunities. Companies worth monitoring include those with proprietary technology platforms and superior pipelines: Ribo Life Sciences, Sirnaomics, SinoMab BioScience, Yabao Pharmaceutical Group, CSPC Pharmaceutical Group, Betta Pharmaceuticals, and Hengrui Medicine. Notable overseas companies include Alnylam, Ionis, and Arrowhead.

**Risk Warnings:** Research and development progress below expectations, product commercialization underperforming projections, and intensified competition leading to price declines.