As a representative class of nucleic acid therapeutics, small nucleic acid drugs boast enormous development potential. Compared with conventional protein-targeted medicines, they feature high specificity, potent efficacy and durable therapeutic effects.

Based on mechanisms of action, small nucleic acid drugs fall into five core categories: siRNA, miRNA, saRNA, antisense oligonucleotides and aptamers. Their applicable indications span oncology, multiple rare diseases including amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), viral disorders, nephropathies and cardiovascular ailments, supporting extensive clinical application prospects. Rare diseases and cancers remain the most mature application segments for small nucleic acid therapeutics.

This landscape map features only those companies that have successfully secured funding.

3SBIO's Envidar® Combined with GEMOX Demonstrates Survival Benefit in Phase III Biliary Tract Cancer Trial — Results Presented at ASCO 2026