Summary
siRNA and ASOs are emerging as major RNA-based therapeutic platforms, enabling targeted disease treatment by silencing or modulating gene expression.
siRNA holds a commercial advantage in revenue generation and dosing convenience. Marketed siRNA drugs generated approximately $4.2 billion in disclosed global net sales in 2025, driven by AMVUTTRA and LEQVIO.
ASOs retain a strong commercial presence, particularly in neurology and rare genetic diseases. Approved therapies such as SPINRAZA and Sarepta's exon-skipping drugs have established durable revenue streams.
siRNA's late-stage pipeline offers substantial growth potential, but important clinical r...
