Start with these research questions
How to read the evidence
Do not classify every RNA technology as mRNA. Separate payload, delivery route, tissue, species, and development stage.
Sources, selection, and corrections →Follow siRNA, ASOs, splice modulation, conjugation, delivery, analytics, and manufacturing.
Do not classify every RNA technology as mRNA. Separate payload, delivery route, tissue, species, and development stage.
Sources, selection, and corrections →The first six companies in this field’s editorial order, not a separate selection or capability ranking. The full directory uses the same data and order.
Manage field companies & order →RNAi developer. This update adds the US label scope and delivery routes for vutrisiran and patisiran; consult the original labels for eligibility.
RNA-targeted drug developer. Added ION717 and obudanersen targets, diseases and company-reported study phases; these remain investigational.
RNA medicines developer. On October 27, 2026, Wave reported WVE-007 in Phase 2a and WVE-006 in Phase 1b/2a; its registrational trial remained planned.
Arrowhead develops RNAi therapeutics using its TRiM platform, which combines RNA triggers, targeting ligands, linkers, and chemical modifications. Liver and extrahepatic programs must be assessed by candidate and indication.
Silence Therapeutics develops siRNA medicines through its mRNAi GOLD platform. The name refers to RNA interference and gene silencing; it should not be classified as an mRNA-expression platform.
Dyne describes its FORCE platform as a transferrin-receptor-binding Fab, linker, and therapeutic payload for muscle and CNS delivery. Oligonucleotide conjugation and candidate-specific clinical results should be assessed separately.
Original online dates are retained. Guides use accessible articles or abstracts and do not replace reading the paper.
This review examines RNA species, structure and function, chemical modifications, and delivery systems in the context of therapeutic development. It connects molecular design with stability and distribution and discusses clinical applications available at the time of writing. Its historical approval overview should not be interpreted as a current, exhaustive drug list.
Dalton W Staller; Flobater I Gawargi; Sanjali S Panigrahi; Paras K Mishra; Ram I Mahato
DOI: 10.1016/j.pharmr.2025.100065
Manually selected historical and recent original announcements, not presented as today’s news. Source dates, including historical dates, are retained.
Wave's quarterly update describes planning for a registrational WVE-006 RNA-editing study in AATD and ongoing WVE-007 siRNA Phase 2a work. The company also reports FDA support for its LC-MS assay in the planned WVE-006 program. Regulatory feedback and trial plans do not constitute marketing approval or comparative efficacy evidence.
Original announcement ↗