RNAi developer. This update adds the US label scope and delivery routes for vutrisiran and patisiran; consult the original labels for eligibility.
United StatesFull profile →
Oligonucleotide Therapeutics & Delivery · Companies
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Disease tags identify mentions in recorded facts or pipeline text, not approved indications or a complete pipeline. Platform service providers may have no disease-specific tag.
8 matching companiesShowing 1–8 / 8
RNA-targeted drug developer. Added ION717 and obudanersen targets, diseases and company-reported study phases; these remain investigational.
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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.
United StatesFull profile →
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.
Develops RNA-activation and siRNA medicines. Representative programs include RAG-01 for NMIBC, RAG-18 for DMD and RAG-17 for SOD1-associated ALS. Stages are tied to dated issuer disclosures; this is not a complete pipeline or independent efficacy validation.
Mainland ChinaFull profile →
Wholly owned Nippon Shinyaku subsidiary developing and commercializing rare-disease medicines, including DMD exon-skipping programs.
United StatesFull profile →
Coverage expands from sourced records; this database does not claim all biopharma companies.