Table 1.
Treatments using non-coding RNA are being developed.
| Name of the Product | Name of the Manufacturer | Functionalization | Route of Administration | Indication | Target Delivery Organ | Target mRNA or miRNA | Phase | Clinical Trial Identification Number |
|---|---|---|---|---|---|---|---|---|
| Vupanorsen | Ionis Pharmaceuticals, Pfizer | ASO (Antisense oligonucleotides) (GalNAc-conjugated) | Subcutaneous | Familial Chylomicronaemia Syndrome (FCS) | Liver | ANGPTL3 (angiopoietin-like 3) mRNA | Phase 2 | NCT03371355 |
| Olpasiran | Amgen | siRNA (Small interfering RNA) (GalNAc-conjugated) | Subcutaneous | Elevated Lp(a) | Liver | LPA (Lipoprotein A) mRNA | Phase 2 | NCT04270760 |
| SLN360 | Silence Therapeutics | siRNA (GalNAc-conjugated) | Subcutaneous | Elevated Lp(a) | Liver | LPA mRNA | Phase 1 | NCT04606602 |
| MRG-110 | miRagen, Servier | ASO (LNA) | Intradermal | Neovascularization, wound healing | Vasculature | miR-92a-3p | Phase 1 | NCT03494712 |
| ARO-ANG3 | Arrowhead Pharmaceuticals | siRNA (liver targeted) | Subcutaneous | Mixed dyslipidaemia | Liver | ANGPTL3 mRNA | Phase 2 | NCT04832971 |
| MRG-110 | miRagen, Servier | ASO (LNA) | Intradermal | Neovascularization, wound healing | Vasculature | miR-92a-3p | Phase 1 | NCT03494712 |
| CDR132L | Cardior | ASO (LNA) | Intravenous | Heart failure | Heart | miR-132-3p | Phase 1b | NCT04045405 |
| elacarsen | Lonis Pharmaceuticals | ASO (PS, 2′-MOE, GalNAc-conjugated) | Subcutaneous | Elevated Lp(a) | Liver | LPA mRNA | Phase 3 | NCT04023552 |
| Vutrisiran | Anylam Pharmaceuticals | siRNA (GalNAc-conjugated) | Subcutaneous | TT-amyloid with cardiomyopathy | Liver | Transthyretin mRNA | Phase 3 | NCT03759379 |
| Phase 3 | NCT04153149 | |||||||
| Teprasiran | Quark Pharmaceuticals | siRNA | Intravenous | Acute kidney injury | Kidney | p53 mRNA | Phase 2 | NCT02610283 |
| Phase 3 | NCT03510897 |