Table 3.
Oligonucleotide therapeutics in pre-clinical studies
| Name (company) | Targeted gene | Targeting agent | Targeting cell | Disease | Animal model | Reference |
|---|---|---|---|---|---|---|
| GalNAc-Stk25ASO (Sprint Bioscience AB) | STK25 | ASO/ASO-GalNAc conjugate | hepatocyte | NASH/T2D | murine | Cansby et al.316 |
| MST3-targeting ASO | MST3 | ASO | hepatocyte | NAFLD | murine | Caputo et al.318 |
| GalNAc-ASO-ADGRF1 | ADGRF1 | ASO-GalNAc conjugate | hepatocyte | NAFLD | murine | Wu et al.320 |
| GalNAc-ASO-PCSK7 | PCSK7 | ASO-GalNAc conjugate | hepatocyte | NAFLD | murine | Sachan et al.323 |
| AVO101 (Avogadro Pharmaceuticals) | TLR9 | ASO | N/A | NASH | primate | Shepard et al.329 |
| NCST | nicastrin | 2′-O-MOE modified ASO | hepatocyte | NASH | murine | Zhu et al.332 |
| LNP-siMCJ/GalNAc-siMCJ | MCJ | LNP/siRNA-GalNAc conjugate | hepatocyte | NASH | murine | Barbier-Torres et al.290 |
| PAMAM-AEG-1si | AEG-1 | nanoplexes conjugating PAMAM-PEG-Gal | hepatocyte | NASH | murine | Srivastava et al.370 |
| GalNAc-siTAZ | TAZ | GalNAc-siRNA | hepatocyte | NASH | murine | Wang et al.375 |
| mLNP-siHMGB1 | HMGB1 | mannose-modified siRNA loaded LNP | Kupffer cell | NASH | murine | Zhou et al.289 |
| OLX702A (OliX Pharmaceuticals) | N/A | asiRNA-GalNAc conjugate | N/A | NASH | primate | OliX Pharmaceuticals385 |
| anti-miR-132 (Regulus Therapeutics) | miR-132 | 2′-F and 2′-O-Me modified anti-miRNA | hepatocyte | NASH | murine | Papazyan et al.394 |
| RES-010 (Resalis Therapeutics) | miR-22 | LNA modified anti-miRNA | hepatocyte | NASH/NAFLD | murine | Thibonnier et al.406 |
| anti-miR-33 | miR-33 | amido-bridged nucleic acids (AmNAs)420 | hepatocyte | NASH | murine | Miyagawa et al.413 |
| MiR-10b-5p mimic (RosVivo Therapeutics) | miR-10b-5p | miRNA mimic | N/A | NAFLD/T2D/obesity/GI | N/A | RosVivo Therapeutics418 |
STK25, serine/threonine protein kinase 25; MST3, mammalian sterile 20-like 3; ADGRF1, adhesion G-protein-coupled receptor F1; PCSK7, proprotein convertase subtilisin/kexin type 7; TLR9, Toll-like receptor 9; MCJ, methylation-controlled J protein; AEG-1, astrocyte elevated gene 1; TAZ, transcriptional co-activator with PDZ-binding motif; HMGB1, high-mobility group box 1.