Table 1.
Antisense oligonucleotides (ASOs) approved or in clinical development.
| Drug Name | Target Gene | Mode of Action | Therapy Area | Latest Stage of Development | Company |
|---|---|---|---|---|---|
| Nusinersen | SMN2 | Splicing modulation | Neurology | Marketed | Biogen |
| Eteplirsen | DMD | Splicing modulation | Muscular disorders | Marketed | Sarepta Therapeutics |
| Inotersen | TTR | Expression inhibition | Metabolic disorders | Marketed | Akcea Therapeutics |
| Viltolarsen | DMD | Splicing modulation | Muscular disorders | Marketed | NS Pharma |
| Casimersen | DMD | Splicing modulation | Muscular disorders | Marketed | Sarepta Therapeutics |
| Golodirsen | DMD | Splicing modulation | Muscular disorders | Marketed | Sarepta Therapeutics |
| Mipomersen | APOB | Expression inhibition | Metabolic disorders | Marketed | Kastle Therapeutics |
| Volanesorsen | APOC3 | Expression inhibition | Metabolic disorders | Marketed | Akcea Therapeutics |
| Fomivirsen | CMV virus IE2 | Expression inhibition | Infectious disease | Marketed and withdrawn | Novartis |
| Aganirsen | IRS1 | Expression inhibition | Ophthalmology and metabolic disorders | Phase III | Gene Signal |
| Alicaforsen | ICAM1 | Expression inhibition | Gastrointestinal | Phase III | Atlantic Healthcare |
| Eplontersen | TTR | Expression inhibition | Metabolic disorders | Phase III | Akcea Therapeutics |
| ION-363 | FUS | Expression inhibition | Neurology | Phase III | Ionis Pharmaceuticals |
| Olezarsen | APOC3 | Expression inhibition | Cardiovascular and metabolic disorders | Phase III | Akcea Therapeutics |
| Pelacarsen | LPA | Expression inhibition | Cardiovascular and metabolic disorders | Phase III | Novartis |
| Sepofarsen | CEP290 | Splicing modulation | Ophthalmology | Phase III | ProQR Therapeutics |
| Tofersen | SOD1 | Expression inhibition | Neurology | Phase III | Biogen |
| Tominersen | HTT | Expression inhibition | Neurology | Phase III | Roche |
| Trabedersen | TGFB2 | Expression inhibition | Oncology | Phase III | Oncotelic |
| Zilganersen | GFAP | Expression inhibition | Neurology | Phase III | Ionis Pharmaceuticals |
| ASM-8 | CCR3 and CSF2RB | Expression inhibition | Respiratory | Phase II | Pharmaxis |
| Atesidorsen | GHR | Expression inhibition | Hormonal disorders | Phase II | Antisense Therapeutics |
| ATL-1102 | ITGA4 | Expression inhibition | Neurology and muscular disorders | Phase II | Antisense Therapeutics |
| AZD-8233 | PCSK9 | Expression inhibition | Metabolic disorders | Phase II | AstraZeneca |
| AZD-8701 | FOXP3 | Expression inhibition | Oncology | Phase II | AstraZeneca |
| Bepirovirsen | Viral HBV | Expression inhibition | Infectious disease | Phase II | Ionis Pharmaceuticals |
| BIIB-080 | MAPT | Expression inhibition | Neurology | Phase II | Biogen |
| Cepadacursen | PCSK9 | Expression inhibition | Metabolic disorders | Phase II | Civi Biopharma |
| Cimderlirsen | GHR | Expression inhibition | Hormonal disorders | Phase II | Ionis Pharmaceuticals |
| CODA-001 | GJA1 | Expression inhibition | Ophthalmology | Phase II | Eyevance Pharmaceuticals |
| Danvatirsen | STAT3 | Expression inhibition | Oncology | Phase II | AstraZeneca |
| Donidalorsen | KLKB1 | Expression inhibition | Immunology and infectious disease | Phase II | Ionis Pharmaceuticals |
| DYN-101 | DYN2 | Expression inhibition | Muscular disorders | Phase II | Dynacure |
| GTX-102 | UBE2A | Expression inhibition | Neurology | Phase II | GeneTx Biotherapeutics |
| ION-224 | DGAT2 | Expression inhibition | Gastrointestinal | Phase II | Ionis Pharmaceuticals |
| ION-253 | Undisclosed | Expression inhibition | Gastrointestinal | Phase II | Johnson & Johnson |
| ION-464 | SNCA | Expression inhibition | Neurology | Phase II | Ionis Pharmaceuticals |
| ION-541 | ATXN2 | Expression inhibition | Neurology | Phase II | Ionis Pharmaceuticals |
| ION-859 | LRRK2 | Expression inhibition | Neurology | Phase II | Ionis Pharmaceuticals |
| IONIS-AGTLRx | AGT | Expression inhibition | Cardiovascular | Phase II | Ionis Pharmaceuticals |
| IONIS-FB-LRx | CFB | Expression inhibition | Genitourinary system and ophthalmology | Phase II | Ionis Pharmaceuticals |
| IONIS-FXILRx | F11 | Expression inhibition | Cardiovascular, hematology, and genitourinary system | Phase II | Ionis Pharmaceuticals |
| IONIS-GCGRRx | GCGR | Expression inhibition | Metabolic disorders | Phase II | Ionis Pharmaceuticals |
| IONIS-HBVLRx | Viral HBV | Expression inhibition | Infectious disease | Phase II | Ionis Pharmaceuticals |
| IONIS-PKKRx | KLKB1 | Expression inhibition | Neurology | Phase II | Ionis Pharmaceuticals |
| IONISAR-2.5Rx | AR | Expression inhibition | Oncology | Phase II | Ionis Pharmaceuticals |
| IONISENAC-2.5Rx | SCNN1A | Expression inhibition | Respiratory | Phase II | Ionis Pharmaceuticals |
| IONISTMPRSS-6LRx | TMPRSS6 | Expression inhibition | Hematology | Phase II | Ionis Pharmaceuticals |
| ISTH-0036 | TGFB2 | Expression inhibition | Ophthalmology | Phase II | Isarna Therapeutics |
| NS-089 | DMD | Splicing modulation | Muscular disorders | Phase II | Nippon Shinyaku |
| Prexigebersen | GRB2 | Expression inhibition | Oncology | Phase II | Bio-Path Holdings |
| QR-1123 | RHO | Expression inhibition | Ophthalmology | Phase II | ProQR Therapeutics |
| QRX-421a | USH2A | Splicing modulation | Ophthalmology | Phase II | ProQR Therapeutics |
| Renadirsen | DMD | Splicing modulation | Muscular disorders | Phase II | Daiichi Sankyo |
| SRP-5051 | DMD | Splicing modulation | Muscular disorders | Phase II | Sarepta Therapeutics |
| STK-001 | SCN1A | Splicing modulation | Neurology | Phase II | Stoke Therapeutics |
| Vupanorsen | ANGPTL3 | Expression inhibition | Metabolic disorders | Phase II | Pfizer |
| WVE-003 | HTT | Expression inhibition | Neurology | Phase II | Wave Life Sciences |
| WVE-004 | C9orf72 | Expression inhibition | Neurology | Phase II | Wave Life Sciences |
| WVEN-531 | DMD | Splicing modulation | Muscular disorders | Phase II | Wave Life Sciences |