Examples of PS or chimeric antisense oligonucleotides approved or in clinical trials.
| Compound | Chemical structurea | Disease | Status (clinical phase) | Company |
|---|---|---|---|---|
| Fomivirsen (Vitravene®, ISIS-2922)82 | PS | CMV retinitis | Approved | Ionis Pharmaceuticals |
| Mipomersen, (Kynamro®, ISIS-301012)99 | 2′-OMoE chimera | Homozygous familial hypercholesterolemia (HoFH) | Approved | Ionis Pharmaceuticals |
| Nusinersen (Spinraza®)100 | 2′-OMoE chimera | Spinal muscular atrophy (SMA) | Approved | Biogen/Ionis Pharmaceuticals |
| Inotersen (Tegsedi®)101 | 2′-OMoE chimera | Hereditary transthyretin amyloidosis (hATTR) | Approved | Akcea Therapeutics/Ionis Pharmaceuticals |
| Milasen | PS | Batten disease | Approved | Boston Hospital (crowdfunding) |
| Volanesorsen (Waylivra®)102 | 2′-OMoE chimera | Hypertriglycidemia, familial chylomicronemia syndrome and familial partial lipodystrophy | Approved | Ionis Pharmaceuticals |
| Oblimersen (Genasense, Augmerosen, G-3139)103 | PS | Chronic lymphocytic leukemia, malignant melanoma, multiple myeloma, non-small cell lung cancer, acute myeloid leukemia | III | Genta Inc. & Aventis Pharma |
| Trabedersen (AP-12009)104 | PS | Oncology-glioblastoma | III | Antisense Pharma |
| Aganirsen (GS-101)105 | PS | Corneal neovascularization | III | Gene Signal |
| Affinitak (ISIS-3521, LY-900003, aprinocarsen)106 | PS | Non-small cell lung cancer | III | Ionis Pharmaceuticals & Eli Lilly |
| Custirsen (OGX-011, ISIS-112989, TV-1011)59 | 2′-OMoE chimera | Non-small cell lung cancer, prostate and breast cancer | III | OncoGeneX 42 |
| Drisapersen (PRO-051, GSK-2402968) | 2′-OMoE chimera | Duchenne muscular dystrophy | III | Prosensa Therapeutics & GlaxoSmithKline |
| ProMune46 (CPG-7909, PF-3512676)107 | PS | Non-small cell lung cancer | III | Pfizer |
| 1018-ISS108 | PS | Ragweed allergy, hepatitis B, non-Hodgkin's lymphoma and colorectal neoplasms | III | Dynavax Technologies |
a
PS and OMoE refer to the phosphorothioate internucleoside linkage and 2′-O-(2-methoxyethyl) modification of the deoxyribose respectively.