Drug / Trade name | Mode of action | Backbone / Modifications | Disease | Target | Route |
---|---|---|---|---|---|
Viltolarse /Viltepso | ASO | PMO | DMD | Dystrophin, exon 53 | i/v |
Fomivirsen / Vitravene | ASO | PSO | Cytomegalovirus retinitis | Cytomegalovirus infection | i/vt |
Mipomersen / Kynamro | ASO | PSO, MOE | Homozygous familial hypercholesterolemia | APO B-100 | s/c |
Iotersen / Tegsedi | ASO | PSO, MOE | hATTR amyloidosis-polyneuropathy | Transthyretin | s/c |
Eteplirsen / Exondys 51 | ASO | PMO | DMD | Dystrophin, exon 51 | i/v |
Golodirsen / Vyondys 53 | ASO | PMO | DMD | Dystrophin, exon 53 | i/v |
Nusinersen / Spinraza | ASO | PSO, MOE | Spinal muscular dystrophy | SMN2 | i/t |
Patisiran / Onpattro | siRNA | 2’-O-Me | hATTR amyloidosis-polyneuropathy | Transthyretin | i/v |
Pegaptanib / Macugen* | Aptamer | 2’-O-Me, 2’-F, PEG | Macular degeneration | VEGF | i/vt |
Lumasiran / Oxlumo | siRNA | 2’-O-Me, 2’-F, PSO | Primary Hyperoxaluria type I | Hydroxy Acid oxidase | s/c |
Casimersen / Amondys 45 | ASO | PMO | DMD | Dystrophin, exon 45 | i/v |
Defibrotide / Defitelio | DNA | ssDNA from pig intestines | Hepatic Veno-Occlusive disease | multiple mechanisms | i/v |
Inclisiran / Leqvio | siRNA | 2’-O-Me, 2’-F, PSO, trGalNAc | Familial Hypercholesterolemia | PCSK9 | i/m |
Volanesorsen / Waylivra** | ASO | PSO, MOE | Familial Chylomicronemia | Apolipoprotein C-III | s/c |
Givosiran / Givlaari | siRNA | 2’-O-Me, 2’-F, trGalNAc | Acute Hepatic Porphyria | 5’-aminolevulinic acid synthase 1 | s/c |