Table 3 Approved ASO-based therapies for genetic diseases
From: Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs
Trade name (drug name) | Strategy | Approval year | Disease | Vector - modification | Targeted sequence | Company |
|---|---|---|---|---|---|---|
Vitravene™ (Fomivirsen) | ASO | 1998†2002 | Cytomegalovirus retinitis | RNA - Phosphorothioate | CMV IE2 | Ionis Pharmaceuticals |
Kynamro™ (Mipomersen) | ASO | 2013†2018 | Homozygous familial hypercholesterolemia | RNA - Phosphorothoiate/2′O-methoxyethyl | ApoB-100 | Sanofi and Ionis Pharmaceuticals |
Exondys51™ (Eteplirsen) | ASO | 2016 | Duchenne Muscular Dystrophy | RNA - Phosphorodiamidate morpholino | Exon 51 of DMD | Sarepta Therapeutics |
Spinraza™ (Nusinersen) | ASO | 2016 | Spinal Muscular Atrophy | RNA - Phosphorothioate/2′O-methoxyethylated | Exon 7 of SMN2 | Biogen |
Tegsedi™ (Inotersen) | ASO | 2018 | Hereditary Transthyretin-related Amyloidosis | RNA - Phosphorothioate/2′O-methoxyethylated | Transthyretin (TTR) | Ionis Pharmaceuticals |
Vyondys53™ (Golodirsen) | ASO | 2019 | Duchenne Muscular Dystrophy | RNA - Phosphorodiamidate morpholino | Exon 53 of DMD | Sarepta Therapeutics |
Waylivra™ (Volanesorsen) | ASO | 2019 | Adult Familial Chylomicronemia syndrome | RNA - Phosphorothioate/2′O-methoxyethyl | ApoC3 | Ionis Pharmaceuticals |
Viltepso™ (viltolarsen) | SSO | 2020 | Duchenne Muscular Dystrophy | RNA - Phosphorodiamidate morpholino | Exon 53 of DMD | Nippon Shinyaku |
Amondys45™ (casimersen/ SRP-4045) | ASO | 2021 | Duchenne Muscular Dystrophy | RNA -Phosphorodiamidate morpholino | Exon 45 of DMD | Sarepta Therapeutics |
Milasen™ | ASO | 2019 | Batten Disease | RNA -Phosphorothioate/2′-O-methoxyethyl | i6.SA cryptic splice-acceptor site in MFSD8 gene | Boston Children Hospital |
Wainua™ (eplontersen) | ASO | 2023 | Transthyretin-mediated amyloidosis | GalNAC-conjugated antisense oligonucleotide | Transthyretin (TTR) | Ionis Pharmaceuticals + AstraZeneca |
Qalsody™ (tofersen) | ASO | 2023 | SOD1 mutations associated amyotrophic lateral sclerosis (ALS) | RNA - Phosphorothioate/2′O-methoxyethylated | Superoxide dismutase 1 (SOD1) | Ionis Pharmaceuticals + Biogen |
