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. 2022 Jan 10;342:241–279. doi: 10.1016/j.jconrel.2022.01.008

Table 1.

Marketed RNA products.

Type Product Gene Target Indication Approval Year Company
ASO Vitravene (Fomivirsen) Cytomegalovirus gene (UL123) Cytomegalovirus infection 1998 Ionis Pharmaceuticals
Kynamro (Mipomersen) ApoB-100 Hypercholesterolemia 2013 Ionis Pharmaceuticals
Exondys 51 (Eteplirsen) Dystrophin
(exon 51)
Duchenne muscular dystrophy 2016 Sarepta Therapeutics
Spinraza (Nusinersen) SMN2 Spinal muscular atrophy 2016 Ionis Pharmaceuticals
Tegsedi (inotersen) TTR TTR-mediated amyloidosis 2018 Ionis Pharmaceuticals
Waylivra (Volanesorsen) ApoCIII Familial chylomicronemia syndrome 2019 Ionis Pharmaceuticals/Akcea
Vyondy 53 (Golodirsen) Dystrophin
(exon 53)
Duchenne muscular dystrophy 2019 Sarepta Therapeutics
Viltepso (viltolarsen) Dystrophin
(exon 53)
Duchenne muscular dystrophy 2020 Nippon Shinyaku
AMONDYS 45 (casimersen) Dystrophin
(exon 45)
Duchenne muscular dystrophy 2021 Sarepta Therapeutics
siRNA Onpattro (patisiran) TTR TTR-mediated amyloidosis 2018 Alnylam Pharmaceuticals
Givlaari (givosiran) ALAS1 Acute hepatic porphyrias 2019 Alnylam Pharmaceuticals
OXLUMO (lumasiran) Glycolate oxidase Primary hyperoxaluria type 1 2020 Alnylam Pharmaceuticals
Inclisiran (Leqvio) PCSK9 Hypercholesterolemia 2020 Novartis/ Alnylam Pharmaceuticals
Aptamer Macugen (Pegaptanib) VEGF-165 Age-related macular degeneration and diabetic macular edema 2004 Eyetech Pfizer
mRNA Comirnaty (tozinameran) SARS-CoV-2 spike protein mRNA COVID-19 2020 BioNTech/Pfizer
mRNA-1273 SARS-CoV-2 spike protein mRNA COVID-19 2020 Moderna/NIAID/BARDA

Abbreviation: Apo: Apolipoprotein; SMN2: Survival of motor neuron 2, TTR: Transthyretin, ALAS1: Aminolevulinate synthase 1, PCSK9: Proprotein convertase subtilisin/kexin type 9, VEGF: Vascular endothelial growth factor, COVID-19: Coronavirus disease 2019.