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Table 1 Approved RNA-based drugs in market at present

From: Research progress on non-protein-targeted drugs for cancer therapy

Class

Drug name

Target

Disease

Year of approval

ASO

Nusinersen

Exon 7 of SMN2

SMA

2016

Eteplirsen

Exon 51 of DMD

DMD

2016

Inotersen

TTR mRNA

FAP

2018

Volanesorsen

ApoC3

FCS

2019

Golodirsen

Exon 53 of DMD

DMD

2019

Vitolarsen

Exon 53 of DMD

DMD

2020

Casimersen

Exon 45 of DMD

DMD

2021

siRNA

Patisiran

TTR mRNA

FAP

2018

Givosiran

ALAS1 mRNA

AHP

2020

Lumasiran

HAO1 mRNA

PH1

2020

Inclisiran

PCSK9

Hypercholesterolaemia

2020

mRNA

BNT162b2

Spike protein

SARA-CoV-2

2020

mRNA-1273

Spike protein

SARA-CoV-2

2020

  1. SMN2 survival pf motor neuron-2, SMA spinal muscular atrophy, TTR transthyretin, FAP familial amyloid polyneuropathy, ApoC3 apolipoprotein C3, FCS Familial chylomicronemia syndrome, DMD duchene muscular dystrophy, ALAS1 aminolevulinate synthase 1, AHP Acute hepatic porphyria, HAO1 Hydroxyacid oxidase 1, PH1 Primary hyperoxaluria type 1, PCSK9 proprotein convertase subtilisin/kexin 9, SARS-CoV-2 syndrome coronavirus 2