Table 2.

Oligonucleotide drugs approved for therapeutical use

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

ASO Antisense Oligonucleotide, i/v intravenous, i/vt intravitreous, s/c subcutaneous, i/m intramuscular, i/th intrathecal, MOE 2’-O-methoxyethyl, 2’-FP 2’-deoxy-2’-fluoro, PSO Phosphorothioate, PMO Phosphorodiamidate morpholino, trGalNAc triantennary N-acetylgalactosamine, * discontinued, ** approved in Europe, DMD Duchenne Muscular Dystrophy, hATTR Hereditary Transthyretin, PCSK9 Proprotein Convertase Subtilisin/Kexin type-9