Table 2 Oligonucleotide drugs approved for therapeutical use

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