A Collection of Approved Antisense Therapeutic Drugs 2024

Antisense oligonucleotides (ASOs) are short, synthetic, single-stranded oligodeoxynucleotides. ASOs can alter RNA and reduce, restore, or modify protein expression through several mechanisms. ASO-mediated therapies target the source of the pathogenesis, thereby having a higher chance of success than therapies targeting downstream pathways. An improved understanding of antisense pharmacology enabled the translation of these therapeutics into the clinic. Several ASO-mediated therapies have now received approval from the US Food and Drug Administration. However, enabling successful ASO therapies in the clinic requires optimizing ASO delivery, target engagement, and safety profiles. 

Synthetic antisense oligonucleotides can modulate RNA function, influencing gene expression levels, exon skipping, and epitranscriptomics. However, understanding the function of various RNAs and the proteins they interact with can take time and effort. 

The ASO technology can potentially change the therapeutic landscape for many neurological and non-neurological conditions soon. Antisense technology promises to deliver therapeutics for treating diseases by targeting RNA. 

Modifications in approved oligonucleotide-based drugs are mainly based on a few sugar and backbone modifications. Modifications use in earlier ASO drugs are 2’-fluoro (2’-F), 2’-O-Methyl (2’-O-Me), phosphorothioate (PS) chemistries and 2’-O-methoxyethyl (2’-O-MOE) RNA and neutral phosphorodiamidate morpholino oligomer (PMO) backbone analogs.

Bio-Synthesis offers a comprehensive suite of technologies to enable your RNA research and help you to answer these critical questions.

Selected References

ASO Insights

Egli, M., Manoharan, M.; Chemistry, structure and function of approved oligonucleotide therapeutics, Nucleic Acids Research, Volume 51, Issue 6, 11 April 2023, Pages 2529–2573, NAR

Rinaldi, C., Wood, M. Antisense oligonucleotides: the next frontier for treatment of neurological disorders. Nat Rev Neurol 14, 9–21 (2018). Nature

Table 1: Approved Antisense Drugs



Approval Date






 Fomivirsen (Vitravene

 5'-GCGTTTGCTCTTCTTCTTGCG-3', Phosphorothioate Oligonucleotide






 Pegaptanib (Macugen)





 Macular Degeneration

 Mipomersen (Kynamro


Phosphorothioate Oligonucleotide. Mipomersen



 Homozygous Familial 


 Defibrotide (Defitelio)

 Deoxyribonucleic acid derivative extracted from mammalian organs.






 Hepatic Veno-

 Occlusive Disease

 Eteplirsen (Exondys 51)

 Eteplirsen is a morpholino antisense oligomer which triggers excision of exon 51 during pre-mRNA splicing of the dystrophin RNA transcript.









 Nusinersen (Spinraza)


 Phosphorothioate Oligonucleotide. Nusinersen








 HEPLISAV-B is a hepatitis B vaccine composed of recombinant hepatitis B virus surface antigen particles (rHBsAg) mixed with a synthetic oligonucleotide containing CpG motifs that stimulate innate immunity through TLR9, containing CpG oligonucleotide as adjuvant!






 Hepatitis B

 Patisiran (Onpattro)








 Inotersen (Tegsedi)

 ASO with sequence TCTTG GTTACATGAA ATCCC, where C is methylated C, and the first and third section (bases 1-5 and 16–20, separated from the middle section by spaces) are MOE-modified.









 Volanesorsen (Waylivra)

This triglyceride-reducing drug is a second-generation 2'-O-methoxyethyl (2'-MOE) chimeric antisense therapeutic oligonucleotide (ASO) targeting the messenger RNA for apolipoprotein C3 (apo-CIII).


3'—A*—G*—mC*—T*—T*—dmCdTdTdGdTdmCdmCdAdGdmCT*—T*—T*—A*—T*—5',  * = 2'-O-(2-methoxyethyl), m = 5-methyl, d = 2'-deoxy









 syndrome (FCS)

 (also known as

 type I  hyperlipo-


 Givosiran (Givlaari)




 Acute Hepatic 


 Golodirsen (Vyondys 53)



5′-{P-[4-({2-[2-(2-hydroxyethoxy)-ethoxy]-ethoxy}-carbonyl)-piperazin-1-yl]-N,N-dimethylphosphonamidate}; Formula: C305H481N138O112P25








 Viltolarsen (Viltepso

 Morpholino oligonucleotide (PMO)








 Lumasiran (Oxlumo


 Lumasiran sodium: C530H669F10N173O320P43S6Na43, Mw 17,286 Da.






 Type 1 (PH1)

 Inclisiran (LeqvioTM)


 Abbreviations: Af = adenine 2'-F ribonucleotide; Cf = cytosine 2'-F ribonucleotide; Gf = guanine 2'-F ribonucleotide; Am = adenine 2'-OMe ribonucleotide; Cm = cytosine 2'-OMe ribonucleotide; Gm = guanine 2'-OMe ribonucleotide; Um = uracil 2'-OMe ribonucleotide; L96 = triantennary GalNAc (N-acetyl-galactosamine). 





 Casimersen (Amondys 45)


 C268H424N124O95P22. Mw 7584.5 daltons.








 Vutrisiran (Amvuttra)

 Medication for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, targeting the mRNA of transthyretin. Vutrisiran sodium: C530H672F9N171Na43O323P43S6; Mw: 17,290 Da. Free acid: C530H715F9N171O323P43S6; Mw: 16,345 Da.





 TTR, liver

 Nedosiran (Rivfloza)

Nedosirna sodium: C662H808F19N231O413P57S6Na57, Mw: 22,238 Da, freely soluble in water.


 DB17635PMC, EplontersenFDA






 Eplontersen (Wainua)









 " Bio-Synthesis provides a full spectrum of high quality custom oligonucleotide modification services including 5'-triphosphate and back-bone modifications, conjugation to fatty acids, biotinylation by direct solid-phase chemical synthesis or enzyme-assisted approaches to obtain artificially modified oligonucleotides, such as BNA antisense oligonucleotidesmRNAs or siRNAs, containing a natural or modified backbone, as well as base, sugar and internucleotide linkages.

Bio-Synthesis also provides biotinylated mRNA and long circular oligonucleotides".