Eplontersen sodium

Modify Date: 2025-08-25 14:27:47

Eplontersen sodium Structure
Eplontersen sodium structure
Common Name Eplontersen sodium
CAS Number 2131025-75-5 Molecular Weight 9046.10
Density N/A Boiling Point N/A
Molecular Formula C296H417N77Na20O156P20S13 Melting Point N/A
MSDS N/A Flash Point N/A

 Use of Eplontersen sodium


Eplontersen sodium is a triantennary N-acetyl galactosamine (GalNAc3-7a)-conjugated antisense oligonucleotide targeting transthyretin (TTR) mRNA to inhibit production of both variant and wild-type TTR protein. Misfolded TTR induces amyloid fibrils formation in the heart and peripheral nerves, leads to amyloid TTR (ATTR) amyloidosis diseases[1][2][3].

 Names

Name Eplontersen sodium

 Eplontersen sodium Biological Activity

Description Eplontersen sodium is a triantennary N-acetyl galactosamine (GalNAc3-7a)-conjugated antisense oligonucleotide targeting transthyretin (TTR) mRNA to inhibit production of both variant and wild-type TTR protein. Misfolded TTR induces amyloid fibrils formation in the heart and peripheral nerves, leads to amyloid TTR (ATTR) amyloidosis diseases[1][2][3].
Related Catalog
References

[1]. Aimo A, et al. RNA-targeting and gene editing therapies for transthyretin amyloidosis. Nat Rev Cardiol. 2022 Mar 23.  

[2]. Diep JK, et al. Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis. Br J Clin Pharmacol. 2022 Jul 22.   

[3]. Prakash Thazha P, et al. Antisense oligonucleotides to hepatitis B virus RNA or transthyretin mRNA conjugated with N-acetylgalactosamine targeting moieties: World Intellectual Property Organization, WO2014179627[P]. 2014-11-06.

 Chemical & Physical Properties

Molecular Formula C296H417N77Na20O156P20S13
Molecular Weight 9046.10
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