Eplontersen (sodium)

Eplontersen sodium the sodium salt form of Eplontersen (HY-148089) . 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].

Product Specifications

CAS Number

[2131025-75-5]

UNSPSC

12352208

Target

Transthyretin (TTR)

Type

Oligonucleotides

Related Pathways

Neuronal Signaling

Applications

Neuroscience-Neuromodulation

Field of Research

Neurological Disease

Assay Protocol

https://www.medchemexpress.com/eplontersen-sodium.html

Purity

96.92

Solubility

10 mM in H2O

Smiles

[Eplontersen(sodium)]

Molecular Formula

C296H417N77Na20O156P20S13

Molecular Weight

8606.5 (free acid)

References & Citations

[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.