2. Neuronal Signaling
  3. Transthyretin (TTR)
  4. 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.

For research use only. We do not sell to patients.

DNA, d([2′-O-(2-methoxyethyl)]m5rU-sp-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rG-G-sp-T-sp-T-sp-A-sp-m5C-sp-A-sp-T-sp-G-sp-A-sp-A-sp-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rC), 5′-[26-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[6-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]hexyl]amino]-3-oxopropoxy]methyl]-8,12,19-trioxo-16-oxa-7,13,20-triazahexacos-1-yl hydrogen phosphate], sodium salt

Eplontersen sodium Chemical Structure

CAS No. : 2131025-75-5

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Eplontersen sodium Related Antibodies

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Description

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].

In Vitro

Eplontersen sodium mediates N-acetylgalactosamine moiety targeting the oligonucleotide to cells bearing an asialoglycoprotein receptor[3].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Eplontersen sodium Related Antibodies
In Vivo

Eplontersen sodium (682884) (0.6, 2, 6 mg/kg, s.c., once a week for 3 weeks) inhibits TTR protein expression in a dose-dependent manner in vivo, without affecting normal growth in transgenic C57BL/6 mice expressing human transthyretin (TTR)[3].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Transgenic C57BL/6 mice expressing human transthyretin[3]
Dosage: 0.6-6 mg/kg
Administration: sc, once a week for 3 weeks; performed tail bleeds at various time point; sacrificed mice 72 h following the final administration
Result: Decreased TTR mRNA level to 15%, and reduced plasma TTR protein levels to 21% at 6 mg/kg on day 17 after injection.
Showed no significant effect on plasma ALT and AST level, no inhibition on body weight, organ weight, spleen weight, and kidney weight as well.
Molecular Weight

9046.10

Formula

C296H417N77Na20O156P20S13
CAS No.
Appearance

Solid

Color

White to off-white

SMILES

[Eplontersen (sodium)]
Shipping

Room temperature in continental US; may vary elsewhere.
Storage

-20°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Purity & Documentation
References
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Eplontersen sodium Related Classifications

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Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Eplontersen2131025-75-5Transthyretin (TTR)thyroxine-binding prealbuminN-acetyl galactosamineGalNAc3transthyretinTTRMisfolded TTRamyloid fibrilsheartperipheral nervesageingantisense oligonucleotideasialoglycoprotein receptorhepatitis B virusATTR variant polyneuropathyATTR cardiomyopathyInhibitorinhibitorinhibit

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