Eplontersen
Modify Date: 2024-01-16 17:24:46
Eplontersen structure
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Common Name | Eplontersen | ||
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| CAS Number | 1637600-16-8 | Molecular Weight | N/A | |
| Density | N/A | Boiling Point | N/A | |
| Molecular Formula | N/A | Melting Point | N/A | |
| MSDS | N/A | Flash Point | N/A | |
Use of EplontersenEplontersen 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]. |
| Name | Eplontersen |
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| Description | Eplontersen 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]. |
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| Related Catalog | |
| Target |
TTR[1], asialoglycoprotein receptor[3] |
| In Vitro | Eplontersen mediates N-acetylgalactosamine moiety targeting the oligonucleotide to cells bearing an asialoglycoprotein receptor[3]. |
| In Vivo | Eplontersen (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]. Animal Model: Transgenic C57BL/6 mice expressing human transthyretin (TTR) (8-week-old)[3] Dosage: 0.6, 2, 6 mg/kg Administration: Subcutaneous injection; 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. |
| References |
| No Any Chemical & Physical Properties |