Eplontersen

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Eplontersen

本产品不向个人销售，仅用作科学研究，不用于任何人体实验及非科研性质的动物实验。

CAS NO:

1637600-16-8

包装与价格：

包装	价格(元)
100mg	电议
250mg	电议
500mg	电议

产品介绍

Eplontersen 是一种三触角的、N-乙酰半乳糖胺 (GalNAc3)-缀合的反义寡核苷酸，能够靶向甲状腺素运载蛋白 TTR 的 mRNA，抑制变体和野生型 TTR 产生。错误折叠的 TTR 诱导心脏和周围神经的淀粉样原纤维形成，并介导淀粉样 TTR (ATTR) 蛋白变性疾病。

生物活性

Eplontersen is a triantennary N-acetyl galactosamine (GalNAc₃-7a)-conjugated antisense oligonucleotide targetingtransthyretin (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].

IC₅₀& Target

TTR^[1], asialoglycoprotein receptor^[3]

体外研究
(In Vitro)

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

体内研究
(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.

Clinical Trial

CAS 号

1637600-16-8

运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

Please store the product under the recommended conditions in the Certificate of Analysis.