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Vutrisiran
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。
包装:1mg
规格:98%
市场价:12789元
分子量:16345

产品介绍
Vutrisiran (ALN-TTRsc02) 是一种肝脏导向的、研究性的、小的干扰核糖核酸药物。 Vutrisiran 可用于转甲状腺素蛋白 (TTR) 介导的淀粉样变性研究。
货号:ajcx38842
CAS:1867157-35-4
分子式:N/A
分子量:16345
溶解度:N/A
纯度:98%
存储:Store at -20°C
库存:现货

Background:

Vutrisiran (ALN-TTRsc02) is a liver-directed, investigational, small interfering ribonucleic acid (siRNA) agent. Vutrisiran can be used for transthyretin (TTR)-mediated amyloidosis research[1].

Vutrisiran (previously ALN-TTRSC02) is a second-generation investigational RNAi therapeutic under development for the study of ATTR amyloidosis. Vutrisiran contains an siRNA that targets a sequence within the TTR mRNA which is conserved across wt and all known TTR variants. However, the Vutrisiran siRNA utilizes enhanced stabilization chemistry (ESC) and is conjugated to a triantennary GalNAc ligand, with the aim of enabling infrequent, subcutaneous (SC) dosing[1].

In non-human primates, single SC doses of Vutrisiran 0.3 and 1 mg/kg achieved mean maximum TTR reductions (nadirs) of 55% and 96%, respectively, with serum TTR reductions persisting beyond 4 months for the 1 mg/kg dose. In the same study, monthly doses of 1 and 3 mg/kg maintained a reduction of TTR levels at 96%, relative to baseline[1].

[1]. Bahru A Habtemariam, et al. Single-Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N-acetylgalactosamine-Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects. Clin Pharmacol Ther. 2021 Feb;109(2):372-382.