Inclisiran RNAi therapeutic targeting PCSK9 Treatment of hypercholesterolemia

被引：0

作者：

Paton, D. M. ^{[1
]}

机构：

[1] Univ Auckland, Dept Pharmacol & Clin Pharmacol, Pharmacol, Auckland, New Zealand

来源：

DRUGS OF THE FUTURE | 2017年 / 42卷 / 06期

关键词：

ALN-PCSsc; Inclisiran; Proprotein convertase subtilisin/kexin type (PCSK9); Hypercholesterolemia; siRNAs; SUBTILISIN/KEXIN TYPE 9; DENSITY-LIPOPROTEIN CHOLESTEROL; LDL CHOLESTEROL; CARDIOVASCULAR RISK; SIRNA DELIVERY; PCSK9; THERAPEUTICS; ASSOCIATION; INTERFERENCE; METAANALYSIS;

D O I：

10.1358/dof.2017.042.06.2623106

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Alnylam has reported the first use in humans of a small interfering RNA (siRNA) to reduce levels of LDL cholesterol (LDL-C). The siRNA, inclisiran, is administered subcutaneously and causes marked reductions in serum levels of both proprotein convertase subtilisin/kexin type 9 (PCSK9) and LDL-C that last for up to 180 days allowing its administration every 6 months. To facilitate the delivery of the siRNA to the liver, Alnylam first developed a sophisticated lipid delivery system then conjugated the siRNA to a triantennary N-acetylgalactosamine that allows the delivery of the conjugate to hepatocytes through an interaction with the asialoglycoprotein receptor. Inclisiran is now the subject of phase II and III trials.

引用

页码：335 / 343

页数：9

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