靶向HSD17B13的RNA干扰治疗代谢功能障碍相关脂肪性肝炎的1期随机双盲研究

IF 26.8 1区医学 Q1 GASTROENTEROLOGY & HEPATOLOGY

Journal of Hepatology Pub Date : 2025-06-27 DOI:10.1016/j.jhep.2025.05.031

Arun J. Sanyal, Jorg Taubel, Prajakta Badri, Sarah Bond, Nune Makarova, Weizhi Zhao, Swati Duggal, Farshad Kajbaf, Benjamin A. Olenchock, John M. Gansner

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引用次数: 0

摘要

全基因组关联研究已经发现羟基类固醇17- β脱氢酶13基因（HSD17B13）的功能缺失变异与慢性肝病风险降低相关。在这项i期研究中，我们评估了rapirosiran的安全性、耐受性、药代动力学和药效学。rapirosiran是一种n -乙酰半乳糖胺（GalNAc）偶联的小干扰RNA，靶向肝脏表达的HSD17B13 mRNA。

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Phase 1 randomized double-blind study of an RNA interference therapeutic targeting HSD17B13 for metabolic dysfunction–associated steatohepatitis

Genome-wide association studies have identified loss-of-function variants in the hydroxysteroid 17-beta dehydrogenase 13 gene (HSD17B13) associated with reduced risk of chronic liver disease. In this Phase 1 study, we evaluated safety, tolerability, pharmacokinetics, and pharmacodynamics of rapirosiran, an investigational, N-acetylgalactosamine (GalNAc)-conjugated small interfering RNA targeting liver-expressed mRNA for HSD17B13.

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来源期刊

Journal of Hepatology 医学-胃肠肝病学

CiteScore

46.10

自引率

4.30%

发文量

2325

审稿时长

30 days

期刊介绍： The Journal of Hepatology is the official publication of the European Association for the Study of the Liver (EASL). It is dedicated to presenting clinical and basic research in the field of hepatology through original papers, reviews, case reports, and letters to the Editor. The Journal is published in English and may consider supplements that pass an editorial review.