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这是首个针对慢性乙型肝炎病毒感染的RNAi治疗RG6346的人类随机研究

Edward J Gane¹, Won Kim², Tien Huey Lim³

¹New Zealand Liver Transplant Unit, Auckland City Hospital and University of Auckland, Auckland, New Zealand.

Journal of hepatology

|July 31, 2023

概括

一种RNA干扰疗法RG6346在I阶段研究中显示出良好的安全性和乙型肝炎表面抗原 (HBsAg) 的显著降低. 这些发现支持其在慢性乙型肝炎病毒 (HBV) 感染中的功能治疗的发展.

科学领域:

肝病学和病毒学.
抗RNA干扰治疗药物抗RNA干扰治疗
传染性疾病传染性疾病

背景情况:

目前的慢性乙型肝炎病毒 (HBV) 疗法具有有限的功能治愈率.
针对HBV的新型治疗对于实现持续的病毒清除至关重要.
RG6346是一种与N-乙-D-银胺 (GalNAc) 结合的RNA干扰剂,向HBV S区域.

研究的目的:

评估RG6346.6的安全性,耐受性,药理动力学和药理动力学.
评估RG6346在降低乙型肝炎表面抗原 (HBsAg) 水平方面的有效性.
为了支持RG6346的临床开发,用于慢性HBV (CHB) 感染.

主要方法:

这是一项首次在人类身上进行的适应性,随机,双盲,I期研究 (NCT03772249).
三组参与者:健康的志愿者 (A组),核化物模拟原始的CHB患者 (B组) 和核化物抑制的CHB患者 (C组).
RG6346 用于各种单剂或多剂量,与安慰剂对照.

主要成果:

RG6346是安全的,耐受性高达4个月,最常见的不良事件是轻微的注射部位反应.
在C组观察到HBsAg的显著降低,91.7%的患者实现了≥1 log10 IU/ml的降低.
在B型肝炎核心相关抗原,HBV RNA,HBV DNA和HBeAg的中度至显著持续减少.

结论:

良好的安全性和药理动力学数据支持RG6346的临床发展.
RG6346显示出作为有限抗病毒疗法的骨干疗法的潜力.
目标是在CHB患者中实现持续的HBsAg损失 (功能治愈).

关键词:

基因表达基因表达基因表达乙型肝炎病毒的感染.免疫清除免疫清除药理学药理学是指药理学的学科.治疗用途治疗用途

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