With the first RNA interference (RNAi) drug (ONPATTRO (patisiran)) on the market, we witness the RNAi therapy field reaching a critical turning point, when further improvements in drug candidate design and delivery pipelines should enable fast delivery of novel life changing treatments to patients. Nevertheless, ignoring parallel development of RNAi dedicated in vitro pharmacological profiling aiming to identify undesirable off-target activity may slow down or halt progress in the RNAi field. Since academic research is currently fueling the RNAi development pipeline with new therapeutic options, the objective of this article is to briefly summarize the basics of RNAi therapy, as well as to discuss how to translate basic research into better understanding of related drug candidate safety profiles early in the process.

中文翻译：

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编辑重点：理解脱靶效应是 RNAi 治疗成功的关键

随着市场上第一种 RNA 干扰 (RNAi) 药物 (ONPATTRO (patisiran)) 的上市，我们见证了 RNAi 治疗领域达到了一个关键的转折点，候选药物设计和输送管道的进一步改进将能够快速提供改变生活的新颖治疗方法给患者。然而，忽视旨在识别不良脱靶活性的 RNAi 专用体外药理学分析的并行开发可能会减慢或停止 RNAi 领域的进展。由于学术研究目前正在通过新的治疗方案推动 RNAi 开发，本文的目的是简要总结 RNAi 治疗的基础知识，并讨论如何尽早将基础研究转化为更好地理解相关候选药物的安全性。正在进行中。