2年前FDA批准的第一个RNA药物，治疗hereditary transthyretin-mediated amyloidosi

https://cen.acs.org/pharmaceuticals/drug-discovery/FDA-approves-first-ever-RNAi/96/i33

hereditary transthyretin-mediated amyloidosis 是一种家族病，非常罕见。确诊也不容易，心肺功能，肝脏，消化系统等都会出现衰竭。

The concept behind RNAi therapeutics is simple enough: prevent a protein of interest from being made by intercepting its related messenger RNA. To do that, researchers design complementary “silent interfering” RNA, or siRNA, which are double-stranded molecules running 20 to 25 nucleotides in length.

使用的技术是 gene-editing technology CRISPR

One technical hurdle was figuring out how to safely deliver siRNA to the right tissues. To reach its target in the liver, the siRNA in Onpattro is packaged in a lipid nanoparticle, a vessel that required years of engineering. Still more work went into optimizing the chemistry of the RNA molecules themselves.