Approval of the first-ever rnai therapeutics and its technological development history

Recently, die United States Food and Drug Administration (FDA) and the fcuropean Commission (EC) approved ONPATTRO'" (Patisiran) lipid complex injection, an RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals, Inc., for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. ONPATTRO is die first approved RNAi therapeutics all over the world, indicating the whole development processes have been well established. It also means a novel form of drug molecule comes to beside from bench, following the small molecules and monoclonal antibodies. Based on this milestone achievement, the basic information of die drug and indication, the mechanism and properties of RNAi therapeutics, its complicated development history, as well as die latest advances of delivery and modification technologies, were thoroughly reviewed and discussed in this paper, which provides a rough picture for ONPATTRO and RNAi therapy.