Running interference: lumasiran and other RNA interference therapeutics for kidney diseases

Abstract

RNA interference (RNAi) is a biological process that directly modulates gene activity. RNAi-based therapeutic agents, which bind specifically to target messenger RNA (mRNA) sequences, allow downstream silencing of the respective target protein. In recent years, RNAi-based agents have become a major novel tool for the treatment of several rare diseases. In 2018, the first RNAi drug approved by the FDA in the United States was patisiran for hereditary transthyretin (TTR) amyloidosis, followed by the approval of givosiran for acute hepatic porphyria, and lumasiran for primary hyperoxaluria.