Abstract

A decade ago , drug firms were becoming disenchanted with RNA interference (RNAi) technology, which uses small interfering RNA (siRNA) molecules to turn down the expression of disease-causing genes. But the RNAi company Alnylam Pharmaceuticals has launched three commercial siRNA therapies in the past 3 years, and companies are investing in the genetic technology once again. GlaxoSmithKline has signed an agreement with the RNAi company Arrowhead Pharmaceuticals to develop an siRNA therapy for nonalcoholic steatohepatitis (NASH), a form of fatty liver disease. The therapy, currently in a Phase 1/2 clinical trial, targets a gene, HSD17B13 , that encodes an enzyme linked to inflammation in NASH. GSK will pay Arrowhead $120 million initially and up to $910 million in milestone payments. And Novo Nordisk will acquire the RNAi company Dicerna Pharmaceuticals for $3.3 billion . The two firms began collaborating in 2019 on siRNAs that target more than 30 genes in

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