Early Movers in RNAi

The first few Big Pharma moves into RNA interference were tentative and tightly circumscribed, low risk target- or therapeutic area-specific alliances built around indications where localized delivery wasn’t insurmountable. The fundamental idea of RNAi—using double-stranded RNA oligonucleotides to silence gene expression via destruction of certain mRNA sequences—was promising if early stage. And serious chemistry hurdles needed to be overcome to deliver the potential therapies to their site of action in the body. Even though just about every pharmaceutical company was probably using RNAi as a laboratory tool to validate drug targets, few were eager to place big bets on its therapeutic utility. Big Pharma, which had barely wrapped its collective mind around investing big in more conventional large molecules, hesitated.