DsiRNA-0401

DsiRNA-0401 is a Dicer-substrate small interfering RNA (DsiRNA), a 27-mer double-stranded RNA designed to be processed by the Dicer enzyme into active siRNA for potent RNA interference (RNAi)-mediated gene silencing. It demonstrates superior potency compared to conventional 21-mer siRNAs in cellular assays, particularly for targets like HPRT, by enhancing uptake and knockdown efficiency in various cell types including airway epithelia when combined with delivery enhancers like PI3K inhibitors. Developed in the context of Dicerna Pharmaceuticals' early DsiRNA platform, it exemplifies the technology's potential for therapeutic applications against undruggable gene targets, though no specific clinical target or indication is associated with this exact candidate.