DsiRNA: Dicerna’s Therapy Platform
Discovering and delivering innovative RNAi therapies.
Dicerna has assembled a stable of innovative technologies with the goal of realizing the full clinical and commercial potential of RNAi therapies. Our innovative GalXCTM RNAi technology platform fuels a pipeline of promising, precisely targeted therapies designed to overcome many of the challenges of earlier generations of RNAi therapeutics.
Due to the asymmetry of the DsiRNA molecules, an enzyme known as Dicer binds them preferentially in only one orientation, resulting in the preferential incorporation of the Guide Strand into the RNAi machinery (known as RISC, or RNA-Induced Silencing Complex). Before incorporation into RISC, Dicer cleaves the blunt end of the DsiRNA, and the cleaved-off end of the molecule is discarded and degraded. With DsiRNAs, this small cleaved region lacks any function.
The extended region at one end of the DsiRNA-EX molecule is generally on the part of the molecule cleaved off by Dicer, and this region is engineered to provide additional pharmaceutical functionality to the DsiRNA-EX molecules. For example, we can engineer enhanced immunosilencing activity, and can manufacture this region to bind serum albumin, improving the circulating half-life of a DsiRNA-EX molecule.