Using natural pathways to silence disease-causing genes.
Dicerna discovers and develops drugs designed to silence or “knockdown” the expression of a targeted gene in a way that is highly selective, specific, precise and reversible.
Our drugs harness the power of the natural RNA interference (RNAi) pathway, a pathway within cells whose purpose is to silence genes. In this biological process, the double-stranded RNA molecules trigger the potent and specific destruction of messenger RNAs (mRNAs) of disease-driving genes. GalXC-based therapies are processed by the Dicer enzyme, which is the natural initiation point for RNAi within the human cell. By using the Dicer enzyme as the entry point into the RNAi, we seek to optimize the activity of the RNAi pathway so that it operates in the most specific and potent fashion.