Dicerna Pharmaceuticals is a biopharmaceutical company focused on the discovery and development of innovative treatments for rare inherited diseases involving the liver and for cancers that are genetically defined. Dicerna’s entire drug pipeline is generated from our proprietary RNA interference (RNAi) technology platform, which is believed to improve upon existing RNAi technologies.
RNAi is a highly potent and specific mechanism for silencing the activity of a targeted disease-driving gene. RNAi offers the potential to go beyond traditional therapeutic modalities, such as small molecules and monoclonal antibodies, and to attack targets such as transcription factors that are expressed exclusively inside cells and lack good small-molecule binding pockets. Dicerna’s discovery approach is based on proprietary double-stranded RNA molecules, called Dicer Substrate siRNA (DsiRNA™), which the Company believes maximizes RNAi potency.
The Company is developing DCR-PH1 for Primary Hyperoxaluria 1 (PH1), a rare, inherited autosomal recessive disorder of metabolism in the liver that results in irreparable damage to the kidneys and DCR-MYC for MYC-related cancers including hepatocellular carcinoma.