Dicerna™ Pharmaceuticals, Inc., is a biopharmaceutical company using RNA interference, or RNAi, to create medicines that silence disease-causing genes. The Company’s proprietary GalXC™ technology is being applied to develop potent, selective, and safe RNAi therapies to treat diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases. Dicerna aims to extend its capabilities beyond the liver to address a broad range of diseases, focusing on target genes where connections between gene and disease are well documented. Dicerna intends to discover, develop, and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners.
The company’s lead drug, DCR-PHXC, is the only RNAi investigational drug in development for the treatment of all types of primary hyperoxaluria (PH), a family of severe, rare, genetic liver disorders characterized by overproduction of oxalate. In patients with PH, the kidneys are unable to eliminate the large amount of oxalate produced. Oxalate accumulation can result in severe damage to kidneys and other organs resulting in ESRD and mortality.