Dicerna Pharmaceuticals

The core of Dicerna's business is the discovery and development of therapies that use RNAi to silence genes responsible for causing or contributing to a range of diseases. The company's GalXC™ technology platform is engineered to create stable, potent, and highly targeted RNAi therapies. This platform has been instrumental in building a pipeline of drug candidates. Initially, the focus was on liver-targeted treatments for rare conditions, cardiometabolic diseases, viral infections, and chronic liver ailments. Building on this foundation, the GalXC-Plus™ platform extends the application of RNAi to tissues beyond the liver, opening up potential treatments for neurodegenerative diseases and pain. The company's business model involves both advancing its internal pipeline of drug candidates and forming strategic collaborations with major pharmaceutical companies. These partnerships provide non-dilutive funding through upfront payments, milestone achievements, and potential future royalties, while also expanding the reach and application of its RNAi technology. Dicerna's primary clients are these large pharmaceutical partners and, ultimately, patients with serious unmet medical needs across various therapeutic areas.