Developing next-generation RNAi therapies.
Dicerna has a growing pipeline of product candidates to address unmet medical needs in rare diseases, chronic liver diseases, cardiovascular disease, and viral liver infectious diseases. Our optimized, subcutaneously administered GalXCTM molecules are designed to potently and selectively silence genes that are implicated in these disorders. In total, we have qualified over thirty disease-associated genes across therapeutic areas where we believe an RNAi-based inhibitor may provide substantial benefit to patients, and have parallel-tracked the discovery and optimization of GalXC conjugate inhibitors against half of these identified targets.
We choose to attack disease targets where we have high confidence that successful silencing of the target gene will provide substantial benefit to patients, and for which we can move rapidly through the drug approval process. Whether focusing on our core area of rare diseases or on serious, life-threatening, chronic diseases, the common threads are the expression of faulty genes in the liver and a substantial need for new treatment options. In making these choices, we are working to improve the lives of patients suffering from potentially debilitating conditions.