RNAi therapies to target the cause of disease
We are developing a pipeline of RNAi therapies that aim to restore health by addressing the underlying cause of disease. We strive to deliver life-changing therapies as efficiently as possible to meet the urgent needs of individuals living with rare diseases and common diseases that have a genetic component.
Key core programs
Nedosiran (formerly reffered to as DCR-PHXC) is the only investigational RNAi therapy in development for treatment of all three known types of primary hyperoxaluria (PH).
RG6346 (formerly referred to as DCR-HBVS) is in development with Roche for treatment of chronic hepatitis B virus (HBV) infection. Unlike current therapies that typically provide long-term suppression, RG6346 has the potential to provide a functional cure for patients living with chronic HBV.
DCR-A1AT is in development for treatment of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease.
