Harnessing RNAi to fight disease
At Dicerna, we use RNA interference, or RNAi, to create medicines that silence or turn off the genes that cause disease. Using our GalXC technology platform, we develop potent and patient-friendly RNAi therapies – in-house and with our partners – for both rare diseases and common diseases that have a genetic component.
Our GalXC™ pipeline and platform
Our proprietary GalXC technology opens a world of possibilities for creating targeted and potent RNAi therapies. These therapies are thought to be more convenient for patients because they can be delivered under the skin and have a long duration of action.
We currently have three drug candidates in clinical trials:
- Nedosiran (DCR-PHXC): all three known types of primary hyperoxaluria (PH)
- DCR-HBVS (RG6346): hepatitis B virus (HBV) infection
- DCR-A1AT: alpha-1 antitrypsin deficiency-associated liver disease
Our commitment to patients
We believe all patients deserve safe and effective medicines that can improve the quality and duration of their lives. Our goal is to understand and ease the unique disease-related burdens of patients and their caregivers as we create new medicines with the potential to restore their health.