Pipeline

Pipeline

Developing next-generation RNAi therapies.

Dicerna has a growing pipeline of product candidates to address unmet medical needs in diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases. Our optimized, subcutaneously administered GalXCTM molecules are designed to potently and selectively silence genes that are implicated in these disorders.

Key Clinical Programs

Primary Hyperoxaluria (PH)

Dicerna is developing DCR-PHXC, a subcutaneously delivered GalXCTM candidate for the treatment of patients with all three genetic forms of primary hyperoxaluria (PH), a family of severe, rare, genetic liver disorders characterized by overproduction of oxalate, a natural chemical in the body that is normally eliminated as waste through the kidneys. DCR-PHXC is being tested in a variety of PHYOX trials:

  • PHYOX1 (DCR-PHXC-101): Single ascending dose study of DCR-PHXC in healthy volunteers (HVs) and patients with PH 1 and 2
  • PHYOX2 (DCR-PHXC-201): Double-blind, randomized, placebo-controlled, pivotal trial (2:1 randomization) in ~36 patients with PH1 and PH2
  • PHYOX3 (DCR-PHXC-301): Long-term, multi-dose, open-label, rolloverextension trial allowing readouts of multi-dose data and long-term access to DCR-PHXC for patients who previously participated in PHYOX study

For more information about our PHYOX trials, please visit PHYOXTrials.com.

 

Chronic Hepatitis B Virus (HBV) Infection 

Dicerna’s DCR-HBVS is a GalXC RNAi platform-based product candidate for the treatment of chronic hepatitis B virus (HBV) infection. Current therapies for HBV rarely lead to a long-term immunological cure as measured by the clearance of HBV surface antigen (HBsAg) and sustained HBV deoxyribonucleic acid (DNA) suppression in patient plasma or blood. DCR-HBVS is currently being tested in the DCR-HBVS-101 clinical trial, a randomized, placebo-controlled study designed to evaluate the safety and tolerability of DCR-HBVS in HVs and in patients with non-cirrhotic chronic HBV.

For more information about our DCR-HBVS clinical trial, please visit clinicaltrials.gov, identifier: NCT03772249.

 

An Undisclosed Rare Disease Involving the Liver

We are developing a GalXC-based therapeutic, targeting a liver-expressed gene involved in a serious rare disease. For competitive reasons, we have not yet publicly disclosed the target gene or disease. The disease is a genetic disorder, where mutations in the disease gene lead to the production of an abnormal protein. The protein causes progressive liver damage and fibrosis, in some cases leading to cirrhosis and liver failure, and we believe that silencing of the disease gene will prevent production of the abnormal protein and thereby slow or stop progression of the liver fibrosis. This program is currently in Investigational New Drug (IND)-enabling studies. Submissions of regulatory filings are planned in the second quarter of 2019.