Dicerna Pharma Submits IND Application For DCR-PH1 - Quick Facts

(RTTNews) - Dicerna Pharmaceuticals Inc. (DRNA) announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration or FDA for DCR-PH1, the company's therapeutic candidate for the treatment of primary hyperoxaluria type 1 or PH1. PH1 is a severe, rare, inherited disorder of the liver that often results in kidney failure and for which there are no approved therapies.

DCR-PH1 incorporates small interfering RNA (siRNA) formulated in a proprietary lipid nanoparticle (LNP) technology that is being investigated as a system for efficient delivery to the liver after intravenous (IV) administration. Dicerna obtained rights to this delivery technology by way of a licensing agreement with Arbutus Biopharma Corporation, formerly Tekmira Pharmaceuticals Corporation.

PH1 is a rare, inherited genetic disorder of the AGXT gene in the liver which causes excess oxalate production. The AGXT gene encodes for the liver enzyme alanine:glyoxylate-aminotransferase (AGT). The kidneys are unable to eliminate the large amount of oxalate that is produced and the accumulation can result in severe damage to the kidneys and other organs. Currently, there are no approved therapies for the treatment of PH1 in the US and the EU.