Dicerna Pharmaceuticals, Inc., a leading developer of RNA interference (RNAi) therapeutics, today announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for DCR-PH1, the company's therapeutic candidate for the treatment of primary hyperoxaluria type 1 (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.
“The IND submission is an important milestone for the DCR-PH1 program, as it takes us one step closer to bringing this novel therapy to patients with primary hyperoxaluria type 1,” said Pankaj Bhargava, M.D., chief medical officer at Dicerna. “We look forward to working with the FDA during the review process in order to rapidly initiate clinical development of DCR-PH1.”
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.