Date: 2015-09-02
Type of information: Submission of a clinical trial application
phase: 1
Announcement: submission of a clinical trial application
Company: Dicerna Pharmaceuticals (USA - MA)
Product: DCR-PH1 - synthetic double-stranded (hybridized duplex) ribonucleic acid oligonucleotide specific to hydroxyacid oxidase 1 gene
Action
mechanism: RNAi/oligonucleotide . DCR-PH1 is engineered to address the pathology of primary hyperoxaluria type 1 by targeting and destroying the messenger RNA (mRNA) produced by HAO1, a gene implicated in the pathogenesis of PH1. HAO1 encodes glycolate oxidase, a protein involved in producing oxalate. By reducing oxalate production, this approach seeks to prevent the complications of primary hyperoxaluria type 1. DCR-PH1 incorporates a proprietary, lipid nanoparticle (LNP) technology which allows it to be efficiently delivered to the liver after intravenous (IV) administration. Dicerna obtained rights to this delivery technology by way of a licensing agreement signed in November 2014 with Arbutus Biopharma Corporation, formerly Tekmira Pharmaceuticals Corporation. In preclinical studies, DCR-PH1 has been shown to induce potent and long-term inhibition of HAO1 and to significantly reduce levels of urinary oxalate, while demonstrating long-term efficacy and tolerability in animal models of primary hyperoxaluria type 1.
Disease: primary hyperoxaluria type 1 (PH1)
Therapeutic area: Rare diseases - Genetic diseases - Kidney diseases
Country: USA
Trial details:
Latest
news: * On September 2, 2015, Dicerna Pharmaceuticals, a leading developer of RNA interference (RNAi) therapeutics, announced the submission of an investigational new drug (IND) application to the FDA for DCR-PH1, the company\'s therapeutic candidate for the treatment of primary hyperoxaluria type 1 (PH1).
