DCR-PH1

DCR-PH1 is an investigational RNA interference (RNAi) therapeutic developed for the treatment of primary hyperoxaluria type 1 (PH1), a rare, inherited liver disorder characterized by excessive oxalate production leading to kidney and systemic organ damage. The drug utilizes small interfering RNA (siRNA) formulated in a proprietary lipid nanoparticle delivery system to specifically target and degrade messenger RNA produced by the HAO1 gene, which encodes glycolate oxidase (GO)—an enzyme upstream in the oxalate synthesis pathway. By silencing HAO1 mRNA, DCR-PH1 aims to reduce hepatic oxalate production and mitigate disease pathology. Preclinical studies demonstrated potent inhibition of GO expression and normalization of oxalate levels in animal models. Clinical development was discontinued after early-phase trials due to strategic reasons[3][4][5].