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news.Dicerna Pharmaceuticals has submitted an investigational new drug (IND) application to the US Food and Drug Administration (FDA) for its DCR-PH1 therapeutic candidate.
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DCR-PH1 can be used to treat primary hyperoxaluria type 1 (PH1), which is a rare, inherited disorder of the liver that leads to kidney failure.
It features small interfering RNA (siRNA) formulated in a lipid nanoparticle (LNP) technology. It is being examined as a system for efficient delivery to the liver after intravenous (IV) administration.
Dicerna secured rights to the technology through a licensing deal with Arbutus Biopharma, formerly Tekmira Pharmaceuticals.
Dicerna Pharmaceuticals chief medical officer Pankaj Bhargava said: "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.
"We look forward to working with the FDA during the review process in order to rapidly initiate clinical development of DCR-PH1."
DCR-PH1, which is in preclinical development, targets and destroys the messenger RNA (mRNA) produced by HAO1, a gene implicated in the pathogenesis of PH1.
HAO1 encodes glycolate oxidase, an enzyme involved in oxalate production. In preclinical studies, DCR-PH1 induced inhibition of HAO1 and reduced urinary oxalate levels in animal models of PH1.