Skip to main content
Premium Trial:

Request an Annual Quote

Dicerna Presents Preclinical Data on Primary Hyperoxaluria Type 1 Drug

Premium

NEW YORK (GenomeWeb) – Dicerna Pharmaceuticals this week announced preclinical data showing that its investigational primary hyperoxaluria type 1 (PH1) therapy DCR-PH1 could silence expression of its target by as much as 97 percent in a mouse model of the disease.

PH1 is a rare, inherited autosomal-recessive condition characterized by the liver's inability to metabolize a precursor of oxalate due to disruption of an enzyme called alanine-glyoxylate aminotransferase 1 (AGT1). As a result, calcium oxalate builds up in renal tubules causing kidney stones and fibrosis.

Dicerna's drug is designed to inhibit HAO1, a gene that produces glycolate oxidase, which is an enzyme upstream of AGT1.

In the preclinical studies, which were detailed at the 11th International Primary Hyperoxaluria Workshop, Dicerna and academic collaborators specifically demonstrated that a single dose of DCR-PH1 led to a 97 percent reduction of the HAO1 transcript in the liver and significant reductions in urinary oxalate to near baseline levels.

Dicerna has said it expects to begin Phase I testing of DCR-PH1 in 2015.

The Scan

Purnell Choppin Dies

Purnell Choppin, a virologist who led the Howard Hughes Medical Institute, has died at 91, according to the Washington Post.

Effectiveness May Decline, Data From Israel Suggests

The New York Times reports that new Israeli data suggests a decline in Pfizer-BioNTech SARS-CoV-2 vaccine effectiveness against Delta variant infection, though protection against severe disease remains high.

To See Future Risk

Slate looks into the use of polygenic risk scores in embryo screening.

PLOS Papers on Methicillin-Resistant Staphylococcus, Bone Marrow Smear Sequencing, More

In PLOS this week: genomic analysis of methicillin-resistant Staphylococcus pseudintermedius, archived bone marrow sequencing, and more.