GRNA-001

GRNA-001 is an investigational, in vivo CRISPR-based gene editing therapy developed by Arbor Biotechnologies for the treatment of primary hyperoxaluria type 1 (PH1). PH1 is a rare genetic disorder caused by a deficiency of the liver enzyme alanine:glyoxylate aminotransferase (AGT), which leads to the overproduction of oxalate and subsequent kidney failure. GRNA-001 utilizes a proprietary CRISPR nuclease and a guide RNA (gRNA) delivered via lipid nanoparticles (LNPs) to the liver. The therapy is designed to permanently silence the HAO1 gene, which encodes glycolate oxidase. By knocking out HAO1, GRNA-001 reduces the levels of glyoxylate, the substrate for oxalate production, thereby lowering systemic oxalate levels and preventing renal damage. It is currently being evaluated in a Phase 1/2 clinical trial.