GT-EPIC

GT-EPIC is an investigational gene therapy candidate designed for the treatment of primary hyperoxaluria type 1 (PH1). Developed by Generation Bio, the therapy utilizes the company's proprietary non-viral genetic medicine platform, which features a closed-ended DNA (ceDNA) construct delivered via a cell-targeted lipid nanoparticle (ctLNP). The mechanism of action involves the delivery of a functional copy of the AGXT gene to hepatocytes. This gene encodes the enzyme alanine-glyoxylate aminotransferase (AGT), which is deficient or dysfunctional in patients with PH1. By restoring AGT activity in the peroxisomes of liver cells, GT-EPIC aims to normalize the metabolism of glyoxylate, thereby preventing the overproduction and subsequent systemic accumulation of oxalate, which leads to severe kidney damage and systemic oxalosis.