BGT-OTCD for Ornithine Transcarbamylase (OTC) deficiency

BGT-OTCD is a liver-targeted AAV-LK03 gene therapy designed to provide a potentially curative solution to OTC deficiency patients following a one-time intravenous injection. AAV-LK03 was selected for its high tropism for liver cells and its success in other liver disorders such as haemophilia A.