BGT-OTCD for Ornithine Transcarbamylase Deficiency (OTCD)

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