BGT-OTCD

BGT-OTCD is an investigational adeno-associated virus (AAV) gene therapy being developed by Bloomsbury Genetic Therapies for the treatment of Ornithine Transcarbamylase Deficiency (OTCD). The therapy utilizes the AAV-LK03 capsid, which has been engineered for high tropism and transduction efficiency in human hepatocytes. BGT-OTCD delivers a functional copy of the human *OTC* gene to the liver, aiming to restore ornithine transcarbamylase enzyme activity. By re-establishing the urea cycle's function, the therapy is designed to prevent the accumulation of toxic ammonia levels, thereby reducing the risk of life-threatening metabolic crises and long-term neurological damage in patients. It is currently in Phase 1/2 clinical development.