BBM-G102

BBM-G102 (CRG003) is an adeno-associated virus (AAV) gene therapy developed for the treatment of late-onset Pompe disease (LOPD). It is designed to deliver a functional human acid alpha-glucosidase (GAA) gene to liver cells, effectively turning the liver into a "bio-factory" for the long-term production and secretion of the active GAA enzyme into the systemic circulation. Once secreted, the enzyme is taken up by skeletal and cardiac muscle tissues to degrade accumulated lysosomal glycogen, addressing the underlying enzyme deficiency that causes the disease. This approach aims to provide a durable therapeutic effect from a single intravenous administration, potentially replacing the need for chronic biweekly enzyme replacement therapy (ERT).