CAP-001

CAP-001 is an investigational adeno-associated virus (AAV) gene therapy developed by Capsida Biotherapeutics for the treatment of Hunter syndrome (Mucopolysaccharidosis type II, MPS II). The therapy utilizes a novel, engineered AAV capsid designed to cross the blood-brain barrier and achieve broad, non-invasive delivery to the central nervous system (CNS) following intravenous administration. CAP-001 delivers a functional human iduronate-2-sulfatase (IDS) transgene to restore enzyme activity, thereby addressing the underlying cause of MPS II—the accumulation of glycosaminoglycans (GAGs) that leads to progressive physical and neurological decline. Preclinical data have demonstrated that CAP-001 provides significantly enhanced biodistribution and more uniform brain coverage compared to traditional AAV9 vectors, with the potential to treat both the systemic and neurological manifestations of the disease. While initially developed as a pilot program to validate Capsida's AAV engineering platform, it has served as a proof-of-concept for the company's ability to target CNS diseases systemically.