CCB-001

CCB-001 is a gene therapy drug candidate developed to deliver a functional type I collagen gene (COL1A1) for the treatment of osteogenesis imperfecta, a genetic disorder characterized by brittle bones. The therapy uses gene transference technology to introduce the correct version of the COL1A1 gene into patient cells, aiming to address the underlying cause of disease rather than just managing symptoms. Initially developed by Mayo Clinic and now under development by Castle Creek Biosciences, CCB-001 is positioned as a disease-modifying approach for congenital disorders affecting skin and musculoskeletal systems[1][2].