In the field of metal-based anticancer agents, gold-based drugs have emerged as one of the most promising research directions following platinum-based drugs, owing to their unique physicochemical properties and distinct anticancer mechanisms. This review introduces Au(I) complexes, Au(III) complexes and gold clusters separately, summarizing the recent advances in the anticancer research of gold complexes and gold clusters with well-defined structures. Special emphasis is placed on the ligand design and structure–activity relationships of Au(I) complexes, the synthetic strategies and anticancer mechanisms of Au(III) complexes, as well as the characteristics of gold cluster drugs. Furthermore, the future development directions of gold-based anticancer agents are prospected, including the development of novel targeted ligands, the combination of cancer therapies, and in-depth elucidation of their mechanisms of action.