In comparison with conventional therapies, nanomedicine shows prominent clinical performance, with better therapeutic efficacy and less off-target toxicity. As an important component of nanomedicine, gold nanoparticle (GNP)-based nanodrugs have attracted considerable interest because of their excellent performance given by the unique structure. Although no pharmaceutical formulations of GNP-associated nanodrugs have been officially marketed yet, a substantial amount of research on this aspect is being carried out, producing numerous GNP-based drug delivery systems with potential clinical applications. In this review, we present an overview of our progress on GNP-based nanodrugs combined with other achievements in biomedical applications, including drug-conjugated GNPs prepared for disease treatments and specific tumour targeting, structure–efficacy relationship (SER) studies on GNP-conjugated nanodrugs, and therapeutic hybrid nanosystems composed of GNPs. In addition, we also put forward some proposals to guide future work in developing GNP-based nanomedicine. We hope that this review will offer some useful experience for our peers and GNP-based nanodrugs will be utilized in the clinic with further persistent efforts.