To conduct a comprehensive investigation on the developmental trajectory, current status, and research hotspots in gene therapy drugs both domestically and internationally, thus to provide references for promoting technological innovation and facilitating the translation of research outcomes.

This study retrieved data from the CNKI, Web of Science Core Collection, and ClinicalTrials.gov databases, incorporating 1 890 Chinese articles, 7 598 English articles, and 1 806 clinical trial records. Visualization analysis of the current landscape and trends in the field of gene therapy drugs was conducted using VOSviewer and CiteSpace.

Research in this field has demonstrated sustained growth. Domestic research hotspots are primarily focused on the application of CRISPR-Cas9 technology, the development of nanocarriers, and targeted cancer therapies. In recent years, international research has shown heightened interest in exploratory technologies such as gene editing, rare disease treatments, and artificial intelligence. These findings provide a theoretical basis for understanding the evolution of the gene therapy drug field.

Gene therapy drug research has entered a phase of rapid development. Emerging hotspots include AI-enabled vector design and the optimization of off-target effects. Future research holds great potential through the deep integration of molecular biology, materials science, and information technology. Nevertheless, challenges such as vector delivery efficiency, immunogenicity control, and scalable production processes remain the central to current research efforts and represent key areas for ongoing investigation.