Gene therapy has shown significant potential for treating genetic, oncologic, and infectious diseases, but comprehensive preclinical safety evaluation remains essential before clinical application. Toxicology studies assess potential adverse effects, biodistribution, and overall safety of gene therapy products. Among available preclinical models, non-human primates (NHPs) are widely regarded as one of the most translational because of their close genetic, physiological, and immunological similarity to humans.
NHP toxicology studies are designed to evaluate the safety and biological effects of gene therapy through carefully planned dosing strategies, clinical observations, laboratory assessments, histopathology, molecular analyses, and imaging. Compared with rodent models, NHPs provide more clinically relevant information on vector distribution, immune responses, long-term transgene expression, and potential toxicities, supporting more reliable prediction of human outcomes.
Recent studies have demonstrated the value of NHPs in evaluating gene therapies for diseases such as hemophilia and neurodegenerative disorders, showing sustained therapeutic gene expression with favorable safety profiles. As gene therapy technologies continue to evolve, NHP models remain an important component of preclinical development, helping optimize dosing strategies, improve safety assessment, and generate robust data to support regulatory submissions and clinical translation.
