Nucleic acid therapeutics, including DNA, RNA, siRNA, ASOs, and mRNA, have emerged as powerful treatment modalities for genetic, infectious, and oncologic diseases. Their success, however, depends on effective delivery systems that protect nucleic acids from degradation and enable efficient targeting of specific tissues and cells. Both viral and non-viral delivery platforms have been developed to improve stability, cellular uptake, and therapeutic efficacy.
Non-human primates (NHPs) are among the most valuable preclinical models for evaluating nucleic acid drug delivery systems because of their close genetic, physiological, and immunological similarity to humans. They provide highly translational data on biodistribution, pharmacokinetics, pharmacodynamics, immunogenicity, and safety, helping bridge the gap between rodent studies and clinical trials.
NHP studies have supported the development of mRNA vaccines, siRNA and antisense therapies, and gene-editing technologies by demonstrating effective delivery, sustained biological activity, and favorable safety profiles. As nucleic acid therapeutics continue to evolve, NHP models remain essential for optimizing delivery strategies, improving translational predictability, and accelerating the development of next-generation genetic medicines.
