Lipid Nanoparticles: A Comprehensive Assessment Of Liver Enzymes And Immunopathology Markers In Nonhuman Primates

By Gabriela Campoy, Sydney P. Petersen, Narine Lalayeva, Julie Forget, and Norbert Makori

Lipid nanoparticles (LNPs) have emerged as a groundbreaking advancement in drug delivery technology, serving as carriers for non-viral targeted therapies. Their unique ability to encapsulate and transport a wide range of molecules across cell membranes while shielding them from degradation has paved the way for innovations in gene therapies and gene editing. However, ensuring the safety and efficacy of these new products is crucial, and despite regulatory guidance from the Food and Drug Administration (FDA), the limited availability of published data makes it challenging to design comprehensive preclinical studies that enable proper sampling and monitoring of critical parameters.

Nonhuman primates, due to their close physiological similarities to humans, are considered an ideal test system for evaluating toxicity. This review seeks to fill knowledge gaps and provide valuable insights that can contribute to the safe and effective use of LNPs in gene therapy and editing applications.