Selective organ-targeted chemotherapy

The term "Selective organ-targeted chemotherapy" does not refer to a specific molecular target, receptor, or therapeutic molecule. Instead, it describes a drug delivery strategy or technology platform designed to deliver therapeutic agents to particular organs or tissues within the body. This concept is most prominently illustrated by SORT (Selective Organ Targeting) nanoparticles, which are a sophisticated delivery system. SORT nanoparticles are not a biological target that drugs act upon; rather, they are a means by which drugs are transported to their intended biological targets more precisely. The technology modifies lipid nanoparticles (LNPs) to achieve tissue-specific delivery, overcoming challenges in systemic drug administration by enabling tailored biodistribution.

Other names

SORT (Selective Organ Targeting) nanoparticlesOrgan-targeted chemotherapyOrgan-specific chemotherapy

Selective organ-targeted chemotherapy, particularly exemplified by SORT (Selective Organ Targeting) nanoparticles, functions as a drug delivery strategy. The SORT technology involves adding a fifth component (a 'SORT molecule') to conventional four-component lipid nanoparticles (LNPs). This SORT molecule, typically a small molecule or peptide, alters the biodistribution of the LNPs. The mechanism involves a three-step endogenous targeting process: 1) desorption of PEG lipid from the LNP surface, 2) distinct serum protein adsorption to the LNP based on the SORT molecule's chemical structure, and 3) subsequent receptor-mediated uptake in specific target tissues or organs (e.g., lungs, spleen, liver) due to the altered protein corona. This system enables the targeted delivery of various therapeutic agents, such as mRNA and CRISPR-Cas systems, to desired organs, thereby enhancing efficacy and reducing off-target effects.