Tailored therapies: Exploring macromolecule based delivery science for personalized medicine

Abstract

The advent of personalized medicine has revolutionized healthcare, transitioning from a one-size-fits-all model to tailored therapies that address the unique genetic, environmental, and lifestyle factors of individual patients. Central to this shift is the field of macromolecule-based delivery science which involves the use of biologically derived or synthetically engineered macromolecules, like proteins, nucleic acids, and polysaccharides. These macromolecules have shown immense potential in improving the specificity, efficacy and safety of therapeutic interventions, paving the way for more precise and effective treatments. Macromolecule-based delivery systems are designed to overcome the limitations of traditional small-molecule drugs, such as poor solubility, rapid degradation, nonspecific distribution and potential toxicity. Unlike small molecules, macromolecules can be engineered for high specificity toward target cells or tissues, reducing off-target effects and enhancing therapeutic outcomes. The ability to modify and functionalize these macromolecules with various chemical and biological entities allows for the development of multifunctional delivery platforms capable of addressing complex pathophysiological conditions. These systems have been instrumental in targeting specific antigens on cancer cells, leading to highly effective therapies with reduced systemic toxicity. In addition, RNA-based therapies offer innovative solutions for gene silencing and protein replacement, providing hope for patients with previously untreatable genetic disorders.