Each cell in the body has its own unique delivery system that scientists are working on harnessing to move revolutionary biological drugs — molecules like proteins, RNA and combinations of the two — to specific diseased parts of the body.
A recent study conducted by Northwestern University explored a new approach to enhance the transport of therapeutic proteins by leveraging the body’s natural delivery mechanisms. By utilizing biophysical principles, researchers were able to significantly improve the loading and delivery of these proteins, surpassing traditional loading methods by enabling molecules to be loaded up to 240 times more efficiently.
The study, published in the journal Nature Communications in July, demonstrated the manipulation of the body’s internal transport system to effectively deliver engineered proteins to target cells, thereby inducing changes in gene expression within the cells. This breakthrough paves the way for a more precise and targeted biological drug delivery system.
One of the key challenges in the development of biological medicines is ensuring the protection and accurate delivery of fragile molecules to diseased cells while avoiding any adverse effects on healthy cells. By combining the efforts of two labs within Northwestern’s Center for Synthetic Biology – led by biomedical engineer Neha Kamat and chemical and biological engineer Josh Leonard – the study achieved a breakthrough in addressing this critical bottleneck.
The research focused on harnessing the structure of lipid rafts on cell membranes, which provided a reliable method for loading protein cargo into extracellular vesicles (EVs). The team discovered that by engineering proteins to associate with lipid rafts, they could dramatically enhance the loading efficiency of therapeutic cargo into vesicles, enabling more effective delivery to other cells.
Furthermore, the study showcased a practical application of this novel method by genetically modifying cells to produce a specific protein, loading it into EVs, and successfully delivering it to target cells to modulate gene expression. This approach demonstrated the reversible function of the cargo, allowing it to be released from the EV membrane and perform its intended biological function within the recipient cell.
Excited about the potential applications of this strategy, the researchers emphasized its versatility in loading various therapeutic cargos, such as CRISPR gene-editing systems, for disease treatments in fields like immunotherapy and regenerative medicine.
With the ability to engineer EVs to selectively deliver functional biomedicines to specific diseased cells, the study opens up new possibilities for treating a wide range of diseases. The findings hold promise for the development of targeted and efficient therapeutic delivery systems that could revolutionize the treatment of various medical conditions.
