Synthetic biologists at Northwestern University have pioneered a flexible delivery platform that addresses a significant hurdle in the deployment of cell and gene therapies. Traditional methods of delivering biological treatments into the body have proven challenging, limiting the accessibility of these innovative therapies for diseases such as cancer, cystic fibrosis, diabetes, heart disease, and HIV/AIDS.
The novel platform leverages extracellular vesicles (EVs), tiny nanoparticles produced naturally by all cells, mimicking processes used by viruses for drug delivery. Through synthetic biology, researchers designed DNA "programs" to instruct producer cells to self-assemble custom EVs with specific surface features, and load them with biological drugs. In proof-of-concept experiments, this system successfully delivered CRISPR gene-editing agents to T cells, notoriously difficult to target.
Published in Nature Biomedical Engineering, this study marks the first use of EVs to deliver cargo into T cells, according to Joshua N. Leonard, who led the study.
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The new platform, named GEMINI (Genetically Encoded Multifunctional Integrated Nanovesicles), offers a suite of technologies for genetically engineering cells to produce multifunctional EVs tailored to diverse patient needs. Leonard envisions its application in rapidly generating optimal delivery vehicles for a range of developers, from academic spinouts to mature biotechnology companies.
The versatility of GEMINI lies in its ability to genetically encode cargo and surface compositions of nanovesicles, turning complex biological challenges into more manageable DNA engineering problems. This breakthrough holds promise for accelerating the development and evaluation of gene and cell therapies, ultimately benefiting a wide range of patients.