Extracellular vesicles (EVs), including exosomes, are being studied extensively and have emerged as a promising novel technology for the delivery of complex therapeutic payloads such as nucleic acids or proteins. EVs are naturally occurring within the body and have several potential advantages over other delivery vehicles such as LNPs or viral vectors, such as their ability to evade the immune system response and therefore support repeat dosing, as well as their capacity to access biological barriers and deliver a wide range of cargo to specific target tissue types given their tropism profiles. These attributes make EVs favorable candidates to engineer as drug delivery systems.
Here, we discuss a plug-and-play platform approach to express biomolecules using engineered EVs. Therapeutic cargo may be loaded onto or within EVs via identified scaffold proteins using novel plasmid design. This concept was explored in the development of therapeutics such as exoSTING®, exoASO-STAT6® and exoIL-12™, which have completed Phase 1 clinical studies within oncology applications. Alternatively, small molecules may be linked to EVs using a proprietary linker technology. A dedicated cell line for EV production was also generated, which resulted in a clone capable of high particle titer, integrity, and purity. These technologies will be made available to the wider scientific community under a research license, or via Lonza’s CDMO service offerings.