The nanoVAST

a non-viral delivery system for genome editors and other cargo

Cell-specific cargo delivery is a key remaining problem in the field of targeted therapeutics, such as gene therapy, RNA-modification, and drug delivery. Despite decades of promise, there is a glaring lack of effective, cell-type-specific drug and/or gene therapy delivery systems approved for use in humans.

Presently, delivery relies on lipid nanoparticles (LNPs) which encapsulate RNA with high efficiency, but broadly lack in specificity. The ideal carrier system would involve LNP-efficient cargo encapsulation together with specific targeting in the absence of oncogenicity or immunogenicity that is a concern of viral LNPs.

Panosome has developed such a particle – the nanoVAST: a patented vesicular phospholipid bilayer derived from the VAST (a recipient of a European Research Council Proof of Concept award). Thus, the surface of these non-viral LNPs can be coated using the VAST sortase-based chemistry to attach peptides, small molecules, and proteins that can direct the nanoparticle to specific cells. The nanoVAST is highly fusogenic with target membranes, permitting cargo delivery directly to the cytoplasm.

Tens of millions of people are affected by diseases that could be treated through gene therapy or targeted drug delivery. With the advent of base editing technologies and attempts at the commercialization of such technologies over the last decade by many companies, precision therapeutics is beginning to fulfill its promise.

With nanoVAST, Panosome is poised to provide a game changer in precision payload delivery. Such a scalable and non-viral precision delivery tool would unlock the therapeutic potential to treat many currently untreatable diseases.