Bio-mimetic guided design of novel helper lipids for gene therapy

There has been a shift in the pharma industry towards development of medicines acting within the target cell, typically the in cytosol, thereby creating demand for advanced delivery vehicles that deliver their payload intracellularly. Generally, these vehicles are taken into the cell's endosome via a process known as endocytosis. In order for the therapeutic agent to exert its effect, the vehicle (and its payload) must escape the endosome into the cytosol. Currently only 2% of vehicles are released from the endosome into the cytosol. This study wishes to determine the detailed structure of lipid nanoparticles containing a novel bio-lipid, known as LPBA, that exhibits good transfection ability by disrupting the endosomal membrane using SANS in combination with contrast variation with a view to understanding how the lipid exerts its activity.