We design viral vector systems that can deliver complex, multigene cargos into diverse cell types: insect cells, mammalian cells, primary cells and whole organs, for a variety of applications. We have expertise in viral vector genome engineering including development of pseudotyping strategies, target homing and cell entry strategies for the most difficult cell and organ types. Our goal in these applications is the same: to develop methods to deliver DNA code of ever increasing size and complexity into cells.
The first method for delivering a multigene readout into diverse mammalian and primary cells lines is described in Multigene Delivery in Primary Cells_Nature Communications_2016.
GB-007 is a small molecule drug candidate targeting a GPCR with a well understood biology. We began by developing a cellular discovery assay that was an order of magnitude more sensitive than all prior art systems. Our computational chemistry team probed putative novel extracellular drug binding sites on the GPCR and used in silico methods to propose molecules which were screened using our cellular discovery assay. We then applied MultiBac to produce the target and solve its structure in complex with early hits (below images), guiding both chemistry and computational chemistry and leading to preclinical candidate GB-007.