Our philosophy is that the best way for us to identify our drug targets is to define and evaluate genetic variants in patients at highest risk for disease. We identify our targets genetically and then use bench research to quantify and define the defect at a functional level. Once we have identified the problem, we explore multiple relevant technologies—antibody, recombinant protein, or gene therapy—to develop drug candidates to address the specific problem.
We use a combination of in vitro, cell-based and in vivo experiments to expand our biological understanding of the targets and of the specific variants within those targets. Our work allows us understand the direction of modulation that is required. Where variants result in a loss of protein or activity of a protein, we use recombinant proteins or gene therapy to restore a functional level of protein. Where variants result in higher levels of protein or a gain in the activity, we use antibodies or other technologies to reduce the level of the protein.