During this period, the collaborative team worked to complete high-throughput screening and identified new chemotypes able to elevate Rab9 expression. Optimization of one the chemotypes yielded potent and selective molecules with good pharmacokinetic molecules. These molecules were able to normalize the phenotype of several lysosomal storage disorders (such as Fabry and Niemann Pick C) in vitro, as well as ameliorate these diseases in several in vivo mouse models. Target deconvolution and advanced characterization of these molecules identified a primary mechanism of action, and they are currently undergoing further development under an outlicense with Amathus Therapeutics.
