qHTS to Identify Inhibitors of DYT1 Inclusion Formation

National Center For Advancing Translational Sciences

Investigators

Linked publications & trials

Abstract

During this period, the collaborative team fully evaluated a top set of hits from the initial screen, prioritizing them for hit-to-lead development. A subset of these top compounds were also tested in advanced cell-based models and secondary assays to further characterize their activity. Top hits were selected for preliminary SAR assessments, and following two rounds of structure activity studies, one top series was selected for full hit-to-lead evaluation. This chemotype has undergone systematic structural exploration to improve potency, to identify the molecular target, and to address physicochemical liabilities. A subset of top molecules have advanced into ex-vivo studies for efficacy evaluation.