qHTS to Identify Inhibitors of DYT1 Inclusion Formation

National Center For Advancing Translational Sciences

Investigators

Linked publications, trials & patents

Abstract

The NCATS Early Translation Branch (ETB) hosts a broad and comprehensive program for the discovery of drug candidates directed towards rare diseases and pharmacological tools to probe the function of the human genome. ETB conducts research to understand the underlying principles driving the translation of basic research discoveries into tangible improvements in human health. During this period, the collaborative team focused on extensive SAR studies of a lead chemical series that was discovered from qHTS. After multiple rounds of SAR, the team has developed lead candidates with 1) potent activity in the primary cellular phenotypic assay, 2) reduced metabolism, and 3) brain exposure in mice. These compounds will be evaluated in more disease relevant models. Active and inactive chemical biology probes were also synthesized and are being evaluated in target-identification studies.