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 SAR studies of a lead chemical series that was discovered from qHTS. The team has developed potent analogs with significant activity and amenable ADME properties, and additional rounds of medicinal chemistry optimization are underway to improve compound pharmacokinetics. Target engagement probes have also been synthesized, which will be evaluated in target-identification studies.