Assay for Small-Molecule Antagonists of the TSH Receptor

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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 project team continued SAR-driven optimization of the lead molecule to improve metabolic stability. Pharmacokinetic studies were carried out, and the levels of T4 were also measured to corroborate activity of antagonists. Further optimization and scale-up is currently underway, with a view to testing the current best lead in the first-ever mouse model of Graves' Disease.