Assay for Small-Molecule Antagonists of the TSH Receptor

NCATS早期翻译分支（ETB）拥有一个广泛而全面的计划，用于发现针对罕见疾病的候选药物和探索人类基因组功能的药理学工具。ETB开展研究，以了解推动基础研究发现转化为人类健康切实改善的基本原则。 在此期间，项目团队继续SAR驱动的先导分子优化，以提高代谢稳定性。进行了药代动力学研究，并测量了T4的水平以证实拮抗剂的活性。目前正在进一步优化和扩大规模，以期在有史以来第一个格雷夫斯病小鼠模型中测试目前最好的铅。

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.