Abstract
Animal models play a pivotal role in drug development and serve to better understand not only the origins and pathology of the diseases but also the safety and dynamics of the drug in the body. Transgenic animals have become a key tool to study the mode of action of the drugs and recapitulate the etiology and pahtology of the diseases. Here, we would like to introduce how we used different animal models to reveal the therapeutic potentials of a compound and ultimately pushed the compound into Phase II clinical trial.