RU-SKI 43 is primarily utilized in research settings to study and modulate the activity of the Hedgehog signaling pathway, which plays a critical role in cell differentiation, proliferation, and tissue patterning. It acts as an inhibitor of the Smoothened receptor, a key component of this pathway, making it a valuable tool for investigating the molecular mechanisms underlying various biological processes and diseases. In cancer research, RU-SKI 43 is particularly significant for its potential to suppress tumor growth, especially in cancers where the Hedgehog pathway is dysregulated, such as basal cell carcinoma and medulloblastoma. Additionally, it is employed in developmental biology studies to understand the role of Hedgehog signaling in embryonic development and organogenesis. Its application extends to drug discovery, where it serves as a reference compound for developing new therapeutic agents targeting Hedgehog-driven pathologies.