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KAAD-cyclopamine
- Development stage
- Discontinued
- Lead developer
- Johns Hopkins University
- Modality
- Small Molecules
01
Overview
KAAD-cyclopamine is a potent, cell-permeable semisynthetic derivative of the plant-derived steroidal alkaloid cyclopamine. It acts as a highly potent antagonist of the Smoothened (Smo) receptor, a key component of the Hedgehog (Hh) signaling pathway. Developed originally at Johns Hopkins University, KAAD-cyclopamine was designed to improve the solubility and biological potency of natural cyclopamine, demonstrating 10- to 20-fold greater potency in suppressing Hedgehog signaling (with an IC50 of approximately 20 nM). It is widely used as a research tool to study Hedgehog pathway-dependent processes, including embryonic development and various cancers such as medulloblastoma, basal cell carcinoma, and breast cancer.
- Other names
- Cyclopamine-KAADShh Signaling Antagonist IIStemolecule KAAD-Cyclopamine
02
Targets
SMO (Smoothened)
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