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CPA006 is a small molecule cyclopamine derivative that acts as an inhibitor of the Hedgehog (Hh) signaling pathway. Developed as a more soluble and efficacious alternative to naturally occurring cyclopamine, it targets the Smoothened (SMO) protein to block Hh-mediated carcinogenesis. Research presented at AACR 2010 demonstrated its efficacy in reducing tumor mass and decreasing Hh target gene expression in mouse models of basal cell carcinoma (BCC) and pancreatic cancer. It has been studied for topical application in skin dysplasia and systemic or orthotopic treatment in pancreatic cancer models.
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