Several Shh pathway inhibitors have been developed, including cyclopamine, a natural product inhibitor of Smo, and GANT61, a small molecule inhibitor of Gli1. However, these inhibitors have limitations, such as poor pharmacokinetic properties or limited efficacy. Therefore, there is a need for the development of more effective and selective Shh pathway inhibitors.
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\subsectionBiological Activity
\subsectionAbstract
SONE-220 is a novel small molecule inhibitor of the Shh pathway that exhibits potent inhibitory activity and good selectivity. The compound shows good pharmacokinetic properties and in vivo efficacy in a medulloblastoma mouse model. These findings suggest that SONE-220 is a promising candidate for the treatment of Shh-driven cancers. SONE-220