BCH-HSP-C01，25MG，SML4139-25MG，Sigma

Biochem/physiol Actions

Restores ATG9A function and modulates intracellular trafficking and autophagy.


BCH-HSP-C01, a novel pharmacotherapeutic agent, is engineered to rectify ATG9A-related pathologies. Its primary mode of action entails the regulation of intracellular trafficking mechanisms, with a pronounced effect on the RAB protein family, integral to vesicular transport processes. Exhibiting both temporal and concentration-dependent characteristics, BCH-HSP-C01 optimally enhances ATG9A translocation within a 72–96 hour window post-administration, achieving an EC50 ~ 5 µM in hiPSC-derived neurons from AP-4-HSP patients. Furthermore, BCH-HSP-C01 markedly increases autophagic flux, as indicated by the elevated levels of LC3-II following its application and restores neuronal phenotypes of adapter protein complex 4 (AP-4)-deficiency.