Quality Segment
assay
≥98%
form
powder
color
white to beige
solubility
DMSO: 2 mg/mL, clear
storage temp.
2-8°C
SMILES string
C1(SC(N2CCC(NC=N3)=C3C2)=N4)=C4C=CC=C1
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.
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.
存储类别
11 - Combustible Solids
wgk
WGK 3
flash_point_f
Not applicable
flash_point_c
Not applicable