GCase Activator 3

An activator of GCase (AC50 = 1.49 µM), increases GCase protein levels and activity and reduces mutant GCase protein misfolding in GBA1N370S/84GG-expressing patient-derived fibroblasts from patients with Gaucher disease, increases GCase protein levels and activity in iPSC-derived and GBA1N370S-expressing iPSCs differentiated into midbrain dopaminergic neurons from a patient with Parkinson's disease. SMILES: CN(C[C@H]1COC2=CC=CC=C2O1)C3=NC(C4=CN=CC=C4)=NC5=C3C=CC=C5. InChi Code: InChI=1S/C23H20N4O2/c1-27(14-17-15-28-20-10-4-5-11-21(20)29-17)23-18-8-2-3-9-19(18)25-22(26-23)16-7-6-12-24-13-16/h2-13,17H,14-15H2,1H3/t17-/m0/s1. InChi Key: JFOVNJQCUHRYOI-KRWDZBQOSA-N