Lucerastat

Research use only, not for human use.

A galactose analogue that is an UDP-glucose ceramide glucosyltransferase (UGCG/GCS) inhibitor (IC50=41.4 uM).

A galactose analogue that is an UDP-glucose ceramide glucosyltransferase (UGCG/GCS) inhibitor (IC50=41.4 uM); prevents glucosylceramide biosynthesis both in vitro and in mouse models of lysosome storage disorders, without affecting α-glucosidase I and II or β-glucocerebrosidase (IC50>1 mM); shows potential as substrate reduction therapy for glycolipid storage disorders such as Gaucher disease and Fabry disease.Other Indication Phase 1 Clinical.

Fabry patient-derived fibroblasts with the genotypes R301G (residual -GalA activity; 20%) R220X (<3%) and W162X (<1%). Cell Viability Assay.Cell Line:Fabry patient-derived fibroblasts with the genotypes R301G (residual-GalA activity; 20%) R220X (<3%) and W162X (<1%). Concentration:Incubation Time:9 days.Result:Dose-dependently inhibited GCS, reducing glucosylceramide and increasing sphingomyelin.

Lucerastat (1200 mg/kg/day food admix), a GCS inhibitor, reduces Gb3 in the absence of residual-GalA activity. Animal Model:Fabry mice (Gla-/0 and Gla-/-, n = 5 or 6 for each gender).Dosage:1200 mg/kg/day food admix.Administration:Food admix for 20 weeks.Result:Reduced lipid storage in two major organs affected by FD: mean Gb3 in the kidneys (-33%, p<0.01). and α-galactose- terminated glycosphingolipids in the dorsal root ganglia (-48%, p<0.05). In the liver of the Fabry mice, mean glucosylceramide (GlcCer (24:0)) was reduced (-59%, p<0.001) in addition to Gb3 (24:1) (-37%, p<0.05) demonstrating substrate reduction through GCS inhibition.

N-Butyldeoxygalactonojirimycin | ACT-434964 | OGT-923

Others

Other Targets

Other Targets

Other Indications

Other Disease

141206-42-0

219.281

C10H21NO4

>98% (HPLC)

In Vitro:?H2O : 24 mg/mL (109.45 mM)

O[C@H]1[C@@H](CO)N(CCCC)C[C@H](O)[C@H]1O

(2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol

(-20℃)

With Ice Pack

≥ 2 years