molecules of the month

The Sanofi glucosylceramide synthase (GCS) inhibitor, venglustat, is an oral, brain-penetrant clinical candidate for GBA-mutant Parkinson’s disease and other diseases where GBA-mutations are relevant. Though the molecule did not demonstrate efficacy within one year in early GBA-PD patients and was discontinued in this indication (NCT02906020), it continues to be tested in various rare genetic diseases including Gaucher disease type 3 (NCT02843035), Fabry disease (NCT02228460), GM2 gangliosidosis (NCT04421451), and autosomal-dominant polycystic kidney disease (NCT04705051), as its safety has already been demonstrated.