Synthesis of a Hydroxy-15-Azasterol

Abstract

Niemann-Pick type C (NPC) is a lysosomal storage disorder that will cause eventual brain damage with limited treatment options available. Though clinical trials are undergoing with repurposed pharmaceuticals, no novel chemotype exists purely for the treatment of NPC. In 2021, an azasterol was found to bind to both NPC1 and NPC2 proteins and is considered as a cholesterol mimic. A convergent synthesis to obtain a hydroxy-15-azasterol was completed in 12 steps, from 2-oxepanone.