Other LSD Program (Zacharon Pharmaceuticals)

Zacharon Pharmaceuticals' Other LSD Program is a preclinical-stage development effort focused on small molecule inhibitors of the glycan biosynthesis pathway. These therapeutic candidates are designed to treat a broad spectrum of lysosomal storage disorders (LSDs) through substrate reduction therapy (SRT), a mechanism that decreases the production of pathological glycans to prevent their toxic accumulation. The program specifically targets several rare genetic conditions, including Mucopolysaccharidosis types IVA (Morquio A), IVB (Morquio B), VI (Maroteaux-Lamy), VII (Sly), and IX, as well as Metachromatic Leukodystrophy (MLD) and Niemann-Pick type C (NPC). By modulating the synthesis of glycosaminoglycans and other glycans, these therapies aim to address the underlying biochemical imbalance in patients where enzyme replacement therapy may be challenging or unavailable. Following the acquisition of Zacharon by BioMarin in 2013, these early-stage assets were integrated into the BioMarin pipeline.

Other names

unnamed small molecule (Zacharon Pharmaceuticals)glycan biosynthesis inhibitor (Zacharon Pharmaceuticals)