Other Lysosomal Storage Diseases
In addition to our most advanced programs, Zacharon's therapeutic strategy can be readily applied to other lysosomal storage diseases by varying the target glycan. This strategy represents the first small molecule approach to treating these diseases. This fundamental difference of using a small molecule creates the potential to penetrate the central nervous system and other critical tissues largely unaddressed by existing therapies.
The following diagram outlines the biosynthetic pathway of two classes of glycans. The relevant target for glycan biosynthesis which can reduce downstream lysosomal accumulation in patients with specific lysosomal storage diseases is shown. In many cases, multiple diseases can be treated with one glycan-targeted drug. For example, lysosomal accumulation in MPS I, MPS II, MPS IIIA, IIIB and IIIC can all be reduced by targeting the same specific step in glycan biosynthesis.
Zacharon is conducting important preclinical development activities to enable new small molecule therapies for a range of lysosomal storage diseases
