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Realizing the promise of glycobiology

Zacharon's Unique Approach

The glycan-targeted small molecule drug development platform established by Zacharon has enabled an entirely new treatment strategy for patients with lysosomal storage diseases. Rather than attempting to correct the enzyme deficiency (historical approach), Zacharon is developing small molecule drugs which selectively modify the structure of the glycan so that the deficient enzyme is not required for degradation.

This strategy, termed "substrate optimization therapy", represents the first small molecule approach to treating lysosomal storage 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. By modulating the target glycan, this strategy can be readily applied to many lysosomal storage diseases, creating the opportunity to address substantial unmet need.

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.