ExSAR is developing EXR-202 for the treatment of Gaucher disease. As with EXR-101, EXR-202 is also a small molecule pharmacological chaperone that binds to and stabilizes acid beta-glucocerebrosidase (GCase). EXR-202 has been approved by European and other foreign regulatory agencies for other indications, can be administered orally and has shown remarkable efficacy in cell lines derived from GCase patient fibroblasts. An IND is in preparation and Phase I clinical trial planned for in 2009.

Lysosomal Storage Disorders and Protein Misfolding | Product Pipeline | Clinical Trials
EXR-101 for Late-Onset Tay-Sachs | Tay-Sachs and EXR-101 | EXR-202 for Gaucher disease | Gaucher Disease and EXR-202