FLT201: Initial Clinical Data

Gaucher disease type 1 is a debilitating, chronic and progressive disorder with life-altering symptoms Deficiency of GCase enzyme needed to metabolize Gb-1 in the lysosome Normal GCase function Normal metabolism of Gb-1 GCase deficiency Gb-1 not metabolized Harmful accumulation of Gb-1 and its substrate lyso-Gb1 in cells ¹Weinreb, et al., 2008 GCase = glucocerebrosidase. Gb-1 = glucosylceramide. Lyso-Gb-1 = glucosylsphingosine. Affects multiple organs, leading to wide range of symptoms and shortening life span¹ Bone pain and fractures Enlarged liver Reduced lung function *** Anemia and thrombocytopenia Enlarged spleen FREELNE 6

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