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Nixing Niemann-Pick

Vtesse repurposing cyclodextrin for ultra-Orphan Niemann-Pick disease

May 4, 2015 7:00 AM UTC

Vtesse Inc. is hoping to change the course of disease in Niemann-Pick type C, a fatal ultra-Orphan lysosomal storage disorder, using a compound plucked from NIH's Therapeutics for Rare and Neglected Diseases repurposing project.

Niemann-Pick type C is caused by the progressive accumulation of unesterified cholesterol and glycosphingolipids in the CNS and visceral organs. The accumulation stems from mutations in the Niemann-Pick disease type C1 (NPC1) or NPC2 genes, which encode proteins involved in the intracellular trafficking of cholesterol and other lipids. ...