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HPBCD (VTS-270), 2-hydroxypropyl-b-cyclodextrin

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Company Vtesse Inc.
Description2-hydroxypropyl-b-cyclodextrin targeting cholesterol and sphingolipid storage
Molecular Target
Mechanism of Action 
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase II/III
Standard IndicationGlycosphingolipid storage disorders
Indication DetailsTreat Niemann-Pick disease type C1 (NPC1)
Regulatory Designation U.S. - Breakthrough Therapy (Treat Niemann-Pick disease type C1 (NPC1));
U.S. - Orphan Drug (Treat Niemann-Pick disease type C1 (NPC1));
U.S. - Rare Pediatric Disease (Treat Niemann-Pick disease type C1 (NPC1));
EU - Orphan Drug (Treat Niemann-Pick disease type C1 (NPC1));
EU - Promising Innovative Medicine (Treat Niemann-Pick disease type C1 (NPC1))
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