NPC12-G

NPC12-G (also known as G-NPC12) is an investigational nanoparticle-encapsulated formulation of 2-hydroxypropyl-beta-cyclodextrin (HPBCD) developed by G-Nanosystems for the treatment of Niemann-Pick Disease Type C (NPC). NPC is a rare lysosomal storage disorder characterized by the accumulation of cholesterol and glycosphingolipids in various tissues, particularly the brain. While HPBCD is known to mobilize sequestered cholesterol, its ability to cross the blood-brain barrier (BBB) is limited when administered systemically. NPC12-G utilizes G-Nanosystems' proprietary G-technology to enhance the delivery of HPBCD across the BBB, potentially allowing for effective neurological treatment via intravenous administration rather than the more invasive intrathecal route required for standard HPBCD formulations. The drug has received Orphan Drug Designation from both the FDA and EMA.

Other names

nanoparticle-encapsulated 2-hydroxypropyl-beta-cyclodextrinnanoparticle-encapsulated2-hydroxypropyl-beta-cyclodextrinnanoparticle-encapsulated-2-hydroxypropyl-beta-cyclodextrin