BGT-NPC for Niemann-Pick Disease Type C (NPC)

BGT-NPC is a neuron-targeted AAV9 investigational gene therapy designed to provide a potentially curative solution to NPC patients following a one-time injection in the cerebrospinal fluid (CSF). AAV9 for administration in the CSF was selected for its promise in other whole-brain neurological disorders such as mucopolysaccharoidosis type II or giant axonal neuropathy.