BGT-NPC

BGT-NPC is an adeno-associated virus (AAV) serotype 9-based gene therapy being developed by Bloomsbury Genetic Therapies for the treatment of Niemann-Pick Disease Type C (NPC). The therapy is designed to deliver a functional copy of the NPC1 gene to the central nervous system and other affected tissues to correct the underlying genetic defect. NPC is a rare, progressive lysosomal storage disorder characterized by the inability of the body to transport cholesterol and other lipids, leading to their accumulation in various organs, particularly the brain. By restoring NPC1 protein function, BGT-NPC aims to halt or reverse the neurodegeneration and systemic symptoms associated with the disease. It is currently in preclinical development and was developed based on research from University College London (UCL).