AAV9-GBE1

AAV9-GBE1 is an investigational adeno-associated virus serotype 9 (AAV9) gene therapy designed to deliver a functional copy of the human glycogen branching enzyme 1 (GBE1) gene. It is being developed for the treatment of Adult Polyglucosan Body Disease (APBD) and Glycogen Storage Disease Type IV (GSD IV). APBD is a rare, progressive neurodegenerative disorder caused by mutations in the GBE1 gene, leading to deficient GBE activity and the accumulation of abnormal, poorly branched glycogen aggregates known as polyglucosan bodies (PBs) in the central and peripheral nervous systems and muscles. AAV9-GBE1 is designed to restore GBE1 expression and enzyme activity, thereby reducing polyglucosan accumulation and improving neuromuscular and metabolic functions. Preclinical studies in mouse models of APBD have demonstrated dose-dependent improvements in histological and functional parameters following intrathecal or systemic administration.

Other names

AAV9-delivered human GBE1AAV-9-delivered human GBE1AAV 9-delivered human GBE1AAV9-hGBE1AAV-9-hGBE1AAV 9-hGBE1