vanglusagene ensiparvovec

Vanglusagene ensiparvovec (SPK-3006) is an investigational gene therapy developed by Spark Therapeutics (a member of the Roche Group) for the treatment of late-onset Pompe disease (LOPD). It utilizes a bio-engineered adeno-associated virus (AAV) capsid, AAV-LK03, to deliver a functional human acid alpha-glucosidase (GAA) gene to hepatocytes. This "liver-depot" approach is designed to enable the liver to continuously produce and secrete the GAA enzyme into the bloodstream, where it can be taken up by muscle cells via the cation-independent mannose-6-phosphate receptor. By providing a steady endogenous source of the enzyme, vanglusagene ensiparvovec aims to clear glycogen accumulation in cardiac and skeletal muscles, potentially offering a more durable and effective alternative to traditional enzyme replacement therapy (ERT), which requires frequent lifelong infusions.

Other names

SPK-3006SPK3006SPK 3006RG6358RG-6358RG 6358