PGB-D102

PGB-D102 is an investigational adeno-associated virus (AAV)-based gene therapy developed for the treatment of Duchenne muscular dystrophy (DMD). The therapy is designed as a one-time intravenous infusion that delivers a functional transgene encoding a shortened version of the dystrophin protein, often referred to as micro-dystrophin, to muscle cells. By restoring the expression of a functional dystrophin-like protein, PGB-D102 aims to stabilize muscle cell membranes, prevent further muscle degeneration, and improve motor function in patients. It is currently undergoing exploratory clinical evaluation in China to assess its safety, tolerability, and preliminary efficacy in ambulatory male children with DMD.