moss-GAA

**moss-GAA** is a recombinant form of human acid alpha-glucosidase (GAA) produced in the moss _Physcomitrella patens_. It is developed as an **enzyme replacement therapy** (ERT) for **Glycogen Storage Disease Type II (Pompe disease)**, a lysosomal storage disorder characterized by GAA deficiency leading to glycogen accumulation, particularly in cardiac and skeletal muscle. Unlike currently approved ERTs, moss-GAA can be glycoengineered (such as the GnGn variant) to enhance tissue uptake, particularly by skeletal muscle, and is produced using a plant-based expression platform that provides advantages in terms of safety (no animal-derived components) and controllable glycosylation profiles. Preclinical studies have shown that moss-GAA can efficiently clear glycogen accumulation from muscle cells, with some variants outperforming conventional therapies in cellular uptake and efficacy[1][2][3][4][5][6].

Other names

moss-GAA GnGn