Moss-GBA for patients suffering
from Gaucher's disease
Moss-GBA is the proprietary drug candidate of Greenovation for enzyme replacement therapy of patients suffering from Gaucher's disease, which is the most common lysosomal storage disease in humans.
This proprietary moss-expressed protein of the human enzyme glucocerebrosidase (GBA) is intended for treatment of patients suffering from hereditary GBA-deficiency called Gaucher's disease.
GBA deficiency leads to accumulation of glucosylceramide particularly in white blood cells, most often macrophages. Particularly spleen, liver, kidneys, lungs, brain, and bone marrow are affected by this accumulation.
Therapy of Gaucher's disease is done by regular infusion of a biotechnologically produced substitute of the missing enzyme. This treatment approach is called enzyme replacement therapy (ERT).
Moss-GBA is produced in the moss system Physcomitrella patents and therefore benefits from optimized
N-glycosylation. Up to 100 % of the mannose-terminated glycans lead to efficient uptake via mannose-receptors, and sterically optimized glycans enable efficient receptor binding, thus leading to improved enzyme uptake into target cells.
Our moss-GBA product is currently in preclinical testing.