GD-BT6822

GD-BT6822 is an investigational enzyme replacement therapy (ERT) being developed by BioArctic for the treatment of Gaucher disease, including its neuropathic forms. The therapy utilizes BioArctic's proprietary BrainTransporter™ technology to deliver the enzyme glucocerebrosidase (GCase) across the blood-brain barrier (BBB). Gaucher disease is a rare genetic disorder caused by a deficiency in GCase, which leads to the toxic accumulation of glucosylceramide in various organs. While current ERTs effectively address peripheral symptoms, they are unable to cross the BBB to treat central nervous system manifestations. GD-BT6822 is designed to overcome this limitation by facilitating active transport into the brain, potentially providing a comprehensive treatment for both the systemic and neurological aspects of the disease. The program was initiated in late 2022 and is currently in the research stage.

Other names

GCase with BrainTransporter