As the Heilbronn based biotechnological company Greenovation announced recently, it has permission from the German Federal Institute for Drugs and Medical Devices to start a phase I clinical trial in Europe for its first drug candidate moss-aGal. The protein could be used as an enzyme replacement therapy for patients who suffer from Fabry disease, a rare genetic lysosomal storage disorder.
“This milestone validates our BryoTechnology approach, confirming that it can serve as a production system for biopharmaceuticals”, commented CEO Dr. Thomas Fischmuth on the decision of Germany’s regulatory authority. “We have reached this goal through many years of hard work from all of our team.”
Moss-aGal is a recombinant form of human alpha galactosidase. Greenovation has used its proprietary BryoTechnology to express the moss-aGal protein in Physcomitrella patens. The technology attaches mannose molecules to the surface of the moss aGal protein. This improves the uptake of the proteins in the human body, mediated via mannose receptors on cell surfaces. As demonstrated by preclinical studies, this novel approach is very effective.
Fabry disease is caused by an inborn deficiency of the alpha-galactosidase (aGal) enzyme. In healthy persons aGal breaks down a specific fatty acid, globotriaosylceramide (Gb3). In patients with Fabry disease, however, Gb3 is accumulating continuously within the cells. Severe symptoms like pain and heart, skin and kidney complications occur, which can lead to organ failure. The missing enzyme can be replaced with regular intravenous administrations of a biopharmaceutically produced substitute as the world’s first moss-produced drug candidate with its promising potential could it be in the future.