Greenovation, a German Biotech company based in Freiburg, has announced a phase I clinical trial application for its drug candidate moss-alpha-galactosidase (moss-aGal). This moss-made biopharmaceutical compound is intended for use as enzyme replacement therapy in patients suffering from Fabry Disease. The trial design should provide safety data on the molecule and initial information about efficacy.
Fabry Disease is a rare genetic lysosomal storage disorder, caused by subnormal or absent activity of the lysosomal enzyme α-galactosidase A. It affects about 1–5 in 10,000 people worldwide. Because of the lacking enzyme activity glycosphingolipids are accumulated in most tissues and cell types leading to symptoms like extreme pain, renal impairment and cardiomyopathy. The usual enzyme replacement therapy is based on the infusion of enzyme substitutes, which are biotechnologically produced in different mammalian cell lines. These substitutes exhibit a remarkable heterogeneity in N-glycosylation with only a minor fraction being relevant for target cell uptake.
In contrast, Greenovation developed moss-aGal on its own Bryotechnology platform, using the moss system Physcomitrella patens. These organisms show an inherent N-glycan homogeneity, which should facilitate the uptake of the moss-made compound into human target cells. Preclinical studies demonstrated enhanced cell uptake and convenient organ distribution of moss-aGal. More than 95% of the moss N-glycans are relevant for the process. Furthermore, the amino acid sequence of the moss-made compound is identical to the human enzyme.
“Our vision is to provide moss-made enzymes that allow a safer and better treatment of patients suffering from rare diseases. So we are very excited to move our first drug candidate moss-aGal into phase I,” says Dr. Thomas Frischmuth, CEO of Greenovation.
Besides moss-aGal the biotech company is also developing biopharmaceuticals relevant for the treatment of Gaucher’s Disease, Pompe Disease and atypical hemolytic uremic syndrome (HUS), and is running antibody programs for enhanced antibody dependent cellular cytotoxicity (ADCC).