Enzyme replacement therapies
Some of the worst human genetic diseases are caused by missing enzymes. They can, however, be easily substituted – but only if the substitute is human-like and efficient. Moss plants are perfectly suited to successful humanization of enzymes. Capitalizing on this, we develop enzymes that are tolerated by the human immune system and easily taken up into the relevant cells, making a world of a difference to patients.
For an injected replacement enzyme in the blood stream to be taken up into the cells, its N-glycan structure (sugar residues on the surface) is crucial. The right pattern and homogeneity determine the therapeutic success. Studies have shown that 95 % of moss N-glycans are relevant for best uptake of the recombinant enzyme into the cell and for optimal organ distribution.
Repleva AGAL/RPV-001: glyco-improved Fabry-ERT
Repleva AGAL for patients suffering from Fabry disease
In Fabry disease, patients are deficient of the enzyme a-galactosidase (aGAL). Without aGAL, glycogen accumulates in blood vessels and organs, which causes excruciating pain and other severe symptoms. Repleva-aGAL cell uptake is faster than mammalian cell-based products, and is better targeted at the relevant organs.
Research
Preclinical
Phase I
Phase II
Repleva-AGAL has completed clinical stage I.
Repleva GAA/RPV-002: glyco-improved Pompe-ERT
Repleva GAA for Pompe disease
In another rare glycogen storage disorder, Pompe disease, the enzyme a-glucosidase (GAA) is not working properly. The subsequent glycogen build-up in muscle cells leads to respiratory insufficiency and is often fatal in the first 12 months of life. Moss-produced recombinant GAA shows superior uptake into muscular cells, thanks to the inherent glycan pattern of moss.
Research
Preclinical
Phase I
Phase II
Repleva GAA is in preclinical stages.