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. Repleca-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.

Contact us for additional information

I consent to the processing of my personal data for the purpose of establishing contact in accordance with Article 6 paragraph 1 lit a of the Basic Data Protection Regulation (DSGVO).

In accordance with Article 13 of the Basic Data Protection Regulation (DSGVO), we would like to provide you with comprehensive information about the processing of your personal data. You can read all relevant information in our data protection policy.