ERT producer platform

Lysosomal storage diseases (LSDs) are comparatively rare but grave genetic diseases, affecting mostly children who often die at a young and unpredictable age if untreated. Diverse and often severe symptoms are associated with LSDs. For example Fabry disease, a LSD characterized by deficiency in -galactosidase, is associated with pain crises, fever, fatigue and conditions such as ventricular hypertrophy. Fabry disease has a prevalence of roughly 1 in 50.000 in the European Union with associated costs of currently 200,000 € per patient per year, totalling more than 2 billion € annual health expenditures. There are no cures for LSDs currently available. A significant advance for the treatment of LSDs comes through enzyme replacement therapy (ERT). In ERT, the enzyme lacking in the patients is replaced by intrevenous infusion containing high-quality preparations of active enzyme.

Recombinant expression of biotherapeutics in high quality is critical for the treatment of LSDs by ERT. Overproduction in particular in yeast combines the eukaryotic capacity of performing post-translational modifications, notably glycosylation, with the microbial ability to grow to high cell densities in inexpensive media. Many therapeutic proteins require specific glycosylation patterns. A correct glycosylation pattern is a key parameter to ensure correct folding and function of a given protein biotherapeutic and its capacity to remain circulating in the bloodstream, as well as for interacting with the appropriate receptors.

The GLYCOMAM platform of Bioingenium in conjunction with large-scale production of selected proteins, ComplexINC will set the stage to produce sufficient amounts of suitable protein biotherapeutics for further tests and (pre)clinical studies towards treatment by ERT of LSDs.