Pompe disease is a rare, congenital metabolic muscle disorder. Patients diagnosed with Pompe disease are lacking the enzyme alpha-glucosidase, which is involved in the breakdown of glycogen in muscle cells. Oxyrane Belgium and the Dutch company BioscienZ decided to join forces to optimise the production process of a candidate therapeutic product for Pompe disease. This collaboration, financially supported by CrossRoads2, has the ultimate goal to offer a cost-effective alternative for the currently best available therapy.
The alpha-glucosidase enzyme is responsible for the breakdown of glycogen, a polysaccharide stored in muscle cells. Pompe disease is characterised by a deficiency of the alpha-glucosidase enzyme, causing accumulation of glycogen in the lysosomes of muscle cells. As a result, Pompe diseased patients experience progressive muscle weakness, especially affecting heart, skeletal muscles, liver and nervous system. In the KEPTYL project, Oxyrane (Ghent, Belgium) and BioscienZ (Breda, The Netherlands) are collaborating to develop a technology platform with the potential to produce alpha-glucosidase in a cost-effective way.
Proven lab results
Oxyrane successfully developed a yeast based expression platform to support the production of an efficacious alpha-glucosidase enzyme that can be targeted to the right cellular compartment in the muscle cell. This enzyme proved to be highly effective in a Pompe disease mouse model. The main challenge now is to optimise the production process in terms of product yield to offer an economical and affordable alternative for the currently best available treatment for Pompe disease (yearly cost per patient/year exceeds 300.000 euro).
To support the development of the envisioned cost-effective production process, Oxyrane will rely on the expertise of BioscienZ, a Dutch company with a proven track record in qualitative screening methods, process development and upscaling of bio-technological processes. During this CrossRoads2-project, the yeast with the highest production capacity will be selected for process upscaling in bioreactors. This project serves the ultimate goal to provide Pompe diseased patients with the most effective and affordable treatment.Download full press release Return to press releases/News >