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Clinical Trials

Date: 2011-11-19

Type of information:

phase:

Announcement:

Company: UGent and Vrije Universiteit (Belgium) Oxyrane (Belgium-UK-USA)

Product: Enzyme Replacement Therapy (ERT)

Action mechanism:

Disease: Pompe disease and other metabolic disorders

Therapeutic area: Genetic diseases - Rare diseases - Metabolic diseases

Country:

Trial details:

Latest news: VIB researchers from UGent and Vrije Universiteit Brussel, together with a team of the firm Oxyrane have developed a new technology that can lead to a more efficient and possibly also cheaper therapy for diseases such as Pompe disease. The enzyme for the treatment of Pompe\'s disease is absorbed 17 times as efficiently by patient cells in vitro than the current therapeutic drug. In a Pompe disease mouse model the enzyme also worked better than the current therapeutic product. Based on these results Oxyrane will start clinical trials for Pompe disease, this means that it will be tested in humans for the first time.
Nico Callewaert (VIB-UGent): \"By combining natural processes and proteins of two microbes and a plant we have improved the technology to produce enzymes that patients with metabolic disorders can\'t produce themselves. We played with the sugar structures on the enzymes. As a result these enzymes were directed much more efficient to the locations in the cells where they actually have to work. It shows once again what medical biotechnology can accomplish.\"
Han Remaut (VIB-VUB): \"The lacking piece of the jigsaw in this technology was a protein we discovered in a soil bacterium. Thanks to the detailed picture we obtained from the 3D structure of this novel protein, we now understand how it comes to its unique reaction specificity and how we could further improve it\".

Is general: Yes