Conception of chemical chaperones

Last Updated on Wednesday, 12 January 2011 09:22

Conception and evaluation of new chemical chaperones of the N-acetyglucosaminidase in the context of the development of a treatment for Sanfilippo Syndrome (MPS III B).

This research and development project, organized by the team of Professor Matthieu Sollogoub (CNRS / UMR, Université Pierre and Marie Curie, Paris), consists of developing glycosidic inhibitors of the N-acetyglucosaminidase.

It is expected that these new molecules will be able to correct the structure and intracellular transport of certain mutated forms of the N-acetyglucosaminidase and therefore restore the ability to degrade heparan sulfate. The total budget of nearly 300,000 Euros will be used initially for the development of the method of production of these new glycosidic structures. In the next stage, funding will be devoted to evaluating these molecules in the cellular in-vitro models of the disease.

It should be noted that the Foundation has taken appropriate measures to protect intellectual property (patents) issuing from the work it finances.