DEVELOPMENT OF MOLECULES THAT REVERSE THE MOLECULAR PHENOTYPE OF ARSACS

In News Archives by ARSACS

The IRIC HTS platform completed a screen of 16 000 molecules on a cell line engineered by Dr Brais’ team showing the characteristic vimentin bundling of ARSACS. This led to the identification of a set of potential candidate compounds, of which the two most promising lead to an almost complete disappearance of the bundling without signs of cellular toxicity. Medicinal chemistry efforts are presently focused on designing new molecules based on the identified active chemotype to improve the desired properties to be tested in a pre-clinical using the already existing transgenic mouse model to demonstrate efficacy. In parallel, Dr Brais’ team developed native assays to translate the molecules’ activity into more physiological systems which will shed light on the biology mechanisms of the disease and how the candidate drugs work.

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