A xpo1 inhibitor for the treatment of myelodysplastic syndromes

The present invention provides methods and pharmaceutical compositions designed to intervene in this defective process and to promote or restore erythrocyte maturation in individuals suffering from a myelodysplastic syndrome. The methods involve maintaining the activity of GATA-1 by preventing sequestration of Hsp70 in the cytoplasm. Accordingly, it is an object of this invention to provide methods of restoring or increasing erythrocyte maturation in a subject suffering from a myelodysplastic syndrome by preventing proteolytic inactivation of GATA-1. In some embodiments, preventing is achieved by administering to the subject a compound that inhibits the XPO1 nuclear transporter.

Keywords: Oncology - Myelodysplastic Syndromes - Small molecule - XPO1 inhibitor
Patent Application number: European Procedure (Patents) (EPA) - 10 Juin 2014 - 14 305 873.3
Inventors:
FONTENAY Michaela,ARLET Jean-benoît,GUILLEM Flavia,COURTOIS Genevieve

Reference:

BIO14070-T1

Business Developper
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Aymeric Empereur
Business Developer
Rare disease: No
Second indication: No

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