Invention Grant
- Patent Title: Methods and pharmaceutical compositions for the treatment of myelodysplastic syndromes
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Application No.: US15316890Application Date: 2015-06-09
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Publication No.: US09675590B2Publication Date: 2017-06-13
- Inventor: Olivier Hermine , Flavia Guillem , Jean-Benoit Arlet , Genevève Courtois , Michaela Fontenay
- Applicant: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , FONDATION IMAGINE , ASSISTANCE PUBLIQUE-HÔPITAUX DE PARIS (APHP) , UNIVERSITÉ PARIS DESCARTES , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS)
- Applicant Address: FR Paris FR Paris FR Paris FR Paris FR Paris
- Assignee: Institut National de la Sante et de la Recherche Medicale (INSERM),Fondation Imagine,Assistance Publique-Hopitaux de Paris (APHP),Universite Paris Descartes,Centre National de la Recherche Scientifique (CNRS)
- Current Assignee: Institut National de la Sante et de la Recherche Medicale (INSERM),Fondation Imagine,Assistance Publique-Hopitaux de Paris (APHP),Universite Paris Descartes,Centre National de la Recherche Scientifique (CNRS)
- Current Assignee Address: FR Paris FR Paris FR Paris FR Paris FR Paris
- Agency: Whitham Curtis & Cook, PC
- Priority: EP14305873 20140610
- International Application: PCT/EP2015/062807 WO 20150609
- International Announcement: WO2015/189186 WO 20151217
- Main IPC: A61K31/422
- IPC: A61K31/422 ; A61K31/366

Abstract:
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.
Public/Granted literature
- US20170100378A1 METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT OF MYELODYSPLASTIC SYNDROMES Public/Granted day:2017-04-13
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