发明申请
- 专利标题： METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT OF SYSTEMIC MASTOCYTOSIS
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申请号： US17324690申请日： 2021-05-19
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公开(公告)号： US20210308106A1公开(公告)日： 2021-10-07
- 发明人: Olivier HERMINE , Flavia GUILLEM , Gandhi DAMAJ , Sophia LADRAA
- 申请人： INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) , FONDATION IMAGINE , UNIVERSITE PARIS DESCARTES , ASSISTANCE PUBLIQUE-HOPITAUX DE PARIS (APHP) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , UNIVERSITE DE CAEN NORMANDIE , CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE CAEN
- 申请人地址： FR Paris; FR Paris; FR Paris; FR Paris; FR Paris; FR Caen; FR Caen
- 专利权人： INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE),FONDATION IMAGINE,UNIVERSITE PARIS DESCARTES,ASSISTANCE PUBLIQUE-HOPITAUX DE PARIS (APHP),CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS),UNIVERSITE DE CAEN NORMANDIE,CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE CAEN
- 当前专利权人： INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE),FONDATION IMAGINE,UNIVERSITE PARIS DESCARTES,ASSISTANCE PUBLIQUE-HOPITAUX DE PARIS (APHP),CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS),UNIVERSITE DE CAEN NORMANDIE,CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE CAEN
- 当前专利权人地址： FR Paris; FR Paris; FR Paris; FR Paris; FR Paris; FR Caen; FR Caen
- 优先权： EP16306181.5 20160916
- 主分类号： A61K31/4196
- IPC分类号： A61K31/4196 ; A61K31/506 ; A61P37/00 ; A61K31/397 ; A61K31/4245
摘要：
The present invention relates to methods and pharmaceutical compositions for the treatment of systemic mastocytosis. The inventors showed the effect of KPT-251 treatment on SCF-dependent human Mast cell (MC) line without KIT mutation (WT ROSA) and on two factor-independent MC lines with KIT mutations: ROSA Δ 417-419 insY and ROSA D816V. KPT is a Selective Inhibitor of Nuclear Export (SINE) that specifically inhibits the activity of the exportin-1 (XPO1). KPT-251 treatment induces minimal toxicity in non-cancerous hematopoietic cells both in vitro and in vivo. In particular, the present invention relates a method of treating systemic mastocytosis in patient in need thereof comprising administering to the patient a therapeutically effective amount of a XPO1 inhibitor.
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