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The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Patent No.:

US 11065230 B1

PDFFull Text
Date of Patent:
Jul. 20, 2021

Filed:

Sep. 15, 2017

Methods and pharmaceutical compositions for the treatment of systemic mastocytosis

Applicants:

Inserm (Institut National DE LA Sante ET DE LA Recherche Medicale), Paris, FR;

Fondation Imagine, Paris, FR;

Universite Paris Descartes, Paris, FR;

Assistance Publique-hopitaux DE Paris (Aphp), Paris, FR;

Centre National DE LA Recherche Scientifique (Cnrs), Paris, FR;

Universite DE Caen Normandie, Caen, FR;

Centre Hospitalier Regional Universitaire DE Caen, Caen, FR;

Inventors:

Olivier Hermine, Paris, FR;

Flavia Guillem, Paris, FR;

Gandhi Damaj, Paris, FR;

Sophia Ladraa, Paris, FR;

Assignees:

INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), Paris, FR;

FONDATION IMAGINE, Paris, FR;

UNIVERSITE PARIS DESCARTES, Paris, FR;

ASSISTANCE PUBLIQUE—HOPITAUX DE PARIS (APHP), Paris, FR;

CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS), Paris, FR;

UNIVERSITE DE CAEN NORMANDIE, Caen, FR;

CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE CAEN, Caen, FR;

Attorney:

W&C IP

Primary Examiner:

Theodore R Howell

Int. Cl.
CPC ...
A61K 31/4196 (2006.01); A61P 37/00 (2006.01); A61K 31/506 (2006.01); A61K 31/397 (2006.01); A61K 31/4245 (2006.01); A61K 31/7105 (2006.01); A61P 35/04 (2006.01); A61K 31/497 (2006.01);
U.S. Cl.
CPC ...
A61K 31/4196 (2013.01); A61K 31/397 (2013.01); A61K 31/4245 (2013.01); A61K 31/506 (2013.01); A61P 37/00 (2018.01); A61K 31/497 (2013.01); A61K 31/7105 (2013.01); A61P 35/04 (2018.01); C12N 2310/14 (2013.01); C12N 2310/531 (2013.01);
Abstract

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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