Compositions and methods for targeted delivery to cells
US-2024390271-A1 · Nov 28, 2024 · US
Methods and pharmaceutical compositions for the treatment of systemic mastocytosis
US2019247416A1 · US · A1
| Field | Value |
|---|---|
| Publication number | US-2019247416-A1 |
| Application number | US-201716330956-A |
| Country | US |
| Kind code | A1 |
| Filing date | Sep 15, 2017 |
| Priority date | Sep 16, 2016 |
| Publication date | Aug 15, 2019 |
| Grant date | — |
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- Title
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- Abstract
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Abstract
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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.
First claim
Opening claim text (preview).
1 . A method of treating systemic mastocytosis in a patient in need thereof comprising administering to the patient a therapeutically effective amount of a XPO1 inhibitor. 2 . The method of claim 1 wherein the patient suffers from indolent SM (ISM), SM with associated clonal hematologic non-MC disease, aggressive SM (ASM), or MC leukemia (MCL). 3 . The method of claim 1 wherein the patient harbors the KIT mutation D816V or Delta 417-419 insY. 4 . The method of claim 1 wherein the XPO1 inhibitor is KPT-330, KPT-276 or KPT-251. 5 . The method of claim 1 wherein the XPO1 inhibitor is a short hairpin RNA (shRNA), a small interfering RNA (siRNA) or an antisense oligonucleotide which inhibits the expression of XPO1.
Assignees
Inventors
Classifications
Antineoplastic agents · CPC title
interfering nucleic acids [NA] · CPC title
specific for metastasis · CPC title
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)} · CPC title
- A61K31/7105Primary
Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links · CPC title
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- What does patent US2019247416A1 cover?
- 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) tha…
- Who is the assignee on this patent?
- Inst Nat Sante Rech Med, Fond Imagine, Univ Paris Descartes, and 4 more
- What technology area does this patent fall under?
- Primary CPC classification A61K31/7105. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Thu Aug 15 2019 00:00:00 GMT+0000 (Coordinated Universal Time) (A1). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).