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Methods and pharmaceutical compositions for the treatment of beta-thalassemias
US9861615B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9861615-B2 |
| Application number | US-201415039230-A |
| Country | US |
| Kind code | B2 |
| Filing date | Nov 28, 2014 |
| Priority date | Nov 28, 2013 |
| Publication date | Jan 9, 2018 |
| Grant date | Jan 9, 2018 |
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- Title
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Abstract
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The present invention relates to methods and pharmaceutical compositions for the treatment of beta-thalassemias. In particular, the present invention relates to an XPO1 inhibitor for use in a method for treating beta-thalassemia in a subject in need thereof.
First claim
Opening claim text (preview).
The invention claimed is: 1. A method of restoring or increasing erythrocyte maturation in a subject suffering from β-thalassemia major (β-TM) by preventing proteolytic inactivation of GATA-1, wherein preventing is achieved by administering to the subject a compound that is an XPO1 inhibitor of an XPO1 nuclear transporter, wherein the XPO1 inhibitor is selected from the group consisting of: 2. The method of claim 1 , wherein the XPO1 inhibitor is KPT-330 which has a formula: 3. A method of restoring or increasing erythrocyte maturation in a subject suffering from β-thalassemia major (β-TM) by preventing proteolytic inactivation of GATA-1, wherein preventing is achieved by administering to the subject a compound that is an XPO1 inhibitor of an XPO1 nuclear transporter, wherein the XPO1 inhibitor has a formula 4. A method of treating beta-thalassemia in a subject in need thereof, or for promoting or restoring erythrocyte maturation and/or increasing the production of (α/γ) 2 Hb erythrocytes in a subject suffering from beta-thalassemia, comprising administering to the subject a therapeutically effective amount of an XPO1 inhibitor wherein the XPO1 is selected from the group consisting of 5. A method of restoring or increasing erythrocyte maturation in a subject suffering from β-thalassemia major (β-TM) by preventing proteolytic inactivation of GATA-1, wherein preventing is achieved by administering to the subject a compound that is an XPO1 inhibitor of an XPO1 nuclear transporter, wherein the XPO1 inhibitor is KPT-330 which has a formula: 6. A method of treating beta-thalassemia in a subject in need thereof, or for promoting or restoring erythrocyte maturation and/or increasing the production of (α/γ) 2 Hb erythrocytes in a subject suffering from beta-thalassemia, comprising administering to the subject a therapeutically effective amount of an XPO1 inhibitor wherein the XPO1 inhibitor has a formula:
Assignees
Inventors
Classifications
Drugs for disorders of the blood or the extracellular fluid · CPC title
Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title
Antianaemics · CPC title
Stem cells · CPC title
Thidiazoles · CPC title
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Frequently asked questions
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- What does patent US9861615B2 cover?
- The present invention relates to methods and pharmaceutical compositions for the treatment of beta-thalassemias. In particular, the present invention relates to an XPO1 inhibitor for use in a method for treating beta-thalassemia in a subject in need thereof.
- Who is the assignee on this patent?
- Inserm (Institut National De La Sante Et De La Rech Medicale), Univ Paris Descartes, Assist Publique-Hopitaux De Paris (Aphp), and 3 more
- What technology area does this patent fall under?
- Primary CPC classification A61K31/4196. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Tue Jan 09 2018 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). 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).