JP6527149B2 - ベータ−サラセミアの処置のための方法及び医薬組成物 - Google Patents
ベータ−サラセミアの処置のための方法及び医薬組成物 Download PDFInfo
- Publication number
- JP6527149B2 JP6527149B2 JP2016534904A JP2016534904A JP6527149B2 JP 6527149 B2 JP6527149 B2 JP 6527149B2 JP 2016534904 A JP2016534904 A JP 2016534904A JP 2016534904 A JP2016534904 A JP 2016534904A JP 6527149 B2 JP6527149 B2 JP 6527149B2
- Authority
- JP
- Japan
- Prior art keywords
- xpo1
- pharmaceutical composition
- compound
- inhibits
- phenyl
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Expired - Fee Related
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/4196—1,2,4-Triazoles
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/397—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having four-membered rings, e.g. azetidine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/4245—Oxadiazoles
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/433—Thidiazoles
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
- A61K31/4439—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
- A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
- A61K31/454—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
- A61K31/4965—Non-condensed pyrazines
- A61K31/497—Non-condensed pyrazines containing further heterocyclic rings
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
- A61K31/5375—1,4-Oxazines, e.g. morpholine
- A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
- G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
- G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
- G01N33/5073—Stem cells
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2500/00—Screening for compounds of potential therapeutic value
- G01N2500/02—Screening involving studying the effect of compounds C on the interaction between interacting molecules A and B (e.g. A = enzyme and B = substrate for A, or A = receptor and B = ligand for the receptor)
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2500/00—Screening for compounds of potential therapeutic value
- G01N2500/10—Screening for compounds of potential therapeutic value involving cells
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Hematology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Urology & Nephrology (AREA)
- Molecular Biology (AREA)
- Cell Biology (AREA)
- Diabetes (AREA)
- Food Science & Technology (AREA)
- Analytical Chemistry (AREA)
- Microbiology (AREA)
- Tropical Medicine & Parasitology (AREA)
- Developmental Biology & Embryology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP13306628 | 2013-11-28 | ||
| EP13306628.2 | 2013-11-28 | ||
| PCT/EP2014/075980 WO2015079031A1 (en) | 2013-11-28 | 2014-11-28 | Methods and pharmaceutical compositions for the treatment of beta-thalassemias |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017502929A JP2017502929A (ja) | 2017-01-26 |
| JP2017502929A5 JP2017502929A5 (https=) | 2017-12-28 |
| JP6527149B2 true JP6527149B2 (ja) | 2019-06-05 |
Family
ID=49713040
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016534904A Expired - Fee Related JP6527149B2 (ja) | 2013-11-28 | 2014-11-28 | ベータ−サラセミアの処置のための方法及び医薬組成物 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US9861615B2 (https=) |
| EP (1) | EP3074007B1 (https=) |
| JP (1) | JP6527149B2 (https=) |
| ES (1) | ES2739637T3 (https=) |
| WO (1) | WO2015079031A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020247654A1 (en) * | 2019-06-06 | 2020-12-10 | The Henry M. Jackson Foundation For The Advancement Of Military Medicine, Inc. | Setbp1 and xpo1 inhibitors for the treatment of sickle cell disease and beta-thalassemia |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2542089B1 (en) * | 2010-03-05 | 2017-01-18 | Karyopharm Therapeutics, Inc. | Nuclear transport modulators and uses thereof |
| EP2665362B1 (en) * | 2011-01-17 | 2016-03-30 | Karyopharm Therapeutics, Inc. | Olefin containing nuclear transport modulators and uses thereof |
| EP4234545A3 (en) * | 2011-07-29 | 2023-09-06 | Karyopharm Therapeutics Inc. | Hydrazide containing nuclear transport modulators and uses thereof |
| JP6006794B2 (ja) * | 2011-07-29 | 2016-10-12 | カリオファーム セラピューティクス,インコーポレイテッド | 核内輸送調節因子およびその使用 |
| WO2013020024A2 (en) * | 2011-08-03 | 2013-02-07 | Karyopharm Therapeutics, Inc. | Maleimide compounds and methods of treatment |
-
2014
- 2014-11-28 ES ES14808905T patent/ES2739637T3/es active Active
- 2014-11-28 JP JP2016534904A patent/JP6527149B2/ja not_active Expired - Fee Related
- 2014-11-28 EP EP14808905.5A patent/EP3074007B1/en not_active Not-in-force
- 2014-11-28 WO PCT/EP2014/075980 patent/WO2015079031A1/en not_active Ceased
- 2014-11-28 US US15/039,230 patent/US9861615B2/en not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| ES2739637T3 (es) | 2020-02-03 |
| US9861615B2 (en) | 2018-01-09 |
| JP2017502929A (ja) | 2017-01-26 |
| EP3074007A1 (en) | 2016-10-05 |
| EP3074007B1 (en) | 2019-07-03 |
| WO2015079031A1 (en) | 2015-06-04 |
| US20170020847A1 (en) | 2017-01-26 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| Zhou et al. | Microglial debris is cleared by astrocytes via C4b-facilitated phagocytosis and degraded via RUBICON-dependent noncanonical autophagy in mice | |
| JP5342834B2 (ja) | 骨髄線維症処置剤 | |
| EP2679221B1 (en) | Method to enhance tissue regeneration | |
| US20210317461A1 (en) | Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof | |
| JP2026048892A (ja) | 状態および疾患の処置のためのアンチセンスオリゴマー | |
| JP7712673B2 (ja) | 神経の疾患を検出し、予防し、回復に向かわせ、治療する方法 | |
| JP2018531046A (ja) | 核酸ベースのtia−1阻害剤 | |
| US11345738B2 (en) | Compounds for treating neurodegenerative proteinopathies | |
| JP2025525099A (ja) | 状態および疾患の治療のための化合物 | |
| WO2017180841A1 (en) | Treatment of cerebral cavernous malformations | |
| JP2026507323A (ja) | アンチセンスオリゴマー製剤 | |
| JP6527149B2 (ja) | ベータ−サラセミアの処置のための方法及び医薬組成物 | |
| WO2021081580A1 (en) | Treatment of renal cystic disease | |
| US20220143128A1 (en) | Methods for treating traumatic brain injury | |
| US9675590B2 (en) | Methods and pharmaceutical compositions for the treatment of myelodysplastic syndromes | |
| JPWO2015046574A1 (ja) | 虚血後の再灌流に起因する出血を予防するための薬剤 | |
| WO2015174443A1 (ja) | インターロイキン1α(IL-1α)を含有する巨核球分化誘導及び血小板産生促進剤 | |
| WO2015035414A1 (en) | Treating neurological disorders | |
| US20160304881A1 (en) | Ddr1 antagonist or an inhibitor of ddr1 gene expression for use in the prevention or treatment of crescentic glomerulonephritis | |
| US20250066784A1 (en) | Therapeutic oligonucleotides | |
| Chavez et al. | Novel Therapies for | |
| JP2025540914A (ja) | Nlrp12およびnlrc5モジュレーター、ならびにそれらを使用して疾患を調節する方法 | |
| WO2025064402A1 (en) | Method for treating sickle cell disease by targeting senescent cells | |
| WO2024059335A1 (en) | Methods of treating age-related and inherited retinal dysfunctions | |
| TW202327566A (zh) | 包含藻褐素及小分子褐藻醣膠之組合物用於製備治療或改善慢性腎病之醫藥品的用途及包含藻褐素、小分子褐藻醣膠及左旋肉鹼之組合物及包含其的醫藥品 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20171117 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20171117 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20180807 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20181106 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20190108 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20190403 |
|
| TRDD | Decision of grant or rejection written | ||
| A01 | Written decision to grant a patent or to grant a registration (utility model) |
Free format text: JAPANESE INTERMEDIATE CODE: A01 Effective date: 20190416 |
|
| A61 | First payment of annual fees (during grant procedure) |
Free format text: JAPANESE INTERMEDIATE CODE: A61 Effective date: 20190509 |
|
| R150 | Certificate of patent or registration of utility model |
Ref document number: 6527149 Country of ref document: JP Free format text: JAPANESE INTERMEDIATE CODE: R150 |
|
| LAPS | Cancellation because of no payment of annual fees |