RU2011122461A - Модуляторы атф-связывающих кассетных транспортеров - Google Patents
Модуляторы атф-связывающих кассетных транспортеров Download PDFInfo
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- RU2011122461A RU2011122461A RU2011122461/04A RU2011122461A RU2011122461A RU 2011122461 A RU2011122461 A RU 2011122461A RU 2011122461/04 A RU2011122461/04 A RU 2011122461/04A RU 2011122461 A RU2011122461 A RU 2011122461A RU 2011122461 A RU2011122461 A RU 2011122461A
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- 150000001875 compounds Chemical class 0.000 claims abstract 31
- 238000000034 method Methods 0.000 claims abstract 17
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims abstract 13
- 239000000203 mixture Substances 0.000 claims abstract 12
- 108010006533 ATP-Binding Cassette Transporters Proteins 0.000 claims abstract 10
- 102000005416 ATP-Binding Cassette Transporters Human genes 0.000 claims abstract 10
- 201000010099 disease Diseases 0.000 claims abstract 9
- 230000000694 effects Effects 0.000 claims abstract 6
- 208000035475 disorder Diseases 0.000 claims abstract 4
- 206010014561 Emphysema Diseases 0.000 claims abstract 3
- 210000004027 cell Anatomy 0.000 claims abstract 3
- 239000003795 chemical substances by application Substances 0.000 claims abstract 3
- 230000007812 deficiency Effects 0.000 claims abstract 3
- 108010039209 Blood Coagulation Factors Proteins 0.000 claims abstract 2
- 102000015081 Blood Coagulation Factors Human genes 0.000 claims abstract 2
- 201000003883 Cystic fibrosis Diseases 0.000 claims abstract 2
- 108010071289 Factor XIII Proteins 0.000 claims abstract 2
- 208000033981 Hereditary haemochromatosis Diseases 0.000 claims abstract 2
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- 201000005660 Protein C Deficiency Diseases 0.000 claims abstract 2
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims abstract 2
- 239000003242 anti bacterial agent Substances 0.000 claims abstract 2
- 229940121363 anti-inflammatory agent Drugs 0.000 claims abstract 2
- 239000002260 anti-inflammatory agent Substances 0.000 claims abstract 2
- -1 antibiotic Substances 0.000 claims abstract 2
- 230000003115 biocidal effect Effects 0.000 claims abstract 2
- 239000003114 blood coagulation factor Substances 0.000 claims abstract 2
- 229940019700 blood coagulation factors Drugs 0.000 claims abstract 2
- 239000000168 bronchodilator agent Substances 0.000 claims abstract 2
- 210000000170 cell membrane Anatomy 0.000 claims abstract 2
- 230000007547 defect Effects 0.000 claims abstract 2
- 239000003937 drug carrier Substances 0.000 claims abstract 2
- 239000003623 enhancer Substances 0.000 claims abstract 2
- 239000003172 expectorant agent Substances 0.000 claims abstract 2
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims abstract 2
- 208000013746 hereditary thrombophilia due to congenital protein C deficiency Diseases 0.000 claims abstract 2
- 150000002632 lipids Chemical class 0.000 claims abstract 2
- 229940066491 mucolytics Drugs 0.000 claims abstract 2
- 230000007170 pathology Effects 0.000 claims abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 claims abstract 2
- 239000012472 biological sample Substances 0.000 claims 3
- 239000012634 fragment Substances 0.000 claims 3
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
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- 208000029402 Bulbospinal muscular atrophy Diseases 0.000 claims 1
- 208000010693 Charcot-Marie-Tooth Disease Diseases 0.000 claims 1
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 208000020406 Creutzfeldt Jacob disease Diseases 0.000 claims 1
- 208000003407 Creutzfeldt-Jakob Syndrome Diseases 0.000 claims 1
- 208000010859 Creutzfeldt-Jakob disease Diseases 0.000 claims 1
- 208000003556 Dry Eye Syndromes Diseases 0.000 claims 1
- 206010013883 Dwarfism Diseases 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 206010060378 Hyperinsulinaemia Diseases 0.000 claims 1
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- 208000027747 Kennedy disease Diseases 0.000 claims 1
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- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 1
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- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 1
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- XOFYZVNMUHMLCC-ZPOLXVRWSA-N prednisone Chemical compound O=C1C=C[C@]2(C)[C@H]3C(=O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 XOFYZVNMUHMLCC-ZPOLXVRWSA-N 0.000 claims 1
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- 239000004576 sand Substances 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 229940126214 compound 3 Drugs 0.000 abstract 1
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- C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
- C07D405/02—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings
- C07D405/12—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing two hetero rings linked by a chain containing hetero atoms as chain links
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Abstract
1. Соединение, выбранное из2. Соединение3. Соединение4. Соединение5. Фармацевтическая композиция, содержащая(i) соединение по п.1 и(ii) фармацевтически приемлемый носитель.6. Композиция по п.5, дополнительно содержащая дополнительное средство, выбранное из муколитического средства, бронхолитического средства, антибиотика, антибактериального средства, противовоспалительного средства, корректора CFTR, усилителя CFTR или питательного средства.7. Композиция по п.5 или 6, в которой соединение представляет собой8. Композиция по п.5 или 6, в которой соединение представляет собой9. Композиция по п.5 или 6, в которой соединение представляет собой10. Способ повышения количества функцинальных ABC-транспортеров в мембране клетки, включающий стадию контактирования указанной клетки с соединением, выбранным из11. Способ по п.10, в котором ABC-транспортер представляет собой CFTR.12. Способ по п.10 или 11, в котором соединение представляет собой13. Способ по п.10 или 11, в котором соединение представляет собой14. Способ по п.10 или 11, в котором соединение представляет собой15. Способ лечения состояния, заболевания или расстройства у пациента, в патологии которого задействована активность ABC-транспортера, включающий стадию введения указанному пациенту соединения, выбранного из16. Способ по п.15, в котором указанное состояние, заболевание или расстройство выбрано из муковисцидоза, наследственной эмфиземы, наследственного гемохроматоза, недостаточностей факторов свертывания крови и фибринстабилизирующего фактора, таких как дефицит белка С, наследственный ангионеврологический отек 1 типа, дефектов в процессинге липидов, таких как семейная гиперхолестеринем�
Claims (25)
5. Фармацевтическая композиция, содержащая
(i) соединение по п.1 и
(ii) фармацевтически приемлемый носитель.
6. Композиция по п.5, дополнительно содержащая дополнительное средство, выбранное из муколитического средства, бронхолитического средства, антибиотика, антибактериального средства, противовоспалительного средства, корректора CFTR, усилителя CFTR или питательного средства.
11. Способ по п.10, в котором ABC-транспортер представляет собой CFTR.
16. Способ по п.15, в котором указанное состояние, заболевание или расстройство выбрано из муковисцидоза, наследственной эмфиземы, наследственного гемохроматоза, недостаточностей факторов свертывания крови и фибринстабилизирующего фактора, таких как дефицит белка С, наследственный ангионеврологический отек 1 типа, дефектов в процессинге липидов, таких как семейная гиперхолестеринемия, хиломикронемия 1 типа, абеталипопротеинемия, лизосомных болезней, таких как болезнь клеточных включений/псевдо-Хюрлера, мукополисахаридозы, болезнь Сандхоффа/Тея-Сакса, болезнь Криглера-Найяра II типа, полиэндокринопатия/гиперинсулинемия, сахарный диабет, карликовость Ларона, дефицит милеопероксидазы, первичный гипопаратиреоидизм, меланома, гликогеноз CDG 1 типа, наследственная эмфизема, врожденный гипертиреоидизм, несовершенный остеогенез, наследственная гипофибриногенемия, недостаточность ACT, несахарный диабет (di), нейрофизиологический di, нефрогенный DI, синдром Шарко-Мари-Тута, болезнь Пелицеуса-Мерцбахера, нейродегенеративных заболеваний, таких как болезнь Альцгеймера, болезнь Паркинсона, боковой амиотрофический склероз, прогрессирующий супрануклеарный паралич, болезнь Пика, некоторых неврологических расстройств, связанных с обменом глутамина, таких как болезнь Гентингтона, спинномозговая атаксия I типа, спинально-бульбарная мышечная атрофия, дентаторубральная и паллидолюисовая атрофия и миотоническая дистрофия, а также губчатых энцефалопатий, таких как наследственная болезнь Крейтцфельдта-Якоба, болезнь Фабри, синдром Штраусслера-Шейнкера, хроническое обструктивное заболевание легких (COPD), болезнь сухих глаз и синдром Шегрена.
20. Набор для измерения активности ABC-транспортера или его фрагмента в биологическом образце in vitro или in vivo, содержащий
(i) первую композицию, содержащую соединение, выбранное из соединения по п.1 и
(ii) инструкции для
а) контактирования композиции с биологическим образцом;
b) измерения активности указанного ABC-транспортера или его фрагмента.
21. Набор по п.20, дополнительно содержащий инструкции для
а) контактирования дополнительной композиции с биологическим образцом;
b) измерения активности указанного ABC-транспортера или его фрагмента в присутствии указанного дополнительного соединения и
c) сравнения активности ABC-транспортера в присутствии дополнительного соединения с плотностью ABC-транспортера в присутствии указанной первой композиции.
22. Набор по п.20, предназначенный для измерения плотности CFTR.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US11215208P | 2008-11-06 | 2008-11-06 | |
| US11214508P | 2008-11-06 | 2008-11-06 | |
| US61/112,152 | 2008-11-06 | ||
| US61/112,145 | 2008-11-06 | ||
| PCT/US2008/012689 WO2010053471A1 (en) | 2008-11-06 | 2008-11-12 | Modulators of atp-binding cassette transporters |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2013157877/04A Division RU2013157877A (ru) | 2008-11-06 | 2013-12-25 | Модуляторы атф-связывающих кассетных транспортеров |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2011122461A true RU2011122461A (ru) | 2012-12-20 |
| RU2512682C2 RU2512682C2 (ru) | 2014-04-10 |
| RU2512682C3 RU2512682C3 (ru) | 2024-02-09 |
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