EA201791624A1 - СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНК - Google Patents
СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНКInfo
- Publication number
- EA201791624A1 EA201791624A1 EA201791624A EA201791624A EA201791624A1 EA 201791624 A1 EA201791624 A1 EA 201791624A1 EA 201791624 A EA201791624 A EA 201791624A EA 201791624 A EA201791624 A EA 201791624A EA 201791624 A1 EA201791624 A1 EA 201791624A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- compounds
- mrna
- improve
- splising
- methods
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D473/00—Heterocyclic compounds containing purine ring systems
- C07D473/26—Heterocyclic compounds containing purine ring systems with an oxygen, sulphur, or nitrogen atom directly attached in position 2 or 6, but not in both
- C07D473/32—Nitrogen atom
- C07D473/34—Nitrogen atom attached in position 6, e.g. adenine
-
- 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
- A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
- A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with 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
- A61K31/495—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
- A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
- A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
- A61K31/52—Purines, e.g. adenine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
- C07D417/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings
- C07D417/12—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings linked by a chain containing hetero atoms as chain links
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
- C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
- C07D471/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D473/00—Heterocyclic compounds containing purine ring systems
- C07D473/02—Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6
- C07D473/16—Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6 two nitrogen atoms
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
- C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
- C07D495/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D513/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00
- C07D513/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for in groups C07D463/00, C07D477/00 or C07D499/00 - C07D507/00 in which the condensed system contains two hetero rings
- C07D513/04—Ortho-condensed systems
Abstract
Описаны соединения, которые могут применяться для улучшения сплайсинга мРНК в клетке. Иллюстративные описанные в настоящем тексте соединения могут применяться для улучшения сплайсинга мРНК в генах, содержащих по меньшей мере один экзон, заканчивающийся нуклеотидной последовательностью CAA. Описаны также способы получения указанных соединений и способы лечения заболеваний центральной нервной системы.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562104547P | 2015-01-16 | 2015-01-16 | |
US201562180380P | 2015-06-16 | 2015-06-16 | |
PCT/US2016/013553 WO2016115434A1 (en) | 2015-01-16 | 2016-01-15 | Compounds for improving mrna splicing |
Publications (2)
Publication Number | Publication Date |
---|---|
EA201791624A1 true EA201791624A1 (ru) | 2018-06-29 |
EA037663B1 EA037663B1 (ru) | 2021-04-28 |
Family
ID=56406434
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA201791624A EA037663B1 (ru) | 2015-01-16 | 2016-01-15 | СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНК |
Country Status (16)
Country | Link |
---|---|
US (3) | US10676475B2 (ru) |
EP (2) | EP4115882A1 (ru) |
AU (2) | AU2016206589B2 (ru) |
BR (1) | BR112017015019A2 (ru) |
CA (2) | CA3199430A1 (ru) |
CL (2) | CL2017001823A1 (ru) |
DK (1) | DK3244891T3 (ru) |
EA (1) | EA037663B1 (ru) |
ES (1) | ES2928714T3 (ru) |
HU (1) | HUE059891T2 (ru) |
IL (2) | IL253291B (ru) |
MX (2) | MX2017009295A (ru) |
PL (1) | PL3244891T3 (ru) |
PT (1) | PT3244891T (ru) |
TW (2) | TWI727939B (ru) |
WO (1) | WO2016115434A1 (ru) |
Families Citing this family (20)
Publication number | Priority date | Publication date | Assignee | Title |
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EP4115882A1 (en) * | 2015-01-16 | 2023-01-11 | The General Hospital Corporation | Compounds for improving mrna splicing |
ES2879995T3 (es) | 2015-12-10 | 2021-11-23 | Ptc Therapeutics Inc | Métodos para el tratamiento de la enfermedad de Huntington |
CN110300590A (zh) * | 2016-10-21 | 2019-10-01 | 林伯士拉克许米公司 | Tyk2抑制剂及其用途 |
JP7129095B2 (ja) * | 2017-02-20 | 2022-09-01 | 国立大学法人京都大学 | スプライシング異常に起因する遺伝性疾病のための医薬組成物及び治療方法 |
EA201992878A1 (ru) | 2017-06-05 | 2020-05-08 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения болезни хантингтона |
BR112019027717A2 (pt) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics, Inc. | métodos para tratar a doença de huntington |
MX2019015580A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
CN112135815A (zh) | 2018-03-27 | 2020-12-25 | Ptc医疗公司 | 用于治疗亨廷顿氏病的化合物 |
AU2019294478B2 (en) | 2018-06-27 | 2023-03-23 | Ptc Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating Huntington's disease |
KR20210087033A (ko) * | 2018-10-01 | 2021-07-09 | 젠자임 코포레이션 | UDP 글리코실트랜스퍼라제 억제제로서의 티에노[3,2-b]피리딘 유도체 및 사용 방법 |
SG11202108519TA (en) | 2019-02-13 | 2021-09-29 | Ptc Therapeutics Inc | Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia |
EP3924050A1 (en) | 2019-02-13 | 2021-12-22 | PTC Therapeutics, Inc. | Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia |
SG11202109036WA (en) * | 2019-03-01 | 2021-09-29 | Revolution Medicines Inc | Bicyclic heteroaryl compounds and uses thereof |
MX2021010323A (es) * | 2019-03-01 | 2021-12-10 | Revolution Medicines Inc | Compuestos bicíclicos de heterociclilo y usos de este. |
MX2022005525A (es) | 2019-11-08 | 2022-06-08 | Revolution Medicines Inc | Compuestos de heteroarilo bicíclicos y usos de estos. |
KR20220113700A (ko) * | 2019-12-12 | 2022-08-16 | 피티씨 테라퓨틱스, 인크. | 가족성 자율신경실조증 치료를 위한 화합물 |
WO2021138678A1 (en) | 2020-01-02 | 2021-07-08 | The General Hospital Corporation | Rna splicing modulation |
CN111777612B (zh) * | 2020-06-05 | 2022-04-19 | 广东达元绿洲食品安全科技股份有限公司 | 一种6-苄基腺嘌呤半抗原、人工抗原及其在免疫检测中的应用 |
WO2022169864A1 (en) * | 2021-02-05 | 2022-08-11 | Ptc Therapeutics Inc. | Methods for treating spinocerebellar ataxia type 3 |
CN113151296B (zh) * | 2021-03-22 | 2022-09-13 | 云南中烟工业有限责任公司 | 一种烟草热激蛋白相关的基因及其应用 |
Family Cites Families (29)
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US3041340A (en) | 1962-06-26 | Method of preparing substituted | ||
US3037980A (en) * | 1955-08-18 | 1962-06-05 | Burroughs Wellcome Co | Pyrrolopyrimidine vasodilators and method of making them |
GB9613736D0 (en) | 1996-07-01 | 1996-09-04 | Hancock Tony | Transport cradle for a skid-supported helicopter |
AP1258A (en) | 1998-02-26 | 2004-03-15 | Aventis Pharma Inc | 6,9-Disubstituted 2-[trans-(4-aminocyclohexyl) amino] purines. |
CZ294535B6 (cs) | 2001-08-02 | 2005-01-12 | Ústav Experimentální Botaniky Avčr | Heterocyklické sloučeniny na bázi N6-substituovaného adeninu, způsoby jejich přípravy, jejich použití pro přípravu léčiv, kosmetických přípravků a růstových regulátorů, farmaceutické přípravky, kosmetické přípravky a růstové regulátory tyto sloučeniny obsahující |
AU2003304276A1 (en) | 2002-09-10 | 2005-01-21 | The Regents Of The University Of California | Methods and devices for determining a cell characteristic, and applications employing same |
CA2540681C (en) | 2003-10-03 | 2014-08-12 | The General Hospital Corporation | Methods for altering mrna splicing and treating familial dysautonomia and other mechanistically related disorders |
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JP2011136925A (ja) * | 2009-12-28 | 2011-07-14 | Dainippon Sumitomo Pharma Co Ltd | 含窒素二環性化合物 |
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EA035030B1 (ru) | 2011-11-18 | 2020-04-20 | Сарепта Терапьютикс, Инк. | Модифицированные морфолиновые аналоги олигонуклеотидов |
CZ306894B6 (cs) | 2013-02-08 | 2017-08-30 | Univerzita PalackĂ©ho v Olomouci | 2-Substituované-6-biarylmethylamino-9-cyklopentyl-9H-purinové deriváty, jejich použití jako léčiva a farmaceutické přípravky tyto sloučeniny obsahující |
WO2014124458A1 (en) | 2013-02-11 | 2014-08-14 | The Regents Of The University Of California | Compositions and methods for treating neurodegenerative diseases |
US9938279B2 (en) | 2013-04-09 | 2018-04-10 | Energenesis Biomedical Co., Ltd | Method for treating disease or condition susceptible to amelioration by AMPK activators and compounds of formula which are useful to activate AMP-activated protein kinase (AMPK) |
WO2015005491A1 (ja) | 2013-07-12 | 2015-01-15 | 国立大学法人京都大学 | 疾病の発症又は進行の一因となる異常スプライシングを抑制できる物質のスクリーニング方法 |
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WO2016011394A1 (en) | 2014-07-18 | 2016-01-21 | The General Hospital Corporation | Imaging agents for neural flux |
EP4115882A1 (en) * | 2015-01-16 | 2023-01-11 | The General Hospital Corporation | Compounds for improving mrna splicing |
-
2016
- 2016-01-15 EP EP22178466.3A patent/EP4115882A1/en active Pending
- 2016-01-15 US US15/543,826 patent/US10676475B2/en active Active
- 2016-01-15 EA EA201791624A patent/EA037663B1/ru unknown
- 2016-01-15 CA CA3199430A patent/CA3199430A1/en active Pending
- 2016-01-15 WO PCT/US2016/013553 patent/WO2016115434A1/en active Application Filing
- 2016-01-15 PT PT167379445T patent/PT3244891T/pt unknown
- 2016-01-15 MX MX2017009295A patent/MX2017009295A/es unknown
- 2016-01-15 HU HUE16737944A patent/HUE059891T2/hu unknown
- 2016-01-15 CA CA2973949A patent/CA2973949C/en active Active
- 2016-01-15 AU AU2016206589A patent/AU2016206589B2/en active Active
- 2016-01-15 ES ES16737944T patent/ES2928714T3/es active Active
- 2016-01-15 EP EP16737944.5A patent/EP3244891B9/en active Active
- 2016-01-15 BR BR112017015019-0A patent/BR112017015019A2/pt active Search and Examination
- 2016-01-15 TW TW105101315A patent/TWI727939B/zh not_active IP Right Cessation
- 2016-01-15 PL PL16737944.5T patent/PL3244891T3/pl unknown
- 2016-01-15 DK DK16737944.5T patent/DK3244891T3/da active
- 2016-01-15 TW TW110113924A patent/TW202204345A/zh unknown
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2017
- 2017-07-03 IL IL253291A patent/IL253291B/en unknown
- 2017-07-13 CL CL2017001823A patent/CL2017001823A1/es unknown
- 2017-07-14 MX MX2022015737A patent/MX2022015737A/es unknown
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2019
- 2019-08-01 CL CL2019002167A patent/CL2019002167A1/es unknown
- 2019-11-26 IL IL270945A patent/IL270945A/en unknown
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2020
- 2020-05-18 US US16/877,254 patent/US11702417B2/en active Active
- 2020-11-26 AU AU2020277224A patent/AU2020277224B2/en active Active
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2023
- 2023-04-07 US US18/297,569 patent/US20230279007A1/en active Pending
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