MX2022015737A - Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm). - Google Patents
Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm).Info
- Publication number
- MX2022015737A MX2022015737A MX2022015737A MX2022015737A MX2022015737A MX 2022015737 A MX2022015737 A MX 2022015737A MX 2022015737 A MX2022015737 A MX 2022015737A MX 2022015737 A MX2022015737 A MX 2022015737A MX 2022015737 A MX2022015737 A MX 2022015737A
- Authority
- MX
- Mexico
- Prior art keywords
- compounds
- mrna splicing
- improving mrna
- improving
- methods
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 4
- 108020004999 messenger RNA Proteins 0.000 title abstract 3
- 210000003169 central nervous system Anatomy 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
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
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Neurosurgery (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Life Sciences & Earth Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
Abstract
Se proveen compuestos útiles para mejorar el empalme del ARNm en una célula. Los compuestos ejemplares provistos en la presente son útiles para mejorar el empalme de ARNm en genes que comprenden por lo menos un exón que termina en la secuencia de nucleótidos CAA. También se proveen métodos para preparar los compuestos y métodos de tratamiento de enfermedades del sistema nervioso central.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562104547P | 2015-01-16 | 2015-01-16 | |
| US201562180380P | 2015-06-16 | 2015-06-16 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2022015737A true MX2022015737A (es) | 2023-01-18 |
Family
ID=56406434
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017009295A MX2017009295A (es) | 2015-01-16 | 2016-01-15 | Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm). |
| MX2022015737A MX2022015737A (es) | 2015-01-16 | 2017-07-14 | Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm). |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017009295A MX2017009295A (es) | 2015-01-16 | 2016-01-15 | Compuestos para mejorar el empalme del acido ribonucleico mensajero (arnm). |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US10676475B2 (es) |
| EP (2) | EP4115882A1 (es) |
| AU (2) | AU2016206589B2 (es) |
| BR (1) | BR112017015019A2 (es) |
| CA (2) | CA3199430A1 (es) |
| CL (2) | CL2017001823A1 (es) |
| DK (1) | DK3244891T3 (es) |
| EA (1) | EA037663B1 (es) |
| ES (1) | ES2928714T3 (es) |
| HU (1) | HUE059891T2 (es) |
| IL (2) | IL253291B (es) |
| MX (2) | MX2017009295A (es) |
| PL (1) | PL3244891T3 (es) |
| PT (1) | PT3244891T (es) |
| TW (2) | TW202204345A (es) |
| WO (1) | WO2016115434A1 (es) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
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| PL3244891T3 (pl) * | 2015-01-16 | 2022-12-27 | The General Hospital Corporation | Związki poprawiające splicing mRNA |
| CN110946865B (zh) | 2015-12-10 | 2024-01-26 | Ptc医疗公司 | 用于治疗亨廷顿病的方法 |
| AU2017345736B2 (en) | 2016-10-21 | 2022-04-07 | Takeda Pharmaceutical Company Limited | TYK2 inhibitors and uses thereof |
| CN114432318A (zh) * | 2017-02-20 | 2022-05-06 | 国立大学法人京都大学 | 用于起因于剪接异常的遗传性疾病的药物组合物及治疗方法 |
| EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
| MX2019015578A (es) | 2017-06-28 | 2020-07-28 | Ptc Therapeutics Inc | Metodos para tratar la enfermedad de huntington. |
| CN111163838B (zh) | 2017-06-28 | 2023-03-28 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| JP7399870B2 (ja) | 2018-03-27 | 2023-12-18 | ピーティーシー セラピューティクス, インコーポレイテッド | ハンチントン病を処置するための化合物 |
| PL3814357T3 (pl) | 2018-06-27 | 2024-09-16 | Ptc Therapeutics, Inc. | Związki heterocykliczne i heteroarylowe do leczenia choroby huntingtona |
| KR20210038845A (ko) | 2018-06-27 | 2021-04-08 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료를 위한 헤테로아릴 화합물 |
| KR102903997B1 (ko) * | 2018-10-01 | 2025-12-26 | 젠자임 코포레이션 | UDP 글리코실트랜스퍼라제 억제제로서의 티에노[3,2-b]피리딘 유도체 및 사용 방법 |
| WO2020167624A1 (en) | 2019-02-13 | 2020-08-20 | Ptc Therapeutics, Inc. | Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia |
| CA3129067A1 (en) | 2019-02-13 | 2020-08-20 | Ptc Therapeutics, Inc. | Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia |
| KR20210146287A (ko) * | 2019-03-01 | 2021-12-03 | 레볼루션 메디슨즈, 인크. | 이환식 헤테로아릴 화합물 및 이의 용도 |
| CN113727758A (zh) * | 2019-03-01 | 2021-11-30 | 锐新医药公司 | 双环杂环基化合物及其用途 |
| CN114245794B (zh) | 2019-05-13 | 2024-09-13 | Ptc医疗公司 | 用于治疗亨廷顿氏病的化合物 |
| BR112022008858A2 (pt) | 2019-11-08 | 2022-09-06 | Revolution Medicines Inc | Composto, composição farmacêutica e métodos para inibir sos1 em um sujeito, para inibir a interação de sos1 e uma proteína, para tratar ou prevenir uma doença e para tratar ou prevenir câncer |
| WO2021118929A1 (en) * | 2019-12-12 | 2021-06-17 | Ptc Therapeutics, Inc. | Compounds for treating familial dysautonomia |
| EP4084796A1 (en) | 2020-01-02 | 2022-11-09 | 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 |
| EP4288441A4 (en) * | 2021-02-05 | 2024-12-11 | 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 |
| JP2024533323A (ja) | 2021-09-07 | 2024-09-12 | ピーティーシー セラピューティクス, インコーポレイテッド | 神経変性疾患を処置するための方法 |
| CA3230996A1 (en) | 2021-09-07 | 2023-03-16 | Ptc Therapeutics, Inc. | Methods for treating neurogenerative diseases |
| WO2025227159A1 (en) * | 2024-04-26 | 2025-10-30 | The General Hospital Corporation | Gene editing treatment for correcting splicing defect in familial dysautonomia |
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| US3041340A (en) | 1962-06-26 | Method of preparing substituted | ||
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| GB9613736D0 (en) | 1996-07-01 | 1996-09-04 | Hancock Tony | Transport cradle for a skid-supported helicopter |
| US6933280B2 (en) * | 1996-10-08 | 2005-08-23 | Gerardo Castillo | Peptides for the treatment of Alzheimer's disease and other beta-amyloid protein fibrillogenesis disorders |
| WO1999043676A2 (en) | 1998-02-26 | 1999-09-02 | Aventis Pharmaceuticals 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 |
| AU2004278773B2 (en) | 2003-10-03 | 2011-05-19 | The General Hospital Corporation | Methods for altering mRNA splicing and treating familial dysautonomia and other mechanistically related disorders |
| US20050239806A1 (en) * | 2004-01-13 | 2005-10-27 | Ambit Biosciences Corporation | Pyrrolopyrimidine derivatives and analogs and their use in the treatment and prevention of diseases |
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| EP1889847A1 (en) * | 2006-07-10 | 2008-02-20 | DeveloGen Aktiengesellschaft | Pyrrolopyrimidines for pharmaceutical compositions |
| AU2008231304B2 (en) * | 2007-03-23 | 2011-05-12 | Amgen Inc. | Heterocyclic compounds and their uses |
| GB0706632D0 (en) | 2007-04-04 | 2007-05-16 | Cyclacel Ltd | New purine derivatives |
| US8642660B2 (en) | 2007-12-21 | 2014-02-04 | The University Of Rochester | Method for altering the lifespan of eukaryotic organisms |
| JP2011518219A (ja) * | 2008-04-22 | 2011-06-23 | ポートラ ファーマシューティカルズ, インコーポレイテッド | タンパク質キナーゼの阻害剤 |
| JP2012504157A (ja) | 2008-09-30 | 2012-02-16 | アストラゼネカ アクチボラグ | 複素環式jakキナーゼ阻害剤 |
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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 |
| US20130072506A1 (en) | 2011-09-16 | 2013-03-21 | Lenka ZAHAJSKA | 6,8-disubstituted purine compositions |
| BR112014011875B1 (pt) * | 2011-11-18 | 2022-01-04 | Sarepta Therapeutics, Inc | Oligonucleotídeos funcionalmente modificados e subunidades dos mesmos |
| 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 |
| US10167286B2 (en) | 2014-02-11 | 2019-01-01 | Mitokinin, Inc. | Compositions and methods using the same for treatment of neurodegenerative and mitochondrial disease |
| WO2016011394A1 (en) | 2014-07-18 | 2016-01-21 | The General Hospital Corporation | Imaging agents for neural flux |
| PL3244891T3 (pl) * | 2015-01-16 | 2022-12-27 | The General Hospital Corporation | Związki poprawiające splicing mRNA |
-
2016
- 2016-01-15 PL PL16737944.5T patent/PL3244891T3/pl unknown
- 2016-01-15 TW TW110113924A patent/TW202204345A/zh unknown
- 2016-01-15 EP EP22178466.3A patent/EP4115882A1/en not_active Withdrawn
- 2016-01-15 AU AU2016206589A patent/AU2016206589B2/en not_active Ceased
- 2016-01-15 TW TW105101315A patent/TWI727939B/zh not_active IP Right Cessation
- 2016-01-15 CA CA3199430A patent/CA3199430A1/en active Pending
- 2016-01-15 MX MX2017009295A patent/MX2017009295A/es unknown
- 2016-01-15 ES ES16737944T patent/ES2928714T3/es active Active
- 2016-01-15 EA EA201791624A patent/EA037663B1/ru unknown
- 2016-01-15 EP EP16737944.5A patent/EP3244891B9/en active Active
- 2016-01-15 HU HUE16737944A patent/HUE059891T2/hu unknown
- 2016-01-15 CA CA2973949A patent/CA2973949C/en active Active
- 2016-01-15 US US15/543,826 patent/US10676475B2/en not_active Expired - Fee Related
- 2016-01-15 DK DK16737944.5T patent/DK3244891T3/da active
- 2016-01-15 BR BR112017015019-0A patent/BR112017015019A2/pt active Search and Examination
- 2016-01-15 PT PT167379445T patent/PT3244891T/pt unknown
- 2016-01-15 WO PCT/US2016/013553 patent/WO2016115434A1/en not_active Ceased
-
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
-
2019
- 2019-08-01 CL CL2019002167A patent/CL2019002167A1/es unknown
- 2019-11-26 IL IL270945A patent/IL270945A/en unknown
-
2020
- 2020-05-18 US US16/877,254 patent/US11702417B2/en active Active
- 2020-11-26 AU AU2020277224A patent/AU2020277224B2/en not_active Ceased
-
2023
- 2023-04-07 US US18/297,569 patent/US20230279007A1/en not_active Abandoned
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