CL2019002167A1 - Compuesto de la fórmula (ic) o una sal farmacéuticamente aceptable del mismo, o una composición farmacéutica que comprende dicho compuesto o una sal farmacéuticamente aceptable del mismo, y al menos un portador farmacéuticamente aceptable, y su uso en el tratamiento de disautonomía familiar al mejorar el empalme de marn. (divisional solicitud 201701823.) - Google Patents
Compuesto de la fórmula (ic) o una sal farmacéuticamente aceptable del mismo, o una composición farmacéutica que comprende dicho compuesto o una sal farmacéuticamente aceptable del mismo, y al menos un portador farmacéuticamente aceptable, y su uso en el tratamiento de disautonomía familiar al mejorar el empalme de marn. (divisional solicitud 201701823.)Info
- Publication number
- CL2019002167A1 CL2019002167A1 CL2019002167A CL2019002167A CL2019002167A1 CL 2019002167 A1 CL2019002167 A1 CL 2019002167A1 CL 2019002167 A CL2019002167 A CL 2019002167A CL 2019002167 A CL2019002167 A CL 2019002167A CL 2019002167 A1 CL2019002167 A1 CL 2019002167A1
- Authority
- CL
- Chile
- Prior art keywords
- pharmaceutically acceptable
- compound
- acceptable salt
- marn
- improving
- Prior art date
Links
Classifications
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- 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
EN LA PRESENTE SE PROPORCIONAN COMPUESTOS EFICACES PARA MEJORAR EL EMPALME DE MARN EN UNA CÉLULA. COMPUESTOS EJEMPLARES QUE SE PROPORCIONAN EN LA PRESENTE SON EFICACES PARA MEJORAR EL EMPALME DE MARN EN GENES QUE COMPRENDEN AL MENOS UN EXÓN QUE TERMINA EN LA SECUENCIA DE NUCLÉOTIDOS CAA. SE PROPORCIONAN MÉTODOS PARA PREPARAR LOS COMPUESTOS Y MÉTODOS PARA TRATAR 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 |
---|---|
CL2019002167A1 true CL2019002167A1 (es) | 2019-12-13 |
Family
ID=56406434
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2017001823A CL2017001823A1 (es) | 2015-01-16 | 2017-07-13 | Compuestos para mejorar el empalme de marn |
CL2019002167A CL2019002167A1 (es) | 2015-01-16 | 2019-08-01 | Compuesto de la fórmula (ic) o una sal farmacéuticamente aceptable del mismo, o una composición farmacéutica que comprende dicho compuesto o una sal farmacéuticamente aceptable del mismo, y al menos un portador farmacéuticamente aceptable, y su uso en el tratamiento de disautonomía familiar al mejorar el empalme de marn. (divisional solicitud 201701823.) |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2017001823A CL2017001823A1 (es) | 2015-01-16 | 2017-07-13 | Compuestos para mejorar el empalme de marn |
Country Status (16)
Country | Link |
---|---|
US (3) | US10676475B2 (es) |
EP (2) | EP3244891B9 (es) |
AU (2) | AU2016206589B2 (es) |
BR (1) | BR112017015019A2 (es) |
CA (2) | CA2973949C (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 (20)
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PL3244891T3 (pl) * | 2015-01-16 | 2022-12-27 | The General Hospital Corporation | Związki poprawiające splicing mRNA |
KR20220100719A (ko) | 2015-12-10 | 2022-07-15 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 또는 개선을 위한 조성물 |
JP7082120B2 (ja) | 2016-10-21 | 2022-06-07 | ニンバス ラクシュミ, インコーポレイテッド | Tyk2阻害剤およびその使用 |
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 |
WO2018226622A1 (en) | 2017-06-05 | 2018-12-13 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
CA3067592A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | Methods for treating huntington's disease |
CN111182898B (zh) | 2017-06-28 | 2024-04-16 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
EA202092001A1 (ru) | 2018-03-27 | 2021-01-29 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Соединения для лечения болезни гентингтона |
SG11202012674PA (en) | 2018-06-27 | 2021-01-28 | Ptc Therapeutics Inc | Heterocyclic and heteroaryl compounds for treating huntington's disease |
EP3861000A1 (en) * | 2018-10-01 | 2021-08-11 | Genzyme Corporation | Thieno[3,2-b]pyridine derivatives as udp glycosyltransferase inhibitors and methods of use |
US20220135568A1 (en) | 2019-02-13 | 2022-05-05 | Ptc Therapeutics, Inc. | Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia |
CN113795304A (zh) | 2019-02-13 | 2021-12-14 | Ptc医疗公司 | 用于治疗家族性自主神经功能障碍的噻吩并[3,2-b]吡啶-7-胺化合物 |
KR20210146288A (ko) * | 2019-03-01 | 2021-12-03 | 레볼루션 메디슨즈, 인크. | 이환식 헤테로사이클릴 화합물 및 이의 용도 |
KR20210146287A (ko) * | 2019-03-01 | 2021-12-03 | 레볼루션 메디슨즈, 인크. | 이환식 헤테로아릴 화합물 및 이의 용도 |
MX2022005525A (es) | 2019-11-08 | 2022-06-08 | Revolution Medicines Inc | Compuestos de heteroarilo bicíclicos y usos de estos. |
PE20221581A1 (es) | 2019-12-12 | 2022-10-06 | Ptc Therapeutics Inc | Compuestos para tratar la disautonomia familiar |
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 |
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 |
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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 |
JP2002504553A (ja) | 1998-02-26 | 2002-02-12 | アベンティス・ファーマスーティカルズ・インコーポレイテツド | 6,9−ジ置換2−[トランス−(4−アミノシクロヘキシル)アミノ]プリン |
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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 |
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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 |
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PL3244891T3 (pl) * | 2015-01-16 | 2022-12-27 | The General Hospital Corporation | Związki poprawiające splicing mRNA |
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2016
- 2016-01-15 PL PL16737944.5T patent/PL3244891T3/pl unknown
- 2016-01-15 TW TW110113924A patent/TW202204345A/zh unknown
- 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 EA EA201791624A patent/EA037663B1/ru unknown
- 2016-01-15 DK DK16737944.5T patent/DK3244891T3/da active
- 2016-01-15 CA CA2973949A patent/CA2973949C/en active Active
- 2016-01-15 MX MX2017009295A patent/MX2017009295A/es unknown
- 2016-01-15 EP EP16737944.5A patent/EP3244891B9/en active Active
- 2016-01-15 ES ES16737944T patent/ES2928714T3/es active Active
- 2016-01-15 EP EP22178466.3A patent/EP4115882A1/en active Pending
- 2016-01-15 AU AU2016206589A patent/AU2016206589B2/en active Active
- 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 HU HUE16737944A patent/HUE059891T2/hu unknown
- 2016-01-15 US US15/543,826 patent/US10676475B2/en active Active
- 2016-01-15 WO PCT/US2016/013553 patent/WO2016115434A1/en active Application Filing
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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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