EA201791624A1 - СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНК - Google Patents

СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНК

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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
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EA
Eurasian Patent Office
Prior art keywords
compounds
mrna
improve
splising
methods
Prior art date
Application number
EA201791624A
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English (en)
Other versions
EA037663B1 (ru
Inventor
Сьюзан А. Слогенхаупт
Грехэм Джонсон
Уилльям Д. Пакетт
Вэй Чжан
Хуан Маруган
Original Assignee
Те Дженерал Хоспитал Корпорейшн
Юнайтед Стейтс Дипартмент Оф Хелт Энд Хьюман Сервисиз
Priority date (The priority date 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 date listed.)
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Application filed by Те Дженерал Хоспитал Корпорейшн, Юнайтед Стейтс Дипартмент Оф Хелт Энд Хьюман Сервисиз filed Critical Те Дженерал Хоспитал Корпорейшн
Publication of EA201791624A1 publication Critical patent/EA201791624A1/ru
Publication of EA037663B1 publication Critical patent/EA037663B1/ru

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D473/00Heterocyclic compounds containing purine ring systems
    • C07D473/26Heterocyclic compounds containing purine ring systems with an oxygen, sulphur, or nitrogen atom directly attached in position 2 or 6, but not in both
    • C07D473/32Nitrogen atom
    • C07D473/34Nitrogen atom attached in position 6, e.g. adenine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52Purines, e.g. adenine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D405/00Heterocyclic 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
    • C07D405/02Heterocyclic 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
    • C07D405/12Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D417/00Heterocyclic 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
    • C07D417/02Heterocyclic 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
    • C07D417/12Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic 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
    • C07D471/02Heterocyclic 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
    • C07D471/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D473/00Heterocyclic compounds containing purine ring systems
    • C07D473/02Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6
    • C07D473/16Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6 two nitrogen atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic 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
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D495/00Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02Heterocyclic 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
    • C07D495/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D513/00Heterocyclic 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
    • C07D513/02Heterocyclic 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
    • C07D513/04Ortho-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.
EA201791624A 2015-01-16 2016-01-15 СОЕДИНЕНИЯ ДЛЯ УЛУЧШЕНИЯ СПЛАЙСИНГА мРНК EA037663B1 (ru)

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

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Family Applications (1)

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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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2005069865A2 (en) 2004-01-13 2005-08-04 Ambit Biosciences Corporation Pyrrolopyrimidine derivatives and analogs and their use in the treatment and prevention of diseases
GB0526246D0 (en) 2005-12-22 2006-02-01 Novartis Ag Organic compounds
EP1989206B1 (en) 2006-02-02 2012-07-04 Millennium Pharmaceuticals, Inc. Inhibitors of e1 activating enzyme
EP1889847A1 (en) 2006-07-10 2008-02-20 DeveloGen Aktiengesellschaft Pyrrolopyrimidines for pharmaceutical compositions
MX2009009968A (es) 2007-03-23 2009-10-08 Amgen Inc Compuestos heterociclicos y sus usos.
GB0706632D0 (en) 2007-04-04 2007-05-16 Cyclacel Ltd New purine derivatives
EP2219646A4 (en) 2007-12-21 2010-12-22 Univ Rochester METHOD FOR MODIFYING THE LIFETIME OF EUKARYOTIC ORGANISMS
US8258144B2 (en) * 2008-04-22 2012-09-04 Portola Pharmaceuticals, Inc. Inhibitors of protein kinases
RU2011116928A (ru) 2008-09-30 2012-11-20 Астразенека Аб (Se) Гетероциклические ингибиторы jак киназы
CA2748943A1 (en) * 2009-02-09 2010-08-12 Supergen, Inc. Pyrrolopyrimidinyl axl kinase inhibitors
WO2010118367A2 (en) 2009-04-10 2010-10-14 Progenics Pharmaceuticals, Inc. Antiviral pyrimidines
WO2011041655A1 (en) * 2009-10-01 2011-04-07 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Quinazolin-4-amine derivatives; and methods of use
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
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
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
EP4115882A1 (en) * 2015-01-16 2023-01-11 The General Hospital Corporation Compounds for improving mrna splicing

Also Published As

Publication number Publication date
CA2973949A1 (en) 2016-07-21
AU2016206589A1 (en) 2017-07-27
MX2022015737A (es) 2023-01-18
IL253291A0 (en) 2017-09-28
ES2928714T3 (es) 2022-11-22
HUE059891T2 (hu) 2023-01-28
CL2019002167A1 (es) 2019-12-13
TW202204345A (zh) 2022-02-01
CA2973949C (en) 2023-07-11
US20200283441A1 (en) 2020-09-10
IL270945A (en) 2020-01-30
MX2017009295A (es) 2018-03-07
AU2020277224A1 (en) 2020-12-24
TW201636341A (zh) 2016-10-16
CL2017001823A1 (es) 2018-04-13
US10676475B2 (en) 2020-06-09
TWI727939B (zh) 2021-05-21
IL253291B (en) 2021-10-31
EP4115882A1 (en) 2023-01-11
EP3244891B9 (en) 2022-11-16
WO2016115434A1 (en) 2016-07-21
US20180118748A1 (en) 2018-05-03
ES2928714T9 (es) 2022-11-29
CA3199430A1 (en) 2016-07-21
AU2016206589B2 (en) 2020-09-10
EP3244891A1 (en) 2017-11-22
EA037663B1 (ru) 2021-04-28
US11702417B2 (en) 2023-07-18
PL3244891T3 (pl) 2022-12-27
BR112017015019A2 (pt) 2018-01-30
EP3244891A4 (en) 2018-12-19
AU2020277224B2 (en) 2022-12-22
PT3244891T (pt) 2022-10-20
DK3244891T3 (da) 2022-10-24
EP3244891B1 (en) 2022-07-20
US20230279007A1 (en) 2023-09-07

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