CN113795304A - 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 - Google Patents

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 Download PDF

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Publication number
CN113795304A
CN113795304A CN202080028449.2A CN202080028449A CN113795304A CN 113795304 A CN113795304 A CN 113795304A CN 202080028449 A CN202080028449 A CN 202080028449A CN 113795304 A CN113795304 A CN 113795304A
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China
Prior art keywords
methyl
amino
chloro
thieno
pyridin
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Pending
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CN202080028449.2A
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English (en)
Chinese (zh)
Inventor
张南京
M·A·阿诺德
A·达卡
G·M·卡普
T·T·梁
J·纳拉辛汉
N·A·纳雷什金
J·王
X·张
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PTC Therapeutics Inc
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PTC Therapeutics Inc
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Publication of CN113795304A publication Critical patent/CN113795304A/zh
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    • 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
    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4365—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system having sulfur as a ring hetero atom, e.g. ticlopidine
    • 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

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202080028449.2A 2019-02-13 2020-02-10 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 Pending CN113795304A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962805283P 2019-02-13 2019-02-13
US62/805,283 2019-02-13
PCT/US2020/017430 WO2020167628A1 (en) 2019-02-13 2020-02-10 Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia

Publications (1)

Publication Number Publication Date
CN113795304A true CN113795304A (zh) 2021-12-14

Family

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CN202080028449.2A Pending CN113795304A (zh) 2019-02-13 2020-02-10 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

Country Status (17)

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US (1) US12384797B2 (enExample)
EP (1) EP3924049B1 (enExample)
JP (1) JP7587511B2 (enExample)
KR (1) KR102907028B1 (enExample)
CN (1) CN113795304A (enExample)
AU (1) AU2020222881C1 (enExample)
BR (1) BR112021015853A2 (enExample)
CA (1) CA3129067A1 (enExample)
CL (1) CL2021002137A1 (enExample)
CO (1) CO2021010598A2 (enExample)
EA (1) EA202192170A1 (enExample)
IL (1) IL285399B2 (enExample)
MX (1) MX2021009667A (enExample)
PE (1) PE20220139A1 (enExample)
SG (1) SG11202108519TA (enExample)
UA (1) UA129890C2 (enExample)
WO (1) WO2020167628A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020167624A1 (en) 2019-02-13 2020-08-20 Ptc Therapeutics, Inc. Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia
AU2020222881C1 (en) 2019-02-13 2026-01-22 Ptc Therapeutics, Inc. Thioeno(3,2-b) pyridin-7-amine compounds for treating familial dysautonomia
KR20220113700A (ko) * 2019-12-12 2022-08-16 피티씨 테라퓨틱스, 인크. 가족성 자율신경실조증 치료를 위한 화합물
WO2021231571A1 (en) 2020-05-13 2021-11-18 Chdi Foundation, Inc. Htt modulators for treating huntington's disease
CA3204868A1 (en) * 2021-01-14 2022-07-21 Charbel MOUSSA Usp13 inhibitors and methods of use thereof
CN112920053B (zh) * 2021-02-01 2022-07-01 暨明医药科技（苏州）有限公司 一种手性α-甲基芳乙胺的制备方法
US20240051968A1 (en) 2021-02-05 2024-02-15 Ptc Therapeutics, Inc. Methods for treating spinocerebellar ataxia type 3
EP4288059A4 (en) * 2021-02-05 2025-01-08 PTC Therapeutics, Inc. Compounds for treating spinocerebellar ataxia type 3
US20240041886A1 (en) * 2021-02-05 2024-02-08 Ptc Therapeutics, Inc. Methods for treating spinocerebellar ataxia type 3
TW202313623A (zh) 2021-06-03 2023-04-01 大陸商江蘇恆瑞醫藥股份有限公司 具有整合酶抑制活性的吡啶酮化合物及其藥用用途
CA3231007A1 (en) * 2021-09-07 2023-03-16 Scott J. Barraza Methods for treating neurogenerative diseases
EP4426702A1 (en) * 2021-11-04 2024-09-11 Skyhawk Therapeutics, Inc. Condensed-pyrazine amine derivatives for treating sca3
AU2022394333A1 (en) 2021-11-17 2024-06-27 Chdi Foundation, Inc. HTT modulators for treating Huntington’s disease
EP4514807B1 (en) 2022-04-27 2025-12-17 Skyhawk Therapeutics, Inc. Compositions useful for modulating splicing
US20260027118A1 (en) 2022-05-12 2026-01-29 Skyhawk Therapeutics, Inc. Compositions useful for modulating splicing
CN119997954A (zh) * 2022-06-22 2025-05-13 Ptc医疗公司 用于治疗脊髓小脑性共济失调3型的化合物
EP4568962A2 (en) * 2022-08-09 2025-06-18 PTC Therapeutics, Inc. Methods for modulating rna splicing

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3020829A1 (en) * 2013-07-12 2016-05-18 Kyoto University Method for screening substance capable of inhibiting abnormal splicing causative of onset or progress of disease
WO2016115434A1 (en) * 2015-01-16 2016-07-21 The General Hospital Corporation Compounds for improving mrna splicing

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE1470356A1 (de) 1964-01-15 1970-04-30 Thomae Gmbh Dr K Neue Thieno[3,2-d]pyrimidine und Verfahren zu ihrer Herstellung
JP3126541B2 (ja) 1993-03-26 2001-01-22 千寿製薬株式会社 ベンゾチアゾール誘導体、その製法及びその用途
US5654307A (en) 1994-01-25 1997-08-05 Warner-Lambert Company Bicyclic compounds capable of inhibiting tyrosine kinases of the epidermal growth factor receptor family
NZ335823A (en) 1997-04-22 2001-06-29 Neurocrine Biosciences Inc Thiophenopyridines, preparation as CRF receptor antagonists
SK6652000A3 (en) 1997-11-11 2002-05-09 Pfizer Prod Inc Thienopyrimidine and thienopyridine derivatives useful as anticancer agents
CL2003002287A1 (es) 2002-11-25 2005-01-14 Wyeth Corp COMPUESTOS DERIVADOS DE TIENO[3,2-b]-PIRIDINA-6-CARBONITRILOS Y TIENEO[2,3-b]-PIRIDINA-5-CARBONITRILOS, COMPOSICION FARMACEUTICA, PROCEDIMIENTO DE PREPARACION Y COMPUESTOS INTERMEDIARIOS, Y SU USO EN EL TRATAMIENTO DEL CANCER, APOPLEJIA, OSTEOPOROSIS
WO2004065392A1 (en) 2003-01-24 2004-08-05 Smithkline Beecham Corporation Condensed pyridines and pyrimidines and their use as alk-5 receptor ligands
WO2005007083A2 (en) 2003-06-18 2005-01-27 Smithkline Beecham Corporation Chemical compounds
WO2005033290A2 (en) 2003-10-03 2005-04-14 The General Hospital Corporation Methods for altering mrna splicing and treating familial dysautonomia and other mechanistically related disorders
JP2009007341A (ja) 2007-06-01 2009-01-15 Mitsubishi Tanabe Pharma Corp 医薬組成物
EP2014663A1 (de) 2007-07-12 2009-01-14 Bayer Schering Pharma AG Thienopyrimidylamine als Modulatoren des EP2-Rezeptors
WO2009085226A2 (en) 2007-12-21 2009-07-09 Sirtris Pharmaceuticals, Inc. Inhibitors of cdc2-like kinases (clks) and methods of use thereof
KR101616140B1 (ko) 2008-03-05 2016-04-27 다케다 야쿠힌 고교 가부시키가이샤 복소환 화합물
WO2010118367A2 (en) 2009-04-10 2010-10-14 Progenics Pharmaceuticals, Inc. Antiviral pyrimidines
US20130209549A1 (en) 2010-07-21 2013-08-15 University Of South Florida Materials and methods for treating neurodegenerative diseases
US20130317045A1 (en) 2010-09-01 2013-11-28 Ambit Biosciences Corporation Thienopyridine and thienopyrimidine compounds and methods of use thereof
DE102011111400A1 (de) 2011-08-23 2013-02-28 Merck Patent Gmbh Bicyclische heteroaromatische Verbindungen
EA025186B1 (ru) 2012-06-29 2016-11-30 Пфайзер Инк. НОВЫЕ 4-(ЗАМЕЩЕННЫЙ АМИНО)-7Н-ПИРРОЛО[2,3-d]ПИРИМИДИНЫ В КАЧЕСТВЕ ИНГИБИТОРОВ LRRK2
AU2014214561C1 (en) 2013-02-11 2019-01-17 Mitokinin, Inc. Compositions and methods for treating neurodegenerative diseases
EP2964654A4 (en) 2013-03-07 2016-08-10 Glaxosmithkline Llc THIEN [3,2-D] PYRIMIDIN-6-CARBOXAMIDE AND ANALOGUE AS SIRTUIN MODULATORS
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
CN103242341B (zh) 2013-04-19 2015-12-09 中国科学院广州生物医药与健康研究院 噻吩并2，4取代嘧啶类化合物及其药物组合物与应用
WO2015053624A2 (en) 2013-10-07 2015-04-16 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions
KR102390370B1 (ko) 2014-02-12 2022-04-26 삼성전자주식회사 축합환 화합물 및 이를 포함한 유기 발광 소자
SG11201609016VA (en) 2014-04-30 2016-11-29 Incyte Corp Processes of preparing a jak1 inhibitor and new forms thereto
BR122023023802A2 (pt) 2015-04-29 2023-12-26 Janssen Pharmaceutica Nv Compostos azabenzimidazóis, seu uso como moduladores do receptor de ampa e composições farmacêuticas que os compreendem
CN108137641B (zh) 2015-08-13 2022-04-01 默沙东公司 作为sting激动剂的环状双核苷酸化合物
WO2017053781A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
CA3119019C (en) 2015-12-10 2024-06-11 Ptc Therapeutics, Inc. Compounds and methods for treating huntington's disease
US10034861B2 (en) 2016-07-04 2018-07-31 H. Lundbeck A/S 1H-pyrazolo[4,3-b]pyridines as PDE1 inhibitors
EP3494229B1 (en) 2016-08-03 2023-10-25 Meiragtx UK II Limited High throughput cell-based screening for aptamers
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
GB201700814D0 (en) 2017-01-17 2017-03-01 Liverpool School Tropical Medicine Compounds
CN110312528B (zh) 2017-02-20 2022-02-18 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
IL274702B2 (en) 2017-11-27 2026-04-01 Coda Biotherapeutics Inc Compounds and methods for neurological diseases
US12503467B2 (en) 2018-12-10 2025-12-23 Ideaya Biosciences, Inc. 2-oxoquinazoline derivatives as methionine adenosyltransferase 2A inhibitors
CN109819066A (zh) 2019-01-31 2019-05-28 平安科技（深圳）有限公司 一种基于地理位置的智能解析域名方法及装置
AU2020222881C1 (en) 2019-02-13 2026-01-22 Ptc Therapeutics, Inc. Thioeno(3,2-b) pyridin-7-amine compounds for treating familial dysautonomia
CR20210426A (es) 2019-02-13 2021-09-30 Denali Therapeutics Inc Compuestos, composiciones y métodos
WO2020167624A1 (en) 2019-02-13 2020-08-20 Ptc Therapeutics, Inc. Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia
EP3929185A4 (en) 2019-02-19 2023-02-15 Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. NITROGEN CONDENSED CYCLIC COMPOUND, METHOD OF PRODUCTION THEREOF AND USE THEREOF
WO2020245233A1 (en) 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
KR102537214B1 (ko) 2019-11-26 2023-05-31 주식회사 고영테크놀러지 자기 공명 이미지들에서 정중시상 평면을 결정하기 위한 방법 및 장치
KR20220113700A (ko) 2019-12-12 2022-08-16 피티씨 테라퓨틱스, 인크. 가족성 자율신경실조증 치료를 위한 화합물
US20230117831A1 (en) 2020-01-08 2023-04-20 The Broad Institute, Inc. Multi-functional chimeric molecules
US20240051968A1 (en) 2021-02-05 2024-02-15 Ptc Therapeutics, Inc. Methods for treating spinocerebellar ataxia type 3
EP4288059A4 (en) 2021-02-05 2025-01-08 PTC Therapeutics, Inc. Compounds for treating spinocerebellar ataxia type 3
WO2022169864A1 (en) 2021-02-05 2022-08-11 Ptc Therapeutics Inc. Methods for treating spinocerebellar ataxia type 3
US20240024490A1 (en) 2021-04-09 2024-01-25 The Broad Institute, Inc. Bifunctional molecules for selective modification of target substrates
JP2024533264A (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 神経原性疾患を処置するための方法
MX2024002886A (es) 2021-09-07 2024-05-24 Ptc Therapeutics Inc Metodos para tratar enfermedades neurodegenerativas.
CA3231007A1 (en) 2021-09-07 2023-03-16 Scott J. Barraza Methods for treating neurogenerative diseases
EP4426702A1 (en) 2021-11-04 2024-09-11 Skyhawk Therapeutics, Inc. Condensed-pyrazine amine derivatives for treating sca3
CN119997954A (zh) 2022-06-22 2025-05-13 Ptc医疗公司 用于治疗脊髓小脑性共济失调3型的化合物

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3020829A1 (en) * 2013-07-12 2016-05-18 Kyoto University Method for screening substance capable of inhibiting abnormal splicing causative of onset or progress of disease
WO2016115434A1 (en) * 2015-01-16 2016-07-21 The General Hospital Corporation Compounds for improving mrna splicing

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US20220135586A1 (en) 2022-05-05
AU2020222881B2 (en) 2025-07-10
EA202192170A1 (ru) 2021-11-15
PE20220139A1 (es) 2022-01-27
EP3924049B1 (en) 2026-08-12
BR112021015853A2 (pt) 2021-10-05
CA3129067A1 (en) 2020-08-20
US12384797B2 (en) 2025-08-12
AU2020222881C1 (en) 2026-01-22
IL285399B2 (en) 2025-06-01
MX2021009667A (es) 2021-12-10
AU2020222881A1 (en) 2021-09-09
WO2020167628A1 (en) 2020-08-20
KR20210137040A (ko) 2021-11-17
JP2022520822A (ja) 2022-04-01
CL2021002137A1 (es) 2022-02-25
IL285399A (en) 2021-09-30
KR102907028B1 (ko) 2026-01-05
SG11202108519TA (en) 2021-09-29
CO2021010598A2 (es) 2021-08-30
JP7587511B2 (ja) 2024-11-20
IL285399B1 (en) 2025-02-01
EP3924049A1 (en) 2021-12-22
UA129890C2 (uk) 2025-09-03

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