JP7587511B2 - 家族性自律神経失調症を処置するためのチオエノ［３，２－ｂ］ピリジン－７－アミン化合物 - Google Patents

家族性自律神経失調症を処置するためのチオエノ［３，２－ｂ］ピリジン－７－アミン化合物 Download PDF

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JP7587511B2
JP7587511B2 JP2021547446A JP2021547446A JP7587511B2 JP 7587511 B2 JP7587511 B2 JP 7587511B2 JP 2021547446 A JP2021547446 A JP 2021547446A JP 2021547446 A JP2021547446 A JP 2021547446A JP 7587511 B2 JP7587511 B2 JP 7587511B2
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methyl
pyridin
amino
chloro
thieno
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ナンジン ジャン
マイケル エー アーノルド
アマル ダッカ
ギャリー ミッチェル カープ
トム トゥアン ルオン
ジャナ ナラシムハン
ニコライ エー ナリシュキン
ジャシ ワン
シャオヤン ジャン
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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
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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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  • Health & Medical Sciences (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
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  • 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)
JP2021547446A 2019-02-13 2020-02-10 家族性自律神経失調症を処置するためのチオエノ［３，２－ｂ］ピリジン－７－アミン化合物 Active JP7587511B2 (ja)

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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

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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
WO2016115434A1 (en) 2015-01-16 2016-07-21 The General Hospital Corporation Compounds for improving mrna splicing
WO2020155491A1 (zh) 2019-01-31 2020-08-06 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

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
US9879014B2 (en) * 2013-07-12 2018-01-30 Kyoto University Method for screening substance capable of inhibiting abnormal splicing causative of onset or progress of disease
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
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
WO2016115434A1 (en) 2015-01-16 2016-07-21 The General Hospital Corporation Compounds for improving mrna splicing
WO2020155491A1 (zh) 2019-01-31 2020-08-06 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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
REGISTRY（STN）[online]，2018年01月01日，[検索日2023年12月15日], CAS登録番号 2167522-70-3など

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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
CN113795304A (zh) 2021-12-14
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
IL285399B1 (en) 2025-02-01
EP3924049A1 (en) 2021-12-22
UA129890C2 (uk) 2025-09-03

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