KR102907028B1 - 가족성 자율신경실조증 치료를 위한 티오에노[3,2-b]피리딘-7-아민 화합물 - Google Patents

가족성 자율신경실조증 치료를 위한 티오에노[3,2-b]피리딘-7-아민 화합물

Info

Publication number
KR102907028B1
KR102907028B1 KR1020217028864A KR20217028864A KR102907028B1 KR 102907028 B1 KR102907028 B1 KR 102907028B1 KR 1020217028864 A KR1020217028864 A KR 1020217028864A KR 20217028864 A KR20217028864 A KR 20217028864A KR 102907028 B1 KR102907028 B1 KR 102907028B1
Authority
KR
South Korea
Prior art keywords
methyl
pyridin
amino
chloro
thieno
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
KR1020217028864A
Other languages
English (en)
Korean (ko)
Other versions
KR20210137040A (ko
Inventor
난징 장
마이클 에이. 아놀드
아말 다카
그레이 미첼 카프
톰 투안 루엉
자나 나라심한
니콜라이 에이. 나리쉬킨
지아쉬 왕
샤오옌 장
Original Assignee
피티씨 테라퓨틱스, 인크.
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.)
Filing date
Publication date
Application filed by 피티씨 테라퓨틱스, 인크. filed Critical 피티씨 테라퓨틱스, 인크.
Publication of KR20210137040A publication Critical patent/KR20210137040A/ko
Application granted granted Critical
Publication of KR102907028B1 publication Critical patent/KR102907028B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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
    • A61K31/4365Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020217028864A 2019-02-13 2020-02-10 가족성 자율신경실조증 치료를 위한 티오에노[3,2-b]피리딘-7-아민 화합물 Active KR102907028B1 (ko)

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 (2)

Publication Number Publication Date
KR20210137040A KR20210137040A (ko) 2021-11-17
KR102907028B1 true KR102907028B1 (ko) 2026-01-05

Family

ID=69960696

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217028864A Active KR102907028B1 (ko) 2019-02-13 2020-02-10 가족성 자율신경실조증 치료를 위한 티오에노[3,2-b]피리딘-7-아민 화합물

Country Status (17)

Country Link
US (1) US12384797B2 (https=)
EP (1) EP3924049A1 (https=)
JP (1) JP7587511B2 (https=)
KR (1) KR102907028B1 (https=)
CN (1) CN113795304A (https=)
AU (1) AU2020222881C1 (https=)
BR (1) BR112021015853A2 (https=)
CA (1) CA3129067A1 (https=)
CL (1) CL2021002137A1 (https=)
CO (1) CO2021010598A2 (https=)
EA (1) EA202192170A1 (https=)
IL (1) IL285399B2 (https=)
MX (1) MX2021009667A (https=)
PE (1) PE20220139A1 (https=)
SG (1) SG11202108519TA (https=)
UA (1) UA129890C2 (https=)
WO (1) WO2020167628A1 (https=)

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
SG11202108519TA (en) 2019-02-13 2021-09-29 Ptc Therapeutics Inc Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia
CN115209955A (zh) * 2019-12-12 2022-10-18 Ptc医疗公司 治疗家族性自主神经功能障碍的化合物
BR112022023025A2 (pt) 2020-05-13 2023-03-28 Chdi Foundation Inc Moduladores de htt para tratar doença de huntington
EP4277626A4 (en) * 2021-01-14 2024-12-11 Georgetown University 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
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
US20240051968A1 (en) * 2021-02-05 2024-02-15 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
EP4398987A4 (en) * 2021-09-07 2025-07-09 Ptc Therapeutics Inc METHODS OF TREATMENT OF NEURODEGENERATIVE DISEASES
US20250127789A1 (en) * 2021-11-04 2025-04-24 Skyhawk Therapeutics, Inc. Compositions useful for modulating splicing
US12281116B2 (en) 2021-11-17 2025-04-22 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
WO2023212237A1 (en) 2022-04-27 2023-11-02 Skyhawk Therapeutics, Inc. Compositions useful for modulating splicing
US20260001889A1 (en) 2022-05-12 2026-01-01 Skyhawk Therapeutics, Inc. Compositions useful for modulating splicing
MA71255A (fr) * 2022-06-22 2025-04-30 Ptc Therapeutics, Inc. Composés pour le traitement de l'ataxie spinocérébelleuse de type 3
WO2024036143A2 (en) * 2022-08-09 2024-02-15 Ptc Therapeutics, Inc. Methods for modulating rna splicing

Citations (1)

* 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

Family Cites Families (55)

* 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
EP1028964A1 (en) 1997-11-11 2000-08-23 Pfizer Products 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
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
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
BRPI0912245A2 (pt) 2008-03-05 2019-09-24 Takeda Pharmaceuticals Co composto, prodroga, medicamento, métodos de suprimir produção de açúcar em um mamífero e para a profilaxia ou o tratamento de diabetes em um mamífero, e, uso do composto
WO2010118367A2 (en) 2009-04-10 2010-10-14 Progenics Pharmaceuticals, Inc. Antiviral pyrimidines
WO2012012656A2 (en) 2010-07-21 2012-01-26 University Of South Florida Materials and methods for treating neurodegenerative diseases
EP2611812A1 (en) 2010-09-01 2013-07-10 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
EP2867236B1 (en) 2012-06-29 2017-06-14 Pfizer Inc Novel 4-(substituted-amino)-7h-pyrrolo[2,3-d]pyrimidines as lrrk2 inhibitors
WO2014124458A1 (en) 2013-02-11 2014-08-14 The Regents Of The University Of California Compositions and methods for treating neurodegenerative diseases
CN105339371A (zh) 2013-03-07 2016-02-17 葛兰素史密斯克莱有限责任公司 作为沉默调节蛋白调节剂的噻吩并[3,2-d]嘧啶-6-甲酰胺和类似物
CN110218727A (zh) 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
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
RU2692634C2 (ru) 2013-10-07 2019-06-25 Академиш Зикенхойс Лейден Х.О.Д.Н. Люмк Направляемое антисмысловым олигонуклеотидом удаление сайтов протеолитического расщепления, мутации hchwa-d и увеличенного числа тринуклеотидных повторов
KR102390370B1 (ko) 2014-02-12 2022-04-26 삼성전자주식회사 축합환 화합물 및 이를 포함한 유기 발광 소자
MA39987A (fr) 2014-04-30 2017-03-08 Incyte Corp Procédés de préparation d'un inhibiteur de jak1 et nouvelles formes associées
ES2865330T3 (es) 2015-04-29 2021-10-15 Janssen Pharmaceutica Nv Azabenzimidazoles y su uso como moduladores del receptor AMPA
KR102222186B1 (ko) 2015-08-13 2021-03-03 머크 샤프 앤드 돔 코포레이션 Sting 효능제로서 시클릭 디-뉴클레오티드 화합물
EP4285912A3 (en) 2015-09-25 2024-07-10 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
IL294124B2 (en) 2015-12-10 2024-02-01 Ptc Therapeutics Inc 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
EP3528816A4 (en) 2016-10-21 2020-04-08 Nimbus Lakshmi, Inc. TYK2 INHIBITORS AND USES THEREOF
GB201700814D0 (en) 2017-01-17 2017-03-01 Liverpool School Tropical Medicine Compounds
WO2018151326A1 (ja) 2017-02-20 2018-08-23 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
US10538571B2 (en) 2017-11-27 2020-01-21 Coda Biotherapeutics, Inc. Compositions and methods for neurological diseases
WO2020123395A1 (en) 2018-12-10 2020-06-18 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
SG11202108519TA (en) 2019-02-13 2021-09-29 Ptc Therapeutics Inc Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia
WO2020167624A1 (en) 2019-02-13 2020-08-20 Ptc Therapeutics, Inc. Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia
MA54953A (fr) 2019-02-13 2021-12-22 Denali Therapeutics Inc Composés, compositions et procédés
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
CN113950529A (zh) 2019-06-06 2022-01-18 豪夫迈·罗氏有限公司 靶向atxn3的反义寡核苷酸
US11798161B2 (en) 2019-11-26 2023-10-24 Koh Young Technology Inc. Method and apparatus for determining mid-sagittal plane in magnetic resonance images
CN115209955A (zh) 2019-12-12 2022-10-18 Ptc医疗公司 治疗家族性自主神经功能障碍的化合物
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
EP4288441A4 (en) 2021-02-05 2024-12-11 PTC Therapeutics, Inc. Methods for treating spinocerebellar ataxia type 3
JP2024513566A (ja) 2021-04-09 2024-03-26 ザ・ブロード・インスティテュート・インコーポレイテッド 標的基質の選択的修飾のための二官能性分子
EP4398987A4 (en) 2021-09-07 2025-07-09 Ptc Therapeutics Inc METHODS OF TREATMENT OF NEURODEGENERATIVE DISEASES
CN118251225A (zh) 2021-09-07 2024-06-25 Ptc医疗公司 治疗神经退行性疾病的方法
MX2024002887A (es) 2021-09-07 2024-05-28 Ptc Therapeutics Inc Metodos para tratar enfermedades neurodegenerativas.
US20250127789A1 (en) 2021-11-04 2025-04-24 Skyhawk Therapeutics, Inc. Compositions useful for modulating splicing
MA71255A (fr) 2022-06-22 2025-04-30 Ptc Therapeutics, Inc. Composés pour le traitement de l'ataxie spinocérébelleuse de type 3

Patent Citations (1)

* 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

Also Published As

Publication number Publication date
BR112021015853A2 (pt) 2021-10-05
PE20220139A1 (es) 2022-01-27
UA129890C2 (uk) 2025-09-03
IL285399B2 (en) 2025-06-01
IL285399B1 (en) 2025-02-01
CL2021002137A1 (es) 2022-02-25
AU2020222881B2 (en) 2025-07-10
WO2020167628A1 (en) 2020-08-20
KR20210137040A (ko) 2021-11-17
JP7587511B2 (ja) 2024-11-20
US12384797B2 (en) 2025-08-12
AU2020222881C1 (en) 2026-01-22
EP3924049A1 (en) 2021-12-22
US20220135586A1 (en) 2022-05-05
CO2021010598A2 (es) 2021-08-30
AU2020222881A1 (en) 2021-09-09
CA3129067A1 (en) 2020-08-20
EA202192170A1 (ru) 2021-11-15
IL285399A (en) 2021-09-30
SG11202108519TA (en) 2021-09-29
JP2022520822A (ja) 2022-04-01
MX2021009667A (es) 2021-12-10
CN113795304A (zh) 2021-12-14

Similar Documents

Publication Publication Date Title
KR102907028B1 (ko) 가족성 자율신경실조증 치료를 위한 티오에노[3,2-b]피리딘-7-아민 화합물
US12398140B2 (en) Substituted pyrrolo [2,3-d]pyrimidines for treating familial dysautonomia
US10987359B2 (en) Oxadiazepinone derivatives and methods of treating hepatitis B infections
KR20250035623A (ko) 척추소뇌성 운동실조증 유형 3을 치료하기 위한 화합물
US11198692B2 (en) Dihydro-pyrrolo-pyridine derivatives
EP2694472B1 (en) Sulfonamide derivative and use thereof
JP2016537369A (ja) カゼインキナーゼ1d/e阻害剤としての置換された4,5,6,7−テトラヒドロピラゾロ[1,5−a]ピラジン誘導体
AU2013319845A1 (en) Novel bicyclic pyridinones
JP2025133750A (ja) 家族性自律神経失調症を処置するための化合物
US20230203007A1 (en) Pyridinylacetamide derivatives as sodium channel activators
BR122024013729A2 (pt) Compostos de tieno[3,2-b]piridin-7-amina, seus usos no tratamento de disautonomia familiar e composição farmacêutica compreendendo os mesmos
EP4514807B1 (en) Compositions useful for modulating splicing
US20260001889A1 (en) Compositions useful for modulating splicing

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

A201 Request for examination
PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

E601 Decision to refuse application
PE0601 Decision on rejection of patent

St.27 status event code: N-2-6-B10-B15-exm-PE0601

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

F13 Ip right granted in full following pre-grant review

Free format text: ST27 STATUS EVENT CODE: A-3-4-F10-F13-REX-PX0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PX0701 Decision of registration after re-examination

St.27 status event code: A-3-4-F10-F13-rex-PX0701

F11 Ip right granted following substantive examination

Free format text: ST27 STATUS EVENT CODE: A-2-4-F10-F11-EXM-PR0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

U12 Designation fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U12-OTH-PR1002 (AS PROVIDED BY THE NATIONAL OFFICE)

Year of fee payment: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601

Q13 Ip right document published

Free format text: ST27 STATUS EVENT CODE: A-4-4-Q10-Q13-NAP-PG1601 (AS PROVIDED BY THE NATIONAL OFFICE)