JP2023541040A - 筋ジストロフィーを処置するための組成物及び方法 - Google Patents

筋ジストロフィーを処置するための組成物及び方法 Download PDF

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JP2023541040A
JP2023541040A JP2023515741A JP2023515741A JP2023541040A JP 2023541040 A JP2023541040 A JP 2023541040A JP 2023515741 A JP2023515741 A JP 2023515741A JP 2023515741 A JP2023515741 A JP 2023515741A JP 2023541040 A JP2023541040 A JP 2023541040A
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compound
mmol
sspn
acid
alkyl
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JP2023515741A
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Japanese (ja)
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JP2023541040A5 (https=
Inventor
クロスビー，ラシェル，エイチ．
シュー，シンシア
ジョン，ヴァルギース
カンパーニャ，ジーザス
ジャゴジンスカ，バーバラ
パルフェノバ，リューヴォフ
モコノヴァ，エカテリーナ
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University of California San Diego UCSD
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University of California San Diego UCSD
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D213/00—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members
    • C07D213/02—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members
    • C07D213/04—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D213/60—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D213/78—Carbon atoms having three bonds to hetero atoms, with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C07D213/84—Nitriles
    • C07D213/85—Nitriles in position 3
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D213/00—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members
    • C07D213/02—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members
    • C07D213/89—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members with hetero atoms directly attached to the ring nitrogen atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D215/00—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems
    • C07D215/02—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom
    • C07D215/16—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D215/20—Oxygen atoms
    • C07D215/22—Oxygen atoms attached in position 2 or 4
    • C07D215/227—Oxygen atoms attached in position 2 or 4 only one oxygen atom which is attached in position 2
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D215/00—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems
    • C07D215/02—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom
    • C07D215/16—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D215/36—Sulfur atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D215/00—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems
    • C07D215/02—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom
    • C07D215/16—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D215/38—Nitrogen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D215/00—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems
    • C07D215/02—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom
    • C07D215/16—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D215/48—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D215/00—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems
    • C07D215/02—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom
    • C07D215/16—Heterocyclic compounds containing quinoline or hydrogenated quinoline ring systems having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen atoms or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D215/48—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen
    • C07D215/54—Carbon atoms having three bonds to hetero atoms with at the most one bond to halogen attached in position 3
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings
    • C07D401/12—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings
    • C07D413/12—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pyridine Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
JP2023515741A 2020-09-11 2021-09-08 筋ジストロフィーを処置するための組成物及び方法 Pending JP2023541040A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063077329P 2020-09-11 2020-09-11
US63/077,329 2020-09-11
US202163148823P 2021-02-12 2021-02-12
US63/148,823 2021-02-12
PCT/US2021/049362 WO2022055926A1 (en) 2020-09-11 2021-09-08 Compositions and methods for treating muscular dystrophies

Publications (2)

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JP2023541040A true JP2023541040A (ja) 2023-09-27
JP2023541040A5 JP2023541040A5 (https=) 2024-09-17

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US (1) US20230348394A1 (https=)
EP (1) EP4211131A4 (https=)
JP (1) JP2023541040A (https=)
KR (1) KR20230067637A (https=)
AU (1) AU2021341960A1 (https=)
CA (1) CA3192071A1 (https=)
CL (1) CL2023000698A1 (https=)
CO (1) CO2023004447A2 (https=)
IL (1) IL301122A (https=)
MX (1) MX2023002947A (https=)
SA (1) SA523442922B1 (https=)
WO (1) WO2022055926A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2023007133A (es) 2020-12-17 2023-06-27 Hoffmann La Roche Anticuerpos anti-hla-g y uso de estos.
EP4705297A1 (en) * 2023-05-03 2026-03-11 The Regents Of The University Of California Small molecule therapy for duchenne muscular dystrophy

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994004535A1 (fr) * 1992-08-21 1994-03-03 Kyorin Pharmaceutical Co., Ltd. Derive d'ester carboxylique de trifluoromethylpyrroloindole et procede pour sa fabrication
WO2007008541A2 (en) * 2005-07-08 2007-01-18 Kalypsys, Inc. Cellular cholesterol absorption modifiers
WO2008059214A1 (en) * 2006-11-13 2008-05-22 Astrazeneca Ab Bisamlde derivatives and use thereof as fatty acid synthase inhibitors
JP2010535831A (ja) * 2007-08-15 2010-11-25 スムミト コーポレーション ピーエルシー デュシェンヌ型筋ジストロフィーの治療
WO2012098070A1 (en) * 2011-01-19 2012-07-26 F. Hoffmann-La Roche Ag Quinoline dyrk1 inhibitors
WO2018174288A1 (ja) * 2017-03-24 2018-09-27 大正製薬株式会社 ２（１ｈ）－キノリノン誘導体
JP2019502691A (ja) * 2015-12-17 2019-01-31 アステックス、セラピューティックス、リミテッドＡｓｔｅｘ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｌｉｍｉｔｅｄ Ｈ−ｐｇｄｓ阻害剤としてのキノリン−３−カルボキサミド
WO2019084499A1 (en) * 2017-10-27 2019-05-02 Genea Biocells USA (Holdings), Inc. INHIBITORS OF DUX4 INDUCTION FOR REGULATION OF MUSCLE FUNCTION
WO2019236677A1 (en) * 2018-06-05 2019-12-12 The Regents Of The University Of California Methods for treating muscular dystrophies

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
LT2502911T (lt) * 2004-06-24 2017-09-11 Vertex Pharmaceuticals Incorporated Atp surišančios kasetės transporterių moduliatoriai

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994004535A1 (fr) * 1992-08-21 1994-03-03 Kyorin Pharmaceutical Co., Ltd. Derive d'ester carboxylique de trifluoromethylpyrroloindole et procede pour sa fabrication
WO2007008541A2 (en) * 2005-07-08 2007-01-18 Kalypsys, Inc. Cellular cholesterol absorption modifiers
WO2008059214A1 (en) * 2006-11-13 2008-05-22 Astrazeneca Ab Bisamlde derivatives and use thereof as fatty acid synthase inhibitors
JP2010535831A (ja) * 2007-08-15 2010-11-25 スムミト コーポレーション ピーエルシー デュシェンヌ型筋ジストロフィーの治療
WO2012098070A1 (en) * 2011-01-19 2012-07-26 F. Hoffmann-La Roche Ag Quinoline dyrk1 inhibitors
JP2019502691A (ja) * 2015-12-17 2019-01-31 アステックス、セラピューティックス、リミテッドＡｓｔｅｘ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｌｉｍｉｔｅｄ Ｈ−ｐｇｄｓ阻害剤としてのキノリン−３−カルボキサミド
WO2018174288A1 (ja) * 2017-03-24 2018-09-27 大正製薬株式会社 ２（１ｈ）－キノリノン誘導体
WO2019084499A1 (en) * 2017-10-27 2019-05-02 Genea Biocells USA (Holdings), Inc. INHIBITORS OF DUX4 INDUCTION FOR REGULATION OF MUSCLE FUNCTION
WO2019236677A1 (en) * 2018-06-05 2019-12-12 The Regents Of The University Of California Methods for treating muscular dystrophies

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
"CID 855355", DATABASE PUBCHEM COMPOUND ［ONLINE］, JPN7025003700, 9 July 2005 (2005-07-09), ISSN: 0005672270 *
"RN:1100333-54-7", REGISTRY（STN）［ONLINE］, JPN7025003703, 3 February 2009 (2009-02-03), ISSN: 0005672266 *
"RN:1290417-03-6,RN:1288334-59-7,RN:1363813-11-9,RN:2183230-98-8,RN:1299645-51-4,RN:1294888-83-7,RN:1", REGISTRY（STN）［ONLINE］, JPN7025003698, 1 May 2011 (2011-05-01), ISSN: 0005672267 *
"RN:443744-97-6", REGISTRY（STN）［ONLINE］, JPN7025003704, 13 August 2002 (2002-08-13), ISSN: 0005817906 *
"RN:842960-38-7,RN:1794433-37-6", REGISTRY（STN）［ONLINE］, JPN7025003702, 6 March 2005 (2005-03-06), ISSN: 0005672268 *
"RN:943076-33-3,RN:1100333-54-7", REGISTRY（STN）［ONLINE］, JPN7025003701, 22 July 2007 (2007-07-22), ISSN: 0005672269 *
"SID 105219246", DATABASE PUBCHEM COMPOUND ［ONLINE］, JPN7025003699, 22 February 2011 (2011-02-22), ISSN: 0005672271 *

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Publication number Publication date
KR20230067637A (ko) 2023-05-16
IL301122A (en) 2023-05-01
AU2021341960A1 (en) 2023-04-20
SA523442922B1 (ar) 2024-08-25
CA3192071A1 (en) 2022-03-17
US20230348394A1 (en) 2023-11-02
CL2023000698A1 (es) 2023-10-30
WO2022055926A1 (en) 2022-03-17
EP4211131A4 (en) 2025-01-15
EP4211131A1 (en) 2023-07-19
CO2023004447A2 (es) 2023-04-27
MX2023002947A (es) 2023-06-06

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