JP2024502469A - フリードライヒ運動失調症を治療するための方法及び化合物 - Google Patents

フリードライヒ運動失調症を治療するための方法及び化合物 Download PDF

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JP2024502469A
JP2024502469A JP2023541510A JP2023541510A JP2024502469A JP 2024502469 A JP2024502469 A JP 2024502469A JP 2023541510 A JP2023541510 A JP 2023541510A JP 2023541510 A JP2023541510 A JP 2023541510A JP 2024502469 A JP2024502469 A JP 2024502469A
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アンサリ,アシーム
バーツ,アビー
ジェフリーズ,ショーン
シャー,プラティック
ジャン,チェンジ
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デザイン セラピューティクス,インク.
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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D401/00Heterocyclic 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
    • C07D401/14Heterocyclic 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 three or more hetero rings
    • 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
    • C07D495/12Heterocyclic 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 three hetero rings
    • C07D495/14Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D498/00Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D498/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D519/00Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

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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)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Indole Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Photoreceptors In Electrophotography (AREA)
  • Polyethers (AREA)
  • Medicinal Preparation (AREA)
JP2023541510A 2021-01-08 2022-01-07 フリードライヒ運動失調症を治療するための方法及び化合物 Pending JP2024502469A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163135427P 2021-01-08 2021-01-08
US63/135,427 2021-01-08
PCT/US2022/011560 WO2022150555A2 (en) 2021-01-08 2022-01-07 Methods and compounds for treating friedreich's ataxia

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JP2024502469A true JP2024502469A (ja) 2024-01-19
JP2024502469A5 JP2024502469A5 (https=) 2025-01-09

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JP2023541510A Pending JP2024502469A (ja) 2021-01-08 2022-01-07 フリードライヒ運動失調症を治療するための方法及び化合物

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US (1) US20240124491A1 (https=)
EP (1) EP4274583A4 (https=)
JP (1) JP2024502469A (https=)
CN (1) CN117241805A (https=)
AR (1) AR124589A1 (https=)
AU (1) AU2022206424A1 (https=)
CA (1) CA3204523A1 (https=)
IL (1) IL304322A (https=)
MX (1) MX2023008153A (https=)
TW (1) TW202241892A (https=)
WO (1) WO2022150555A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024502469A (ja) * 2021-01-08 2024-01-19 デザイン セラピューティクス,インク. フリードライヒ運動失調症を治療するための方法及び化合物
JP2025502064A (ja) * 2022-01-06 2025-01-24 デザイン セラピューティクス,インク. フリードライヒ運動失調症を治療するための化合物及び方法
CN119708005A (zh) * 2023-09-27 2025-03-28 北京沐华生物科技有限责任公司 一类brd4蛋白降解剂化合物及其制备方法和应用

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060270727A1 (en) * 2005-05-03 2006-11-30 Christian Melander Small molecule therapeutics and uses therefor
WO2015074081A1 (en) * 2013-11-18 2015-05-21 Bair Kenneth W Benzopiperazine compositions as bet bromodomain inhibitors
WO2017172914A1 (en) * 2016-03-30 2017-10-05 Wisconsin Alumni Research Foundation Methods and compositions for modulating frataxin expression
JP2018509468A (ja) * 2015-03-13 2018-04-05 ザ ユニバーシティ オブ ダンディーThe University Of Dundee 増殖性、自己免疫性または炎症性疾患の処置に有用な1−[(シクロペンチルまたは2−ピロリジニル)カルボニルアミノメチル]−4−(1,3−チアゾール−5−イル)ベンゼンの誘導体
JP2019511482A (ja) * 2016-04-15 2019-04-25 アッヴィ・インコーポレイテッド ブロモドメイン阻害剤
WO2019226836A1 (en) * 2018-05-22 2019-11-28 Design Therapeutics Inc. Methods and compounds for the treatment of genetic disease
WO2020056188A1 (en) * 2018-09-12 2020-03-19 Board Of Regents, The University Of Texas System Combination of parp inhibitor and brd4 inhibitor for the treatment of cancer
JP2023516886A (ja) * 2020-02-03 2023-04-21 デザイン セラピューティクス,インク. 遺伝性疾患の処置のための方法および化合物
JP2023554317A (ja) * 2020-12-11 2023-12-27 デザイン セラピューティクス,インク. 筋強直性ジストロフィー1を調節するための方法及び化合物

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019204781A1 (en) * 2018-04-20 2019-10-24 Design Therapeutics Inc. Methods and compounds for the treatment of genetic disease
JP2024502469A (ja) * 2021-01-08 2024-01-19 デザイン セラピューティクス,インク. フリードライヒ運動失調症を治療するための方法及び化合物

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060270727A1 (en) * 2005-05-03 2006-11-30 Christian Melander Small molecule therapeutics and uses therefor
WO2015074081A1 (en) * 2013-11-18 2015-05-21 Bair Kenneth W Benzopiperazine compositions as bet bromodomain inhibitors
JP2018509468A (ja) * 2015-03-13 2018-04-05 ザ ユニバーシティ オブ ダンディーThe University Of Dundee 増殖性、自己免疫性または炎症性疾患の処置に有用な1−[(シクロペンチルまたは2−ピロリジニル)カルボニルアミノメチル]−4−(1,3−チアゾール−5−イル)ベンゼンの誘導体
WO2017172914A1 (en) * 2016-03-30 2017-10-05 Wisconsin Alumni Research Foundation Methods and compositions for modulating frataxin expression
JP2019511482A (ja) * 2016-04-15 2019-04-25 アッヴィ・インコーポレイテッド ブロモドメイン阻害剤
WO2019226836A1 (en) * 2018-05-22 2019-11-28 Design Therapeutics Inc. Methods and compounds for the treatment of genetic disease
WO2020056188A1 (en) * 2018-09-12 2020-03-19 Board Of Regents, The University Of Texas System Combination of parp inhibitor and brd4 inhibitor for the treatment of cancer
JP2023516886A (ja) * 2020-02-03 2023-04-21 デザイン セラピューティクス,インク. 遺伝性疾患の処置のための方法および化合物
JP2023554317A (ja) * 2020-12-11 2023-12-27 デザイン セラピューティクス,インク. 筋強直性ジストロフィー1を調節するための方法及び化合物

Non-Patent Citations (3)

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Title
BRADBURY,R.H. ET AL.: "Optimization of a Series of Bivalent Triazolopyridazine Based Bromodomain and Extraterminal Inhibito", JOURNAL OF MEDICINAL CHEMISTRY, vol. 59, no. 17, JPN6025042284, 2016, pages 7801 - 7817, ISSN: 0005709764 *
LAW,R.P. ET AL.: "Discovery of Tetrahydroquinoxalines as Bromodomain and Extra-Terminal Domain (BET) Inhibitors with S", JOURNAL OF MEDICINAL CHEMISTRY, vol. 61, no. 10, JPN6025042283, 2018, pages 4317 - 4334, ISSN: 0005709765 *
SHEPPARD,G.S. ET AL.: "Discovery of N-Ethyl-4-[2-(4-fluoro-2,6-dimethyl-phenoxy)-5-(1-hydroxy-1-methyl-ethyl)phenyl]-6-meth", JOURNAL OF MEDICINAL CHEMISTRY, vol. 63, no. 10, JPN6025042282, 2020, pages 5585 - 5623, ISSN: 0005709766 *

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Publication number Publication date
CA3204523A1 (en) 2022-07-14
IL304322A (en) 2023-09-01
MX2023008153A (es) 2023-09-21
TW202241892A (zh) 2022-11-01
AU2022206424A1 (en) 2023-08-24
AU2022206424A9 (en) 2024-09-19
WO2022150555A3 (en) 2022-12-22
CN117241805A (zh) 2023-12-15
WO2022150555A2 (en) 2022-07-14
US20240124491A1 (en) 2024-04-18
EP4274583A4 (en) 2025-01-22
EP4274583A2 (en) 2023-11-15
AR124589A1 (es) 2023-04-12

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