JP2023516886A - 遺伝性疾患の処置のための方法および化合物 - Google Patents

遺伝性疾患の処置のための方法および化合物 Download PDF

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JP2023516886A
JP2023516886A JP2022546698A JP2022546698A JP2023516886A JP 2023516886 A JP2023516886 A JP 2023516886A JP 2022546698 A JP2022546698 A JP 2022546698A JP 2022546698 A JP2022546698 A JP 2022546698A JP 2023516886 A JP2023516886 A JP 2023516886A
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methyl
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modulator molecule
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アンサリ，アシーム
ジェイ 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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    • 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
    • C07D495/12—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 three hetero rings
    • C07D495/14—Ortho-condensed systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/545—Heterocyclic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C07D401/14—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 three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic 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
    • C07D471/02—Heterocyclic 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
    • C07D471/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic 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
    • C07D487/04—Ortho-condensed systems

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
  • Medicinal Preparation (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
JP2022546698A 2020-02-03 2021-02-03 遺伝性疾患の処置のための方法および化合物 Pending JP2023516886A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062969644P 2020-02-03 2020-02-03
US62/969,644 2020-02-03
US202163135476P 2021-01-08 2021-01-08
US63/135,476 2021-01-08
PCT/US2021/016481 WO2021158707A1 (en) 2020-02-03 2021-02-03 Methods and compounds for the treatment of genetic disease

Publications (2)

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JP2023516886A true JP2023516886A (ja) 2023-04-21
JP2023516886A5 JP2023516886A5 (https=) 2024-02-21

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US (2) US20230149550A1 (https=)
EP (1) EP4100404A4 (https=)
JP (1) JP2023516886A (https=)
CN (2) CN121045206A (https=)
CA (1) CA3169810A1 (https=)
TW (1) TW202142261A (https=)
WO (1) WO2021158707A1 (https=)

Cited By (1)

* 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

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WO2023014793A1 (en) * 2021-08-04 2023-02-09 Design Therapeutics, Inc. Process for polyamide synthesis
KR20250016096A (ko) * 2022-04-01 2025-02-03 디자인 테라퓨틱스, 인크. 헌팅턴병을 조절하기 위한 방법 및 화합물
US20250295632A1 (en) * 2022-05-09 2025-09-25 Design Therapeutics, Inc. Compounds and methods for treating friedreich's ataxia
WO2025111500A1 (en) * 2023-11-22 2025-05-30 Ionis Pharmaceuticals, Inc. Modulation of gene expression
WO2025122762A1 (en) * 2023-12-08 2025-06-12 Design Therapeutics, Inc. Compounds and methods for treating sickle cell disease

Citations (6)

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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
JP2019199471A (ja) * 2018-05-14 2019-11-21 国立大学法人京都大学 配列選択的遺伝子活性制御剤
WO2019226836A1 (en) * 2018-05-22 2019-11-28 Design Therapeutics Inc. Methods and compounds for the treatment of genetic disease

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US11759533B2 (en) * 2017-03-29 2023-09-19 Wisconsin Alumni Research Foundation Methods and compositions for modulating gene expression
AU2019368245A1 (en) * 2018-10-23 2021-05-20 Wisconsin Alumni Research Foundation Next generation synthetic transcription factors

Patent Citations (6)

* 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
JP2019199471A (ja) * 2018-05-14 2019-11-21 国立大学法人京都大学 配列選択的遺伝子活性制御剤
WO2019226836A1 (en) * 2018-05-22 2019-11-28 Design Therapeutics Inc. Methods and compounds for the treatment of genetic disease

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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

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TW202142261A (zh) 2021-11-16
CA3169810A1 (en) 2021-08-12
EP4100404A4 (en) 2024-05-22
US20240050576A1 (en) 2024-02-15
EP4100404A1 (en) 2022-12-14
CN115768765A (zh) 2023-03-07
US20230149550A1 (en) 2023-05-18
WO2021158707A1 (en) 2021-08-12
CN121045206A (zh) 2025-12-02

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