CN117279636A - 用于调节强直性肌营养不良1型的方法和化合物 - Google Patents

用于调节强直性肌营养不良1型的方法和化合物 Download PDF

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Publication number
CN117279636A
CN117279636A CN202180093566.1A CN202180093566A CN117279636A CN 117279636 A CN117279636 A CN 117279636A CN 202180093566 A CN202180093566 A CN 202180093566A CN 117279636 A CN117279636 A CN 117279636A
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optionally substituted
pharmaceutically acceptable
solvate
acceptable salt
alkyl
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Chinese (zh)
Inventor
阿西姆·安萨里
肖恩·J·杰弗里斯
普拉蒂克·沙阿
张承智
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Design Treatment Co
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Design Treatment Co
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • 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
    • 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
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4164—1,3-Diazoles
    • A61K31/4178—1,3-Diazoles not condensed 1,3-diazoles and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • 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
    • 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
    • C07D405/14—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 three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Polyesters Or Polycarbonates (AREA)
  • Excavating Of Shafts Or Tunnels (AREA)
  • Medicinal Preparation (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202180093566.1A 2020-12-11 2021-12-11 用于调节强直性肌营养不良1型的方法和化合物 Pending CN117279636A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063124592P 2020-12-11 2020-12-11
US63/124,592 2020-12-11
PCT/US2021/062974 WO2022126000A1 (en) 2020-12-11 2021-12-11 Methods and compounds for modulating myotonic dystrophy 1

Publications (1)

Publication Number Publication Date
CN117279636A true CN117279636A (zh) 2023-12-22

Family

ID=81974018

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180093566.1A Pending CN117279636A (zh) 2020-12-11 2021-12-11 用于调节强直性肌营养不良1型的方法和化合物

Country Status (7)

Country Link
US (1) US20240166693A1 (https=)
EP (1) EP4259133A4 (https=)
JP (1) JP2023554317A (https=)
CN (1) CN117279636A (https=)
AU (1) AU2021396454A1 (https=)
CA (1) CA3201756A1 (https=)
WO (1) WO2022126000A1 (https=)

Families Citing this family (5)

* 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
US20250120928A1 (en) * 2021-07-14 2025-04-17 University Of Maryland, Baltimore Suberoylanilide hydroxamic acid (saha) drugs, conjugates, and nanoparticles, and methods of use thereof
KR20250016096A (ko) * 2022-04-01 2025-02-03 디자인 테라퓨틱스, 인크. 헌팅턴병을 조절하기 위한 방법 및 화합물
US11976066B1 (en) 2023-10-23 2024-05-07 King Faisal University Substituted 7-amino-3-(substituted benzoyl)indolizine-1-carboxylates as anti-tubercular agents
GB202401731D0 (en) * 2024-02-08 2024-03-27 Univ Strathclyde Polyamides

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998037087A1 (en) * 1996-02-26 1998-08-27 California Institute Of Technology Inhibition of major groove dna binding proteins by modified polyamides
US6506906B1 (en) * 1996-02-26 2003-01-14 California Institute Of Technology Preparation and use of bifunctional molecules having DNA sequence binding specificity
US20060270727A1 (en) * 2005-05-03 2006-11-30 Christian Melander Small molecule therapeutics and uses therefor
WO2017172914A1 (en) * 2016-03-30 2017-10-05 Wisconsin Alumni Research Foundation Methods and compositions for modulating frataxin expression
WO2019226835A1 (en) * 2018-05-22 2019-11-28 Design Therapeutics Inc. Methods and compounds for the treatment of genetic disease

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0722088D0 (en) * 2007-11-09 2007-12-19 Spirogen Ltd Pyrrolobenzodiazepines
WO2010001933A1 (ja) * 2008-07-01 2010-01-07 学校法人日本大学 標的遺伝子特異的ヒストン修飾制御剤
US20190343873A1 (en) * 2018-05-14 2019-11-14 Kyoto University Sequence-selective gene expression regulators

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998037087A1 (en) * 1996-02-26 1998-08-27 California Institute Of Technology Inhibition of major groove dna binding proteins by modified polyamides
US6506906B1 (en) * 1996-02-26 2003-01-14 California Institute Of Technology Preparation and use of bifunctional molecules having DNA sequence binding specificity
US20060270727A1 (en) * 2005-05-03 2006-11-30 Christian Melander Small molecule therapeutics and uses therefor
WO2017172914A1 (en) * 2016-03-30 2017-10-05 Wisconsin Alumni Research Foundation Methods and compositions for modulating frataxin expression
WO2019226835A1 (en) * 2018-05-22 2019-11-28 Design Therapeutics Inc. Methods and compounds for the treatment of genetic disease

Also Published As

Publication number Publication date
US20240166693A1 (en) 2024-05-23
WO2022126000A1 (en) 2022-06-16
CA3201756A1 (en) 2022-06-16
EP4259133A4 (en) 2024-11-27
EP4259133A1 (en) 2023-10-18
JP2023554317A (ja) 2023-12-27
AU2021396454A1 (en) 2023-07-20

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