AU2021396454A1 - Methods and compounds for modulating myotonic dystrophy 1 - Google Patents

Methods and compounds for modulating myotonic dystrophy 1 Download PDF

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Publication number
AU2021396454A1
AU2021396454A1 AU2021396454A AU2021396454A AU2021396454A1 AU 2021396454 A1 AU2021396454 A1 AU 2021396454A1 AU 2021396454 A AU2021396454 A AU 2021396454A AU 2021396454 A AU2021396454 A AU 2021396454A AU 2021396454 A1 AU2021396454 A1 AU 2021396454A1
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AU
Australia
Prior art keywords
optionally substituted
alkyl
pharmaceutically acceptable
solvate
acceptable salt
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Pending
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AU2021396454A
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English (en)
Inventor
Aseem Ansari
Sean J. JEFFRIES
Pratik Shah
Chengzhi Zhang
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Design Therapeutics Inc
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Design Therapeutics Inc
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Publication of AU2021396454A1 publication Critical patent/AU2021396454A1/en
Pending legal-status Critical Current

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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

Landscapes

  • 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)
AU2021396454A 2020-12-11 2021-12-11 Methods and compounds for modulating myotonic dystrophy 1 Pending AU2021396454A1 (en)

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
AU2021396454A1 true AU2021396454A1 (en) 2023-07-20

Family

ID=81974018

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021396454A Pending AU2021396454A1 (en) 2020-12-11 2021-12-11 Methods and compounds for modulating myotonic dystrophy 1

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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1260006A (zh) * 1996-02-26 2000-07-12 加利福尼亚州技术学院 双链dna和杂环低聚物之间复合物的形成
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
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
US10517877B2 (en) * 2016-03-30 2019-12-31 Wisconsin Alumni Research Foundation Compounds and methods for modulating frataxin expression
US20190343873A1 (en) * 2018-05-14 2019-11-14 Kyoto University Sequence-selective gene expression regulators
EP4234549A1 (en) * 2018-05-22 2023-08-30 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
CN117279636A (zh) 2023-12-22

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