AU2023205889A1 - Compounds and methods for treating friedreich's ataxia - Google Patents

Compounds and methods for treating friedreich's ataxia Download PDF

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Publication number
AU2023205889A1
AU2023205889A1 AU2023205889A AU2023205889A AU2023205889A1 AU 2023205889 A1 AU2023205889 A1 AU 2023205889A1 AU 2023205889 A AU2023205889 A AU 2023205889A AU 2023205889 A AU2023205889 A AU 2023205889A AU 2023205889 A1 AU2023205889 A1 AU 2023205889A1
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AU
Australia
Prior art keywords
optionally substituted
methyl
alkyl
compound
amido
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Pending
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AU2023205889A
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English (en)
Inventor
Aseem Ansari
Abhijit Bhat
Sean 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 AU2023205889A1 publication Critical patent/AU2023205889A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • 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
    • A61K47/595—Polyamides, e.g. nylon
    • 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
    • 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
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
AU2023205889A 2022-01-06 2023-01-06 Compounds and methods for treating friedreich's ataxia Pending AU2023205889A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263297090P 2022-01-06 2022-01-06
US63/297,090 2022-01-06
US202263382854P 2022-11-08 2022-11-08
US63/382,854 2022-11-08
PCT/US2023/010331 WO2023133284A2 (en) 2022-01-06 2023-01-06 Compounds and methods for treating friedreich's ataxia

Publications (1)

Publication Number Publication Date
AU2023205889A1 true AU2023205889A1 (en) 2024-08-15

Family

ID=87074158

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2023205889A Pending AU2023205889A1 (en) 2022-01-06 2023-01-06 Compounds and methods for treating friedreich's ataxia

Country Status (14)

Country Link
US (1) US20250109132A1 (https=)
EP (1) EP4460302A4 (https=)
JP (1) JP2025502064A (https=)
KR (1) KR20240138079A (https=)
AU (1) AU2023205889A1 (https=)
CA (1) CA3246468A1 (https=)
CL (1) CL2024002029A1 (https=)
CO (1) CO2024010651A2 (https=)
CR (1) CR20240321A (https=)
IL (1) IL314006A (https=)
MX (1) MX2024008474A (https=)
PE (1) PE20241730A1 (https=)
TW (1) TW202334148A (https=)
WO (1) WO2023133284A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025076181A1 (en) * 2023-10-03 2025-04-10 Design Therapeutics, Inc. Methods and compounds for modulating huntington's disease
WO2025076219A1 (en) * 2023-10-03 2025-04-10 Design Therapeutics, Inc. Methods and compounds for modulating huntington's disease
WO2025076236A1 (en) * 2023-10-03 2025-04-10 Design Therapeutics, Inc. Methods and compounds for modulating huntington's disease

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013097052A1 (en) * 2011-12-30 2013-07-04 Abbott Laboratories Bromodomain inhibitors
CN106068266A (zh) * 2013-10-11 2016-11-02 千叶县 以癌症驱动基因的基因突变为靶点进行烷基化的新型烷基化剂
US11759533B2 (en) * 2017-03-29 2023-09-19 Wisconsin Alumni Research Foundation Methods and compositions for modulating gene expression
WO2020253711A1 (zh) * 2019-06-17 2020-12-24 中国科学院上海药物研究所 一种吡咯并吡啶酮类化合物、其制备方法、其组合物和用途
KR102677015B1 (ko) * 2019-07-02 2024-06-19 누베이션 바이오 인크. Bet 억제제로서의 헤테로시클릭 화합물
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

Also Published As

Publication number Publication date
WO2023133284A3 (en) 2023-08-24
CO2024010651A2 (es) 2024-08-08
CA3246468A1 (en) 2023-07-13
CR20240321A (es) 2024-10-16
EP4460302A2 (en) 2024-11-13
CL2024002029A1 (es) 2024-11-04
JP2025502064A (ja) 2025-01-24
KR20240138079A (ko) 2024-09-20
MX2024008474A (es) 2024-07-12
TW202334148A (zh) 2023-09-01
US20250109132A1 (en) 2025-04-03
IL314006A (en) 2024-08-01
PE20241730A1 (es) 2024-08-19
EP4460302A4 (en) 2025-12-24
WO2023133284A2 (en) 2023-07-13

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