CN115996916B - 用于预防和/或治疗包括弗里德赖希共济失调的线粒体疾病的组合物和方法 - Google Patents

用于预防和/或治疗包括弗里德赖希共济失调的线粒体疾病的组合物和方法

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Publication number
CN115996916B
CN115996916B CN202180040296.8A CN202180040296A CN115996916B CN 115996916 B CN115996916 B CN 115996916B CN 202180040296 A CN202180040296 A CN 202180040296A CN 115996916 B CN115996916 B CN 115996916B
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CN115996916A (zh
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D·基夫
郑国柱
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Kant Biomedical Co ltd
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Kant Biomedical Co ltd
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Priority to CN202511246906.6A priority patent/CN121085756A/zh
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C50/00—Quinones
    • C07C50/26—Quinones containing groups having oxygen atoms singly bound to carbon atoms
    • C07C50/28—Quinones containing groups having oxygen atoms singly bound to carbon atoms with monocyclic quinoid structure
    • 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
    • A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/05—Phenols
    • 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
    • A61K31/12—Ketones
    • A61K31/122—Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
    • 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
    • A61K31/695—Silicon compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • 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
    • A61P27/02—Ophthalmic agents
    • 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
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C43/00—Ethers; Compounds having groups, groups or groups
    • C07C43/02—Ethers
    • C07C43/20—Ethers having an ether-oxygen atom bound to a carbon atom of a six-membered aromatic ring
    • C07C43/23—Ethers having an ether-oxygen atom bound to a carbon atom of a six-membered aromatic ring containing hydroxy or O-metal groups
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C50/00—Quinones
    • C07C50/02—Quinones with monocyclic quinoid structure
    • C07C50/06—Quinones with monocyclic quinoid structure with unsaturation outside the quinoid structure
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C50/00—Quinones
    • C07C50/26—Quinones containing groups having oxygen atoms singly bound to carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D317/00—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms
    • C07D317/08—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3
    • C07D317/44—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 ortho- or peri-condensed with carbocyclic rings or ring systems
    • C07D317/46—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 ortho- or peri-condensed with carbocyclic rings or ring systems condensed with one six-membered ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D319/00—Heterocyclic compounds containing six-membered rings having two oxygen atoms as the only ring hetero atoms
    • C07D319/10—1,4-Dioxanes; Hydrogenated 1,4-dioxanes
    • C07D319/14—1,4-Dioxanes; Hydrogenated 1,4-dioxanes condensed with carbocyclic rings or ring systems
    • C07D319/16—1,4-Dioxanes; Hydrogenated 1,4-dioxanes condensed with carbocyclic rings or ring systems condensed with one six-membered ring
    • C07D319/18—Ethylenedioxybenzenes, not substituted on the hetero ring
    • 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
    • C07F7/00—Compounds containing elements of Groups 4 or 14 of the Periodic Table
    • C07F7/02—Silicon compounds
    • C07F7/08—Compounds having one or more C—Si linkages
    • C07F7/0803—Compounds with Si-C or Si-Si linkages
    • C07F7/081—Compounds with Si-C or Si-Si linkages comprising at least one atom selected from the elements N, O, halogen, S, Se or Te
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2601/00—Systems containing only non-condensed rings
    • C07C2601/12—Systems containing only non-condensed rings with a six-membered ring
    • C07C2601/16—Systems containing only non-condensed rings with a six-membered ring the ring being unsaturated

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Engineering & Computer Science (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Diabetes (AREA)
  • Neurology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Rheumatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Hydrogenated Pyridines (AREA)
CN202180040296.8A 2020-04-03 2021-04-02 用于预防和/或治疗包括弗里德赖希共济失调的线粒体疾病的组合物和方法 Active CN115996916B (zh)

Priority Applications (3)

Application Number Priority Date Filing Date Title
CN202511246796.3A CN121085765A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法
CN202510664780.8A CN120554211A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法
CN202511246906.6A CN121085756A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063004639P 2020-04-03 2020-04-03
US63/004,639 2020-04-03
PCT/US2021/025558 WO2021202986A1 (en) 2020-04-03 2021-04-02 Compositions and methods for the prevention and/or treatment of mitochondrial disease, including friedreich's ataxia

Related Child Applications (3)

Application Number Title Priority Date Filing Date
CN202511246796.3A Division CN121085765A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法
CN202511246906.6A Division CN121085756A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法
CN202510664780.8A Division CN120554211A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法

Publications (2)

Publication Number Publication Date
CN115996916A CN115996916A (zh) 2023-04-21
CN115996916B true CN115996916B (zh) 2025-09-23

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CN202180040296.8A Active CN115996916B (zh) 2020-04-03 2021-04-02 用于预防和/或治疗包括弗里德赖希共济失调的线粒体疾病的组合物和方法
CN202511246906.6A Pending CN121085756A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法
CN202511246796.3A Pending CN121085765A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法
CN202510664780.8A Pending CN120554211A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法

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CN202511246906.6A Pending CN121085756A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法
CN202511246796.3A Pending CN121085765A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法
CN202510664780.8A Pending CN120554211A (zh) 2020-04-03 2021-04-02 用于预防和/或治疗线粒体疾病的组合物和方法

Country Status (9)

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US (2) US12037350B2 (https=)
EP (1) EP4126836A1 (https=)
JP (1) JP2023520462A (https=)
KR (1) KR20230005855A (https=)
CN (4) CN115996916B (https=)
AU (1) AU2021246142A1 (https=)
CA (1) CA3176909A1 (https=)
IL (1) IL296985A (https=)
WO (1) WO2021202986A1 (https=)

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CA3222132A1 (en) 2021-06-10 2022-12-15 Stealth Biotherapeutics Inc. Compounds, compositions and methods for the prevention and/or treatment of various mitochondrial diseases or disorders, including friedreich's ataxia
US11541116B1 (en) 2022-01-07 2023-01-03 Kojin Therapeutics, Inc. Methods and compositions for inducing ferroptosis in vivo
WO2024263742A1 (en) * 2023-06-22 2024-12-26 Stealth Biotherapeutics Inc. Combination therapy for the treatment of friedreich's ataxia
WO2025024247A1 (en) * 2023-07-21 2025-01-30 Stealth Biotherapeutics Inc. Methods, compositions and uses related to the treatment and prevention of leigh's syndrome
WO2025101748A1 (en) 2023-11-09 2025-05-15 Stealth Biotherapeutics Inc. Cyclo-hexa-2,5-diene-1,4-diones for use in the treatment of cardiomyopathy

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JP2023520462A (ja) 2023-05-17
CN121085765A (zh) 2025-12-09
CA3176909A1 (en) 2021-10-07
CN121085756A (zh) 2025-12-09
WO2021202986A1 (en) 2021-10-07
CN120554211A (zh) 2025-08-29
US20240400588A1 (en) 2024-12-05
US12037350B2 (en) 2024-07-16
IL296985A (en) 2022-12-01
AU2021246142A1 (en) 2022-10-20
US20210317146A1 (en) 2021-10-14
EP4126836A1 (en) 2023-02-08
KR20230005855A (ko) 2023-01-10
CN115996916A (zh) 2023-04-21

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