CN113660939A - 使用卡西莫森治疗肌营养不良的方法 - Google Patents

使用卡西莫森治疗肌营养不良的方法 Download PDF

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Publication number
CN113660939A
CN113660939A CN202080022966.9A CN202080022966A CN113660939A CN 113660939 A CN113660939 A CN 113660939A CN 202080022966 A CN202080022966 A CN 202080022966A CN 113660939 A CN113660939 A CN 113660939A
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CN
China
Prior art keywords
patient
dose
exon
dystrophin
administered
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Pending
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CN202080022966.9A
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English (en)
Chinese (zh)
Inventor
E·M·凯
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Sarepta Therapeutics Inc
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Sarepta Therapeutics Inc
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Publication of CN113660939A publication Critical patent/CN113660939A/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN202080022966.9A 2019-03-28 2020-03-25 使用卡西莫森治疗肌营养不良的方法 Pending CN113660939A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962825573P 2019-03-28 2019-03-28
US62/825573 2019-03-28
US201962902518P 2019-09-19 2019-09-19
US62/902518 2019-09-19
PCT/US2020/024550 WO2020198268A1 (en) 2019-03-28 2020-03-25 Methods for treating muscular dystrophy with casimersen

Publications (1)

Publication Number Publication Date
CN113660939A true CN113660939A (zh) 2021-11-16

Family

ID=70286009

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080022966.9A Pending CN113660939A (zh) 2019-03-28 2020-03-25 使用卡西莫森治疗肌营养不良的方法

Country Status (15)

Country Link
US (2) US20220152086A1 (https=)
EP (1) EP3946376A1 (https=)
JP (2) JP2022526763A (https=)
KR (1) KR20210145192A (https=)
CN (1) CN113660939A (https=)
AU (2) AU2020244803A1 (https=)
BR (1) BR112021018801A2 (https=)
CA (1) CA3134165A1 (https=)
CL (1) CL2021002437A1 (https=)
CO (1) CO2021014024A2 (https=)
IL (2) IL318548A (https=)
MA (1) MA55515A (https=)
MX (1) MX2021011498A (https=)
SG (1) SG11202108757XA (https=)
WO (1) WO2020198268A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2021025899A1 (en) * 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer pharmaceutical compositions
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping

Citations (3)

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WO2018005805A1 (en) * 2016-06-30 2018-01-04 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
WO2018118627A1 (en) * 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
CN108738310A (zh) * 2015-09-30 2018-11-02 萨勒普塔医疗公司 用于治疗肌营养不良的方法

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Also Published As

Publication number Publication date
MX2021011498A (es) 2022-01-04
JP2025096396A (ja) 2025-06-26
EP3946376A1 (en) 2022-02-09
BR112021018801A2 (pt) 2021-11-23
IL286653A (en) 2021-10-31
IL318548A (en) 2025-03-01
CL2021002437A1 (es) 2022-05-06
US20260007690A1 (en) 2026-01-08
CO2021014024A2 (es) 2021-10-29
KR20210145192A (ko) 2021-12-01
CA3134165A1 (en) 2020-10-01
AU2025256263A1 (en) 2025-11-27
SG11202108757XA (en) 2021-10-28
JP2022526763A (ja) 2022-05-26
US20220152086A1 (en) 2022-05-19
AU2020244803A1 (en) 2021-11-18
MA55515A (fr) 2022-02-09
WO2020198268A1 (en) 2020-10-01

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Application publication date: 20211116