KR20220061915A - Cd49d에 대한 억제성 올리고뉴클레오티드를 사용하는 근이영양증의 치료 방법 - Google Patents

Cd49d에 대한 억제성 올리고뉴클레오티드를 사용하는 근이영양증의 치료 방법 Download PDF

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KR20220061915A
KR20220061915A KR1020217039906A KR20217039906A KR20220061915A KR 20220061915 A KR20220061915 A KR 20220061915A KR 1020217039906 A KR1020217039906 A KR 1020217039906A KR 20217039906 A KR20217039906 A KR 20217039906A KR 20220061915 A KR20220061915 A KR 20220061915A
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muscle
cd49d
subject
oligonucleotide
dosing
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Korean (ko)
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조지 타차스
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안티센스 테라퓨틱스 엘티디
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
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  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020217039906A 2019-05-06 2020-05-06 Cd49d에 대한 억제성 올리고뉴클레오티드를 사용하는 근이영양증의 치료 방법 Ceased KR20220061915A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2019901540A AU2019901540A0 (en) 2019-05-06 Therapeutic uses and methods
AU2019901540 2019-05-06
PCT/AU2020/050445 WO2020223762A1 (en) 2019-05-06 2020-05-06 Methods for treating muscular dystrophy using inhibitory oligonucleotides to cd49d

Publications (1)

Publication Number Publication Date
KR20220061915A true KR20220061915A (ko) 2022-05-13

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KR1020217039906A Ceased KR20220061915A (ko) 2019-05-06 2020-05-06 Cd49d에 대한 억제성 올리고뉴클레오티드를 사용하는 근이영양증의 치료 방법

Country Status (9)

Country Link
US (1) US20220296631A1 (https=)
EP (1) EP3965778A4 (https=)
JP (1) JP2022532098A (https=)
KR (1) KR20220061915A (https=)
CN (1) CN114555095A (https=)
AU (1) AU2020269078A1 (https=)
BR (1) BR112021022208A2 (https=)
CA (1) CA3138945A1 (https=)
WO (1) WO2020223762A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IT202100014333A1 (it) * 2021-06-01 2022-12-01 Materias S R L Nuovo impiego terapeutico di inibitori della iodiotironina deiodinasi di tipo 2 (D2)
WO2024159266A1 (en) * 2023-02-01 2024-08-08 Antisense Therapeutics Ltd Combination compositions and methods for treatment of muscular dystrophy
CN119113214B (zh) * 2024-11-14 2025-02-11 四川大学 一种肌肉修复材料及其制备方法和用途

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2318424B1 (en) * 2008-06-23 2015-02-25 Antisense Therapeutics Ltd Methods for treating multiple sclerosis using antisense oligonucleotides
JP5931720B2 (ja) * 2009-05-08 2016-06-08 クルナ・インコーポレーテッド Ｄｍｄファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療
EP2467159A1 (en) * 2009-08-20 2012-06-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Vla-4 as a biomarker for prognosis and target for therapy in duchenne muscular dystrophy
EP3858993A1 (en) * 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
AU2018286483C1 (en) * 2017-06-16 2024-10-31 Antisense Therapeutics Ltd Methods for treating multiple sclerosis using antisense oligonucleotides
KR20210018820A (ko) * 2018-05-04 2021-02-18 안티센스 테라퓨틱스 엘티디 치료 용도 및 방법

Also Published As

Publication number Publication date
WO2020223762A1 (en) 2020-11-12
BR112021022208A2 (pt) 2022-01-11
EP3965778A1 (en) 2022-03-16
AU2020269078A1 (en) 2022-01-06
CN114555095A (zh) 2022-05-27
JP2022532098A (ja) 2022-07-13
US20220296631A1 (en) 2022-09-22
EP3965778A4 (en) 2023-05-31
CA3138945A1 (en) 2020-11-12

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