JP2022532098A - ＣＤ４９ｄに対する阻害オリゴヌクレオチドを使用して筋ジストロフィーを治療する方法 - Google Patents

ＣＤ４９ｄに対する阻害オリゴヌクレオチドを使用して筋ジストロフィーを治療する方法 Download PDF

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JP2022532098A
JP2022532098A JP2021566041A JP2021566041A JP2022532098A JP 2022532098 A JP2022532098 A JP 2022532098A JP 2021566041 A JP2021566041 A JP 2021566041A JP 2021566041 A JP2021566041 A JP 2021566041A JP 2022532098 A JP2022532098 A JP 2022532098A
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muscle
cd49d
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oligonucleotide
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ジョージ・タチャス
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アンチセンス セラピューティクス リミテッド
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    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • 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
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    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C—CHEMISTRY; METALLURGY
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    • 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)
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  • 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)
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  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
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  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021566041A 2019-05-06 2020-05-06 ＣＤ４９ｄに対する阻害オリゴヌクレオチドを使用して筋ジストロフィーを治療する方法 Ceased JP2022532098A (ja)

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 (2)

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JP2022532098A true JP2022532098A (ja) 2022-07-13
JP2022532098A5 JP2022532098A5 (https=) 2023-05-16

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JP2021566041A Ceased JP2022532098A (ja) 2019-05-06 2020-05-06 ＣＤ４９ｄに対する阻害オリゴヌクレオチドを使用して筋ジストロフィーを治療する方法

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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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011020874A1 (en) * 2009-08-20 2011-02-24 Inserm (Institut National De La Sante Et De La Recherche Medicale) Vla-4 as a biomarker for prognosis and target for therapy in duchenne muscular dystrophy
JP2021522346A (ja) * 2018-05-04 2021-08-30 アンチセンス セラピューティクス リミテッド 治療での使用及び方法

Family Cites Families (4)

* 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
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011020874A1 (en) * 2009-08-20 2011-02-24 Inserm (Institut National De La Sante Et De La Recherche Medicale) Vla-4 as a biomarker for prognosis and target for therapy in duchenne muscular dystrophy
US20120258093A1 (en) * 2009-08-20 2012-10-11 Institut National De La Sante Et De La Recherche Medicale (Inserm) Vla-4 as a biomarker for prognosis and target for therapy in duchenne muscular dystrophy
JP2021522346A (ja) * 2018-05-04 2021-08-30 アンチセンス セラピューティクス リミテッド 治療での使用及び方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
FERNANDA PINTO-MARIZ ET AL.: ""CD49d is a disease progression biomarker and a potential target for immunotherapy in Duchenne musc", SKELETAL MUSCLE, vol. 5, no. 1, JPN6024020203, December 2015 (2015-12-01), XP055650015, ISSN: 0005331015, DOI: 10.1186/s13395-015-0066-2 *
WILSON SAVINO ET AL.: ""Flow Cytometry-Defined CD49d Expression in Circulating T-Lymphocytes Is a Biomarker for Disease Pr", METHODS IN MOLECULAR BIOLOGY, JPN6024020202, 25 October 2017 (2017-10-25), pages 219 - 227, ISSN: 0005331016 *

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Publication number Publication date
WO2020223762A1 (en) 2020-11-12
BR112021022208A2 (pt) 2022-01-11
EP3965778A1 (en) 2022-03-16
KR20220061915A (ko) 2022-05-13
AU2020269078A1 (en) 2022-01-06
CN114555095A (zh) 2022-05-27
US20220296631A1 (en) 2022-09-22
EP3965778A4 (en) 2023-05-31
CA3138945A1 (en) 2020-11-12

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