CN114555095A - 使用针对CD49d的抑制性寡核苷酸治疗肌营养不良症的方法 - Google Patents

使用针对CD49d的抑制性寡核苷酸治疗肌营养不良症的方法 Download PDF

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Publication number
CN114555095A
CN114555095A CN202080049373.1A CN202080049373A CN114555095A CN 114555095 A CN114555095 A CN 114555095A CN 202080049373 A CN202080049373 A CN 202080049373A CN 114555095 A CN114555095 A CN 114555095A
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Prior art keywords
muscle
cd49d
oligonucleotide
subject
dose
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Pending
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CN202080049373.1A
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English (en)
Chinese (zh)
Inventor
G·塔查斯
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Percheron Therapeutics Ltd
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Antisense Therapeutics Ltd
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Publication date
Priority claimed from AU2019901540A external-priority patent/AU2019901540A0/en
Application filed by Antisense Therapeutics Ltd filed Critical Antisense Therapeutics Ltd
Publication of CN114555095A publication Critical patent/CN114555095A/zh
Pending legal-status Critical Current

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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
    • A61K31/7115—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
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/31—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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • 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)
CN202080049373.1A 2019-05-06 2020-05-06 使用针对CD49d的抑制性寡核苷酸治疗肌营养不良症的方法 Pending CN114555095A (zh)

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
CN114555095A true CN114555095A (zh) 2022-05-27

Family

ID=73050454

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080049373.1A Pending CN114555095A (zh) 2019-05-06 2020-05-06 使用针对CD49d的抑制性寡核苷酸治疗肌营养不良症的方法

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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119113214A (zh) * 2024-11-14 2024-12-13 四川大学 一种肌肉修复材料及其制备方法和用途

Families Citing this family (2)

* 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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010008474A2 (en) * 2008-06-23 2010-01-21 Teva Pharmaceutical Industries, Ltd. Methods for treating multiple sclerosis using antisense oligonucleotides
WO2010129861A2 (en) * 2009-05-08 2010-11-11 Curna, Inc. Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
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
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010008474A2 (en) * 2008-06-23 2010-01-21 Teva Pharmaceutical Industries, Ltd. Methods for treating multiple sclerosis using antisense oligonucleotides
WO2010129861A2 (en) * 2009-05-08 2010-11-11 Curna, Inc. Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
CN102459597A (zh) * 2009-05-08 2012-05-16 欧科库尔纳有限责任公司 通过针对dmd家族的天然反义转录物的抑制治疗肌营养蛋白家族相关疾病
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
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
VOLKER LIMMROTH 等: "CD49d antisense drug ATL1102 reduces disease activity in patients with relapsing-remitting MS", NEUROLOGY, vol. 83, pages 1781 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119113214A (zh) * 2024-11-14 2024-12-13 四川大学 一种肌肉修复材料及其制备方法和用途
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

Also Published As

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
JP2022532098A (ja) 2022-07-13
US20220296631A1 (en) 2022-09-22
EP3965778A4 (en) 2023-05-31
CA3138945A1 (en) 2020-11-12

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