JP7652713B2 - 新規の網膜色素変性処置 - Google Patents

新規の網膜色素変性処置 Download PDF

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JP7652713B2
JP7652713B2 JP2021569934A JP2021569934A JP7652713B2 JP 7652713 B2 JP7652713 B2 JP 7652713B2 JP 2021569934 A JP2021569934 A JP 2021569934A JP 2021569934 A JP2021569934 A JP 2021569934A JP 7652713 B2 JP7652713 B2 JP 7652713B2
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cnot3
expression
antisense
antisense oligomer
oligomer
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JP2022534086A5 (enExample
JP2022534086A (ja
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スー フレッチャー，
イアンテ ピットアウト，
ジェイナ グライノク，
スティーブ ディー． ウィルトン，
フレッド ケー． チェン，
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ビジョン ファーマ ピーティーワイ リミテッド
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    • 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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
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    • A—HUMAN NECESSITIES
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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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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    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • General Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Dermatology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
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JP2021569934A 2019-05-27 2020-05-25 新規の網膜色素変性処置 Active JP7652713B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025041378A JP2025085741A (ja) 2019-05-27 2025-03-14 新規の網膜色素変性処置

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
AU2019901804A AU2019901804A0 (en) 2019-05-27 Novel Retinitis Pigmentosa Treatment
AU2019901804 2019-05-27
AU2019903812A AU2019903812A0 (en) 2019-10-10 Novel Retinitis Pigmentosa Treatment
AU2019903812 2019-10-10
PCT/AU2020/050516 WO2020237294A1 (en) 2019-05-27 2020-05-25 Novel retinitis pigmentosa treatment

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JP2022534086A JP2022534086A (ja) 2022-07-27
JP2022534086A5 JP2022534086A5 (enExample) 2023-06-01
JP7652713B2 true JP7652713B2 (ja) 2025-03-27

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JP2021569934A Active JP7652713B2 (ja) 2019-05-27 2020-05-25 新規の網膜色素変性処置
JP2025041378A Pending JP2025085741A (ja) 2019-05-27 2025-03-14 新規の網膜色素変性処置

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US (2) US12559747B2 (enExample)
EP (1) EP3976789A4 (enExample)
JP (2) JP7652713B2 (enExample)
KR (1) KR20220012276A (enExample)
CN (1) CN113891940B (enExample)
AU (1) AU2020285470B2 (enExample)
BR (1) BR112021023936A2 (enExample)
CA (1) CA3141633A1 (enExample)
IL (1) IL288442A (enExample)
SG (1) SG11202112946VA (enExample)
WO (1) WO2020237294A1 (enExample)

Families Citing this family (2)

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Publication number Priority date Publication date Assignee Title
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
WO2025043278A1 (en) * 2023-08-25 2025-03-06 PYC Therapeutics Limited Gene-mediated conditions

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CHI, Xuan et al.，"Safety of antisense oligonucleotide and siRNA-based therapeutics"，Drug Discovery Today，2017年05月，Vol. 22, No. 5，p.823-833，DOI: 10.1016/j.drudis.2017.01.013
PITOUT, Ianthe，Modulation of modifiers of Pre-mRNA splicing: A therapeutic strategy for Amenable Inherited Diseases，BSc (Hons) Doctor of Philosophy (PhD) ，Murdoch University，2018年，21-26,154-161
VENTURINI, Giulia et al.，"CNOT3 Is a Modifier of PRPF31 Mutations in Retinitis Pigmentosa with Incomplete Penetrance"，PLoS Genetics，2012年11月08日，Vol. 8, No. 11，p.e1003040，DOI: 10.1371/journal.pgen.1003040

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US12559747B2 (en) 2026-02-24
AU2020285470A1 (en) 2021-12-16
CN113891940B (zh) 2024-12-20
SG11202112946VA (en) 2021-12-30
AU2020285470B2 (en) 2025-08-28
JP2025085741A (ja) 2025-06-05
JP2022534086A (ja) 2022-07-27
US20250066782A1 (en) 2025-02-27
WO2020237294A1 (en) 2020-12-03
CN113891940A (zh) 2022-01-04
CA3141633A1 (en) 2020-12-03
BR112021023936A2 (pt) 2022-01-25
US20220298506A1 (en) 2022-09-22
IL288442A (en) 2022-01-01
EP3976789A4 (en) 2023-08-09
EP3976789A1 (en) 2022-04-06
KR20220012276A (ko) 2022-02-03

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