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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/0048—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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    • C12N2310/00—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
    • C12N2310/314—Phosphoramidates
    • 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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    • C12N2310/3233—Morpholino-type ring
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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 Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
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
AU2019903812 2019-10-10
AU2019903812A AU2019903812A0 (en) 2019-10-10 Novel Retinitis Pigmentosa Treatment
PCT/AU2020/050516 WO2020237294A1 (en) 2019-05-27 2020-05-25 Novel retinitis pigmentosa treatment

Related Child Applications (1)

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JP2025041378A Division JP2025085741A (ja) 2019-05-27 2025-03-14 新規の網膜色素変性処置

Publications (3)

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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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12559747B2 (en) * 2019-05-27 2026-02-24 Vision Pharma Pty Ltd Retinitis pigmentosa treatment
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
WO2025043278A1 (en) * 2023-08-25 2025-03-06 PYC Therapeutics Limited Gene-mediated conditions
WO2026090034A1 (en) * 2024-10-21 2026-04-30 Vision Pharma Pty Ltd Agents for treatment of optic conditions

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US20020123476A1 (en) 1991-03-19 2002-09-05 Emanuele R. Martin Therapeutic delivery compositions and methods of use thereof
RU2034835C1 (ru) 1992-03-09 1995-05-10 Научно-исследовательский и проектный институт мономеров с опытным заводом СПОСОБ ПОЛУЧЕНИЯ β -КАРОТИНА
US6806084B1 (en) 1992-06-04 2004-10-19 The Regents Of The University Of California Methods for compositions for in vivo gene delivery
WO1997022371A1 (en) 1995-12-18 1997-06-26 Collagen Corporation Crosslinked polymer compositions and methods for their use
US6821724B1 (en) * 1998-09-17 2004-11-23 Affymetrix, Inc. Methods of genetic analysis using nucleic acid arrays
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
AU2011349464B2 (en) 2010-12-20 2016-07-07 The General Hospital Corporation Polycomb-associated non-coding RNAS
WO2013027104A1 (en) 2011-08-25 2013-02-28 Db Equipment As Accessory bag having reinforced sidewalls and variable length
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
US12559747B2 (en) * 2019-05-27 2026-02-24 Vision Pharma Pty Ltd Retinitis pigmentosa treatment
KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
JP2023529026A (ja) * 2020-06-01 2023-07-06 デイナ ファーバー キャンサー インスティチュート，インコーポレイテッド Ｍｈｃ－ｉ発現を調節するための方法及びその免疫療法の使用
CN113584038B (zh) * 2021-09-09 2023-11-14 深圳雅济科技有限公司 一种治疗视网膜疾病的反义寡核苷酸组合及其应用

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
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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Publication number Publication date
US20250066782A1 (en) 2025-02-27
CN113891940A (zh) 2022-01-04
EP3976789A1 (en) 2022-04-06
KR20220012276A (ko) 2022-02-03
WO2020237294A1 (en) 2020-12-03
CN113891940B (zh) 2024-12-20
JP2022534086A (ja) 2022-07-27
US12649922B2 (en) 2026-06-09
JP2025085741A (ja) 2025-06-05
IL288442A (en) 2022-01-01
US20220298506A1 (en) 2022-09-22
AU2020285470A1 (en) 2021-12-16
BR112021023936A2 (pt) 2022-01-25
US12559747B2 (en) 2026-02-24
AU2020285470B2 (en) 2025-08-28
EP3976789A4 (en) 2023-08-09
CA3141633A1 (en) 2020-12-03
SG11202112946VA (en) 2021-12-30

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