KR20220145865A - 엑손 51의 스키핑을 유도하는 안티센스 핵산 - Google Patents

엑손 51의 스키핑을 유도하는 안티센스 핵산 Download PDF

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KR20220145865A
KR20220145865A KR1020227032708A KR20227032708A KR20220145865A KR 20220145865 A KR20220145865 A KR 20220145865A KR 1020227032708 A KR1020227032708 A KR 1020227032708A KR 20227032708 A KR20227032708 A KR 20227032708A KR 20220145865 A KR20220145865 A KR 20220145865A
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antisense oligomer
exon
antisense
skipping
nucleic acid
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Korean (ko)
Inventor
유 혼다
가나메 무치마
다카히로 후쿠이
사키 하세가와
신이치 다케다
요시츠구 아오키
Original Assignee
니뽄 신야쿠 가부시키가이샤
고쿠리쯔겡뀨가이하쯔호징 고꾸리쯔 세이신ㆍ신께이 이료겡뀨센따
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Publication of KR20220145865A publication Critical patent/KR20220145865A/ko
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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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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • 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
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    • 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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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020227032708A 2020-02-28 2021-02-26 엑손 51의 스키핑을 유도하는 안티센스 핵산 Pending KR20220145865A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JPJP-P-2020-033483 2020-02-28
JP2020033483 2020-02-28
PCT/JP2021/007286 WO2021172498A1 (ja) 2020-02-28 2021-02-26 エクソン５１のスキッピングを誘導するアンチセンス核酸

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KR20220145865A true KR20220145865A (ko) 2022-10-31

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US (2) US20230140736A1 (enExample)
EP (1) EP4112083A4 (enExample)
JP (3) JPWO2021172498A1 (enExample)
KR (1) KR20220145865A (enExample)
CN (1) CN115210376A (enExample)
AU (1) AU2021226089A1 (enExample)
BR (1) BR112022017066A2 (enExample)
CA (1) CA3173049A1 (enExample)
CL (3) CL2022002279A1 (enExample)
CO (1) CO2022013685A2 (enExample)
EC (1) ECSP22074446A (enExample)
IL (1) IL295967A (enExample)
MX (1) MX2022010545A (enExample)
PE (1) PE20230237A1 (enExample)
TW (2) TWI901457B (enExample)
WO (1) WO2021172498A1 (enExample)
ZA (1) ZA202209294B (enExample)

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IL293997A (en) * 2019-12-19 2022-08-01 Nippon Shinyaku Co Ltd Antistrand nucleic acids that allow exon skipping
EP4458833A4 (en) 2021-12-27 2025-12-31 Nippon Shinyaku Co Ltd PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping

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WO2004048570A1 (ja) 2002-11-25 2004-06-10 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬
WO2004083432A1 (en) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
WO2006000057A1 (en) 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2009054725A2 (en) 2007-10-26 2009-04-30 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2010048586A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
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WO2015137409A1 (ja) 2014-03-12 2015-09-17 日本新薬株式会社 アンチセンス核酸
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ZA202209294B (en) 2024-04-24
JP2026001070A (ja) 2026-01-06
PE20230237A1 (es) 2023-02-07
TWI868323B (zh) 2025-01-01
CA3173049A1 (en) 2021-09-02
US20230140736A1 (en) 2023-05-04
CO2022013685A2 (es) 2022-10-11
WO2021172498A1 (ja) 2021-09-02
BR112022017066A2 (pt) 2022-11-16
AU2021226089A1 (en) 2022-09-15
JP2022180420A (ja) 2022-12-06
EP4112083A4 (en) 2024-06-26
MX2022010545A (es) 2022-09-21
US20230097387A1 (en) 2023-03-30
CN115210376A (zh) 2022-10-18
JPWO2021172498A1 (enExample) 2021-09-02
TW202200162A (zh) 2022-01-01
CL2024001471A1 (es) 2024-09-27
JP7292636B2 (ja) 2023-06-19
ECSP22074446A (es) 2022-10-31
IL295967A (en) 2022-10-01
CL2022002279A1 (es) 2023-02-10
TWI901457B (zh) 2025-10-11
EP4112083A1 (en) 2023-01-04
TW202515585A (zh) 2025-04-16
CL2024001472A1 (es) 2024-09-27
US11781140B2 (en) 2023-10-10

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