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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Application filed by 니뽄 신야쿠 가부시키가이샤, 고쿠리쯔겡뀨가이하쯔호징 고꾸리쯔 세이신ㆍ신께이 이료겡뀨센따 filed Critical 니뽄 신야쿠 가부시키가이샤
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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • 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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    • C07K14/00—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
    • 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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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Physical Education & Sports Medicine (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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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
JP2020033483 2020-02-28
JPJP-P-2020-033483 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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KR1020227032708A Pending KR20220145865A (ko) 2020-02-28 2021-02-26 엑손 51의 스키핑을 유도하는 안티센스 핵산

Country Status (17)

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

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JP7842395B2 (ja) * 2019-12-19 2026-04-08 日本新薬株式会社 エクソンスキッピングを可能にするアンチセンス核酸
CA3244382A1 (en) 2021-12-27 2025-02-26 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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WO2002024906A1 (en) 2000-09-21 2002-03-28 Academisch Ziekenhuis Leiden Induction of exon skipping in eukaryotic cells
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
WO2010050802A2 (en) 2008-10-27 2010-05-06 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of at least one of the following exons of the human duchenne muscular dystrophy gene: 43, 46, 50- 53.
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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EP4112083A1 (en) 2023-01-04
BR112022017066A2 (pt) 2022-11-16
ECSP22074446A (es) 2022-10-31
IL295967A (en) 2022-10-01
JP2022180420A (ja) 2022-12-06
US20230097387A1 (en) 2023-03-30
CL2022002279A1 (es) 2023-02-10
TW202515585A (zh) 2025-04-16
CA3173049A1 (en) 2021-09-02
JP7292636B2 (ja) 2023-06-19
PE20230237A1 (es) 2023-02-07
CL2024001472A1 (es) 2024-09-27
US11781140B2 (en) 2023-10-10
CN115210376A (zh) 2022-10-18
AU2021226089A1 (en) 2022-09-15
WO2021172498A1 (ja) 2021-09-02
US20230140736A1 (en) 2023-05-04
ZA202209294B (en) 2024-04-24
MX2022010545A (es) 2022-09-21
EP4112083A4 (en) 2024-06-26
TWI901457B (zh) 2025-10-11
CO2022013685A2 (es) 2022-10-11
CL2024001471A1 (es) 2024-09-27
TW202200162A (zh) 2022-01-01
JP2026001070A (ja) 2026-01-06
TWI868323B (zh) 2025-01-01
JPWO2021172498A1 (https=) 2021-09-02

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