KR20220122673A - 엑손 50의 스키핑을 유도하는 안티센스 핵산 - Google Patents

엑손 50의 스키핑을 유도하는 안티센스 핵산 Download PDF

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KR20220122673A
KR20220122673A KR1020227024957A KR20227024957A KR20220122673A KR 20220122673 A KR20220122673 A KR 20220122673A KR 1020227024957 A KR1020227024957 A KR 1020227024957A KR 20227024957 A KR20227024957 A KR 20227024957A KR 20220122673 A KR20220122673 A KR 20220122673A
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Prior art keywords
antisense oligomer
exon
pharmaceutically acceptable
hydrate
acceptable salt
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Korean (ko)
Inventor
유키코 엔야
유타 스나도이
레이코 와키
가나메 무치마
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 KR20220122673A publication Critical patent/KR20220122673A/ko
Pending legal-status Critical Current

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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
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/4965—Non-condensed pyrazines
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Engineering & Computer Science (AREA)
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  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020227024957A 2019-12-26 2020-12-25 엑손 50의 스키핑을 유도하는 안티센스 핵산 Pending KR20220122673A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JPJP-P-2019-236704 2019-12-26
JP2019236704 2019-12-26
PCT/JP2020/048803 WO2021132591A1 (ja) 2019-12-26 2020-12-25 エクソン５０のスキッピングを誘導するアンチセンス核酸

Publications (1)

Publication Number Publication Date
KR20220122673A true KR20220122673A (ko) 2022-09-02

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KR1020227024957A Pending KR20220122673A (ko) 2019-12-26 2020-12-25 엑손 50의 스키핑을 유도하는 안티센스 핵산

Country Status (16)

Country Link
US (2) US20230073008A1 (https=)
EP (1) EP4083208A4 (https=)
JP (2) JPWO2021132591A1 (https=)
KR (1) KR20220122673A (https=)
CN (3) CN114901823A (https=)
AU (1) AU2020411964A1 (https=)
BR (1) BR112022012622A2 (https=)
CA (1) CA3165961A1 (https=)
CO (1) CO2022008664A2 (https=)
IL (1) IL294271A (https=)
MX (1) MX2022007937A (https=)
MY (1) MY209476A (https=)
PH (1) PH12022551431A1 (https=)
TW (2) TWI874546B (https=)
WO (1) WO2021132591A1 (https=)
ZA (1) ZA202206667B (https=)

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IL293997A (en) * 2019-12-19 2022-08-01 Nippon Shinyaku Co Ltd Antistrand nucleic acids that allow exon skipping
CN114901823A (zh) * 2019-12-26 2022-08-12 日本新药株式会社 诱导外显子50的跳读的反义核酸
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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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
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.
WO2011057350A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
WO2013100190A1 (ja) 2011-12-28 2013-07-04 日本新薬株式会社 アンチセンス核酸

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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
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
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.
WO2011057350A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
WO2013100190A1 (ja) 2011-12-28 2013-07-04 日本新薬株式会社 アンチセンス核酸

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PH12022551431A1 (en) 2023-11-20
JP2022133312A (ja) 2022-09-13
EP4083208A1 (en) 2022-11-02
US11655472B2 (en) 2023-05-23
ZA202206667B (en) 2023-12-20
MX2022007937A (es) 2022-07-27
US20230073008A1 (en) 2023-03-09
JP7201192B2 (ja) 2023-01-10
CA3165961A1 (en) 2021-07-01
TWI874546B (zh) 2025-03-01
CO2022008664A2 (es) 2022-07-19
WO2021132591A1 (ja) 2021-07-01
BR112022012622A2 (pt) 2022-09-06
MY209476A (en) 2025-07-11
TW202138560A (zh) 2021-10-16
JPWO2021132591A1 (https=) 2021-07-01
TW202523843A (zh) 2025-06-16
US20220333112A1 (en) 2022-10-20
CN118109468A (zh) 2024-05-31
CN114901823A (zh) 2022-08-12
IL294271A (en) 2022-08-01
CN118109469A (zh) 2024-05-31
EP4083208A4 (en) 2024-01-03
AU2020411964A1 (en) 2022-06-16

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