CN115210376A - 诱导外显子51的跳读的反义核酸 - Google Patents

诱导外显子51的跳读的反义核酸 Download PDF

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Publication number
CN115210376A
CN115210376A CN202180017313.6A CN202180017313A CN115210376A CN 115210376 A CN115210376 A CN 115210376A CN 202180017313 A CN202180017313 A CN 202180017313A CN 115210376 A CN115210376 A CN 115210376A
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Prior art keywords
antisense oligomer
exon
nos
pharmaceutically acceptable
seq
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Pending
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CN202180017313.6A
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Chinese (zh)
Inventor
本多优
鞭马奏萌
福井崇弘
长谷川佐季
武田伸一
青木吉嗣
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Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
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Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
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Publication of CN115210376A publication Critical patent/CN115210376A/zh
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
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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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    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202180017313.6A 2020-02-28 2021-02-26 诱导外显子51的跳读的反义核酸 Pending CN115210376A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2020033483 2020-02-28
JP2020-033483 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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Publication Number Publication Date
CN115210376A true CN115210376A (zh) 2022-10-18

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CN202180017313.6A Pending CN115210376A (zh) 2020-02-28 2021-02-26 诱导外显子51的跳读的反义核酸

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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=)

Families Citing this family (3)

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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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US20100130591A1 (en) * 2008-10-24 2010-05-27 Peter Sazani Multiple exon skipping compositions for dmd
CN103154245A (zh) * 2010-09-01 2013-06-12 日本新药株式会社 反义核酸
US20150045413A1 (en) * 2012-01-27 2015-02-12 Prosensa Technologies B.V. RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Duchenne and Becker Muscular Dystrophy
CN104603271A (zh) * 2012-07-03 2015-05-06 普罗森萨科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
US20170067048A1 (en) * 2014-03-12 2017-03-09 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
CN106661577A (zh) * 2014-06-17 2017-05-10 日本新药株式会社 反义核酸
CN107881175A (zh) * 2011-12-28 2018-04-06 日本新药株式会社 反义核酸
CN108026531A (zh) * 2015-09-15 2018-05-11 日本新药株式会社 反义核酸
CN110290812A (zh) * 2016-12-19 2019-09-27 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript

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JP6519842B2 (ja) 2013-10-04 2019-05-29 国立大学法人神戸大学 福山型筋ジストロフィー治療用アンチセンス核酸
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Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100130591A1 (en) * 2008-10-24 2010-05-27 Peter Sazani Multiple exon skipping compositions for dmd
CN103154245A (zh) * 2010-09-01 2013-06-12 日本新药株式会社 反义核酸
CN107881175A (zh) * 2011-12-28 2018-04-06 日本新药株式会社 反义核酸
US20150045413A1 (en) * 2012-01-27 2015-02-12 Prosensa Technologies B.V. RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Duchenne and Becker Muscular Dystrophy
CN104603271A (zh) * 2012-07-03 2015-05-06 普罗森萨科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
US20170067048A1 (en) * 2014-03-12 2017-03-09 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
CN106661577A (zh) * 2014-06-17 2017-05-10 日本新药株式会社 反义核酸
CN108026531A (zh) * 2015-09-15 2018-05-11 日本新药株式会社 反义核酸
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
CN110290812A (zh) * 2016-12-19 2019-09-27 萨勒普塔医疗公司 用于肌肉萎缩症的外显子跳跃寡聚体缀合物

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EP4112083A1 (en) 2023-01-04
BR112022017066A2 (pt) 2022-11-16
KR20220145865A (ko) 2022-10-31
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
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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