CN114901823A - 诱导外显子50的跳读的反义核酸 - Google Patents

诱导外显子50的跳读的反义核酸 Download PDF

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Publication number
CN114901823A
CN114901823A CN202080090310.0A CN202080090310A CN114901823A CN 114901823 A CN114901823 A CN 114901823A CN 202080090310 A CN202080090310 A CN 202080090310A CN 114901823 A CN114901823 A CN 114901823A
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China
Prior art keywords
antisense oligomer
exon
pharmaceutically acceptable
hydrate
acceptable salt
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Pending
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CN202080090310.0A
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English (en)
Chinese (zh)
Inventor
盐谷由辉子
砂土居裕太
胁玲子
鞭马奏萌
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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Application filed by Nippon Shinyaku Co Ltd, National Center of Neurology and Psychiatry filed Critical Nippon Shinyaku Co Ltd
Priority to CN202410219725.3A priority Critical patent/CN118109468A/zh
Priority to CN202410219814.8A priority patent/CN118109469A/zh
Publication of CN114901823A publication Critical patent/CN114901823A/zh
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
    • 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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • 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
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • 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)
CN202080090310.0A 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸 Pending CN114901823A (zh)

Priority Applications (2)

Application Number Priority Date Filing Date Title
CN202410219725.3A CN118109468A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸
CN202410219814.8A CN118109469A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019-236704 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

Related Child Applications (2)

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CN202410219814.8A Division CN118109469A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸
CN202410219725.3A Division CN118109468A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸

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CN114901823A true CN114901823A (zh) 2022-08-12

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CN202080090310.0A Pending CN114901823A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸
CN202410219725.3A Pending CN118109468A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸
CN202410219814.8A Pending CN118109469A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸

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CN202410219725.3A Pending CN118109468A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸
CN202410219814.8A Pending CN118109469A (zh) 2019-12-26 2020-12-25 诱导外显子50的跳读的反义核酸

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=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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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CN105779453A (zh) * 2008-10-24 2016-07-20 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
US20190177723A1 (en) * 2015-10-09 2019-06-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders

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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
KR20220122673A (ko) 2022-09-02
JPWO2021132591A1 (https=) 2021-07-01
TW202523843A (zh) 2025-06-16
US20220333112A1 (en) 2022-10-20
CN118109468A (zh) 2024-05-31
IL294271A (en) 2022-08-01
CN118109469A (zh) 2024-05-31
EP4083208A4 (en) 2024-01-03
AU2020411964A1 (en) 2022-06-16

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