TWI910261B - Ｒｐｓ２５基因之表現及／或功能調節劑 - Google Patents

Ｒｐｓ２５基因之表現及／或功能調節劑

Info

Publication number
TWI910261B
TWI910261B TW110141414A TW110141414A TWI910261B TW I910261 B TWI910261 B TW I910261B TW 110141414 A TW110141414 A TW 110141414A TW 110141414 A TW110141414 A TW 110141414A TW I910261 B TWI910261 B TW I910261B
Authority
TW
Taiwan
Prior art keywords
antisense oligonucleotide
stranded antisense
aforementioned
permissible salt
pharmaceutically permissible
Prior art date
Application number
TW110141414A
Other languages
English (en)
Chinese (zh)
Other versions
TW202233838A (zh
Inventor
鈴木利佳
浅野成宏
栗田光將
鈴木高尾
山上正輝
阿賈亞 拉姆 什雷斯塔
川野邊峻哲
梅本忠士
Original Assignee
日商住友製藥股份有限公司
日商璐庫薩那生物技術股份有限公司
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 日商住友製藥股份有限公司, 日商璐庫薩那生物技術股份有限公司 filed Critical 日商住友製藥股份有限公司
Publication of TW202233838A publication Critical patent/TW202233838A/zh
Application granted granted Critical
Publication of TWI910261B publication Critical patent/TWI910261B/zh

Links

Classifications

    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW110141414A 2020-11-06 2021-11-05 Ｒｐｓ２５基因之表現及／或功能調節劑 TWI910261B (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2020185944 2020-11-06
JP2020-185944 2020-11-06

Publications (2)

Publication Number Publication Date
TW202233838A TW202233838A (zh) 2022-09-01
TWI910261B true TWI910261B (zh) 2026-01-01

Family

ID=81458361

Family Applications (1)

Application Number Title Priority Date Filing Date
TW110141414A TWI910261B (zh) 2020-11-06 2021-11-05 Ｒｐｓ２５基因之表現及／或功能調節劑

Country Status (6)

Country Link
US (1) US20240018516A1 (https=)
EP (1) EP4252846A4 (https=)
JP (1) JPWO2022097727A1 (https=)
CN (1) CN116507727A (https=)
TW (1) TWI910261B (https=)
WO (1) WO2022097727A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4596694A1 (en) * 2022-09-29 2025-08-06 Institute Of Science Tokyo Nucleic acid molecule containing 5'-cyclopropylene modification
JPWO2024257847A1 (https=) 2023-06-16 2024-12-19
WO2025047953A1 (ja) * 2023-08-30 2025-03-06 株式会社Stratoimmune ＲａｓＧＲＰ４のアンチセンスオリゴヌクレオチド

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JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
US7235358B2 (en) * 2001-06-08 2007-06-26 Expression Diagnostics, Inc. Methods and compositions for diagnosing and monitoring transplant rejection
JP2012522013A (ja) * 2009-03-27 2012-09-20 ザ ユーエービー リサーチ ファウンデーション 調節ｉｒｅｓ媒介翻訳
CA2778171C (en) 2009-10-29 2017-07-25 Osaka University Bridged artificial nucleoside and nucleotide
US8377901B2 (en) * 2010-07-07 2013-02-19 The University Of Manitoba Target host factors for treating viral infection
WO2013163404A1 (en) * 2012-04-27 2013-10-31 The Uab Research Foundation TREATING VIRAL INFECTIONS HAVING VIRAL RNAs TRANSLATED BY A NON-IRES MEDIATED MECHANISM
KR101707437B1 (ko) 2012-09-21 2017-02-16 고꾸리쯔 다이가꾸 호우징 오사까 다이가꾸 구아니딘 가교를 갖는 인공 뉴클레오시드 및 올리고뉴클레오티드
CN106103463B (zh) 2014-02-18 2018-11-30 国立大学法人大阪大学 桥连型核苷和核苷酸
CN112501208A (zh) * 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
BR112020008033A2 (pt) 2017-10-23 2020-10-27 Prevail Therapeutics, Inc. terapias gênicas para doença neurodegenerativa
US11725208B2 (en) * 2017-12-14 2023-08-15 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
BR112020018902A2 (pt) * 2018-03-20 2021-01-26 Tokyo Institute Of Technology oligonucleotídeo antissenso com toxicidade reduzida
EP3919501A4 (en) 2019-02-01 2022-11-23 Osaka University 5'-MODIFIED NUCLEOSIDE AND NUCLEOTIDE THEREOF
WO2020203880A1 (ja) * 2019-03-29 2020-10-08 田辺三菱製薬株式会社 Ｄｕｘ４の発現を調節するための化合物、方法及び医薬組成物
EP4013870A1 (en) * 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof

Non-Patent Citations (1)

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Title
期刊 DeVos, Sarah L. et al. Antisense Oligonucleotides: Treating Neurodegeneration at the Level of RNA Antisense Oligonucleotides: Treating Neurodegeneration at the Level of RNA Volume 10, Issue 3 Neurotherapeutics 2013/05/18 486 – 497

Also Published As

Publication number Publication date
US20240018516A1 (en) 2024-01-18
CN116507727A (zh) 2023-07-28
EP4252846A4 (en) 2025-12-03
JPWO2022097727A1 (https=) 2022-05-12
WO2022097727A1 (ja) 2022-05-12
TW202233838A (zh) 2022-09-01
EP4252846A1 (en) 2023-10-04

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