CN116507727A - Rps25基因的表达和/或功能调节剂 - Google Patents

Rps25基因的表达和/或功能调节剂 Download PDF

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Publication number
CN116507727A
CN116507727A CN202180074713.0A CN202180074713A CN116507727A CN 116507727 A CN116507727 A CN 116507727A CN 202180074713 A CN202180074713 A CN 202180074713A CN 116507727 A CN116507727 A CN 116507727A
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CN
China
Prior art keywords
antisense oligonucleotide
stranded antisense
pharmaceutically acceptable
acceptable salt
base
Prior art date
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Pending
Application number
CN202180074713.0A
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English (en)
Chinese (zh)
Inventor
铃木利佳
浅野成宏
栗田光将
铃木高尾
山上正辉
阿贾亚·拉姆·什雷斯塔
川野边峻哲
梅本忠士
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Lucosana Biotechnology Co ltd
Sumitomo Pharma Co Ltd
Original Assignee
Lucosana Biotechnology Co ltd
Sumitomo Pharmaceuticals Co Ltd
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Application filed by Lucosana Biotechnology Co ltd, Sumitomo Pharmaceuticals Co Ltd filed Critical Lucosana Biotechnology Co ltd
Publication of CN116507727A publication Critical patent/CN116507727A/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
    • A61K31/70—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

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  • 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)
CN202180074713.0A 2020-11-06 2021-11-05 Rps25基因的表达和/或功能调节剂 Pending CN116507727A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2020185944 2020-11-06
JP2020-185944 2020-11-06
PCT/JP2021/040853 WO2022097727A1 (ja) 2020-11-06 2021-11-05 Ｒｐｓ２５遺伝子の発現及び／又は機能調節剤

Publications (1)

Publication Number Publication Date
CN116507727A true CN116507727A (zh) 2023-07-28

Family

ID=81458361

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180074713.0A Pending CN116507727A (zh) 2020-11-06 2021-11-05 Rps25基因的表达和/或功能调节剂

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

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
US20240018516A1 (en) 2024-01-18
TWI910261B (zh) 2026-01-01
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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