JPWO2024071099A1 - - Google Patents

Info

Publication number
JPWO2024071099A1
JPWO2024071099A1 JP2024549412A JP2024549412A JPWO2024071099A1 JP WO2024071099 A1 JPWO2024071099 A1 JP WO2024071099A1 JP 2024549412 A JP2024549412 A JP 2024549412A JP 2024549412 A JP2024549412 A JP 2024549412A JP WO2024071099 A1 JPWO2024071099 A1 JP WO2024071099A1
Authority
JP
Japan
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2024549412A
Other languages
Japanese (ja)
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 filed Critical
Publication of JPWO2024071099A1 publication Critical patent/JPWO2024071099A1/ja
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024549412A 2022-09-29 2023-09-26 Pending JPWO2024071099A1 (https=)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2022155777 2022-09-29
PCT/JP2023/034892 WO2024071099A1 (ja) 2022-09-29 2023-09-26 5'-シクロプロピレン修飾を含む核酸分子

Publications (1)

Publication Number Publication Date
JPWO2024071099A1 true JPWO2024071099A1 (https=) 2024-04-04

Family

ID=90478000

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024549412A Pending JPWO2024071099A1 (https=) 2022-09-29 2023-09-26

Country Status (3)

Country Link
EP (1) EP4596694A1 (https=)
JP (1) JPWO2024071099A1 (https=)
WO (1) WO2024071099A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024257848A1 (ja) * 2023-06-16 2024-12-19 ルクサナバイオテク株式会社 遅発性中枢毒性が低減されているアンチセンスオリゴヌクレオチドの設計方法、及びその製造方法

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5955599A (en) 1995-06-01 1999-09-21 Hybridon, Inc. Process for making oligonucleotides containing o- and s- methylphosphotriester internucleoside linkages
JP2009536222A (ja) 2006-05-05 2009-10-08 アイシス ファーマシューティカルズ, インコーポレーテッド Pcsk9の発現を調節するための化合物および方法
CA2666191C (en) 2006-10-09 2017-07-11 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9
ES2526295T5 (es) 2006-10-18 2021-05-04 Ionis Pharmaceuticals Inc Compuestos antisentido
JP6112569B2 (ja) 2011-12-16 2017-04-12 国立大学法人 東京医科歯科大学 キメラ2重鎖核酸
EP3004347B1 (en) 2013-05-30 2018-09-26 National University Corporation Tokyo Medical and Dental University Double-stranded agents for delivering therapeutic oligonucleotides
WO2015168172A1 (en) 2014-04-28 2015-11-05 Isis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
JP6436477B2 (ja) * 2014-05-13 2018-12-12 国立大学法人大阪大学 癌治療用医薬組成物
WO2016081600A1 (en) 2014-11-18 2016-05-26 Zata Pharmaceuticals, Inc. Phosphoramidite synthones for the synthesis of self-neutralizing oligonucleotide compounds
US11260134B2 (en) 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
JP7394392B2 (ja) 2018-03-14 2023-12-08 国立大学法人 東京医科歯科大学 核酸複合体
JP7262816B2 (ja) 2018-03-22 2023-04-24 国立大学法人 東京医科歯科大学 ヘテロ核酸のbbb通過脂質リガンド
EP3919501A4 (en) 2019-02-01 2022-11-23 Osaka University 5'-MODIFIED NUCLEOSIDE AND NUCLEOTIDE THEREOF
WO2021054370A1 (ja) * 2019-09-18 2021-03-25 国立大学法人東京医科歯科大学 核酸複合体
CN116507727A (zh) * 2020-11-06 2023-07-28 住友制药株式会社 Rps25基因的表达和/或功能调节剂
JP7636937B2 (ja) 2021-03-31 2025-02-27 アイホン株式会社 インターホン装置
WO2022211095A1 (ja) * 2021-03-31 2022-10-06 ルクサナバイオテク株式会社 Fgfr3のアンチセンスオリゴヌクレオチド
US20240247263A1 (en) * 2021-05-06 2024-07-25 Luxna Biotech Co., Ltd. Method for designing oligonucleotide having reduced central toxicity

Also Published As

Publication number Publication date
WO2024071099A1 (ja) 2024-04-04
EP4596694A1 (en) 2025-08-06

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