MX2020009765A - Oligonucleotido antisentido reducido en toxicidad. - Google Patents

Oligonucleotido antisentido reducido en toxicidad.

Info

Publication number
MX2020009765A
MX2020009765A MX2020009765A MX2020009765A MX2020009765A MX 2020009765 A MX2020009765 A MX 2020009765A MX 2020009765 A MX2020009765 A MX 2020009765A MX 2020009765 A MX2020009765 A MX 2020009765A MX 2020009765 A MX2020009765 A MX 2020009765A
Authority
MX
Mexico
Prior art keywords
antisense oligonucleotide
reduced toxicity
cross
nucleotide
linked
Prior art date
Application number
MX2020009765A
Other languages
English (en)
Inventor
Hiroyuki Nakajima
Atsushi Inoue
Yusuke Iriyama
Tatsuro Kanaki
Yoshiaki Masaki
Kohji Seio
Original Assignee
Tokyo Inst Tech
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 Tokyo Inst Tech filed Critical Tokyo Inst Tech
Publication of MX2020009765A publication Critical patent/MX2020009765A/es

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Classifications

    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/319—Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-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
    • C12N2310/323—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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente proporciona un oligonucleótido antisentido con toxicidad reducida; un oligonucleótido antisentido que tiene una región central, una región del lado 5´ y una región del lado 3´, y el oligonucleótido antisentido comprende la región central que tiene un nucleótido (nucleótido con puente 2´-3´) en el cual la posición 2´ y la posición 3´ de una porción de azúcar están puenteadas y/o un nucleótido sin puente (nucleótido sin puente modificado en la posición 3´) que tiene un sustituyente en la posición 3´.
MX2020009765A 2018-03-20 2019-03-20 Oligonucleotido antisentido reducido en toxicidad. MX2020009765A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2018052578 2018-03-20
JP2018129296 2018-07-06
PCT/JP2019/011801 WO2019182037A1 (ja) 2018-03-20 2019-03-20 毒性が低減されたアンチセンスオリゴヌクレオチド

Publications (1)

Publication Number Publication Date
MX2020009765A true MX2020009765A (es) 2021-01-08

Family

ID=67987207

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020009765A MX2020009765A (es) 2018-03-20 2019-03-20 Oligonucleotido antisentido reducido en toxicidad.

Country Status (9)

Country Link
US (1) US20210054377A1 (es)
EP (1) EP3770256A4 (es)
JP (1) JPWO2019182037A1 (es)
CN (1) CN111868244A (es)
AU (1) AU2019237599A1 (es)
BR (1) BR112020018902A2 (es)
CA (1) CA3094303A1 (es)
MX (1) MX2020009765A (es)
WO (1) WO2019182037A1 (es)

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WO2017131124A1 (ja) * 2016-01-26 2017-08-03 日産化学工業株式会社 一本鎖オリゴヌクレオチド
JP7438135B2 (ja) 2018-05-09 2024-02-26 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｆｘｉの発現を低下させるための化合物及び方法
JP7579817B2 (ja) 2019-07-03 2024-11-08 インテグレーティッド ディーエヌエイ テクノロジーズ インコーポレーティッド Ｃｒｉｓｐｒで導入された二本鎖ｄｎａ切断修復の同定、特徴付けおよび定量化
JP7762651B2 (ja) * 2019-10-24 2025-10-30 インテグレーティッド ディーエヌエイ テクノロジーズ インコーポレーティッド 修飾二重鎖ドナー鋳型
US20220251557A1 (en) * 2019-11-27 2022-08-11 Tokyo Institute Of Technology Method for reducing toxicity of antisense nucleic acids
EP4098747A4 (en) * 2020-01-31 2025-03-26 Sanwa Kagaku Kenkyusho Co., Ltd. ATN1 ANTISENSE OLIGONUCLEOTIDE
CN116234908A (zh) * 2020-09-25 2023-06-06 印地安纳大学理事会 促进foxp3s的吗啉代物
EP4252846A4 (en) * 2020-11-06 2025-12-03 Sumitomo Pharma Co Ltd REGULATOR FOR THE EXPRESSION AND/OR FUNCTION OF THE RPS25 GENE
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
JP7607884B2 (ja) * 2021-12-13 2025-01-06 日本新薬株式会社 ＡＴＮ１ ｍＲＮＡ又はｐｒｅ－ｍＲＮＡを標的とするアンチセンスオリゴヌクレオチド
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Also Published As

Publication number Publication date
EP3770256A4 (en) 2021-12-22
US20210054377A1 (en) 2021-02-25
CA3094303A1 (en) 2019-09-26
CN111868244A (zh) 2020-10-30
EP3770256A1 (en) 2021-01-27
AU2019237599A1 (en) 2020-11-12
BR112020018902A2 (pt) 2021-01-26
WO2019182037A1 (ja) 2019-09-26
JPWO2019182037A1 (ja) 2021-03-11

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