MX2020004898A - Ácidos nucleicos para inhibir la expresión de un gen diana que comprende enlaces fosforoditioato. - Google Patents

Ácidos nucleicos para inhibir la expresión de un gen diana que comprende enlaces fosforoditioato.

Info

Publication number
MX2020004898A
MX2020004898A MX2020004898A MX2020004898A MX2020004898A MX 2020004898 A MX2020004898 A MX 2020004898A MX 2020004898 A MX2020004898 A MX 2020004898A MX 2020004898 A MX2020004898 A MX 2020004898A MX 2020004898 A MX2020004898 A MX 2020004898A
Authority
MX
Mexico
Prior art keywords
target gene
nucleic acids
inhibiting expression
phosphorodithioate linkages
products
Prior art date
Application number
MX2020004898A
Other languages
English (en)
Inventor
Lucas Bethge
Judith Hauptmann
Christian Frauendorf
Adrien Weingärtner
Original Assignee
Silence Therapeutics Gmbh
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
Priority claimed from GBGB1800679.1A external-priority patent/GB201800679D0/en
Application filed by Silence Therapeutics Gmbh filed Critical Silence Therapeutics Gmbh
Publication of MX2020004898A publication Critical patent/MX2020004898A/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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

Abstract

La presente invención se refiere a productos y composiciones y sus usos. En particular, la invención se refiere a productos de ácido nucleico que interfieren con la expresión del gen diana o inhiben la expresión del gen diana y los usos terapéuticos de tales productos.
MX2020004898A 2017-11-13 2018-11-13 Ácidos nucleicos para inhibir la expresión de un gen diana que comprende enlaces fosforoditioato. MX2020004898A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP17201408 2017-11-13
GBGB1800679.1A GB201800679D0 (en) 2018-01-16 2018-01-16 Phosphorodithioate linkages
EP18165913 2018-04-05
PCT/EP2018/081101 WO2019092280A1 (en) 2017-11-13 2018-11-13 Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages

Publications (1)

Publication Number Publication Date
MX2020004898A true MX2020004898A (es) 2020-10-05

Family

ID=64316546

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020004898A MX2020004898A (es) 2017-11-13 2018-11-13 Ácidos nucleicos para inhibir la expresión de un gen diana que comprende enlaces fosforoditioato.

Country Status (14)

Country Link
US (2) US11873489B2 (es)
EP (1) EP3710585A1 (es)
JP (1) JP7461886B2 (es)
KR (1) KR102822091B1 (es)
CN (1) CN111527206A (es)
AU (1) AU2018363837B2 (es)
BR (1) BR112020009092A2 (es)
CA (1) CA3081910A1 (es)
IL (1) IL274611B2 (es)
MA (1) MA51707A (es)
MX (1) MX2020004898A (es)
PH (1) PH12020550244A1 (es)
SG (1) SG11202003082UA (es)
WO (1) WO2019092280A1 (es)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
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WO2021060440A1 (ja) * 2019-09-26 2021-04-01 日油株式会社 脂質ナノ粒子の凍結乾燥組成物
CA3197198A1 (en) 2020-11-04 2022-05-12 Ute SCHAEPER Nucleic acids for inhibiting expression of pros1 in a cell
TW202237840A (zh) * 2020-11-04 2022-10-01 瑞士伯恩大學 用於在細胞中抑制pros1之表現的核酸
WO2022184852A1 (en) * 2021-03-03 2022-09-09 Silence Therapeutics Gmbh Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell
AU2022242899A1 (en) * 2021-03-24 2023-11-09 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
EP4367125A1 (en) * 2021-07-09 2024-05-15 Alnylam Pharmaceuticals, Inc. Aminooxy click chemistry (aocc): a versatile bioconjugation approach
TW202400787A (zh) 2022-03-16 2024-01-01 美商安彼瑞可股份有限公司 改良siRNA生物可利用性之GalNAc組合物
KR20240163743A (ko) 2022-03-28 2024-11-19 엠피리코 인크. 변형된 올리고뉴클레오티드
GB202311334D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025021831A1 (en) 2023-07-24 2025-01-30 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
GB202311324D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof

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US7452987B2 (en) * 2002-08-05 2008-11-18 Silence Therapeutics Aktiengesellschaft (Ag) Interfering RNA molecules
JP4605799B2 (ja) * 2003-04-02 2011-01-05 ダーマコン， インコーポレイテッド Ｒｎａ干渉において使用するための修飾ポリヌクレオチド
WO2008011431A2 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)
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WO2008022309A2 (en) 2006-08-18 2008-02-21 F. Hoffmann-La Roche Ag Polyconjugates for in vivo delivery of polynucleotides
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EP3228326A1 (en) 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate

Also Published As

Publication number Publication date
PH12020550244A1 (en) 2021-02-15
CN111527206A (zh) 2020-08-11
JP7461886B2 (ja) 2024-04-04
US20240200071A1 (en) 2024-06-20
US11873489B2 (en) 2024-01-16
BR112020009092A2 (pt) 2020-10-20
KR20200098521A (ko) 2020-08-20
AU2018363837A1 (en) 2020-05-28
KR102822091B1 (ko) 2025-06-17
IL274611B1 (en) 2024-06-01
CA3081910A1 (en) 2019-05-16
RU2020112641A3 (es) 2022-04-08
RU2020112641A (ru) 2021-12-15
AU2018363837B2 (en) 2024-11-14
IL274611A (en) 2020-06-30
JP2021502411A (ja) 2021-01-28
IL274611B2 (en) 2024-10-01
MA51707A (fr) 2020-09-23
SG11202003082UA (en) 2020-05-28
EP3710585A1 (en) 2020-09-23
US20200392495A1 (en) 2020-12-17
WO2019092280A1 (en) 2019-05-16

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