ZA202007564B - Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides - Google Patents

Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides

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Publication number
ZA202007564B
ZA202007564B ZA2020/07564A ZA202007564A ZA202007564B ZA 202007564 B ZA202007564 B ZA 202007564B ZA 2020/07564 A ZA2020/07564 A ZA 2020/07564A ZA 202007564 A ZA202007564 A ZA 202007564A ZA 202007564 B ZA202007564 B ZA 202007564B
Authority
ZA
South Africa
Prior art keywords
anomeric
alpha
bicyclic sugar
oligonucleotides conjugates
sugar nucleosides
Prior art date
Application number
ZA2020/07564A
Other languages
English (en)
Inventor
Wolfgang Renner
Original Assignee
Alpha Anomeric Sas
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 Alpha Anomeric Sas filed Critical Alpha Anomeric Sas
Publication of ZA202007564B publication Critical patent/ZA202007564B/en

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    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/542Carboxylic acids, e.g. a fatty acid or an amino acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
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    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/33Chemical structure of the base
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/337Chemical structure of the base in alpha-anomeric form
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
ZA2020/07564A 2018-05-11 2020-12-04 Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides ZA202007564B (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862670319P 2018-05-11 2018-05-11
PCT/EP2019/062064 WO2019215333A1 (en) 2018-05-11 2019-05-10 Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides

Publications (1)

Publication Number Publication Date
ZA202007564B true ZA202007564B (en) 2021-10-27

Family

ID=66530048

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2020/07564A ZA202007564B (en) 2018-05-11 2020-12-04 Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides

Country Status (11)

Country Link
US (2) US20230132377A9 (enExample)
EP (1) EP3790968A1 (enExample)
JP (1) JP7665509B2 (enExample)
KR (1) KR20210008369A (enExample)
CN (1) CN112424352A (enExample)
AU (1) AU2019266550A1 (enExample)
BR (1) BR112020022620A2 (enExample)
CA (1) CA3098266A1 (enExample)
SG (1) SG11202010841QA (enExample)
WO (1) WO2019215333A1 (enExample)
ZA (1) ZA202007564B (enExample)

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CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
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
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US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス,インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
CR20250257A (es) 2022-12-02 2025-08-28 Shanghai Argo Biopharmaceutical Co Ltd Análogos de ácidos nucleicos bicíclicos abásicos y compuestos oligoméricos preparados a partir de estos
WO2025217304A1 (en) * 2024-04-09 2025-10-16 Bio-Rad Laboratories, Inc. Compositions and methods for detection of contaminant enzymes

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Publication number Publication date
US20220017898A1 (en) 2022-01-20
JP7665509B2 (ja) 2025-04-21
WO2019215333A1 (en) 2019-11-14
AU2019266550A1 (en) 2020-11-26
BR112020022620A2 (pt) 2021-02-17
EP3790968A1 (en) 2021-03-17
US20250250565A1 (en) 2025-08-07
US20230132377A9 (en) 2023-04-27
JP2021522862A (ja) 2021-09-02
CN112424352A (zh) 2021-02-26
CA3098266A1 (en) 2019-11-14
SG11202010841QA (en) 2020-11-27
KR20210008369A (ko) 2021-01-21

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