CA3223577A1 - Oligonucleotides for ifn-.gamma. signaling pathway modulation - Google Patents

Oligonucleotides for ifn-.gamma. signaling pathway modulation Download PDF

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Publication number
CA3223577A1
CA3223577A1 CA3223577A CA3223577A CA3223577A1 CA 3223577 A1 CA3223577 A1 CA 3223577A1 CA 3223577 A CA3223577 A CA 3223577A CA 3223577 A CA3223577 A CA 3223577A CA 3223577 A1 CA3223577 A1 CA 3223577A1
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Canada
Prior art keywords
nucleotides
antisense strand
dsrna
strand
sense strand
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Pending
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CA3223577A
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English (en)
French (fr)
Inventor
Anastasia Khvorova
John E. Harris
Qi Tang
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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Publication of CA3223577A1 publication Critical patent/CA3223577A1/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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • C12N15/1137Non-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 against enzymes
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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
    • C12N15/1138Non-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 against receptors or cell surface proteins
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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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  • 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)
  • Molecular Biology (AREA)
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  • Wood Science & Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3223577A 2021-06-22 2022-06-21 Oligonucleotides for ifn-.gamma. signaling pathway modulation Pending CA3223577A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163213506P 2021-06-22 2021-06-22
US63/213,506 2021-06-22
US202263331563P 2022-04-15 2022-04-15
US63/331,563 2022-04-15
PCT/US2022/034297 WO2022271666A1 (en) 2021-06-22 2022-06-21 OLIGONUCLEOTIDES FOR IFN-γ SIGNALING PATHWAY MODULATION

Publications (1)

Publication Number Publication Date
CA3223577A1 true CA3223577A1 (en) 2022-12-29

Family

ID=84544753

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3223577A Pending CA3223577A1 (en) 2021-06-22 2022-06-21 Oligonucleotides for ifn-.gamma. signaling pathway modulation

Country Status (7)

Country Link
US (1) US20240287514A1 (https=)
EP (1) EP4359537A4 (https=)
JP (1) JP2024523466A (https=)
KR (1) KR20240040724A (https=)
AU (1) AU2022298641A1 (https=)
CA (1) CA3223577A1 (https=)
WO (1) WO2022271666A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
EP4676443A1 (en) * 2023-03-03 2026-01-14 University of Massachusetts Dendritic conjugates for the skin delivery of therapeutic oligonucleotides
TW202516004A (zh) * 2023-06-16 2025-04-16 瑞士商赫孚孟拉羅股份公司 調節jak1表現之雙股寡核苷酸

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006094293A2 (en) * 2005-03-03 2006-09-08 President And Fellows Of Harvard College Slim compositions and methods of use thereof
EP2227558A1 (en) * 2007-11-30 2010-09-15 GlaxoSmithKline Biologicals SA Method for classifying cancer patients as responder or non-responder to immunotherapy
JP5813778B2 (ja) * 2010-11-19 2015-11-17 イデラ ファーマシューティカルズ インコーポレイテッドIdera Pharmaceuticals, Inc. トール様受容体に基づく免疫反応を調節するための免疫調節オリゴヌクレオチド(iro)化合物
CN113811311A (zh) * 2019-03-15 2021-12-17 马萨诸塞大学 用于组织特异性apoe调节的寡核苷酸

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Publication number Publication date
KR20240040724A (ko) 2024-03-28
AU2022298641A1 (en) 2024-02-01
EP4359537A1 (en) 2024-05-01
EP4359537A4 (en) 2026-05-06
JP2024523466A (ja) 2024-06-28
US20240287514A1 (en) 2024-08-29
WO2022271666A1 (en) 2022-12-29

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