CA2991052A1 - Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof - Google Patents

Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof Download PDF

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Publication number
CA2991052A1
CA2991052A1 CA2991052A CA2991052A CA2991052A1 CA 2991052 A1 CA2991052 A1 CA 2991052A1 CA 2991052 A CA2991052 A CA 2991052A CA 2991052 A CA2991052 A CA 2991052A CA 2991052 A1 CA2991052 A1 CA 2991052A1
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CA
Canada
Prior art keywords
substituted
unsubstituted
compound
odn
seq
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Pending
Application number
CA2991052A
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English (en)
French (fr)
Inventor
Marcin Tomasz KORTYLEWSKI
Piotr Marek Swiderski
Dayson Friaca Moreira
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
City of Hope
Original Assignee
City of Hope
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Filing date
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Application filed by City of Hope filed Critical City of Hope
Publication of CA2991052A1 publication Critical patent/CA2991052A1/en
Pending legal-status Critical Current

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    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
    • 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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
    • 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
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CA2991052A 2015-07-02 2016-06-30 Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof Pending CA2991052A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562187878P 2015-07-02 2015-07-02
US62/187,878 2015-07-02
US201562264026P 2015-12-07 2015-12-07
US62/264,026 2015-12-07
PCT/US2016/040361 WO2017004357A1 (en) 2015-07-02 2016-06-30 Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof

Publications (1)

Publication Number Publication Date
CA2991052A1 true CA2991052A1 (en) 2017-01-05

Family

ID=57609287

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2991052A Pending CA2991052A1 (en) 2015-07-02 2016-06-30 Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof

Country Status (6)

Country Link
US (3) US10758624B2 (enExample)
EP (1) EP3316894A4 (enExample)
JP (2) JP7073109B2 (enExample)
CN (2) CN114224907A (enExample)
CA (1) CA2991052A1 (enExample)
WO (1) WO2017004357A1 (enExample)

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WO2017004357A1 (en) * 2015-07-02 2017-01-05 City Of Hope Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
CN110248665A (zh) 2017-02-06 2019-09-17 Mpeg La有限责任公司 具有降低的肾清除的多聚体寡核苷酸
WO2018209270A1 (en) 2017-05-11 2018-11-15 Northwestern University Adoptive cell therapy using spherical nucleic acids (snas)
US11554178B2 (en) 2017-06-30 2023-01-17 City Of Hope Compositions and methods of modulating macrophage activity
WO2019048632A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE
EP3778893A4 (en) * 2018-04-10 2022-04-20 Ractigen Therapeutics P21 GENE EXPRESSION ACTIVATION METHOD
WO2019241766A1 (en) * 2018-06-15 2019-12-19 City Of Hope Oligonucleotide-based proteolysis targeting chimera
CN109762042B (zh) * 2018-09-21 2022-04-08 河北艾克美冀生物科技有限公司 一种治疗癌症的药物、其合成方法和应用
US20230287406A1 (en) * 2019-03-04 2023-09-14 Mpeg La, L.L.C. Multimeric oligonucleotides with enhanced bioactivity
AU2020322440A1 (en) * 2019-07-26 2022-02-24 Mina Therapeutics Limited Compositions and methods of using C/EBP alpha saRNA
WO2021108702A1 (en) 2019-11-26 2021-06-03 City Of Hope Microrna as a therapeutic agent
KR102433981B1 (ko) * 2020-12-15 2022-08-19 대구가톨릭대학교산학협력단 HIF-1α 및 STAT5 전사인자를 억제하는 합성 디코이 올리고핵산 및 이를 유효성분으로 함유하는 아토피 피부염의 예방 또는 치료용 약학적 조성물
WO2022166849A1 (en) * 2021-02-08 2022-08-11 Ractigen Therapeutics Multi-valent oligonucleotide agent and methods of use thereof
WO2023034853A2 (en) * 2021-08-31 2023-03-09 City Of Hope Oligonucleotides having 6-thio-2'-deoxyguanosine residues and uses thereof
CN114908094B (zh) * 2022-05-31 2024-03-12 中山大学 一类反义寡核苷酸分子及其在制备治疗恶性肿瘤药物中的应用
JP2025535719A (ja) * 2022-10-07 2025-10-28 シティ・オブ・ホープ 二本鎖オリゴヌクレオチド及び使用方法
CN120390802A (zh) * 2022-11-29 2025-07-29 隆萨销售股份有限公司 使用靶向stat6的细胞外囊泡-aso的方法
CN118480552B (zh) * 2024-01-18 2024-10-01 成都先衍生物技术有限公司 一种靶向CEBPA基因表达的saRNA及其用途

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WO2017004357A1 (en) * 2015-07-02 2017-01-05 City Of Hope Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof

Also Published As

Publication number Publication date
US11464865B2 (en) 2022-10-11
JP7073109B2 (ja) 2022-05-23
JP2022116006A (ja) 2022-08-09
US10758624B2 (en) 2020-09-01
WO2017004357A1 (en) 2017-01-05
JP7459168B2 (ja) 2024-04-01
US20210038733A1 (en) 2021-02-11
EP3316894A4 (en) 2019-06-19
JP2018519831A (ja) 2018-07-26
CN107980001A (zh) 2018-05-01
US20180185497A1 (en) 2018-07-05
CN114224907A (zh) 2022-03-25
US12611462B2 (en) 2026-04-28
EP3316894A1 (en) 2018-05-09
US20220401563A1 (en) 2022-12-22
CN107980001B (zh) 2021-12-17

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