CN111201024A - 经化学修饰的寡核苷酸 - Google Patents

经化学修饰的寡核苷酸 Download PDF

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Publication number
CN111201024A
CN111201024A CN201880061531.8A CN201880061531A CN111201024A CN 111201024 A CN111201024 A CN 111201024A CN 201880061531 A CN201880061531 A CN 201880061531A CN 111201024 A CN111201024 A CN 111201024A
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cell
nucleic acid
rxrna
cells
chemically modified
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Pending
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CN201880061531.8A
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English (en)
Chinese (zh)
Inventor
阿列克谢·叶利谢耶娃
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Phio Pharmaceuticals Corp
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RXi Pharmaceuticals Corp
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Publication of CN111201024A publication Critical patent/CN111201024A/zh
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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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/32—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
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
CN201880061531.8A 2017-08-07 2018-08-07 经化学修饰的寡核苷酸 Pending CN111201024A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762542043P 2017-08-07 2017-08-07
US62/542,043 2017-08-07
US201762558183P 2017-09-13 2017-09-13
US62/558,183 2017-09-13
PCT/US2018/045671 WO2019032619A1 (en) 2017-08-07 2018-08-07 CHEMICALLY MODIFIED OLIGONUCLEOTIDES

Publications (1)

Publication Number Publication Date
CN111201024A true CN111201024A (zh) 2020-05-26

Family

ID=65271710

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880061531.8A Pending CN111201024A (zh) 2017-08-07 2018-08-07 经化学修饰的寡核苷酸

Country Status (7)

Country Link
US (1) US20200215113A1 (enExample)
EP (1) EP3664817A4 (enExample)
JP (2) JP2020532955A (enExample)
CN (1) CN111201024A (enExample)
AU (1) AU2018313149B2 (enExample)
CA (1) CA3070747A1 (enExample)
WO (1) WO2019032619A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112266911A (zh) * 2020-08-27 2021-01-26 清华大学 核酸分子
CN113151180A (zh) * 2013-12-02 2021-07-23 菲奥医药公司 癌症的免疫治疗

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CN108165548B (zh) 2008-09-22 2022-10-14 菲奥医药公司 减小大小的自递送RNAi化合物
WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
CN103200945B (zh) 2010-03-24 2016-07-06 雷克西制药公司 眼部症候中的rna干扰
CA2947270A1 (en) 2014-04-28 2015-11-05 Rxi Pharmaceuticals Corporation Methods for treating cancer using nucleic acids targeting mdm2 or mycn
KR102689262B1 (ko) 2014-09-05 2024-07-30 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
EP3862005A1 (en) 2015-07-06 2021-08-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
US11021707B2 (en) 2015-10-19 2021-06-01 Phio Pharmaceuticals Corp. Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA
EP4055167A2 (en) * 2019-11-08 2022-09-14 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides targeting bromodomain containing protein 4 (brd4) for immunotherapy
US20210395366A1 (en) 2020-06-18 2021-12-23 Genentech, Inc. Treatment with anti-tigit antibodies and pd-1 axis binding antagonists
JP2024527049A (ja) 2021-07-28 2024-07-19 ジェネンテック， インコーポレイテッド がんを治療するための方法及び組成物
US20240301430A1 (en) * 2021-08-04 2024-09-12 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides
WO2023015264A1 (en) 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
TW202321308A (zh) 2021-09-30 2023-06-01 美商建南德克公司 使用抗ｔｉｇｉｔ抗體、抗ｃｄ３８抗體及ｐｄ—１軸結合拮抗劑治療血液癌症的方法
WO2023130021A1 (en) * 2021-12-30 2023-07-06 Phio Pharmaceuticals Corp. Chemically modified oligonucleotides with improved delivery properties
JP2025523387A (ja) 2022-06-07 2025-07-23 ジェネンテック， インコーポレイテッド 抗ｐｄ－ｌ１アンタゴニストおよび抗ｔｉｇｉｔアンタゴニスト抗体を含む、肺がん治療の有効性を判定するための方法
WO2024064769A1 (en) * 2022-09-21 2024-03-28 Phio Pharmaceuticals Corp. Induction of stem-like activated t cells
WO2025101204A1 (en) * 2023-11-08 2025-05-15 Phio Pharmaceuticals Corp. Intratumoral targeting of pd-1
WO2025117786A1 (en) * 2023-11-28 2025-06-05 Phio Pharmaceuticals Corp. Enhanced combination immune checkpoint inhibition therapy utilizing rnai

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US20160304873A1 (en) * 2013-12-02 2016-10-20 Mirimmune Inc. Immunotherapy of Cancer

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DK2284266T3 (da) * 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
CN108165548B (zh) * 2008-09-22 2022-10-14 菲奥医药公司 减小大小的自递送RNAi化合物
EP2346883B1 (en) * 2008-09-23 2016-03-23 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
US9745574B2 (en) * 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
EP3072977B1 (en) * 2011-04-28 2018-09-19 Life Technologies Corporation Methods and compositions for multiplex pcr
JP6919118B2 (ja) * 2014-08-14 2021-08-18 ノバルティス アーゲー ＧＦＲα−４キメラ抗原受容体を用いる癌の治療
US10533173B2 (en) * 2015-05-05 2020-01-14 Jiangsu Micromedmark Biotech Co., Ltd. Precursor miRNA and applications in tumor therapy thereof
AU2016322934A1 (en) * 2015-09-14 2018-04-12 Compass Therapeutics Llc Compositions and methods for treating cancer via antagonism of the CD155/TIGIT pathway and TGF-beta

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US20160304873A1 (en) * 2013-12-02 2016-10-20 Mirimmune Inc. Immunotherapy of Cancer

Non-Patent Citations (1)

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Title
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113151180A (zh) * 2013-12-02 2021-07-23 菲奥医药公司 癌症的免疫治疗
CN112266911A (zh) * 2020-08-27 2021-01-26 清华大学 核酸分子

Also Published As

Publication number Publication date
AU2018313149B2 (en) 2024-11-14
JP2020532955A (ja) 2020-11-19
WO2019032619A9 (en) 2020-02-13
EP3664817A1 (en) 2020-06-17
JP2024028231A (ja) 2024-03-01
CA3070747A1 (en) 2019-02-14
US20200215113A1 (en) 2020-07-09
EP3664817A4 (en) 2021-09-22
WO2019032619A1 (en) 2019-02-14
AU2018313149A1 (en) 2020-03-05

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Application publication date: 20200526