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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cells
nucleic acid
rxrna
chemically modified
stranded nucleic
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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)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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 (https=)
EP (1) EP3664817A4 (https=)
JP (2) JP7820909B2 (https=)
CN (1) CN111201024A (https=)
AU (1) AU2018313149B2 (https=)
CA (1) CA3070747A1 (https=)
WO (1) WO2019032619A1 (https=)

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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CA2746527A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
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
CN106074591B (zh) 2010-03-24 2020-01-14 菲奥医药公司 眼部症候中的rna干扰
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
CN108135923B (zh) 2015-07-06 2021-03-02 菲奥医药公司 靶向超氧化物歧化酶1（sod1）的核酸分子
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
WO2018195355A1 (en) 2017-04-19 2018-10-25 Rxi Pharmaceuticals Corporation Topical delivery of nucleic acid compounds
CN115135765A (zh) * 2019-11-08 2022-09-30 菲奥医药公司 用于免疫治疗的靶向含布罗莫结构域之蛋白4(brd4)的经化学修饰的寡核苷酸
CN115916348A (zh) 2020-06-18 2023-04-04 基因泰克公司 使用抗tigit抗体和pd-1轴结合拮抗剂的治疗
CN118871463A (zh) 2021-07-28 2024-10-29 基因泰克公司 用于治疗癌症的方法和组合物
WO2023015264A1 (en) * 2021-08-04 2023-02-09 Phio Pharmaceuticals Corp. Immunotherapy of cancer utilizing natural killer cells treated with chemically modified oligonucleotides
JP2024529025A (ja) 2021-08-04 2024-08-01 フィオ ファーマシューティカルズ コーポレーション 化学修飾されたオリゴヌクレオチド
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
EP4537107A2 (en) 2022-06-07 2025-04-16 Genentech, Inc. Method for determining the efficacy of a lung cancer treatment comprising an anti-pd-l1 antagonist and an anti-tigit antagonist antibody
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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EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
CA2746527A1 (en) * 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Rna interference in skin indications
US8691971B2 (en) * 2008-09-23 2014-04-08 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
US9957558B2 (en) * 2011-04-28 2018-05-01 Life Technologies Corporation Methods and compositions for multiplex PCR
CA2958200A1 (en) * 2014-08-14 2016-02-18 Novartis Ag Treatment of cancer using a gfr alpha-4 chimeric antigen receptor
WO2016177343A1 (zh) * 2015-05-05 2016-11-10 江苏命码生物科技有限公司 一种新的前体miRNA及其在肿瘤治疗中的应用
EP3349792A1 (en) * 2015-09-14 2018-07-25 Compass Therapeutics LLC Compositions and methods for treating cancer via antagonism of the cd155/tigit pathway and tgf-

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Non-Patent Citations (1)

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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

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Publication number Publication date
EP3664817A1 (en) 2020-06-17
AU2018313149B2 (en) 2024-11-14
JP2024028231A (ja) 2024-03-01
WO2019032619A1 (en) 2019-02-14
AU2018313149A1 (en) 2020-03-05
JP2020532955A (ja) 2020-11-19
CA3070747A1 (en) 2019-02-14
EP3664817A4 (en) 2021-09-22
WO2019032619A9 (en) 2020-02-13
JP7820909B2 (ja) 2026-02-26
US20200215113A1 (en) 2020-07-09

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Application publication date: 20200526