JP2016537027A - ジスルフィド基を有するポリヌクレオチド構築物 - Google Patents

ジスルフィド基を有するポリヌクレオチド構築物 Download PDF

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JP2016537027A
JP2016537027A JP2016553242A JP2016553242A JP2016537027A JP 2016537027 A JP2016537027 A JP 2016537027A JP 2016553242 A JP2016553242 A JP 2016553242A JP 2016553242 A JP2016553242 A JP 2016553242A JP 2016537027 A JP2016537027 A JP 2016537027A
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optionally substituted
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group
aryl
polynucleotide construct
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JP2016537027A5 (enExample
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ブラッドショー，コート，ダブリュ．
エルテプ，ラクスマン
カバキビ，アイマン
ラム，ソン
リウ，ビン
リウ，ディングオ
ミード，ブライアン，アール．
サカムリ，スクマー
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ソルスティス バイオロジクス，リミティッド
ソルスティス バイオロジクス，リミティッド
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Publication of JP2016537027A publication Critical patent/JP2016537027A/ja
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65586—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system at least one of the hetero rings does not contain nitrogen as ring hetero atom
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/1137—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 enzymes
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • C07F9/65616—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
    • 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
    • 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
    • C12N2310/311—Phosphotriesters
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
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  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2016553242A 2013-11-06 2014-11-06 ジスルフィド基を有するポリヌクレオチド構築物 Pending JP2016537027A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361900685P 2013-11-06 2013-11-06
US61/900,685 2013-11-06
US201461975686P 2014-04-04 2014-04-04
US61/975,686 2014-04-04
US201462008906P 2014-06-06 2014-06-06
US62/008,906 2014-06-06
PCT/US2014/064401 WO2015069932A1 (en) 2013-11-06 2014-11-06 Polynucleotide constructs having disulfide groups

Publications (2)

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JP2016537027A true JP2016537027A (ja) 2016-12-01
JP2016537027A5 JP2016537027A5 (enExample) 2017-12-14

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US (1) US20160257961A1 (enExample)
EP (1) EP3066105A4 (enExample)
JP (1) JP2016537027A (enExample)
CN (1) CN106061981A (enExample)
AU (1) AU2014346658A1 (enExample)
CA (1) CA2929651A1 (enExample)
WO (1) WO2015069932A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020512843A (ja) * 2017-04-14 2020-04-30 トルニネ， インコーポレイテッド 免疫調節ポリヌクレオチド、その抗体コンジュゲート、及びそれらの使用方法
JP2024501857A (ja) * 2020-12-31 2024-01-16 アルナイラム ファーマシューティカルズ， インコーポレイテッド 環状ジスルフィド修飾リン酸ベースのオリゴヌクレオチドプロドラッグ
WO2025160446A1 (en) * 2024-01-25 2025-07-31 Ai Proteins, Inc. Methods and systems for characterizing structural features of a protein

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CN105764491A (zh) 2013-12-09 2016-07-13 度瑞公司 药物活性剂复合物、聚合物复合物，以及包括其的组合物和方法
EP3152308A4 (en) * 2014-06-06 2017-12-27 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups
US20180133327A1 (en) * 2015-03-16 2018-05-17 Amal Therapeutics Sa Cell Penetrating Peptides and Complexes Comprising the Same
CN105175546A (zh) * 2015-05-27 2015-12-23 连祺周 人血管内皮生长因子与表皮生长因子样结构域7的融合蛋白
JPWO2016208773A1 (ja) * 2015-06-26 2018-04-12 協和発酵キリン株式会社 オリゴヌクレオチド誘導体
CN105037739B (zh) * 2015-07-28 2017-10-27 四川大学 具有精氨酸穿膜作用的还原敏感型聚合物及其制备方法与应用
AU2016349457B2 (en) * 2015-11-06 2022-09-22 Qiagen Sciences Llc Nucleotide analogues
WO2017100461A1 (en) * 2015-12-08 2017-06-15 Solstice Biologics, Ltd. Polynucleotide constructs having an auxiliary moiety non-bioreversibly linked to an internucleoside phosphate or phosphorothioate
RU2769314C1 (ru) 2016-03-16 2022-03-30 Амаль Терапьютикс Са Комбинация модулятора иммунных контрольных точек и комплекса, содержащего проникающий в клетку пептид, карго-молекулу и пептидный агонист tlr, для применения в медицине
WO2018004004A1 (ja) * 2016-06-30 2018-01-04 協和発酵キリン株式会社 核酸複合体
CN109803973B (zh) 2016-08-10 2022-05-24 癌靶技术有限责任公司 螯合psma抑制剂
KR20190058477A (ko) 2016-08-17 2019-05-29 솔스티스 바이올로직스, 리미티드 폴리뉴클레오티드 구축물
WO2018039364A1 (en) 2016-08-23 2018-03-01 Dicerna Pharmaceuticals, Inc. Compositions comprising reversibly modified oligonucleotides and uses thereof
HUE067838T2 (hu) 2016-09-21 2024-11-28 Amal Therapeutics Sa Egy sejtpenetráló peptidet, egy multiepitópot és egy TLR peptidagonistát tartalmazó fúzió rák kezelésére
CA3043619A1 (en) * 2016-11-23 2018-05-31 Cancer Targeted Technology Llc Albumin-binding psma inhibitors
WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
AU2019222767A1 (en) 2018-02-14 2020-08-27 Deep Genomics Incorporated Oligonucleotide therapy for Wilson disease
KR20210102204A (ko) * 2018-10-17 2021-08-19 탈락 테라퓨틱스, 인크. 면역 조절 폴리뉴클레오타이드 컨쥬게이트 및 사용 방법
CN109384715B (zh) * 2018-11-19 2021-09-03 复旦大学附属肿瘤医院 前列腺特异性膜抗原的小分子抑制剂的制备方法
KR20220030204A (ko) * 2018-12-28 2022-03-10 서나오믹스, 인크. 치료 분자의 표적화된 전달
CN115175685B (zh) 2019-12-09 2024-12-13 艾姆皮瑞克公司 用于治疗血管生成素样4(angptl4)相关疾病的寡核苷酸
US11920136B2 (en) 2020-02-28 2024-03-05 Tallac Therapeutics, Inc. Transglutaminase-mediated conjugation
CN112402620A (zh) * 2020-12-07 2021-02-26 南开大学 肿瘤微环境还原响应性的纳米药物及其制备方法
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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020512843A (ja) * 2017-04-14 2020-04-30 トルニネ， インコーポレイテッド 免疫調節ポリヌクレオチド、その抗体コンジュゲート、及びそれらの使用方法
JP2024167198A (ja) * 2017-04-14 2024-12-03 タラック セラピューティクス，インコーポレイテッド 免疫調節ポリヌクレオチド、その抗体コンジュゲート、及びそれらの使用方法
JP2024501857A (ja) * 2020-12-31 2024-01-16 アルナイラム ファーマシューティカルズ， インコーポレイテッド 環状ジスルフィド修飾リン酸ベースのオリゴヌクレオチドプロドラッグ
WO2025160446A1 (en) * 2024-01-25 2025-07-31 Ai Proteins, Inc. Methods and systems for characterizing structural features of a protein

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AU2014346658A1 (en) 2016-06-02
WO2015069932A1 (en) 2015-05-14
CA2929651A1 (en) 2015-05-14
US20160257961A1 (en) 2016-09-08
CN106061981A (zh) 2016-10-26

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