CN113994004A - 具有增强生物活性的多聚寡核苷酸 - Google Patents

具有增强生物活性的多聚寡核苷酸 Download PDF

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Publication number
CN113994004A
CN113994004A CN202080033528.2A CN202080033528A CN113994004A CN 113994004 A CN113994004 A CN 113994004A CN 202080033528 A CN202080033528 A CN 202080033528A CN 113994004 A CN113994004 A CN 113994004A
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Prior art keywords
oligonucleotide
subunit
subunits
polyoligonucleotide
stranded
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Pending
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CN202080033528.2A
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English (en)
Chinese (zh)
Inventor
J.M.布朗
K.K.H.纽曼
H-P.沃恩洛彻
P.哈德维格
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MPEG LA LLC
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MPEG LA LLC
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Publication of CN113994004A publication Critical patent/CN113994004A/zh
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    • 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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Genetics & Genomics (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Dispersion Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
CN202080033528.2A 2019-03-04 2020-03-03 具有增强生物活性的多聚寡核苷酸 Pending CN113994004A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962813599P 2019-03-04 2019-03-04
US62/813,599 2019-03-04
US201962824771P 2019-03-27 2019-03-27
US62/824,771 2019-03-27
PCT/US2020/020845 WO2020180897A1 (en) 2019-03-04 2020-03-03 Multimeric oligonucleotides with enhanced bioactivity

Publications (1)

Publication Number Publication Date
CN113994004A true CN113994004A (zh) 2022-01-28

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Application Number Title Priority Date Filing Date
CN202080033528.2A Pending CN113994004A (zh) 2019-03-04 2020-03-03 具有增强生物活性的多聚寡核苷酸

Country Status (8)

Country Link
US (1) US20230287406A1 (https=)
EP (1) EP3935168A4 (https=)
JP (1) JP2022523226A (https=)
CN (1) CN113994004A (https=)
AU (1) AU2020233375A1 (https=)
CA (1) CA3132505A1 (https=)
IL (1) IL286070A (https=)
WO (1) WO2020180897A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3576752A4 (en) 2017-02-06 2020-12-16 Mpeg La, LLC MULTIMERIC OLIGONUCLEOTIDES WITH REDUCED RENAL CLEARANCE
AU2021263932B2 (en) * 2020-04-30 2025-07-03 Mpeg La, L.L.C. Multimeric oligonucleotides with divided strands
US20240318174A1 (en) * 2021-02-08 2024-09-26 Ractigen Therapeutics Multi-Valent Oligonucleotide Agent and Methods of Use Thereof

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE356630T1 (de) * 1998-04-03 2007-04-15 Univ Iowa Res Found Verfahren und produkte zur stimulierung des immunsystems mittels immunotherapeutischer oligonukleotide und zytokine
US8748405B2 (en) * 2007-01-26 2014-06-10 City Of Hope Methods and compositions for the treatment of cancer or other diseases
US20080311040A1 (en) * 2007-03-06 2008-12-18 Flagship Ventures METHODS AND COMPOSITIONS FOR IMPROVED THERAPEUTIC EFFECTS WITH siRNA
KR101141544B1 (ko) * 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
HK1200484A1 (zh) * 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
US9956294B2 (en) * 2013-01-18 2018-05-01 H. Lee Moffitt Cancer Center And Research Institute, Inc. Targeted sensitization of non-del(5q) malignant cells
IL316159A (en) * 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
JP7073109B2 (ja) * 2015-07-02 2022-05-23 シティ・オブ・ホープ ホスホロチオエート化オリゴデオキシヌクレオチドを含む化合物及び組成物、ならびにそれらの使用法
EP3368671A1 (en) * 2015-10-26 2018-09-05 Translate Bio Ma, Inc. Methods and compositions for increasing smn expression
WO2018030338A1 (ja) * 2016-08-08 2018-02-15 浩文 山本 アジュバント組成物
EP3576752A4 (en) * 2017-02-06 2020-12-16 Mpeg La, LLC MULTIMERIC OLIGONUCLEOTIDES WITH REDUCED RENAL CLEARANCE
JP2020522265A (ja) * 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法

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Publication number Publication date
WO2020180897A1 (en) 2020-09-10
IL286070A (en) 2021-10-31
AU2020233375A1 (en) 2021-09-30
CA3132505A1 (en) 2020-09-10
JP2022523226A (ja) 2022-04-21
EP3935168A4 (en) 2024-04-10
US20230287406A1 (en) 2023-09-14
EP3935168A1 (en) 2022-01-12

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