CN112424356A - 用于经口递送寡核苷酸的方法 - Google Patents

用于经口递送寡核苷酸的方法 Download PDF

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Publication number
CN112424356A
CN112424356A CN201980048096.XA CN201980048096A CN112424356A CN 112424356 A CN112424356 A CN 112424356A CN 201980048096 A CN201980048096 A CN 201980048096A CN 112424356 A CN112424356 A CN 112424356A
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modified oligonucleotide
modified
nucleoside
certain embodiments
sugar moiety
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Pending
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CN201980048096.XA
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English (en)
Chinese (zh)
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T·瑞顿
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Legulus Therapeutics Co ltd
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Legulus Therapeutics Co ltd
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
CN201980048096.XA 2018-07-20 2019-07-19 用于经口递送寡核苷酸的方法 Pending CN112424356A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862701317P 2018-07-20 2018-07-20
US62/701,317 2018-07-20
PCT/US2019/042561 WO2020018887A1 (en) 2018-07-20 2019-07-19 Methods for oral delivery of oligonucleotides

Publications (1)

Publication Number Publication Date
CN112424356A true CN112424356A (zh) 2021-02-26

Family

ID=67704556

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980048096.XA Pending CN112424356A (zh) 2018-07-20 2019-07-19 用于经口递送寡核苷酸的方法

Country Status (6)

Country Link
US (1) US20220098581A1 (https=)
EP (1) EP3824087A1 (https=)
JP (1) JP2021531278A (https=)
CN (1) CN112424356A (https=)
TW (1) TW202019441A (https=)
WO (1) WO2020018887A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4282964A1 (en) * 2022-05-23 2023-11-29 Universitat de Valéncia Oligonucleotides conjugated to oleic acid and uses thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014179446A2 (en) * 2013-05-01 2014-11-06 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-122
CN105164261A (zh) * 2013-05-01 2015-12-16 莱古路斯治疗法股份有限公司 用于增强的细胞摄取的化合物和方法
CN106559995A (zh) * 2014-08-07 2017-04-05 莱古路斯治疗法股份有限公司 靶向代谢紊乱的微小rna

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1120707C (zh) 1995-11-22 2003-09-10 约翰斯·霍普金斯大学 增强生物分子的细胞摄取的配体
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
SI2539451T1 (sl) 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna
EP2561102A1 (en) * 2010-12-02 2013-02-27 Katholieke Universiteit Leuven K.U. Leuven R&D Irak-related interventions and diagnosis
AU2011343664B2 (en) 2010-12-17 2015-10-08 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for siRNA
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014179446A2 (en) * 2013-05-01 2014-11-06 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-122
CN105164261A (zh) * 2013-05-01 2015-12-16 莱古路斯治疗法股份有限公司 用于增强的细胞摄取的化合物和方法
CN105378080A (zh) * 2013-05-01 2016-03-02 莱古路斯治疗法股份有限公司 用于调节mir-122的微小rna化合物和方法
US9506030B2 (en) * 2013-05-01 2016-11-29 Regulus Therapeutics Inc. Compounds and methods for enhanced cellular uptake
CN106559995A (zh) * 2014-08-07 2017-04-05 莱古路斯治疗法股份有限公司 靶向代谢紊乱的微小rna

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
FEDRA GOTTARDO ET AL.: "Micro-RNA profiling in kidney and bladder cancers", 《UROL ONCOL》, vol. 25, no. 5 *

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Publication number Publication date
WO2020018887A1 (en) 2020-01-23
US20220098581A1 (en) 2022-03-31
TW202019441A (zh) 2020-06-01
EP3824087A1 (en) 2021-05-26
JP2021531278A (ja) 2021-11-18

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Application publication date: 20210226