JP2021531278A - オリゴヌクレオチドの経口送達のための方法 - Google Patents

オリゴヌクレオチドの経口送達のための方法 Download PDF

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JP2021531278A
JP2021531278A JP2021502604A JP2021502604A JP2021531278A JP 2021531278 A JP2021531278 A JP 2021531278A JP 2021502604 A JP2021502604 A JP 2021502604A JP 2021502604 A JP2021502604 A JP 2021502604A JP 2021531278 A JP2021531278 A JP 2021531278A
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nucleoside
modified
modified oligonucleotide
certain embodiments
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ティモシー ライト
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レグルス セラピューティクス インコーポレイテッド
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
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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
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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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
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    • 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)
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  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
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  • Molecular Biology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
JP2021502604A 2018-07-20 2019-07-19 オリゴヌクレオチドの経口送達のための方法 Pending JP2021531278A (ja)

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 (2)

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JP2021531278A true JP2021531278A (ja) 2021-11-18
JP2021531278A5 JP2021531278A5 (https=) 2022-07-26

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JP2021502604A Pending JP2021531278A (ja) 2018-07-20 2019-07-19 オリゴヌクレオチドの経口送達のための方法

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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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012072685A1 (en) * 2010-12-02 2012-06-07 Katholieke Universiteit Leuven, K.U.Leuven R&D Irak-related interventions and diagnosis
JP2016518841A (ja) * 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド 細胞取り込みの向上のための化合物および方法
JP2016518842A (ja) * 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法
JP2017523790A (ja) * 2014-08-07 2017-08-24 レグルス セラピューティクス インコーポレイテッド 代謝障害のためのマイクロｒｎａの標的化

Family Cites Families (6)

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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
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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012072685A1 (en) * 2010-12-02 2012-06-07 Katholieke Universiteit Leuven, K.U.Leuven R&D Irak-related interventions and diagnosis
JP2016518841A (ja) * 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド 細胞取り込みの向上のための化合物および方法
JP2016518842A (ja) * 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法
JP2017523790A (ja) * 2014-08-07 2017-08-24 レグルス セラピューティクス インコーポレイテッド 代謝障害のためのマイクロｒｎａの標的化

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GASTROENTEROLOGY, vol. 131(6), JPN6023034127, 2006, pages 1786 - 1798, ISSN: 0005133287 *

Also Published As

Publication number Publication date
WO2020018887A1 (en) 2020-01-23
US20220098581A1 (en) 2022-03-31
CN112424356A (zh) 2021-02-26
TW202019441A (zh) 2020-06-01
EP3824087A1 (en) 2021-05-26

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