JP6929269B2 - 多発性嚢胞腎の処置のための方法 - Google Patents

多発性嚢胞腎の処置のための方法 Download PDF

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JP6929269B2
JP6929269B2 JP2018510089A JP2018510089A JP6929269B2 JP 6929269 B2 JP6929269 B2 JP 6929269B2 JP 2018510089 A JP2018510089 A JP 2018510089A JP 2018510089 A JP2018510089 A JP 2018510089A JP 6929269 B2 JP6929269 B2 JP 6929269B2
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certain embodiments
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modified oligonucleotide
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JP2018528945A (ja
JP2018528945A5 (enExample
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ジョン アール． アンドロサヴィッチ
ジョン アール． アンドロサヴィッチ
ビー．ネルソン チャウ
ビー．ネルソン チャウ
ヴィシャル ディー． パテール
ヴィシャル ディー． パテール
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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
レグルス セラピューティクス インコーポレイテッド
ボード オブ リージェンツ オブ ザ ユニバーシティ オブ テキサス システム
ボード オブ リージェンツ オブ ザ ユニバーシティ オブ テキサス システム
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    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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
    • C12N2310/322—2'-R Modification
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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
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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
    • C12N2310/3525—MOE, methoxyethoxy

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  • Health & Medical Sciences (AREA)
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  • Physics & Mathematics (AREA)
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  • Heart & Thoracic Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2018510089A 2015-08-26 2016-08-25 多発性嚢胞腎の処置のための方法 Active JP6929269B2 (ja)

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Application Number Priority Date Filing Date Title
US201562210031P 2015-08-26 2015-08-26
US62/210,031 2015-08-26
PCT/US2016/048603 WO2017035319A1 (en) 2015-08-26 2016-08-25 Methods for treatment of polycystic kidney disease

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JP2018528945A JP2018528945A (ja) 2018-10-04
JP2018528945A5 JP2018528945A5 (enExample) 2019-10-03
JP6929269B2 true JP6929269B2 (ja) 2021-09-01

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US (3) US10633657B2 (enExample)
EP (2) EP4268891A3 (enExample)
JP (1) JP6929269B2 (enExample)
KR (2) KR20250049446A (enExample)
CN (2) CN108135922A (enExample)
AU (1) AU2016312590B2 (enExample)
CA (1) CA2995996A1 (enExample)
DK (1) DK3340993T5 (enExample)
ES (1) ES2954151T3 (enExample)
IL (1) IL257596B (enExample)
MA (1) MA44836A (enExample)
MX (1) MX2018002354A (enExample)
RU (1) RU2742300C2 (enExample)
WO (1) WO2017035319A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3045461A1 (en) * 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
EP3548503A1 (en) * 2016-12-05 2019-10-09 Regulus Therapeutics Inc. Modified oligonucleotides for treatment of polycystic kidney disease
CN120519574A (zh) 2018-08-23 2025-08-22 罗氏创新中心哥本哈根有限公司 微小rna-134生物标志物
WO2024026354A2 (en) * 2022-07-27 2024-02-01 The General Hospital Corporation Methods and compositions for use in treating autosomal recessive polycystic kidney disease (arpkd)
WO2024254639A1 (en) * 2023-06-16 2024-12-19 PYC Therapeutics Limited Compositions and methods for treatment of kidney disease
WO2025110217A1 (ja) * 2023-11-22 2025-05-30 国立大学法人東海国立大学機構 嚢胞性腎疾患の予防及び／又は治療剤

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1648914A4 (en) * 2003-07-31 2009-12-16 Regulus Therapeutics Inc OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA
WO2005089731A2 (en) 2004-03-17 2005-09-29 Novartis Ag Use of renin inhibitors in therapy
JPWO2007126150A1 (ja) * 2006-04-27 2009-09-17 国立大学法人名古屋大学 癌の新規治療用組成物
US20120283411A9 (en) * 2006-06-29 2012-11-08 Ironwood Pharmaceuticals, Inc. Methods and compositions for the treatment of gastrointestinal disorders
MX2009003548A (es) 2006-10-03 2009-04-15 Alnylam Pharmaceuticals Inc Formulaciones que contienen lipidos.
US20090105174A1 (en) * 2007-04-20 2009-04-23 Amgen Inc. Nucleic acids hybridizable to micro rna and precursors thereof
US8569381B2 (en) * 2008-05-23 2013-10-29 Targacept, Inc. Combination therapy for the management of hypertension
WO2014082644A1 (en) * 2012-11-30 2014-06-05 WULFF, Peter, Samuel Circular rna for inhibition of microrna
JP2016534069A (ja) * 2013-10-24 2016-11-04 メイヨ・ファウンデーション・フォー・メディカル・エデュケーション・アンド・リサーチ Ｈｄａｃ６阻害剤での多嚢胞性疾患の治療
US20160362688A1 (en) * 2014-02-12 2016-12-15 Thomas Jefferson University Compositions and methods of using microrna inhibitors
CA3045461A1 (en) 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
EP3548503A1 (en) 2016-12-05 2019-10-09 Regulus Therapeutics Inc. Modified oligonucleotides for treatment of polycystic kidney disease

Also Published As

Publication number Publication date
JP2018528945A (ja) 2018-10-04
WO2017035319A8 (en) 2021-03-11
EP4268891A3 (en) 2024-01-24
DK3340993T3 (da) 2023-08-21
CA2995996A1 (en) 2017-03-02
US20190153442A1 (en) 2019-05-23
IL257596B (en) 2022-08-01
RU2742300C2 (ru) 2021-02-04
EP3340993B1 (en) 2023-06-14
MX2018002354A (es) 2018-12-10
EP3340993A1 (en) 2018-07-04
KR20180080181A (ko) 2018-07-11
US20220025372A1 (en) 2022-01-27
AU2016312590A1 (en) 2018-03-15
IL257596A (en) 2018-04-30
NZ740040A (en) 2024-07-26
MA44836A (fr) 2018-07-04
US11168325B2 (en) 2021-11-09
WO2017035319A1 (en) 2017-03-02
AU2016312590B2 (en) 2021-12-02
RU2018108206A3 (enExample) 2020-01-14
DK3340993T5 (da) 2024-10-07
EP4268891A2 (en) 2023-11-01
CN108135922A (zh) 2018-06-08
ES2954151T3 (es) 2023-11-20
US20200231971A1 (en) 2020-07-23
US10633657B2 (en) 2020-04-28
RU2018108206A (ru) 2019-09-27
HK1256883A1 (en) 2019-10-04
KR20250049446A (ko) 2025-04-11
CN114404440A (zh) 2022-04-29

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