JP2021527713A - 増殖性細胞を抑制する方法 - Google Patents

増殖性細胞を抑制する方法 Download PDF

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JP2021527713A
JP2021527713A JP2021519532A JP2021519532A JP2021527713A JP 2021527713 A JP2021527713 A JP 2021527713A JP 2021519532 A JP2021519532 A JP 2021519532A JP 2021519532 A JP2021519532 A JP 2021519532A JP 2021527713 A JP2021527713 A JP 2021527713A
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nucleic acid
wrn
proliferative
agent
cells
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JP2021527713A5 (https=
JPWO2019241802A5 (https=
Inventor
リサ、ベルモント
ジェフ、ハーガー
裕次郎 畑
ローン、カテガヤ
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Ideaya Biosciences Inc
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Ideaya Biosciences Inc
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Publication of JPWO2019241802A5 publication Critical patent/JPWO2019241802A5/ja
Priority to JP2024109073A priority Critical patent/JP2024153667A/ja
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    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/4015Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil having oxo groups directly attached to the heterocyclic ring, e.g. piracetam, ethosuximide
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JP2021519532A 2018-06-15 2019-06-17 増殖性細胞を抑制する方法 Pending JP2021527713A (ja)

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Application Number Priority Date Filing Date Title
JP2024109073A JP2024153667A (ja) 2018-06-15 2024-07-05 増殖性細胞を抑制する方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862685866P 2018-06-15 2018-06-15
US62/685,866 2018-06-15
PCT/US2019/037559 WO2019241802A2 (en) 2018-06-15 2019-06-17 Methods of inhibiting proliferative cells

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JP2024109073A Division JP2024153667A (ja) 2018-06-15 2024-07-05 増殖性細胞を抑制する方法

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JP2021527713A true JP2021527713A (ja) 2021-10-14
JP2021527713A5 JP2021527713A5 (https=) 2022-06-27
JPWO2019241802A5 JPWO2019241802A5 (https=) 2022-06-27

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JP2021519532A Pending JP2021527713A (ja) 2018-06-15 2019-06-17 増殖性細胞を抑制する方法
JP2024109073A Pending JP2024153667A (ja) 2018-06-15 2024-07-05 増殖性細胞を抑制する方法

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US (1) US20210371855A1 (https=)
EP (1) EP3806850A2 (https=)
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019236448A1 (en) 2018-06-04 2019-12-12 The Broad Institute, Inc. Therapeutic treatment of microsatellite unstable cancers
WO2025239377A1 (ja) * 2024-05-16 2025-11-20 株式会社ジーンケア研究所 WRNヘリカーゼ遺伝子を標的とするsiRNA

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7714162B6 (ja) * 2018-12-03 2025-09-08 タケダ ファーマシューティカルズ ユーエスエー インコーポレイテッド Msh3活性に関連するトリヌクレオチドリピート伸長障害の処置のための方法
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
JP2024508841A (ja) * 2021-02-25 2024-02-28 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 プログラム可能なタンパク質分解のための化合物及び疾患処置のための使用方法
JP7753397B2 (ja) * 2021-05-26 2025-10-14 ノバルティス アーゲー ウェルナー症候群RecQヘリカーゼ(WRN)の阻害に関連する疾患を治療するためのトリアゾロ-ピリミジン類似体
WO2023062575A1 (en) * 2021-10-14 2023-04-20 Ideaya Biosciences, Inc. Cyclic vinyl sulfone compounds as wrn inhibitors
KR20250034120A (ko) * 2022-07-06 2025-03-10 비비디온 테라퓨틱스, 인코포레이티드 Wrn 헬리케이스 억제제를 포함하는 약제학적 조성물
TW202421158A (zh) 2022-11-18 2024-06-01 瑞士商諾華公司 藥物組合及其用途
WO2024140597A1 (zh) * 2022-12-30 2024-07-04 苏州浦合医药科技有限公司 Wrn解旋酶抑制剂
CN120603834A (zh) * 2023-01-18 2025-09-05 成都微芯药业有限公司 双杂环wrn抑制剂、其制备方法及其应用
WO2024153244A1 (zh) * 2023-01-20 2024-07-25 南京再明医药有限公司 含氮类化合物
WO2024215923A2 (en) * 2023-04-11 2024-10-17 Nimbus Wadjet, Inc. Cyclic vinyl sulfone wrn inhibitors
WO2024235844A1 (en) * 2023-05-12 2024-11-21 Institut National de la Santé et de la Recherche Médicale Methods of preventing on-target genotoxicity induced by nucleases
TW202510857A (zh) * 2023-06-01 2025-03-16 英商葛蘭素史密斯克藍智慧財產權有限公司 化合物及其用途
WO2025026382A1 (zh) * 2023-08-01 2025-02-06 海思科医药集团股份有限公司 一种氧代三并环类衍生物及其在医药上的应用
WO2025106693A1 (en) * 2023-11-16 2025-05-22 Eikon Therapeutics, Inc. Methods and compositions for treating cancers with high microsatellite instability levels
WO2025131978A1 (en) * 2023-12-20 2025-06-26 Nerviano Medical Sciences S.R.L. Drug-linker reagents for conjugation
WO2025237361A1 (zh) * 2024-05-16 2025-11-20 海思科医药集团股份有限公司 一种氧代三并环类衍生物及其在医药上的应用
WO2025252190A1 (en) * 2024-06-07 2025-12-11 Cullgen Inc. Targeted degraders of werner syndrome recq helicase

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002068690A1 (en) * 2001-02-23 2002-09-06 Isis Pharmaceuticals, Inc. Antisense modulation of wrn expression
JP2012219085A (ja) * 2011-04-13 2012-11-12 Shiga Univ Of Medical Science 頭頸部癌及び食道癌用抗癌剤及び増強剤
JP2015516144A (ja) * 2012-04-10 2015-06-08 ブイアイビー ブイゼットダブリュVib Vzw 癌におけるマイクロサテライト不安定性を検出しdna塩基除去修復経路の阻害による合成致死性を決定するための新規なマーカー

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6372772B1 (en) * 1997-08-01 2002-04-16 Prolx Pharmaceuticals Corporation Inhibitors of redox signaling and methods of using same
WO2002024692A1 (en) * 2000-09-20 2002-03-28 Karolinska Innovations Ab 1-azabicyclo[2.2.2]octan-3-one derivatives and maleimide derivatives and their use for treating cancer tumors
US7659278B2 (en) * 2002-02-21 2010-02-09 Aprea Ab Use of low molecular weight compounds for preparing a medicament useful in treating mutant p53 mediated diseases
EP1761629A2 (en) * 2004-05-19 2007-03-14 Trustees Of Boston University Modulation of wrn-mediated telomere-initiated cell signaling
WO2011014825A2 (en) * 2009-07-31 2011-02-03 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Antiangiogenic small molecules and methods of use
CA2951816A1 (en) * 2013-06-12 2014-12-18 Oncoimmunin, Inc. Systemic in vivo delivery of oligonucleotides
US20190185849A1 (en) 2016-06-29 2019-06-20 Crispr Therapeutics Ag Compositions and methods for gene editing
US11530413B2 (en) * 2017-07-21 2022-12-20 Novartis Ag Compositions and methods to treat cancer
MX2020013120A (es) * 2018-06-04 2021-07-21 Broad Inst Inc Tratamiento terapéutico de cánceres de microsatélites inestables.
JP7753397B2 (ja) * 2021-05-26 2025-10-14 ノバルティス アーゲー ウェルナー症候群RecQヘリカーゼ(WRN)の阻害に関連する疾患を治療するためのトリアゾロ-ピリミジン類似体

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002068690A1 (en) * 2001-02-23 2002-09-06 Isis Pharmaceuticals, Inc. Antisense modulation of wrn expression
JP2012219085A (ja) * 2011-04-13 2012-11-12 Shiga Univ Of Medical Science 頭頸部癌及び食道癌用抗癌剤及び増強剤
JP2015516144A (ja) * 2012-04-10 2015-06-08 ブイアイビー ブイゼットダブリュVib Vzw 癌におけるマイクロサテライト不安定性を検出しdna塩基除去修復経路の阻害による合成致死性を決定するための新規なマーカー

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
AGGARWAL M; ET AL: "INHIBITION OF HELICASE ACTIVITY BY A SMALL MOLECULE IMPAIRS WERNER SYNDROME HELICASE 以下備考", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. VOL:108, NR:4,, JPN5021010609, 10 January 2011 (2011-01-10), US, pages 1525 - 1530, ISSN: 0005278953 *
KAZUNOBU FUTAMI; ET AL: "RECQL1 AND WRN DNA REPAIR HELICASES: POTENTIAL THERAPEUTIC TARGETS AND PROLIFERATIVE 以下備考", FRONTIERS IN GENETICS, vol. VOL:5, NR:441,, JPN5021010608, 2015, pages 1 - 11, ISSN: 0005278954 *
R. MOLES; ET AL: "WRN-TARGETED THERAPY USING INHIBITORS NSC 19630 AND NSC 617145 INDUCE APOPTOSIS IN 以下備考", JOURNAL OF HEMATOLOGY & ONCOLOGY, vol. 9, no. 121, JPN5021010610, 9 November 2016 (2016-11-09), pages 1 - 11, ISSN: 0005077442 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019236448A1 (en) 2018-06-04 2019-12-12 The Broad Institute, Inc. Therapeutic treatment of microsatellite unstable cancers
WO2025239377A1 (ja) * 2024-05-16 2025-11-20 株式会社ジーンケア研究所 WRNヘリカーゼ遺伝子を標的とするsiRNA

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