JP7483612B2 - ゲノム編集効率を増加するためのキノキサリノン化合物、組成物、方法、およびキット - Google Patents

ゲノム編集効率を増加するためのキノキサリノン化合物、組成物、方法、およびキット Download PDF

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JP7483612B2
JP7483612B2 JP2020539234A JP2020539234A JP7483612B2 JP 7483612 B2 JP7483612 B2 JP 7483612B2 JP 2020539234 A JP2020539234 A JP 2020539234A JP 2020539234 A JP2020539234 A JP 2020539234A JP 7483612 B2 JP7483612 B2 JP 7483612B2
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combination
cells
genome editing
dna
compound
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JP2021511312A (ja
JP2021511312A5 (enExample
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スディプタ マハジャン，
マーク ソール ワインバーグ，
ディエゴ セバスチャン ダストルフォ，
ケビン エム． コットレル，
マーク エー． モリス，
ジョン パトリック マクスウェル，
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Vertex Pharmaceuticals Inc
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Vertex Pharmaceuticals Inc
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    • C07—ORGANIC CHEMISTRY
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    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
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    • C07D241/00—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings
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    • C07D241/38—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings condensed with carbocyclic rings or ring systems with only hydrogen or carbon atoms directly attached to the ring nitrogen atoms
    • C07D241/40—Benzopyrazines
    • C07D241/44—Benzopyrazines with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to carbon atoms of the hetero ring
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    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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    • A61K31/5375—1,4-Oxazines, e.g. morpholine
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JP2020539234A 2018-01-17 2019-01-16 ゲノム編集効率を増加するためのキノキサリノン化合物、組成物、方法、およびキット Active JP7483612B2 (ja)

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US201862618385P 2018-01-17 2018-01-17
US62/618,385 2018-01-17
PCT/US2019/013785 WO2019143677A1 (en) 2018-01-17 2019-01-16 Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency

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JP2021511312A JP2021511312A (ja) 2021-05-06
JP2021511312A5 JP2021511312A5 (enExample) 2022-02-24
JP7483612B2 true JP7483612B2 (ja) 2024-05-15

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US (1) US12269804B2 (enExample)
EP (1) EP3740483B1 (enExample)
JP (1) JP7483612B2 (enExample)
KR (1) KR20200110687A (enExample)
CN (1) CN111770921B (enExample)
AU (1) AU2019209292B2 (enExample)
BR (1) BR112020013626A2 (enExample)
CA (1) CA3088791A1 (enExample)
EA (1) EA202091707A1 (enExample)
ES (1) ES2940786T3 (enExample)
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MA51619A (fr) * 2018-01-17 2021-04-14 Vertex Pharma Inhibiteurs de la protéine kinase dépendante de l'adn
BR112020013626A2 (pt) 2018-01-17 2020-12-01 Vertex Pharmaceuticals Incorporated compostos de quinoxalinona, composições, métodos e kits para aumentar a eficiência de edição de genoma
JP7466448B2 (ja) 2018-01-17 2024-04-12 バーテックス ファーマシューティカルズ インコーポレイテッド Ｄｎａ－ｐｋ阻害剤
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EP4022057A1 (en) * 2019-08-27 2022-07-06 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of disorders associated with repetitive dna
WO2022136306A1 (en) * 2020-12-21 2022-06-30 Aarhus Universitet Dopaminergic neuron progenitor cells or cell derivatives thereof obtained from lineage restricted pluripotent stem cells
CN112750498B (zh) * 2020-12-30 2022-06-24 同济大学 靶向逆转录引物结合位点从而抑制hiv病毒复制的方法
TW202302848A (zh) 2021-02-26 2023-01-16 美商維泰克斯製藥公司 以crispr/sacas9治療第1型肌強直性營養不良之組合物及方法
EP4298221A1 (en) 2021-02-26 2024-01-03 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/slucas9
CA3213120A1 (en) * 2021-03-10 2022-09-15 adMare Therapeutics Society 7-morpholino-1,6-naphthyridin-5-yl derivatives and pharmaceutical compositions thereof useful as dna-pk inhibitor
WO2023018637A1 (en) 2021-08-09 2023-02-16 Vertex Pharmaceuticals Incorporated Gene editing of regulatory elements
CN114685494B (zh) * 2022-03-16 2024-04-16 中国人民解放军军事科学院军事医学研究院 SpCas9抑制剂的合成制备方法及其应用
US20250326754A1 (en) * 2022-05-11 2025-10-23 adMare Therapeutics Society DNA-PK Inhibitor Compounds and Uses Thereof
WO2024229292A2 (en) * 2023-05-02 2024-11-07 Duke University Compositions and methods for engineering mature ipsc-derived and esc-derived hepatocytes
WO2025049254A1 (en) * 2023-08-25 2025-03-06 Juno Therapeutics, Inc. Pyrazolopyridine based inhibitors of dna-dependent protein kinase and compositions and application in gene editing

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