CN111372611A - 修饰rna剪接的方法 - Google Patents

修饰rna剪接的方法 Download PDF

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Publication number
CN111372611A
CN111372611A CN201880052678.0A CN201880052678A CN111372611A CN 111372611 A CN111372611 A CN 111372611A CN 201880052678 A CN201880052678 A CN 201880052678A CN 111372611 A CN111372611 A CN 111372611A
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alkyl
amino
carbonyl
alkoxy
heteroaryl
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阿努拉哈·巴特查里亚
阿迈尔·达卡
克斯廷·埃芬贝格尔
维加亚拉克什米·加贝塔
米纳基·B·贾尼
李文成
尼柯莱·纳雷什金
克里斯多佛·特罗塔
卡利·韦丁格
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PTC Therapeutics Inc
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PTC Therapeutics Inc
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
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    • A61K31/454—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A61K31/551—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having two nitrogen atoms, e.g. dilazep
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    • C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
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    • C12N2320/33—Alteration of splicing

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CN201880052678.0A 2017-06-14 2018-06-13 修饰rna剪接的方法 Pending CN111372611A (zh)

Applications Claiming Priority (3)

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US201762519226P 2017-06-14 2017-06-14
US62/519,226 2017-06-14
PCT/US2018/037412 WO2018232039A1 (en) 2017-06-14 2018-06-13 Methods for modifying rna splicing

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US (1) US11608501B2 (https=)
EP (1) EP3638318A4 (https=)
JP (1) JP2020523365A (https=)
KR (1) KR20200017476A (https=)
CN (1) CN111372611A (https=)
AU (1) AU2018284853A1 (https=)
BR (1) BR112019026508A2 (https=)
CA (1) CA3065547A1 (https=)
EA (1) EA202090034A1 (https=)
IL (1) IL271053A (https=)
WO (1) WO2018232039A1 (https=)

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CN111194215A (zh) * 2017-08-04 2020-05-22 斯基霍克疗法公司 用于调节剪接的方法和组合物
WO2025247338A1 (zh) * 2024-05-30 2025-12-04 纽欧申医药(上海)有限公司 哒嗪环类化合物、其药物组合物及其应用

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JP2020523365A (ja) 2017-06-14 2020-08-06 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを改変する方法
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KR102925387B1 (ko) * 2019-02-04 2026-02-10 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
WO2020163382A1 (en) * 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP2022519637A (ja) * 2019-02-04 2022-03-24 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
EP3920910A4 (en) * 2019-02-04 2022-11-09 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
WO2020163406A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP2022519311A (ja) * 2019-02-05 2022-03-22 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
JP7551629B2 (ja) * 2019-02-05 2024-09-17 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
WO2020163401A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163405A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP7603595B2 (ja) * 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
JP7603594B2 (ja) * 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
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