EA202190116A1 - Агент для профилактики или лечения спинальной мышечной атрофии - Google Patents

Агент для профилактики или лечения спинальной мышечной атрофии

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EA202190116A1
EA202190116A1 EA202190116A EA202190116A EA202190116A1 EA 202190116 A1 EA202190116 A1 EA 202190116A1 EA 202190116 A EA202190116 A EA 202190116A EA 202190116 A EA202190116 A EA 202190116A EA 202190116 A1 EA202190116 A1 EA 202190116A1
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aliphatic heterocycle
optionally substituted
prevention
agent
treatment
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EA202190116A
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Кодзи Фудзи
Такеси Ямасаки
Сунья Судзуки
Кодзи Оно
Хироки Такахаги
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Реборна Биосайенсиз, Инк.
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Publication of EA202190116A1 publication Critical patent/EA202190116A1/ru

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Abstract

Агент для профилактики или лечения спинальной мышечной атрофии по настоящему изобретению включает соединение, представленное формулой (I), или его сольгде каждый из W1, W2 и W3 независимо выбран из группы, состоящей из C-R2, C-R3, C-Rc и C-Rd, и определяется одним из следующих пунктов от (i) до (iv): (i) когда W3 представляет собой C-R2, тогда W1 представляет собой C-R3, W2 представляет собой C-Rc или N, и R1 представляет собой атом водорода; (ii) когда W3 представляет собой C-R3, тогда W1 представляет собой C-R2, W2 представляет собой C-Rc или N, и R1 представляет собой атом водорода, C1-8алкил или C1-8алкокси; (iii) когда W1 представляет собой C-R2, тогда W2 представляет собой C-Rc или N, W3 представляет собой C-Rd, и R1 представляет собой алифатический гетероцикл, содержащий один или более атомов азота, причем алифатический гетероцикл необязательно замещен неароматическим заместителем; и (iv) когда W2 представляет собой C-R2, тогда W1 представляет собой C-Rc, W3 представляет собой C-Rd, и R1 представляет собой алифатический гетероцикл, содержащий один или более атомов азота, причем алифатический гетероцикл необязательно замещен неароматическим заместителем; R2 представляет собой 6-членное или более ароматическое кольцо, необязательно замещенное неароматическим заместителем; R3 представляет собой алифатический гетероцикл, содержащий один или более атомов азота, причем алифатический гетероцикл необязательно замещен неароматическим заместителем; Q1 выбран из C-Ra и N; Q2 выбран из C-Rb и N; и каждый из Ra, Rb, Rc и Rd независимо выбран из группы, состоящей из атома водорода, галогена, C1-8алкила, C1-8алкокси и цианогруппы.
EA202190116A 2018-06-27 2019-06-27 Агент для профилактики или лечения спинальной мышечной атрофии EA202190116A1 (ru)

Applications Claiming Priority (2)

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JP2018122551 2018-06-27
PCT/JP2019/025732 WO2020004594A1 (ja) 2018-06-27 2019-06-27 脊髄性筋萎縮症の予防または治療剤

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US (1) US20220287748A1 (ru)
EP (1) EP3816160A4 (ru)
JP (1) JP7427252B2 (ru)
KR (1) KR20210025625A (ru)
CN (1) CN112469707A (ru)
AU (2) AU2019293757B2 (ru)
BR (1) BR112020026637A2 (ru)
CA (1) CA3105174A1 (ru)
CL (1) CL2020003386A1 (ru)
CO (1) CO2021000808A2 (ru)
CR (1) CR20210050A (ru)
EA (1) EA202190116A1 (ru)
IL (1) IL279786A (ru)
JO (1) JOP20200338A1 (ru)
MX (1) MX2020014116A (ru)
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PH (1) PH12020552263A1 (ru)
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EP3661509A4 (en) 2017-08-04 2021-01-13 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING THE SPLICE
SI3814348T1 (sl) 2018-06-27 2023-10-30 Bristol-Myers Squibb Company Substituirane naftiridinonske spojine, uporabne kot aktivatorji t celic
KR20210039368A (ko) * 2018-07-26 2021-04-09 도메인 테라퓨틱스 치환된 퀴나졸리논 유도체 및 mGluR4의 양성 알로스테릭 조절인자로서의 이의 용도
CA3169709A1 (en) * 2020-02-28 2021-09-02 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
MX2022012676A (es) * 2020-04-08 2023-01-11 Remix Therapeutics Inc Compuestos y metodos para modular el empalme.
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JP2023532331A (ja) 2020-07-02 2023-07-27 リミックス セラピューティクス インコーポレイテッド 核酸をスプライシングするため及び増殖性疾患を処置するための修飾因子としての5-[5-(ピペリジン-4-イル)チエノ[3,2-c]ピラゾール-2-イル]インダゾール誘導体及び関連する化合物並びにスプライシングを調節するための化合物及び方法
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