AU2017363369A1 - Methods for modulating RNA splicing - Google Patents

Methods for modulating RNA splicing Download PDF

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Publication number
AU2017363369A1
AU2017363369A1 AU2017363369A AU2017363369A AU2017363369A1 AU 2017363369 A1 AU2017363369 A1 AU 2017363369A1 AU 2017363369 A AU2017363369 A AU 2017363369A AU 2017363369 A AU2017363369 A AU 2017363369A AU 2017363369 A1 AU2017363369 A1 AU 2017363369A1
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Australia
Prior art keywords
amino
8alkyl
salkyl
hydroxy
heterocyclyl
Prior art date
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Abandoned
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AU2017363369A
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Inventor
Anuradha Bhattacharyya
Amal DAKKA
Kerstin EFFENBERGER
Vijayalakshmi GABBETA
Wencheng Li
Nikolai Naryshkin
Christopher Trotta
Kari WIEDINGER
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PTC Therapeutics Inc
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PTC Therapeutics Inc
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Publication of AU2017363369A1 publication Critical patent/AU2017363369A1/en
Abandoned legal-status Critical Current

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AU2017363369A 2016-11-28 2017-11-27 Methods for modulating RNA splicing Abandoned AU2017363369A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662426619P 2016-11-28 2016-11-28
US62/426,619 2016-11-28
PCT/US2017/063323 WO2018098446A1 (en) 2016-11-28 2017-11-27 Methods for modulating rna splicing

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AU2017363369A1 true AU2017363369A1 (en) 2019-05-30

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AU2017363369A Abandoned AU2017363369A1 (en) 2016-11-28 2017-11-27 Methods for modulating RNA splicing

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US (2) US11702646B2 (https=)
EP (1) EP3544435A4 (https=)
JP (2) JP2019535789A (https=)
CN (1) CN110352007A (https=)
AU (1) AU2017363369A1 (https=)
CA (1) CA3043755A1 (https=)
EA (1) EA201991309A1 (https=)
MX (1) MX2019005588A (https=)
WO (1) WO2018098446A1 (https=)

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EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
EA201991309A1 (ru) 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
WO2019028440A1 (en) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
US20210355509A1 (en) * 2018-08-07 2021-11-18 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods
EP3864161A4 (en) * 2018-10-09 2022-11-23 The University of North Carolina at Chapel Hill Regulated gene editing system
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN114126613A (zh) 2019-02-05 2022-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN113661162A (zh) 2019-02-06 2021-11-16 斯基霍克疗法公司 用于调节剪接的方法和组合物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
EP4004213A1 (en) 2019-07-25 2022-06-01 Novartis AG Regulatable expression systems
EP4017979A4 (en) * 2019-08-19 2024-03-27 Stoke Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION
CN115151540A (zh) 2019-12-02 2022-10-04 风暴治疗有限公司 作为mettl3抑制剂的多杂环化合物
CA3167756A1 (en) * 2020-02-04 2021-08-12 Regeneron Pharmaceuticals, Inc. Treatment of ophthalmic conditions with son of sevenless 2 (sos2) inhibitors
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院(Us) 用于基因表达的诱导型可变剪接调节的组合物和方法
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