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
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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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US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
JP2019535789A (ja) 2016-11-28 2019-12-12 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スプライシングを調節する方法
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
JP7312749B2 (ja) 2017-08-04 2023-07-21 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 スプライシングをモジュレートする方法および組成物
US20210355509A1 (en) * 2018-08-07 2021-11-18 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods
CN113166779A (zh) * 2018-10-09 2021-07-23 北卡罗来纳大学查佩尔希尔分校 调控的基因编辑系统
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
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
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
WO2021014428A1 (en) 2019-07-25 2021-01-28 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
IL293340A (en) 2019-12-02 2022-07-01 Storm Therapeutics Ltd Polyheterocyclic compounds as mettl3 inhibitors
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