JP7411632B2 - Ａｔｘｎ２発現を低減するための化合物及び方法 - Google Patents

Ａｔｘｎ２発現を低減するための化合物及び方法 Download PDF

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JP7411632B2
JP7411632B2 JP2021503566A JP2021503566A JP7411632B2 JP 7411632 B2 JP7411632 B2 JP 7411632B2 JP 2021503566 A JP2021503566 A JP 2021503566A JP 2021503566 A JP2021503566 A JP 2021503566A JP 7411632 B2 JP7411632 B2 JP 7411632B2
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certain embodiments
modified
seq
nucleobases
oligonucleotide
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JP2021530241A5 (https=
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フレアー，スーザン・エム
シング，プリヤム
リゴ，フランク
ジャファー－ネジャド，ペイマン
コルダシェヴィッチ，ホリー
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Ionis Pharmaceuticals Inc
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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JP2021503566A 2018-07-25 2019-07-25 Ａｔｘｎ２発現を低減するための化合物及び方法 Active JP7411632B2 (ja)

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US201862703240P 2018-07-25 2018-07-25
US62/703,240 2018-07-25
PCT/US2019/043424 WO2020023737A1 (en) 2018-07-25 2019-07-25 Compounds and methods for reducing atxn2 expression

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JP2021530241A JP2021530241A (ja) 2021-11-11
JP2021530241A5 JP2021530241A5 (https=) 2022-08-15
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JP7411632B2 true JP7411632B2 (ja) 2024-01-11

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US (3) US11078486B2 (https=)
EP (1) EP3826645A4 (https=)
JP (2) JP7411632B2 (https=)
KR (1) KR20210038589A (https=)
CN (1) CN112423767B (https=)
AR (1) AR115847A1 (https=)
AU (2) AU2019310097A1 (https=)
BR (1) BR112021000308A2 (https=)
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PE (1) PE20211398A1 (https=)
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CN106103717A (zh) 2014-03-19 2016-11-09 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
RS60707B1 (sr) * 2014-04-01 2020-09-30 Biogen Ma Inc Kompozicije za modulaciju ekspresije sod-1
US20190002887A1 (en) 2015-12-31 2019-01-03 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
US20200283800A1 (en) * 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
BR112020013994A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
BR112021000308A2 (pt) 2018-07-25 2021-04-13 Ionis Pharmaceuticals, Inc. Compostos e métodos para redução da expressão de atxn2
WO2021159008A2 (en) * 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
EP4100527A2 (en) * 2020-02-07 2022-12-14 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
US20230212572A1 (en) * 2020-06-09 2023-07-06 Roche Innovation Center Copenhagen A/S Guanosine Analogues for Use in Therapeutics Polynucleotides
US20240294906A1 (en) * 2020-07-29 2024-09-05 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases
BR112023021318A2 (pt) * 2021-04-14 2023-12-19 Aro Biotherapeutics Company Conjugados de domínio fn3-sirna e usos dos mesmos
WO2023102188A1 (en) * 2021-12-03 2023-06-08 Quralis Corporation Gapmer antisense oligonucleotides with modified backbone chemistries
WO2023107893A2 (en) * 2021-12-06 2023-06-15 The Board Of Trustees Of The Leland Stanford Junior University Treatment of neuorodegenerative diseases through the inhibition of ataxin-2
EP4536236A2 (en) * 2022-06-06 2025-04-16 University of Utah Research Foundation Methods of modulating atxn2 expression
US20250179491A1 (en) * 2022-06-24 2025-06-05 Eli Lilly And Company Atxn2 rna interference agents
WO2024056902A2 (en) * 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases
GB202302480D0 (en) * 2023-02-22 2023-04-05 Drishti Discoveries Ltd shRNA for the treatment of disease
WO2025067482A1 (zh) * 2023-09-28 2025-04-03 大睿生物 调控atxn2表达的dsrna分子

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