JP7297320B2 - アンジェルマン症候群アンチセンス治療 - Google Patents

アンジェルマン症候群アンチセンス治療 Download PDF

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JP7297320B2
JP7297320B2 JP2020529238A JP2020529238A JP7297320B2 JP 7297320 B2 JP7297320 B2 JP 7297320B2 JP 2020529238 A JP2020529238 A JP 2020529238A JP 2020529238 A JP2020529238 A JP 2020529238A JP 7297320 B2 JP7297320 B2 JP 7297320B2
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ube3a
nucleic acid
rna
pharmaceutical composition
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JP2021505129A5 (enExample
JP2021505129A (ja
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ビクター ディンドット，スコット
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ザ テキサス エイ・アンド・エム ユニヴァーシティ システム
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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JP2020529238A 2017-12-01 2018-11-30 アンジェルマン症候群アンチセンス治療 Active JP7297320B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023093643A JP7601441B2 (ja) 2017-12-01 2023-06-07 アンジェルマン症候群アンチセンス治療
JP2024206905A JP2025041632A (ja) 2017-12-01 2024-11-28 アンジェルマン症候群アンチセンス治療

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762593431P 2017-12-01 2017-12-01
US62/593,431 2017-12-01
US201862676034P 2018-05-24 2018-05-24
US62/676,034 2018-05-24
PCT/US2018/063416 WO2019109001A1 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

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JP2021505129A JP2021505129A (ja) 2021-02-18
JP2021505129A5 JP2021505129A5 (enExample) 2022-01-06
JP7297320B2 true JP7297320B2 (ja) 2023-06-26

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JP2020529238A Active JP7297320B2 (ja) 2017-12-01 2018-11-30 アンジェルマン症候群アンチセンス治療
JP2023093643A Active JP7601441B2 (ja) 2017-12-01 2023-06-07 アンジェルマン症候群アンチセンス治療
JP2024206905A Pending JP2025041632A (ja) 2017-12-01 2024-11-28 アンジェルマン症候群アンチセンス治療

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JP2024206905A Pending JP2025041632A (ja) 2017-12-01 2024-11-28 アンジェルマン症候群アンチセンス治療

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KR (2) KR102723989B1 (enExample)
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AU (1) AU2018375807B2 (enExample)
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Families Citing this family (9)

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PL3374509T3 (pl) 2015-11-12 2021-05-04 F. Hoffmann-La Roche Ag Oligonukleotydy do indukowania ojcowskiej ekspresji UBE3A
WO2019168950A1 (en) * 2018-02-27 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for treating angelman syndrome
TWI874376B (zh) 2019-03-29 2025-03-01 美商Ionis製藥公司 用於調節ube3a-ats 之化合物及方法
TW202216996A (zh) * 2020-06-29 2022-05-01 美商Ionis製藥公司 調節ｐｌｐ１之化合物及方法
CA3197664A1 (en) * 2020-10-06 2022-04-14 University Of Maryland, Baltimore Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides
EP4473116A2 (en) * 2022-03-07 2024-12-11 University of Connecticut Shrna targeting snord115 locations to restore paternal ube3a gene expression in angelman syndrome
CN120476156A (zh) 2023-01-31 2025-08-12 富士胶片株式会社 固化性组合物、固化物、光学材料、衍射光学元件及化合物
WO2025100980A1 (ko) * 2023-11-08 2025-05-15 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
WO2025217466A1 (en) * 2024-04-11 2025-10-16 Ultragenyx Pharmaceutical Inc. Dosing regimens for antisense treatment of angelman syndrome

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JP2015529635A (ja) 2012-06-25 2015-10-08 アイシス ファーマシューティカルズ， インコーポレーテッド Ｕｂｅ３ａ−ａｔｓ発現の調節
WO2017081223A1 (en) 2015-11-12 2017-05-18 Roche Innovation Center Copenhagen A/S Oligonucleotides for inducing paternal ube3a expression

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EP3717646A4 (en) 2021-12-01
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CO2020006361A2 (es) 2020-08-10
PT3717646T (pt) 2025-07-28
IL274146A (en) 2020-06-30
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JP2023123517A (ja) 2023-09-05
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KR102723989B1 (ko) 2024-11-01
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US11198869B2 (en) 2021-12-14
KR20240161203A (ko) 2024-11-12
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IL274146B1 (en) 2024-07-01
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LT3717646T (lt) 2025-06-10
BR112020009431A2 (pt) 2020-10-13
RU2020121752A (ru) 2021-12-30
AU2018375807A1 (en) 2020-05-07
US20200370046A1 (en) 2020-11-26
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