JP2021522800A - Ａｔｘｎ３発現を低減するための化合物及び方法 - Google Patents

Ａｔｘｎ３発現を低減するための化合物及び方法 Download PDF

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JP2021522800A
JP2021522800A JP2020561893A JP2020561893A JP2021522800A JP 2021522800 A JP2021522800 A JP 2021522800A JP 2020561893 A JP2020561893 A JP 2020561893A JP 2020561893 A JP2020561893 A JP 2020561893A JP 2021522800 A JP2021522800 A JP 2021522800A
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modified
nucleoside
modified oligonucleotide
certain embodiments
oligonucleotide
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JP2021522800A5 (enExample
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フレアー，スーザン・エム
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Ionis Pharmaceuticals Inc
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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
    • C12N15/1137—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 against enzymes
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/10—Applications; Uses in screening processes
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
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  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020561893A 2018-05-09 2019-05-09 Ａｔｘｎ３発現を低減するための化合物及び方法 Pending JP2021522800A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862669238P 2018-05-09 2018-05-09
US62/669,238 2018-05-09
PCT/US2019/031562 WO2019217708A1 (en) 2018-05-09 2019-05-09 Compounds and methods for reducing atxn3 expression

Publications (2)

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JP2021522800A true JP2021522800A (ja) 2021-09-02
JP2021522800A5 JP2021522800A5 (enExample) 2022-05-13

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US (2) US11434488B2 (enExample)
EP (2) EP4663758A3 (enExample)
JP (1) JP2021522800A (enExample)
KR (1) KR20210008497A (enExample)
CN (1) CN112189053B (enExample)
AR (1) AR115375A1 (enExample)
AU (1) AU2019266307A1 (enExample)
BR (1) BR112020021253A2 (enExample)
CA (1) CA3098144A1 (enExample)
CL (1) CL2020002888A1 (enExample)
CO (1) CO2020013433A2 (enExample)
CR (1) CR20200604A (enExample)
EA (1) EA202092500A1 (enExample)
JO (1) JOP20200283A1 (enExample)
MX (1) MX2020011911A (enExample)
PE (1) PE20212131A1 (enExample)
PH (1) PH12020500668A1 (enExample)
SG (1) SG11202010215TA (enExample)
TW (1) TW202003541A (enExample)
UY (1) UY38225A (enExample)
WO (1) WO2019217708A1 (enExample)

Families Citing this family (13)

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JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
EP4663758A3 (en) 2018-05-09 2026-04-29 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
WO2020172559A1 (en) * 2019-02-22 2020-08-27 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
CN113950529A (zh) 2019-06-06 2022-01-18 豪夫迈·罗氏有限公司 靶向atxn3的反义寡核苷酸
US12565653B2 (en) 2019-11-22 2026-03-03 Alnylam Pharmaceuticals, Inc. ATAXIN3 (ATXN3) RNAi agent compositions and methods of use thereof
CN120505310A (zh) * 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
JP7776420B2 (ja) * 2020-05-12 2025-11-26 田辺三菱製薬株式会社 Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物
US20230212572A1 (en) * 2020-06-09 2023-07-06 Roche Innovation Center Copenhagen A/S Guanosine Analogues for Use in Therapeutics Polynucleotides
US20230060373A1 (en) 2020-12-03 2023-03-02 Hoffmann-La Roche Inc. Antisense Oligonucleotides Targeting ATXN3
AR124229A1 (es) 2020-12-03 2023-03-01 Hoffmann La Roche Oligonucleótidos antisentido que actúan sobre atxn3
WO2025252669A1 (en) 2024-06-03 2025-12-11 Evotec International Gmbh Modified oligonucleotides for reducing atxn3 expression

Citations (1)

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JP2015511821A (ja) * 2012-03-12 2015-04-23 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブＲｏｃｈｅ Ｉｎｎｏｖａｔｉｏｎ Ｃｅｎｔｅｒ Ｃｏｐｅｎｈａｇｅｎ Ａ／Ｓ Ａｔｘｎ３発現の調節のための組成物および方法

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