CA3098144A1 - Compounds and methods for reducing atxn3 expression - Google Patents

Compounds and methods for reducing atxn3 expression Download PDF

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Publication number
CA3098144A1
CA3098144A1 CA3098144A CA3098144A CA3098144A1 CA 3098144 A1 CA3098144 A1 CA 3098144A1 CA 3098144 A CA3098144 A CA 3098144A CA 3098144 A CA3098144 A CA 3098144A CA 3098144 A1 CA3098144 A1 CA 3098144A1
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modified
certain embodiments
compound
modified oligonucleotide
seq
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CA3098144A
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French (fr)
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Susan M. Freier
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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
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    • C12N15/09—Recombinant DNA-technology
    • 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
    • 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
    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Hospice & Palliative Care (AREA)
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  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3098144A 2018-05-09 2019-05-09 Compounds and methods for reducing atxn3 expression Pending CA3098144A1 (en)

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 (1)

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CA3098144A1 true CA3098144A1 (en) 2019-11-14

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CA3098144A Pending CA3098144A1 (en) 2018-05-09 2019-05-09 Compounds and methods for reducing atxn3 expression

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US (4) US11434488B2 (enExample)
EP (2) EP3799602A4 (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
WO2019140236A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
MY203356A (en) 2018-01-12 2024-06-25 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
JP2021522800A (ja) 2018-05-09 2021-09-02 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ａｔｘｎ３発現を低減するための化合物及び方法
EP3927827A4 (en) * 2019-02-22 2023-04-26 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
WO2020245233A1 (en) 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
EP4061945A1 (en) * 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
AU2021225957A1 (en) * 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
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
EP4162047A1 (en) * 2020-06-09 2023-04-12 Roche Innovation Center Copenhagen A/S Guanosine analogues for use in therapeutic polynucleotides
WO2022117745A1 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
TW202237843A (zh) 2020-12-03 2022-10-01 瑞士商赫孚孟拉羅股份公司 靶向ａｔｘｎ３之反義寡核苷酸
WO2025252669A1 (en) 2024-06-03 2025-12-11 Evotec International Gmbh Modified oligonucleotides for reducing atxn3 expression

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