JP2024522147A - Ｍｓｈ３活性と関連するヌクレオチドリピート伸長障害の治療方法 - Google Patents

Ｍｓｈ３活性と関連するヌクレオチドリピート伸長障害の治療方法 Download PDF

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JP2024522147A
JP2024522147A JP2023574553A JP2023574553A JP2024522147A JP 2024522147 A JP2024522147 A JP 2024522147A JP 2023574553 A JP2023574553 A JP 2023574553A JP 2023574553 A JP2023574553 A JP 2023574553A JP 2024522147 A JP2024522147 A JP 2024522147A
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oligonucleotide
pharma
acceptable salt
ceutically acceptable
seq
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JP2024522147A5 (https=
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アンソニー バーミンガム、ネッサン
アール． ベッテンコート、ブライアン
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Takeda Pharmaceuticals USA Inc
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Takeda Pharmaceuticals USA Inc
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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1096—Processes for the isolation, preparation or purification of DNA or RNA cDNA Synthesis; Subtracted cDNA library construction, e.g. RT, RT-PCR
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023574553A 2021-06-04 2022-06-03 Ｍｓｈ３活性と関連するヌクレオチドリピート伸長障害の治療方法 Pending JP2024522147A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163196896P 2021-06-04 2021-06-04
US63/196,896 2021-06-04
PCT/US2022/032128 WO2022256631A2 (en) 2021-06-04 2022-06-03 Methods for the treatment of nucleotide repeat expansion disorders associated with msh3 activity

Publications (2)

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JP2024522147A true JP2024522147A (ja) 2024-06-11
JP2024522147A5 JP2024522147A5 (https=) 2025-06-11

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JP2023574553A Pending JP2024522147A (ja) 2021-06-04 2022-06-03 Ｍｓｈ３活性と関連するヌクレオチドリピート伸長障害の治療方法

Country Status (4)

Country Link
US (1) US20240263179A1 (https=)
EP (1) EP4347837A4 (https=)
JP (1) JP2024522147A (https=)
WO (1) WO2022256631A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7714162B6 (ja) * 2018-12-03 2025-09-08 タケダ ファーマシューティカルズ ユーエスエー インコーポレイテッド Ｍｓｈ３活性に関連するトリヌクレオチドリピート伸長障害の処置のための方法
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA201492116A1 (ru) * 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
WO2016172795A1 (en) * 2015-04-27 2016-11-03 Adaerata Limited Partnership Modulating uracil-dna glycosylase and uses thereof
JOP20190197A1 (ar) * 2017-02-24 2019-08-22 Bayer Pharma AG مثبط كيناز ايه تي آر للاستخدام في طريقة لعلاج مرض فرط التكاثر
JP7714162B6 (ja) * 2018-12-03 2025-09-08 タケダ ファーマシューティカルズ ユーエスエー インコーポレイテッド Ｍｓｈ３活性に関連するトリヌクレオチドリピート伸長障害の処置のための方法

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US20240263179A1 (en) 2024-08-08
EP4347837A4 (en) 2025-08-27
WO2022256631A3 (en) 2023-01-12
WO2022256631A2 (en) 2022-12-08
EP4347837A2 (en) 2024-04-10

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