JP7536007B2 - 修飾二重鎖オリゴヌクレオチド - Google Patents

修飾二重鎖オリゴヌクレオチド Download PDF

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JP7536007B2
JP7536007B2 JP2021523902A JP2021523902A JP7536007B2 JP 7536007 B2 JP7536007 B2 JP 7536007B2 JP 2021523902 A JP2021523902 A JP 2021523902A JP 2021523902 A JP2021523902 A JP 2021523902A JP 7536007 B2 JP7536007 B2 JP 7536007B2
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JP2022506503A5 (enExample
JP2022506503A (ja
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フォスター，ドナルド
ケイ シュレーゲル，マーク
ブラウン，クリストファー
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Alnylam Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021523902A 2018-11-09 2019-11-05 修飾二重鎖オリゴヌクレオチド Active JP7536007B2 (ja)

Priority Applications (1)

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JP2024129696A JP2024149701A (ja) 2018-11-09 2024-08-06 修飾二重鎖オリゴヌクレオチド

Applications Claiming Priority (3)

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US201862758094P 2018-11-09 2018-11-09
US62/758,094 2018-11-09
PCT/US2019/059818 WO2020097044A1 (en) 2018-11-09 2019-11-05 Modified double stranded oligonucleotides

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JP2022506503A JP2022506503A (ja) 2022-01-17
JP2022506503A5 JP2022506503A5 (enExample) 2022-09-20
JP7536007B2 true JP7536007B2 (ja) 2024-08-19

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JP2024129696A Pending JP2024149701A (ja) 2018-11-09 2024-08-06 修飾二重鎖オリゴヌクレオチド

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US (1) US12600965B2 (enExample)
EP (1) EP3877524A4 (enExample)
JP (2) JP7536007B2 (enExample)
AU (2) AU2019376628B2 (enExample)
CA (1) CA3118537A1 (enExample)
WO (1) WO2020097044A1 (enExample)

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TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
WO2020097044A1 (en) * 2018-11-09 2020-05-14 Alnylam Pharmaceuticals, Inc. Modified double stranded oligonucleotides
BR112022009216A2 (pt) * 2019-11-13 2022-08-02 Alnylam Pharmaceuticals Inc Métodos e composições para tratar um distúrbio associado a angiotensinogênio (agt)
MX2023008469A (es) * 2021-01-22 2023-11-22 Alnylam Pharmaceuticals Inc Oligonucleotidos de doble hebra modificados.
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
TW202328449A (zh) 2021-09-24 2023-07-16 美商艾拉倫製藥公司 微管相關蛋白ＴＡＵ（ＭＡＰＴ）ｉＲＮＡ試劑組合物及其使用方法
US11879125B2 (en) 2022-03-16 2024-01-23 Empirico Inc. GalNAc compositions for improving siRNA bioavailability
AU2023245603A1 (en) 2022-03-28 2024-11-07 Empirico Inc. Modified oligonucleotides
TW202449152A (zh) * 2023-02-09 2024-12-16 美商艾拉倫製藥股份有限公司 Ｒｅｖｅｒｓｉｒ分子及其使用方法
WO2026046153A1 (zh) * 2024-08-26 2026-03-05 倍臻生物技术（苏州）有限公司 修饰的双链核糖核酸及其应用

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JP2017538679A (ja) 2014-11-10 2017-12-28 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｂ型肝炎ウイルス（ＨＢＶ）ｉＲＮＡ組成物及びその使用方法
JP2018512155A (ja) 2015-04-13 2018-05-17 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法

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EP3730618A1 (en) 2011-11-18 2020-10-28 Alnylam Pharmaceuticals, Inc. Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
KR102234623B1 (ko) 2013-05-22 2021-04-02 알닐람 파마슈티칼스 인코포레이티드 Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법
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JP2017538679A (ja) 2014-11-10 2017-12-28 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｂ型肝炎ウイルス（ＨＢＶ）ｉＲＮＡ組成物及びその使用方法
JP2017535552A (ja) 2014-11-17 2017-11-30 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用方法
JP2018512155A (ja) 2015-04-13 2018-05-17 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法

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AU2019376628A1 (en) 2021-06-03
WO2020097044A1 (en) 2020-05-14
JP2024149701A (ja) 2024-10-18
US12600965B2 (en) 2026-04-14
EP3877524A4 (en) 2023-10-11
AU2019376628B2 (en) 2025-08-07
EP3877524A1 (en) 2021-09-15
CA3118537A1 (en) 2020-05-14
JP2022506503A (ja) 2022-01-17
AU2025259857A1 (en) 2026-01-22
US20210388356A1 (en) 2021-12-16

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