JP7384833B2 - オフターゲット効果が低下した修飾ｒｎａ剤 - Google Patents

オフターゲット効果が低下した修飾ｒｎａ剤 Download PDF

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JP7384833B2
JP7384833B2 JP2020564156A JP2020564156A JP7384833B2 JP 7384833 B2 JP7384833 B2 JP 7384833B2 JP 2020564156 A JP2020564156 A JP 2020564156A JP 2020564156 A JP2020564156 A JP 2020564156A JP 7384833 B2 JP7384833 B2 JP 7384833B2
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muna
gna
dsrna
antisense strand
strand
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JP2021523720A (ja
JP2021523720A5 (enExample
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成夫 松田
マーク・ケー・シュレーゲル
マジャ・ジャナス
ヴァサント・アール・ジャダヴ
マーティン・メイヤー
クラウス・チャリシー
ムティアー・マノハラン
カラントッタティル・ジー・ラジーヴ
ジャヤプラカシュ・ケー・ナイール
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アルニラム・ファーマシューティカルズ・インコーポレーテッド
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    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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JP2020564156A 2018-05-16 2019-05-16 オフターゲット効果が低下した修飾ｒｎａ剤 Active JP7384833B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023189998A JP7684373B2 (ja) 2018-05-16 2023-11-07 オフターゲット効果が低下した修飾ｒｎａ剤
JP2025076285A JP2025113272A (ja) 2018-05-16 2025-05-01 オフターゲット効果が低下した修飾ｒｎａ剤

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862672405P 2018-05-16 2018-05-16
US62/672,405 2018-05-16
US201862719291P 2018-08-17 2018-08-17
US62/719,291 2018-08-17
PCT/US2019/032633 WO2019222479A1 (en) 2018-05-16 2019-05-16 Modified rna agents with reduced off-target effect

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JP2023189998A Division JP7684373B2 (ja) 2018-05-16 2023-11-07 オフターゲット効果が低下した修飾ｒｎａ剤

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JP2021523720A JP2021523720A (ja) 2021-09-09
JP2021523720A5 JP2021523720A5 (enExample) 2022-05-24
JP7384833B2 true JP7384833B2 (ja) 2023-11-21

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JP2020564156A Active JP7384833B2 (ja) 2018-05-16 2019-05-16 オフターゲット効果が低下した修飾ｒｎａ剤
JP2023189998A Active JP7684373B2 (ja) 2018-05-16 2023-11-07 オフターゲット効果が低下した修飾ｒｎａ剤
JP2025076285A Pending JP2025113272A (ja) 2018-05-16 2025-05-01 オフターゲット効果が低下した修飾ｒｎａ剤

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JP2023189998A Active JP7684373B2 (ja) 2018-05-16 2023-11-07 オフターゲット効果が低下した修飾ｒｎａ剤
JP2025076285A Pending JP2025113272A (ja) 2018-05-16 2025-05-01 オフターゲット効果が低下した修飾ｒｎａ剤

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US (3) US12247203B2 (enExample)
EP (1) EP3802827A4 (enExample)
JP (3) JP7384833B2 (enExample)
AU (2) AU2019270152B2 (enExample)
CA (1) CA3099930A1 (enExample)
WO (1) WO2019222479A1 (enExample)

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US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
MX2020011570A (es) 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
EP4114947A1 (en) * 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
KR20230043195A (ko) * 2020-08-04 2023-03-30 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 오프-타겟 활성이 감소된 변형된 siRNA
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
CN113073098B (zh) * 2021-02-20 2024-08-20 北京理工大学 抑制BACE1基因表达的siRNA修饰物及其应用
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
AU2023313030A1 (en) * 2022-07-27 2025-01-09 E-Therapeutics Plc Nucleic acid compounds
WO2024073732A1 (en) * 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
EP4700125A2 (en) * 2023-04-21 2026-02-25 Suzhou Siran Biotechnology Co., Ltd. Oligomeric nucleic acid, and preparation method therefore and use thereof
WO2025064821A2 (en) 2023-09-21 2025-03-27 Ionis Pharmaceuticals, Inc. Compounds and methods for inhibiting lpa
US12454693B1 (en) * 2024-09-27 2025-10-28 Dexcel Pharma Technologies Ltd. Nucleic acids for inhibiting expression of transferrin receptor 2

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WO2015106128A2 (en) 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
JP2017525705A (ja) 2014-08-20 2017-09-07 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． 修飾二本鎖ｒｎａ剤

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JP2025113272A (ja) 2025-08-01
EP3802827A1 (en) 2021-04-14
JP2021523720A (ja) 2021-09-09
US12247203B2 (en) 2025-03-11
US20250207131A1 (en) 2025-06-26
US20220290145A1 (en) 2022-09-15
US12275938B2 (en) 2025-04-15
AU2025206406A1 (en) 2025-08-14
JP7684373B2 (ja) 2025-05-27
JP2024014905A (ja) 2024-02-01
WO2019222479A1 (en) 2019-11-21
CA3099930A1 (en) 2019-11-21
AU2019270152A1 (en) 2020-12-10
EP3802827A4 (en) 2022-08-03
AU2019270152B2 (en) 2025-05-08
US20210238595A1 (en) 2021-08-05

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