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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JP2021523720A5 (https=
JP2021523720A (ja
JPWO2019222479A5 (https=
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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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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
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    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
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    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/53Methods for regulating/modulating their activity reducing unwanted side-effects

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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)

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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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JP2021523720A JP2021523720A (ja) 2021-09-09
JP2021523720A5 JP2021523720A5 (https=) 2022-05-24
JPWO2019222479A5 JPWO2019222479A5 (https=) 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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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 (https=)
EP (1) EP3802827A4 (https=)
JP (3) JP7384833B2 (https=)
AU (2) AU2019270152B2 (https=)
CA (1) CA3099930A1 (https=)
WO (1) WO2019222479A1 (https=)

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WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
WO2019217459A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
WO2021178607A1 (en) * 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
TW202214858A (zh) * 2020-08-04 2022-04-16 大陸商上海拓界生物醫藥科技有限公司 脫靶活性降低的修飾siRNA
WO2022174000A2 (en) 2021-02-12 2022-08-18 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
WO2022212231A2 (en) 2021-03-29 2022-10-06 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
EP4493570A2 (en) 2022-03-16 2025-01-22 Empirico Inc. Galnac compositions for improving sirna bioavailability
KR20240163743A (ko) 2022-03-28 2024-11-19 엠피리코 인크. 변형된 올리고뉴클레오티드
JP2025524135A (ja) * 2022-07-27 2025-07-25 イー-セラピューティクス・ピーエルシー 核酸化合物
JP2025532985A (ja) * 2022-09-30 2025-10-03 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 修飾二本鎖rna剤
WO2024217152A2 (zh) * 2023-04-21 2024-10-24 苏州时安生物技术有限公司 一种寡聚核酸及其制备方法和应用
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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AU2019270152A1 (en) 2020-12-10
US20250207131A1 (en) 2025-06-26
JP7684373B2 (ja) 2025-05-27
US12247203B2 (en) 2025-03-11
AU2025206406A1 (en) 2025-08-14
EP3802827A4 (en) 2022-08-03
US20210238595A1 (en) 2021-08-05
JP2025113272A (ja) 2025-08-01
US12275938B2 (en) 2025-04-15
JP2021523720A (ja) 2021-09-09
JP2024014905A (ja) 2024-02-01
EP3802827A1 (en) 2021-04-14
AU2019270152B2 (en) 2025-05-08
US20220290145A1 (en) 2022-09-15
WO2019222479A1 (en) 2019-11-21
CA3099930A1 (en) 2019-11-21

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