JP7357002B2 - 高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド - Google Patents

高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド Download PDF

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JP7357002B2
JP7357002B2 JP2020557924A JP2020557924A JP7357002B2 JP 7357002 B2 JP7357002 B2 JP 7357002B2 JP 2020557924 A JP2020557924 A JP 2020557924A JP 2020557924 A JP2020557924 A JP 2020557924A JP 7357002 B2 JP7357002 B2 JP 7357002B2
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oligonucleotide
nucleotides
oligonucleotides
pcsk9
antisense strand
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JP2021521796A5 (enExample
JP2021521796A (ja
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ヘンリク ティー デュデック
ジヘ パク
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Dicerna Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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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
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2020557924A 2018-04-18 2019-04-01 高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド Active JP7357002B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023159941A JP2023174697A (ja) 2018-04-18 2023-09-25 高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862659693P 2018-04-18 2018-04-18
US62/659,693 2018-04-18
US201962820558P 2019-03-19 2019-03-19
US62/820,558 2019-03-19
PCT/US2019/025253 WO2019204021A1 (en) 2018-04-18 2019-04-01 Pcsk9 targeting oligonucleotides for treating hypercholesterolemia and related conditions

Related Child Applications (1)

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JP2023159941A Division JP2023174697A (ja) 2018-04-18 2023-09-25 高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド

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JP2021521796A JP2021521796A (ja) 2021-08-30
JP2021521796A5 JP2021521796A5 (enExample) 2022-04-07
JP7357002B2 true JP7357002B2 (ja) 2023-10-05

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JP2020557924A Active JP7357002B2 (ja) 2018-04-18 2019-04-01 高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド
JP2023159941A Withdrawn JP2023174697A (ja) 2018-04-18 2023-09-25 高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド

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US (2) US11566248B2 (enExample)
EP (1) EP3765611A4 (enExample)
JP (2) JP7357002B2 (enExample)
KR (1) KR102749200B1 (enExample)
CN (1) CN112368381A (enExample)
AU (1) AU2019255460A1 (enExample)
CA (1) CA3097585A1 (enExample)
IL (1) IL278086A (enExample)
MX (1) MX2020011006A (enExample)
WO (1) WO2019204021A1 (enExample)

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US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
JP7357002B2 (ja) * 2018-04-18 2023-10-05 ディセルナ ファーマシューティカルズ インコーポレイテッド 高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド
AU2020268798A1 (en) * 2019-05-03 2021-11-04 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
CN115176011B (zh) * 2019-08-27 2025-11-11 赛诺菲 用于抑制pcsk9的组合物和方法
WO2022089486A1 (zh) * 2020-10-28 2022-05-05 江苏柯菲平医药股份有限公司 抑制PCSK9基因表达的siRNA及其修饰物与应用
CN114634929A (zh) * 2020-12-16 2022-06-17 施能康生物科技有限公司 靶向前蛋白转化酶枯草杆菌转化酶的核酸及其用途
WO2023017004A1 (en) * 2021-08-09 2023-02-16 Cargene Therapeutics Pte. Ltd. Inhibitory nucleic acids for pcsk9
CN115992138A (zh) * 2021-09-30 2023-04-21 北京安龙生物医药有限公司 用于治疗与pcsk9相关疾病的靶向寡核苷酸
US20250346902A1 (en) * 2022-01-13 2025-11-13 Cspc Zhongqi Pharmaceutical Technology (Shijiazhuang) Co., Ltd. Rnai agent inhibiting pcsk9 gene expression and application thereof
CN119487191A (zh) * 2022-06-14 2025-02-18 大睿生物医药科技(上海)有限公司 调控PCSK9基因活性的siRNA分子
CN118086311B (zh) * 2023-05-25 2024-08-09 苏州时安生物技术有限公司 抑制PCSK9基因表达的siRNA、其缀合物和药物组合物及用途
WO2024263915A2 (en) * 2023-06-21 2024-12-26 Sanegene Bio Usa Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
CN121006363A (zh) * 2023-09-18 2025-11-25 广州必贝特医药股份有限公司 用于同时抑制两个靶基因表达的siRNA、药物及其应用
CN117106781B (zh) * 2023-10-16 2024-03-22 深圳市茵冠生物科技有限公司 修饰的核酸及其产品和应用
WO2025096355A1 (en) * 2023-10-29 2025-05-08 Corsera Llc Improved sirna for silencing expression of pcsk9 and uses thereof
CN118147134B (zh) * 2023-11-06 2025-02-25 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用
CN117384907B (zh) * 2023-12-11 2024-03-29 上海鼎新基因科技有限公司 抑制PCSK9表达的siRNA分子及其应用
CN118109463B (zh) * 2024-01-31 2025-02-21 北京悦康科创医药科技股份有限公司 靶向PCSK9基因的siRNA及其应用
WO2026026812A1 (zh) * 2024-07-29 2026-02-05 成都先衍生物技术有限公司 一种具有双靶点基因抑制作用的核酸缀合物及其应用
WO2026026811A1 (zh) * 2024-07-29 2026-02-05 成都先衍生物技术有限公司 一种具有双靶点基因抑制作用的核酸缀合物及其应用

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JP2009536827A (ja) 2006-05-11 2009-10-22 アルニラム ファーマシューティカルズ， インコーポレイテッド Ｐｃｓｋ９遺伝子の発現を阻害するための組成物および方法
JP2010503382A (ja) 2006-07-17 2010-02-04 サーナ・セラピューティクス・インコーポレイテッド 低分子干渉核酸（ｓｉＮＡ）を用いたプロタンパク質転換酵素サブチリシンケクシン９（ＰＣＳＫ９）遺伝子発現のＲＮＡ干渉媒介性阻害
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
JP2013545736A (ja) 2010-10-29 2013-12-26 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 Ｐｃｓｋ９遺伝子の阻害のための組成物および方法

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JP7357002B2 (ja) 2018-04-18 2023-10-05 ディセルナ ファーマシューティカルズ インコーポレイテッド 高コレステロール血症及び関連状態を治療するための、ｐｃｓｋ９を標的とするオリゴヌクレオチド

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JP2009536827A (ja) 2006-05-11 2009-10-22 アルニラム ファーマシューティカルズ， インコーポレイテッド Ｐｃｓｋ９遺伝子の発現を阻害するための組成物および方法
JP2010503382A (ja) 2006-07-17 2010-02-04 サーナ・セラピューティクス・インコーポレイテッド 低分子干渉核酸（ｓｉＮＡ）を用いたプロタンパク質転換酵素サブチリシンケクシン９（ＰＣＳＫ９）遺伝子発現のＲＮＡ干渉媒介性阻害
WO2010078536A1 (en) 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
JP2013545736A (ja) 2010-10-29 2013-12-26 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 Ｐｃｓｋ９遺伝子の阻害のための組成物および方法

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WO2019204021A1 (en) 2019-10-24
CA3097585A1 (en) 2019-10-24
EP3765611A4 (en) 2022-05-04
CN112368381A (zh) 2021-02-12
KR102749200B1 (ko) 2025-01-03
JP2021521796A (ja) 2021-08-30
US20230250435A1 (en) 2023-08-10
JP2023174697A (ja) 2023-12-08
US20210238606A1 (en) 2021-08-05
AU2019255460A1 (en) 2020-11-12
EP3765611A1 (en) 2021-01-20
IL278086A (en) 2020-11-30
MX2020011006A (es) 2021-01-20
KR20210005068A (ko) 2021-01-13
US11566248B2 (en) 2023-01-31

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