KR102749200B1 - 고콜레스테롤혈증 및 관련 상태를 치료하기 위한 pcsk9 표적화 올리고뉴클레오티드 - Google Patents

고콜레스테롤혈증 및 관련 상태를 치료하기 위한 pcsk9 표적화 올리고뉴클레오티드 Download PDF

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KR102749200B1
KR102749200B1 KR1020207032782A KR20207032782A KR102749200B1 KR 102749200 B1 KR102749200 B1 KR 102749200B1 KR 1020207032782 A KR1020207032782 A KR 1020207032782A KR 20207032782 A KR20207032782 A KR 20207032782A KR 102749200 B1 KR102749200 B1 KR 102749200B1
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nucleic acid
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artificial sequence
synthetic nucleic
oligonucleotide
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헨릭 티. 두덱
지혜 박
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다이서나 파마수이티컬, 인크.
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    • 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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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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KR1020207032782A 2018-04-18 2019-04-01 고콜레스테롤혈증 및 관련 상태를 치료하기 위한 pcsk9 표적화 올리고뉴클레오티드 Active KR102749200B1 (ko)

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

Publications (2)

Publication Number Publication Date
KR20210005068A KR20210005068A (ko) 2021-01-13
KR102749200B1 true KR102749200B1 (ko) 2025-01-03

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US (2) US11566248B2 (https=)
EP (1) EP3765611A4 (https=)
JP (2) JP7357002B2 (https=)
KR (1) KR102749200B1 (https=)
CN (1) CN112368381A (https=)
AU (1) AU2019255460A1 (https=)
CA (1) CA3097585A1 (https=)
IL (1) IL278086A (https=)
MX (1) MX2020011006A (https=)
WO (1) WO2019204021A1 (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
US11566248B2 (en) 2018-04-18 2023-01-31 Dicerna Pharmaceuticals, Inc. PCSK9 targeting oligonucleotides for treating hypercholesterolemia and related conditions
WO2020226960A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
US20220290156A1 (en) * 2019-08-27 2022-09-15 Sanofi Compositions and methods for inhibiting pcsk9
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
CN120272480A (zh) * 2021-09-30 2025-07-08 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
CN118786216A (zh) * 2022-01-13 2024-10-15 石药集团中奇制药技术（石家庄）有限公司 一种抑制PCSK9基因表达的RNAi剂及其应用
JP2025520520A (ja) * 2022-06-14 2025-07-03 大睿生物 ＰＣＳＫ９遺伝子活性を調節する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
CN121866332A (zh) * 2023-06-21 2026-04-14 美国圣因生物股份有限公司 靶向前蛋白转化酶枯草杆菌蛋白酶kexin 9（pcsk9）的双链rna及其使用方法
CN119040325B (zh) * 2023-09-18 2025-07-18 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
CN117210468B (zh) * 2023-11-06 2024-02-20 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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WO2009114475A2 (en) * 2008-03-09 2009-09-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

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AU2007275365A1 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. RNA interference mediated inhibition of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
JP2010510807A (ja) * 2006-11-27 2010-04-08 アイシス ファーマシューティカルズ， インコーポレーテッド 高コレステロール血症を治療するための方法
WO2008109369A2 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tnf gene expression and uses thereof
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NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
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US11566248B2 (en) * 2018-04-18 2023-01-31 Dicerna Pharmaceuticals, Inc. PCSK9 targeting oligonucleotides for treating hypercholesterolemia and related conditions

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WO2009114475A2 (en) * 2008-03-09 2009-09-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

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CA3097585A1 (en) 2019-10-24
JP2023174697A (ja) 2023-12-08
KR20210005068A (ko) 2021-01-13
IL278086A (en) 2020-11-30
CN112368381A (zh) 2021-02-12
JP7357002B2 (ja) 2023-10-05
AU2019255460A1 (en) 2020-11-12
MX2020011006A (es) 2021-01-20
US20210238606A1 (en) 2021-08-05
US11566248B2 (en) 2023-01-31
EP3765611A4 (en) 2022-05-04
EP3765611A1 (en) 2021-01-20
WO2019204021A1 (en) 2019-10-24
US20230250435A1 (en) 2023-08-10
JP2021521796A (ja) 2021-08-30

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