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
dna
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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    • C12N15/09—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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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • 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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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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
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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
    • C—CHEMISTRY; METALLURGY
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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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  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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 (OSRAM)
EP (1) EP3765611A4 (OSRAM)
JP (2) JP7357002B2 (OSRAM)
KR (1) KR102749200B1 (OSRAM)
CN (1) CN112368381A (OSRAM)
AU (1) AU2019255460A1 (OSRAM)
CA (1) CA3097585A1 (OSRAM)
IL (1) IL278086A (OSRAM)
MX (1) MX2020011006A (OSRAM)
WO (1) WO2019204021A1 (OSRAM)

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WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
MX2020011006A (es) * 2018-04-18 2021-01-20 Dicerna Pharmaceuticals Inc Pcsk9 dirigido a oligonucleótidos para el tratamiento de la hipercolesterolemia y afecciones relacionadas.
AU2020268798A1 (en) 2019-05-03 2021-11-04 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
WO2021037972A1 (en) * 2019-08-27 2021-03-04 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
WO2023051822A1 (zh) * 2021-09-30 2023-04-06 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
EP4464781A1 (en) * 2022-01-13 2024-11-20 CSPC Zhongqi Pharmaceutical Technology (Shijiazhuang) Co., Ltd. Rnai agent inhibiting pcsk9 gene expression and application thereof
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
EP4731764A2 (en) * 2023-06-21 2026-04-29 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
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
WO2026092650A1 (zh) * 2024-11-01 2026-05-07 迈威(上海)生物科技股份有限公司 一种抑制LPA基因表达的RNAi制剂及其应用

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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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CA2658183A1 (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)
EP2455471A3 (en) 2006-11-27 2012-09-12 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
WO2008109369A2 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tnf gene expression and uses thereof
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
AU2010260148A1 (en) * 2009-06-15 2012-02-02 Alnylam Pharmaceuticals, Inc. Lipid formulated dsRNA targeting the PCSK9 gene
CA2816321A1 (en) 2010-10-29 2012-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of pcsk9 genes
MX2020011006A (es) 2018-04-18 2021-01-20 Dicerna Pharmaceuticals Inc Pcsk9 dirigido a oligonucleótidos para el tratamiento de la hipercolesterolemia y afecciones relacionadas.

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

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