MX2020011006A - Pcsk9 dirigido a oligonucleótidos para el tratamiento de la hipercolesterolemia y afecciones relacionadas. - Google Patents

Pcsk9 dirigido a oligonucleótidos para el tratamiento de la hipercolesterolemia y afecciones relacionadas.

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Publication number
MX2020011006A
MX2020011006A MX2020011006A MX2020011006A MX2020011006A MX 2020011006 A MX2020011006 A MX 2020011006A MX 2020011006 A MX2020011006 A MX 2020011006A MX 2020011006 A MX2020011006 A MX 2020011006A MX 2020011006 A MX2020011006 A MX 2020011006A
Authority
MX
Mexico
Prior art keywords
oligonucleotides
related conditions
treating hypercholesterolemia
targeting oligonucleotides
pcsk9 expression
Prior art date
Application number
MX2020011006A
Other languages
English (en)
Spanish (es)
Inventor
Jihye Park
Henryk T Dudek
Original Assignee
Dicerna Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of MX2020011006A publication Critical patent/MX2020011006A/es

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21061Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/533Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
MX2020011006A 2018-04-18 2019-04-01 Pcsk9 dirigido a oligonucleótidos para el tratamiento de la hipercolesterolemia y afecciones relacionadas. MX2020011006A (es)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
MX2020011006A true MX2020011006A (es) 2021-01-20

Family

ID=68239855

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020011006A MX2020011006A (es) 2018-04-18 2019-04-01 Pcsk9 dirigido a oligonucleótidos para el tratamiento de la hipercolesterolemia y afecciones relacionadas.

Country Status (10)

Country Link
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)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101484588B (zh) * 2006-05-11 2013-11-06 阿尔尼拉姆医药品有限公司 抑制pcsk9基因表达的组合物和方法
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)
CA2670563A1 (en) * 2006-11-27 2008-06-05 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
WO2009114475A2 (en) * 2008-03-09 2009-09-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
WO2010078536A1 (en) * 2009-01-05 2010-07-08 Rxi Pharmaceuticals Corporation Inhibition of pcsk9 through rnai
JP5894913B2 (ja) 2009-06-15 2016-03-30 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. Pcsk9遺伝子を標的とする、脂質で製剤化されたdsrna
WO2012058693A2 (en) 2010-10-29 2012-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of pcsk9 genes
DK3569711T3 (da) * 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
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

Also Published As

Publication number Publication date
WO2019204021A1 (en) 2019-10-24
CA3097585A1 (en) 2019-10-24
JP7357002B2 (ja) 2023-10-05
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
KR20210005068A (ko) 2021-01-13
US11566248B2 (en) 2023-01-31

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