DK3710586T3 - Nukleinsyrer til inhibering af ekspression af LPA i en celle - Google Patents

Nukleinsyrer til inhibering af ekspression af LPA i en celle Download PDF

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Publication number
DK3710586T3
DK3710586T3 DK18803644.6T DK18803644T DK3710586T3 DK 3710586 T3 DK3710586 T3 DK 3710586T3 DK 18803644 T DK18803644 T DK 18803644T DK 3710586 T3 DK3710586 T3 DK 3710586T3
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Denmark
Prior art keywords
lpa
cell
nucleic acids
inhibiting expression
inhibiting
Prior art date
Application number
DK18803644.6T
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English (en)
Inventor
Sibylle Dames
Stephan Tenbaum
Adrien Weingärtner
Lucas Bethge
David Anthony Rider
Steffen Schubert
Christian Frauendorf
Judith Hauptmann
Original Assignee
Silence Therapeutics Gmbh
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Priority claimed from EP17201449.0A external-priority patent/EP3483270A1/en
Application filed by Silence Therapeutics Gmbh filed Critical Silence Therapeutics Gmbh
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Publication of DK3710586T3 publication Critical patent/DK3710586T3/da

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • 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 N.A.
    • 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
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
DK18803644.6T 2017-11-13 2018-11-13 Nukleinsyrer til inhibering af ekspression af LPA i en celle DK3710586T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP17201449.0A EP3483270A1 (en) 2017-11-13 2017-11-13 Products and compositions
EP18179175.7A EP3483271A3 (en) 2017-11-13 2018-06-21 Products and compositions
GB201815915 2018-09-28
PCT/EP2018/081106 WO2019092283A1 (en) 2017-11-13 2018-11-13 Nucleic acids for inhibiting expression of lpa in a cell

Publications (1)

Publication Number Publication Date
DK3710586T3 true DK3710586T3 (da) 2023-01-23

Family

ID=66439085

Family Applications (1)

Application Number Title Priority Date Filing Date
DK18803644.6T DK3710586T3 (da) 2017-11-13 2018-11-13 Nukleinsyrer til inhibering af ekspression af LPA i en celle

Country Status (22)

Country Link
US (2) US11319537B2 (da)
EP (2) EP3710586B1 (da)
JP (1) JP7245254B2 (da)
KR (1) KR102553382B1 (da)
CN (1) CN111465694A (da)
AU (1) AU2018363840B2 (da)
BR (1) BR112020009152A2 (da)
CA (1) CA3081905C (da)
DK (1) DK3710586T3 (da)
ES (1) ES2938193T3 (da)
FI (1) FI3710586T3 (da)
HR (1) HRP20230127T1 (da)
HU (1) HUE061265T2 (da)
IL (1) IL274503B1 (da)
LT (1) LT3710586T (da)
MX (1) MX2020004897A (da)
PL (1) PL3710586T3 (da)
RS (1) RS63955B1 (da)
SG (1) SG11202003230UA (da)
SI (1) SI3710586T1 (da)
WO (1) WO2019092283A1 (da)
ZA (1) ZA202003371B (da)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2016370962B2 (en) 2015-12-16 2020-09-24 6K Inc. Spheroidal dehydrogenated metals and metal alloy particles
CA3057565A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
KR20200109311A (ko) 2018-01-16 2020-09-22 다이서나 파마수이티컬, 인크. Aldh2 발현을 억제하기 위한 조성물 및 방법
KR102553386B1 (ko) 2018-11-13 2023-07-07 사일런스 테라퓨틱스 게엠베하 세포에서 lpa의 발현을 저해하기 위한 핵산
CN113748208A (zh) * 2019-04-04 2021-12-03 迪克纳制药公司 用于抑制中枢神经系统中的基因表达的组合物和方法
WO2020223358A1 (en) 2019-04-30 2020-11-05 6K Inc. Mechanically alloyed powder feedstock
WO2021118762A1 (en) 2019-11-18 2021-06-17 6K Inc. Unique feedstocks for spherical powders and methods of manufacturing
WO2021119034A1 (en) * 2019-12-09 2021-06-17 Amgen Inc. RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
JP2023532457A (ja) 2020-06-25 2023-07-28 シックスケー インコーポレイテッド 微細複合合金構造体
TW202220673A (zh) 2020-08-05 2022-06-01 美商戴瑟納製藥股份有限公司 抑制lpa表現之組合物及方法
CN116547068A (zh) 2020-09-24 2023-08-04 6K有限公司 用于启动等离子体的系统、装置及方法
EP4229201A1 (en) 2020-10-16 2023-08-23 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
KR20230095080A (ko) 2020-10-30 2023-06-28 6케이 인크. 구상화 금속 분말을 합성하는 시스템 및 방법
US20230405040A1 (en) * 2020-11-05 2023-12-21 Amgen Inc. METHODS FOR TREATING ATHEROSCLEROTIC CARDIOVASCULAR DISEASE WITH LPA-TARGETED RNAi CONSTRUCTS
EP4237561A1 (en) 2020-12-23 2023-09-06 Argonaute Rna Limited Treatment of cardiovascular disease
CA3229020A1 (en) 2021-09-14 2023-03-23 Stella Khan Treatment of cardiovascular disease
WO2023091985A1 (en) * 2021-11-18 2023-05-25 Dtx Pharma, Inc. Compounds targeting pmp22 for the treatment of charcot-marie-tooth disease
CA3240273A1 (en) * 2021-11-24 2023-06-01 Adarx Pharmaceuticals, Inc. Complement factor b-modulating compositions and methods of use thereof
CN114703184B (zh) * 2022-03-11 2024-06-18 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
WO2023220744A2 (en) 2022-05-13 2023-11-16 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
CN115851723B (zh) * 2022-10-24 2023-10-03 厦门甘宝利生物医药有限公司 一种抑制lpa基因表达的rna抑制剂及其应用

Family Cites Families (11)

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NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US7259150B2 (en) * 2001-08-07 2007-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein (a) expression
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WO2009151644A2 (en) * 2008-06-13 2009-12-17 Yale University Small molecule inhibitors of autotaxin methods of use
JP6139671B2 (ja) * 2012-05-22 2017-05-31 オリックス ファーマシューティカルズ インコーポレイテッドOlix Pharmaceuticals Inc. 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途
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JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
EP3228326A1 (en) * 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
CA3057565A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
CA3118327A1 (en) 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of aldh2 in a cell
KR102553386B1 (ko) 2018-11-13 2023-07-07 사일런스 테라퓨틱스 게엠베하 세포에서 lpa의 발현을 저해하기 위한 핵산

Also Published As

Publication number Publication date
EP3710586A1 (en) 2020-09-23
JP2021502120A (ja) 2021-01-28
LT3710586T (lt) 2023-02-27
AU2018363840B2 (en) 2022-09-29
KR20200088392A (ko) 2020-07-22
ZA202003371B (en) 2023-08-30
US20210123048A1 (en) 2021-04-29
PL3710586T3 (pl) 2023-03-20
JP7245254B2 (ja) 2023-03-23
HUE061265T2 (hu) 2023-06-28
EP4219716A2 (en) 2023-08-02
IL274503A (en) 2020-06-30
CA3081905C (en) 2023-10-03
EP3710586B1 (en) 2022-11-23
US20220290144A1 (en) 2022-09-15
HRP20230127T1 (hr) 2023-03-31
KR102553382B1 (ko) 2023-07-07
AU2018363840A1 (en) 2020-04-16
EP4219716A3 (en) 2024-03-27
US11319537B2 (en) 2022-05-03
IL274503B1 (en) 2024-06-01
CA3081905A1 (en) 2019-05-16
RS63955B1 (sr) 2023-02-28
MX2020004897A (es) 2020-10-05
SI3710586T1 (sl) 2023-03-31
WO2019092283A1 (en) 2019-05-16
ES2938193T3 (es) 2023-04-05
CN111465694A (zh) 2020-07-28
FI3710586T3 (fi) 2023-03-02
BR112020009152A2 (pt) 2020-10-27
SG11202003230UA (en) 2020-05-28

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