PE20211282A1 - Acidos nucleicos para inhibir la expresion de lpa en una celula - Google Patents

Acidos nucleicos para inhibir la expresion de lpa en una celula

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Publication number
PE20211282A1
PE20211282A1 PE2021000650A PE2021000650A PE20211282A1 PE 20211282 A1 PE20211282 A1 PE 20211282A1 PE 2021000650 A PE2021000650 A PE 2021000650A PE 2021000650 A PE2021000650 A PE 2021000650A PE 20211282 A1 PE20211282 A1 PE 20211282A1
Authority
PE
Peru
Prior art keywords
inhibit
expression
cell
lpa
strand
Prior art date
Application number
PE2021000650A
Other languages
English (en)
Inventor
David Anthony Rider
Lucas Bethge
Christian Frauendorf
Adrien Weingaertner
Judith Hauptmann
Sibylle Dames
Steffen Schubert
Stephan Tenbaum
Original Assignee
Silence Therapeutics Gmbh
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
Priority claimed from PCT/EP2018/081106 external-priority patent/WO2019092283A1/en
Application filed by Silence Therapeutics Gmbh filed Critical Silence Therapeutics Gmbh
Publication of PE20211282A1 publication Critical patent/PE20211282A1/es

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7084—Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/532—Closed or circular
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cardiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente invencion se refiere a un acido nucleico para inhibir o interferir en la expresion del gen LPA en una celula, que comprende al menos una region de duplex que comprende al menos una parte de una primera cadena y al menos una parte de una segunda cadena que es al menos parcialmente complementaria a la primera cadena, en donde dicha primera cadena es al menos parcialmente complementaria a al menos una parte de ARN transcrito a partir del gen LPA, en donde dicha primera cadena comprende una secuencia de nucleotidos que se selecciona de SEQ ID NO: 9, 5, 1, 3, 7, 11, 13, 15, entre otras. Tambien provee una composicion farmaceutica que lo comprende; siendo util en el tratamiento, prevencion o reduccion del riesgo de sufrir enfermedad cardiovascular tal como cardiopatia coronaria o estenosis de la aorta o accidente cardiovascular o cualquier otro trastorno, patologia o sindrome relacionado con niveles elevados de LPA
PE2021000650A 2018-11-13 2019-11-13 Acidos nucleicos para inhibir la expresion de lpa en una celula PE20211282A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
PCT/EP2018/081106 WO2019092283A1 (en) 2017-11-13 2018-11-13 Nucleic acids for inhibiting expression of lpa in a cell
EP19174466 2019-05-14
PCT/EP2019/081158 WO2020099476A1 (en) 2018-11-13 2019-11-13 Nucleic acids for inhibiting expression of lpa in a cell

Publications (1)

Publication Number Publication Date
PE20211282A1 true PE20211282A1 (es) 2021-07-19

Family

ID=66554180

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021000650A PE20211282A1 (es) 2018-11-13 2019-11-13 Acidos nucleicos para inhibir la expresion de lpa en una celula

Country Status (29)

Country Link
US (2) US20220002722A1 (es)
EP (2) EP3880818B1 (es)
JP (1) JP7245328B2 (es)
KR (1) KR102553386B1 (es)
CN (1) CN113227372A (es)
AU (1) AU2019380628A1 (es)
BR (1) BR112021009213A2 (es)
CA (1) CA3119239A1 (es)
CL (1) CL2021001225A1 (es)
CO (1) CO2021007372A2 (es)
CR (1) CR20210301A (es)
DK (1) DK3880818T3 (es)
EA (1) EA202190895A1 (es)
ES (1) ES2932295T3 (es)
FI (1) FI3880818T3 (es)
HR (1) HRP20221358T1 (es)
HU (1) HUE060703T2 (es)
IL (1) IL283159A (es)
LT (1) LT3880818T (es)
MA (1) MA58208B1 (es)
MX (1) MX2021005590A (es)
PE (1) PE20211282A1 (es)
PH (1) PH12021550801A1 (es)
PL (1) PL3880818T3 (es)
PT (1) PT3880818T (es)
RS (1) RS63778B1 (es)
SG (1) SG11202103882QA (es)
SI (1) SI3880818T1 (es)
WO (1) WO2020099476A1 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3607069T3 (pl) * 2017-04-05 2023-03-06 Silence Therapeutics Gmbh Produkty i kompozycje
WO2019092283A1 (en) 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of lpa in a cell
EP3880818B1 (en) 2018-11-13 2022-09-28 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of lpa in a cell
EP4229201A1 (en) 2020-10-16 2023-08-23 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
CA3229020A1 (en) * 2021-09-14 2023-03-23 Stella PELENGARIS Treatment of cardiovascular disease
WO2023152194A1 (en) 2022-02-09 2023-08-17 Silence Therapeutics Gmbh Therapeutic inhibition of lpa expression
CN114703184A (zh) * 2022-03-11 2022-07-05 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
WO2024013334A1 (en) * 2022-07-15 2024-01-18 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of agt in a cell
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 (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
US20060160759A1 (en) 2002-09-28 2006-07-20 Jianzhu Chen Influenza therapeutic
CN101278059A (zh) * 2005-07-28 2008-10-01 肿瘤疗法科学股份有限公司 诊断和治疗肾细胞癌的方法
US20110110886A1 (en) 2008-06-13 2011-05-12 Yale University Small molecule inhibitors of autotaxin and methods of use
WO2016149020A1 (en) 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
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
PL3607069T3 (pl) 2017-04-05 2023-03-06 Silence Therapeutics Gmbh Produkty i kompozycje
EP3735461A1 (en) * 2017-11-13 2020-11-11 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of aldh2 in a cell
WO2019092283A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of lpa in a cell
EP3880818B1 (en) 2018-11-13 2022-09-28 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of lpa in a cell

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Publication number Publication date
US20220002722A1 (en) 2022-01-06
IL283159A (en) 2021-06-30
JP2022507282A (ja) 2022-01-18
US11499153B2 (en) 2022-11-15
JP7245328B2 (ja) 2023-03-23
MA58208B1 (fr) 2022-12-30
EP4219712A2 (en) 2023-08-02
KR20210095878A (ko) 2021-08-03
CA3119239A1 (en) 2020-05-22
HUE060703T2 (hu) 2023-04-28
SI3880818T1 (sl) 2023-01-31
CO2021007372A2 (es) 2021-07-09
EP3880818B1 (en) 2022-09-28
RS63778B1 (sr) 2022-12-30
LT3880818T (lt) 2022-12-27
CR20210301A (es) 2021-09-16
PL3880818T3 (pl) 2023-01-23
WO2020099476A1 (en) 2020-05-22
HRP20221358T1 (hr) 2022-12-23
DK3880818T3 (da) 2022-12-05
FI3880818T3 (fi) 2022-12-15
US20220170016A1 (en) 2022-06-02
SG11202103882QA (en) 2021-05-28
CL2021001225A1 (es) 2021-12-24
EP3880818A1 (en) 2021-09-22
ES2932295T3 (es) 2023-01-17
PT3880818T (pt) 2022-12-06
MX2021005590A (es) 2023-01-24
BR112021009213A2 (pt) 2021-08-10
EP4219712A3 (en) 2023-11-22
KR102553386B1 (ko) 2023-07-07
PH12021550801A1 (en) 2021-10-04
EA202190895A1 (ru) 2021-08-10
AU2019380628A1 (en) 2021-05-20
CN113227372A (zh) 2021-08-06

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