CL2021003169A1 - Construcciones de arni para inhibir la expresión de scap y métodos de uso de las mismas - Google Patents

Construcciones de arni para inhibir la expresión de scap y métodos de uso de las mismas

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Publication number
CL2021003169A1
CL2021003169A1 CL2021003169A CL2021003169A CL2021003169A1 CL 2021003169 A1 CL2021003169 A1 CL 2021003169A1 CL 2021003169 A CL2021003169 A CL 2021003169A CL 2021003169 A CL2021003169 A CL 2021003169A CL 2021003169 A1 CL2021003169 A1 CL 2021003169A1
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CL
Chile
Prior art keywords
methods
constructs
arni
scap expression
expression
Prior art date
Application number
CL2021003169A
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English (en)
Inventor
Daniel C H Lin
Justin K Murray
Michael Ollmann
Oliver Homann
Bradley J Herberich
Amrita Das
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Amgen Inc
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Publication date
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Publication of CL2021003169A1 publication Critical patent/CL2021003169A1/es

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    • 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
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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Abstract

La presente invención se refiere a construcciones de ARNi para reducir la expresión del gen SCAP. También se describen métodos de uso de tales construcciones de ARNi para tratar o prevenir una hepatopatía, la esteatosis hepática no alcohólica (EHNA).
CL2021003169A 2019-05-30 2021-11-29 Construcciones de arni para inhibir la expresión de scap y métodos de uso de las mismas CL2021003169A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962854433P 2019-05-30 2019-05-30

Publications (1)

Publication Number Publication Date
CL2021003169A1 true CL2021003169A1 (es) 2023-01-20

Family

ID=71833431

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2021003169A CL2021003169A1 (es) 2019-05-30 2021-11-29 Construcciones de arni para inhibir la expresión de scap y métodos de uso de las mismas

Country Status (17)

Country Link
US (1) US20220307022A1 (es)
EP (1) EP3976786A2 (es)
JP (1) JP2022534402A (es)
KR (1) KR20220016138A (es)
CN (1) CN113924368A (es)
AR (1) AR119061A1 (es)
AU (1) AU2020284254A1 (es)
BR (1) BR112021024080A2 (es)
CA (1) CA3141902A1 (es)
CL (1) CL2021003169A1 (es)
EA (1) EA202193296A1 (es)
IL (1) IL288398A (es)
MX (1) MX2021014465A (es)
SG (1) SG11202113112WA (es)
TW (1) TW202111124A (es)
UY (1) UY38733A (es)
WO (1) WO2020243702A2 (es)

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WO2024023262A2 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc Nucleic acid compounds
WO2024104416A1 (zh) * 2022-11-17 2024-05-23 北京福元医药股份有限公司 抑制SCAP基因表达的siRNA、其缀合物和药物组合物及用途

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Publication number Publication date
CA3141902A1 (en) 2020-12-03
EP3976786A2 (en) 2022-04-06
AU2020284254A1 (en) 2021-12-23
IL288398A (en) 2022-01-01
SG11202113112WA (en) 2021-12-30
UY38733A (es) 2020-11-30
EA202193296A1 (ru) 2022-03-10
WO2020243702A3 (en) 2021-04-22
BR112021024080A2 (pt) 2022-02-08
MX2021014465A (es) 2022-01-06
AR119061A1 (es) 2021-11-17
KR20220016138A (ko) 2022-02-08
CN113924368A (zh) 2022-01-11
TW202111124A (zh) 2021-03-16
JP2022534402A (ja) 2022-07-29
WO2020243702A2 (en) 2020-12-03
US20220307022A1 (en) 2022-09-29

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