MX2021015290A - Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3). - Google Patents

Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3).

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Publication number
MX2021015290A
MX2021015290A MX2021015290A MX2021015290A MX2021015290A MX 2021015290 A MX2021015290 A MX 2021015290A MX 2021015290 A MX2021015290 A MX 2021015290A MX 2021015290 A MX2021015290 A MX 2021015290A MX 2021015290 A MX2021015290 A MX 2021015290A
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MX
Mexico
Prior art keywords
apoc3
methods
expression
apolipoprotein
genes
Prior art date
Application number
MX2021015290A
Other languages
English (en)
Inventor
Kevin Fitzgerald
Stuart Milstein
Brian Bettencourt
Martin Maier
Satya Kuchimanchi
Tuyen Nguyen
Klaus Charisse
Kallanthottathil Rajeev
Muthiah Manoharan
Original Assignee
Alnylam Pharmaceuticals Inc
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Filing date
Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of MX2021015290A publication Critical patent/MX2021015290A/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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/30—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
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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

Abstract

La invención se relaciona con un ácido ribonucleico de doble hebra (ARNds) dirigido a un gen para APOC3, y métodos de uso del ARNds para inhibir la expresión de APOC3.
MX2021015290A 2011-06-21 2013-12-06 Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3). MX2021015290A (es)

Applications Claiming Priority (1)

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US201161499620P 2011-06-21 2011-06-21

Publications (1)

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MX2021015290A true MX2021015290A (es) 2022-01-18

Family

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Family Applications (2)

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MX2013014378A MX344807B (es) 2011-06-21 2012-06-21 Composiciones y metodos para inhibicion de genes para alipoproteina c-iii (apoc3).
MX2021015290A MX2021015290A (es) 2011-06-21 2013-12-06 Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3).

Family Applications Before (1)

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MX2013014378A MX344807B (es) 2011-06-21 2012-06-21 Composiciones y metodos para inhibicion de genes para alipoproteina c-iii (apoc3).

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US (6) US9315813B2 (es)
EP (2) EP2723756B1 (es)
JP (5) JP6236385B2 (es)
KR (4) KR102540778B1 (es)
CN (3) CN103649103A (es)
AU (5) AU2012272860A1 (es)
BR (1) BR112013032645A2 (es)
CA (2) CA3191066A1 (es)
MX (2) MX344807B (es)
RU (1) RU2631805C2 (es)
WO (1) WO2012177947A2 (es)

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