EA202090893A3 - COMPOSITIONS WITH IRNA K PCSK9 AND METHODS OF THEIR APPLICATION - Google Patents

COMPOSITIONS WITH IRNA K PCSK9 AND METHODS OF THEIR APPLICATION

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Publication number
EA202090893A3
EA202090893A3 EA202090893A EA202090893A EA202090893A3 EA 202090893 A3 EA202090893 A3 EA 202090893A3 EA 202090893 A EA202090893 A EA 202090893A EA 202090893 A EA202090893 A EA 202090893A EA 202090893 A3 EA202090893 A3 EA 202090893A3
Authority
EA
Eurasian Patent Office
Prior art keywords
methods
pcsk9
irna
compositions
application
Prior art date
Application number
EA202090893A
Other languages
Russian (ru)
Other versions
EA202090893A2 (en
Inventor
Анна Бородовский
Раджив Г. Каллантхоттатхил
Кевин Фитцжеральд
Мария Франк-Каменетски
Уилльям Куэрбс
Мартин Майер
Клаус Хариссе
Сатиянараяна Кучиманчи
Мутхиах Манохаран
Стюарт Милстейн
Original Assignee
Элнилэм Фармасьютикалз, Инк.
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 Элнилэм Фармасьютикалз, Инк. filed Critical Элнилэм Фармасьютикалз, Инк.
Publication of EA202090893A2 publication Critical patent/EA202090893A2/en
Publication of EA202090893A3 publication Critical patent/EA202090893A3/en

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    • 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
    • C12N15/11—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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    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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
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    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Abstract

Настоящее изобретение относится к средствам для RNAi, например двухцепочечным средствам для RNAi, нацеленным на ген PCSK9, и способам применения таких средств для RNAi для ингибирования экспрессии PCSK9 и способам лечения субъектов с нарушением липидного обмена, таким как гиперлипидемия.The present invention relates to RNAi agents, eg, double-stranded RNAi agents targeting the PCSK9 gene, and methods of using such RNAi agents to inhibit PCSK9 expression, and methods of treating subjects with lipid disorders such as hyperlipidemia.

EA202090893A 2013-10-17 2013-12-05 COMPOSITIONS WITH IRNA K PCSK9 AND METHODS OF THEIR APPLICATION EA202090893A3 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201361892188P 2013-10-17 2013-10-17

Publications (2)

Publication Number Publication Date
EA202090893A2 EA202090893A2 (en) 2021-06-30
EA202090893A3 true EA202090893A3 (en) 2021-09-30

Family

ID=76807402

Family Applications (2)

Application Number Title Priority Date Filing Date
EA202090893A EA202090893A3 (en) 2013-10-17 2013-12-05 COMPOSITIONS WITH IRNA K PCSK9 AND METHODS OF THEIR APPLICATION
EA201591075A EA037110B9 (en) 2013-10-17 2013-12-05 PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Family Applications After (1)

Application Number Title Priority Date Filing Date
EA201591075A EA037110B9 (en) 2013-10-17 2013-12-05 PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Country Status (1)

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EA (2) EA202090893A3 (en)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA201270019A1 (en) * 2009-06-15 2012-06-29 Элнилэм Фармасьютикалз, Инк. BENTROVAL RNA INCLUDED IN LIPID COMPOSITION AND WHICH IS THE PCSK9 GENE
EP2633046A4 (en) * 2010-10-29 2015-05-06 Alnylam Pharmaceuticals Inc Compositions and methods for inhibition of pcsk9 genes

Also Published As

Publication number Publication date
EA202090893A2 (en) 2021-06-30
EA201591075A1 (en) 2015-11-30
EA037110B1 (en) 2021-02-08
EA037110B9 (en) 2022-03-29

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