EA202090893A2 - КОМПОЗИЦИИ С иРНК К PCSK9 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ - Google Patents

КОМПОЗИЦИИ С иРНК К PCSK9 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ

Info

Publication number
EA202090893A2
EA202090893A2 EA202090893A EA202090893A EA202090893A2 EA 202090893 A2 EA202090893 A2 EA 202090893A2 EA 202090893 A EA202090893 A EA 202090893A EA 202090893 A EA202090893 A EA 202090893A EA 202090893 A2 EA202090893 A2 EA 202090893A2
Authority
EA
Eurasian Patent Office
Prior art keywords
methods
pcsk9
mrna
compositions
application
Prior art date
Application number
EA202090893A
Other languages
English (en)
Other versions
EA202090893A3 (ru
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/ru
Publication of EA202090893A3 publication Critical patent/EA202090893A3/ru

Links

Classifications

    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • 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
    • C12N2310/312Phosphonates
    • 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
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • 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
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

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.
EA202090893A 2013-10-17 2013-12-05 Композиции с ирнк к pcsk9 и способы их применения EA202090893A3 (ru)

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 true EA202090893A2 (ru) 2021-06-30
EA202090893A3 EA202090893A3 (ru) 2021-09-30

Family

ID=76807402

Family Applications (2)

Application Number Title Priority Date Filing Date
EA201591075A EA037110B9 (ru) 2013-10-17 2013-12-05 КОМПОЗИЦИИ С иРНК К PCSK9 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
EA202090893A EA202090893A3 (ru) 2013-10-17 2013-12-05 Композиции с ирнк к pcsk9 и способы их применения

Family Applications Before (1)

Application Number Title Priority Date Filing Date
EA201591075A EA037110B9 (ru) 2013-10-17 2013-12-05 КОМПОЗИЦИИ С иРНК К PCSK9 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ

Country Status (1)

Country Link
EA (2) EA037110B9 (ru)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20120050429A (ko) * 2009-06-15 2012-05-18 알닐람 파마슈티칼스 인코포레이티드 Pcsk9 유전자를 표적으로 하는 지질 제형된 dsrna
WO2012058693A2 (en) * 2010-10-29 2012-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of pcsk9 genes

Also Published As

Publication number Publication date
EA202090893A3 (ru) 2021-09-30
EA037110B1 (ru) 2021-02-08
EA037110B9 (ru) 2022-03-29
EA201591075A1 (ru) 2015-11-30

Similar Documents

Publication Publication Date Title
ZA202110854B (en) Tmprss6 irna compositions and methods of use thereof
MX2019009283A (es) Composiciones de arni de proteina convertasa subtilisina kexina 9 (pcsk9) y metodos de uso de las mismas.
EA201592223A1 (ru) КОМПОЗИЦИИ НА ОСНОВЕ iRNA SERPINAl И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
MX2021006053A (es) Composiciones de angiotensinogeno (agt) arni y metodos de uso de las mismas.
EA201890571A1 (ru) Способы и композиции для лечения нарушения, ассоциированного с геном пропротеинконвертазы субтилизин/кексинового типа (pcsk9)
PH12019550076A1 (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EA201591707A1 (ru) КОМПОЗИЦИИ иРНК КОМПОНЕНТА КОМПЛЕМЕНТА C5 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
TR201901886T4 (tr) DNA-PK inhibitörleri.
DOP2022000085A (es) Composiciones y métodos para inhibir la expresión del gen alas1
EA201591616A1 (ru) Трифторметил-замещенные аннелированные пиримидины и их применение
EA201391416A1 (ru) Новые соединения в качестве модуляторов протеинкиназ
EA201691032A1 (ru) Производные изохромена в качестве ингибиторов фосфоинозитид-3-киназ
EA201492289A1 (ru) Композиции, содержащие аптамер против pdgf и антагонист vegf
EA201692313A1 (ru) Производные индолизина в качестве ингибиторов фосфоинозитид-3-киназ
EA201491357A1 (ru) Анти-lrp5 антитела и способы их применения
EA202091865A1 (ru) Композиции и способы для снижения экспрессии tau
MX2021010716A (es) Composiciones y metodos para inhibir la expresion del gen lect2.
CY1122403T1 (el) Μορφες δοσολογιας και θεραπευτικες χρησεις της l-4-χλωροκυνουρενινης
CY1121000T1 (el) Sirna και χρηση αυτου σε μεθοδους και συνθεσεις για την θεραπεια ή/και προληψη οφθαλμικων καταστασεων
EA201591725A1 (ru) Твердые липидные наночастицы (ii)
EA201500927A1 (ru) Твердые липидные наночастицы (i)
EA201492280A1 (ru) Стабильный состав пексиганана
EA201491455A1 (ru) ЭФФЕКТОРЫ β-АРРЕСТИНА, КОМПОЗИЦИИ, ИХ СОДЕРЖАЩИЕ, И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
EA202090893A2 (ru) КОМПОЗИЦИИ С иРНК К PCSK9 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
EA201690709A1 (ru) Применение одипарцила в лечении мукополисахаридоза