CY1120195T1 - Συνθεσεις irna toy pcsk9 και μεθοδοι χρησης αυτων - Google Patents

Συνθεσεις irna toy pcsk9 και μεθοδοι χρησης αυτων

Info

Publication number
CY1120195T1
CY1120195T1 CY20181100085T CY181100085T CY1120195T1 CY 1120195 T1 CY1120195 T1 CY 1120195T1 CY 20181100085 T CY20181100085 T CY 20181100085T CY 181100085 T CY181100085 T CY 181100085T CY 1120195 T1 CY1120195 T1 CY 1120195T1
Authority
CY
Cyprus
Prior art keywords
methods
pcsk9
irna
toy
compositions
Prior art date
Application number
CY20181100085T
Other languages
Greek (el)
English (en)
Inventor
Anna Borodovsky
Rajeev G. KALLANTHOTTATHIL
Kevin Fitzgerald
Maria Frank-Kamenetsky
William Querbes
Martin Maier
Klaus CHARISSE
Satyanarayana Kuchimanchi
Muthiah Manoharan
Stuart Milstein
Original Assignee
Alnylam Pharmaceuticals, Inc.
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=49841845&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1120195(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Alnylam Pharmaceuticals, Inc. filed Critical Alnylam Pharmaceuticals, Inc.
Publication of CY1120195T1 publication Critical patent/CY1120195T1/el

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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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • C—CHEMISTRY; METALLURGY
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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
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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
    • C12N2310/30—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
CY20181100085T 2012-12-05 2018-01-23 Συνθεσεις irna toy pcsk9 και μεθοδοι χρησης αυτων CY1120195T1 (el)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261733518P 2012-12-05 2012-12-05
US201361793530P 2013-03-15 2013-03-15
US201361886916P 2013-10-04 2013-10-04
US201361892188P 2013-10-17 2013-10-17
PCT/US2013/073349 WO2014089313A1 (en) 2012-12-05 2013-12-05 PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Publications (1)

Publication Number Publication Date
CY1120195T1 true CY1120195T1 (el) 2018-12-12

Family

ID=49841845

Family Applications (2)

Application Number Title Priority Date Filing Date
CY20181100085T CY1120195T1 (el) 2012-12-05 2018-01-23 Συνθεσεις irna toy pcsk9 και μεθοδοι χρησης αυτων
CY2021017C CY2021017I1 (el) 2012-12-05 2021-06-04 Συνθεσεις irna toy pcsk9 και μεθοδοι χρησης αυτων

Family Applications After (1)

Application Number Title Priority Date Filing Date
CY2021017C CY2021017I1 (el) 2012-12-05 2021-06-04 Συνθεσεις irna toy pcsk9 και μεθοδοι χρησης αυτων

Country Status (30)

Country Link
US (2) US10125369B2 (enExample)
EP (3) EP3336187A1 (enExample)
JP (5) JP6574383B2 (enExample)
KR (2) KR20200035490A (enExample)
CN (2) CN108220295B (enExample)
AR (1) AR093835A1 (enExample)
AU (4) AU2013355237C1 (enExample)
BR (1) BR112015013105B1 (enExample)
CA (1) CA2892160C (enExample)
CY (2) CY1120195T1 (enExample)
DK (1) DK2929031T5 (enExample)
ES (1) ES2657608T3 (enExample)
FR (1) FR21C1021I2 (enExample)
HK (1) HK1256621A1 (enExample)
HR (1) HRP20180126T2 (enExample)
HU (2) HUE035887T2 (enExample)
IL (3) IL326966A (enExample)
LT (2) LT2929031T (enExample)
LU (1) LUC00209I2 (enExample)
MX (2) MX367076B (enExample)
NL (1) NL301107I2 (enExample)
NO (1) NO2021024I1 (enExample)
NZ (1) NZ709013A (enExample)
PL (1) PL2929031T4 (enExample)
PT (1) PT2929031T (enExample)
RS (1) RS56783B9 (enExample)
SI (1) SI2929031T1 (enExample)
SM (1) SMT201800039T1 (enExample)
WO (1) WO2014089313A1 (enExample)
ZA (1) ZA201503829B (enExample)

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WO2019126990A1 (zh) * 2017-12-26 2019-07-04 广州市锐博生物科技有限公司 一种抑制PCSK9基因表达的siRNA分子及其应用
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CN110959011B (zh) 2017-12-29 2023-03-28 苏州瑞博生物技术股份有限公司 缀合物及其制备方法和用途
MY207352A (en) 2018-03-21 2025-02-21 Regeneron Pharma 17-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
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BR112021002440A2 (pt) 2018-08-10 2021-05-04 University Of Massachusetts oligonucleotídeos modificados visando snps
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CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
JP7610848B2 (ja) 2019-05-22 2025-01-09 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
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CN113795582B (zh) 2019-05-24 2024-05-24 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
CN110540992B (zh) * 2019-08-21 2021-02-09 武汉泽智生物医药有限公司 增强靶基因沉默效率的siRNA分子及其应用
US20210115442A1 (en) * 2019-08-23 2021-04-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN115176011B (zh) * 2019-08-27 2025-11-11 赛诺菲 用于抑制pcsk9的组合物和方法
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