AU2013251494A1 - Serpinc1 iRNA compositions and methods of use thereof - Google Patents

Serpinc1 iRNA compositions and methods of use thereof Download PDF

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Publication number
AU2013251494A1
AU2013251494A1 AU2013251494A AU2013251494A AU2013251494A1 AU 2013251494 A1 AU2013251494 A1 AU 2013251494A1 AU 2013251494 A AU2013251494 A AU 2013251494A AU 2013251494 A AU2013251494 A AU 2013251494A AU 2013251494 A1 AU2013251494 A1 AU 2013251494A1
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AU
Australia
Prior art keywords
dsrna
subject
cell
nucleotide
irna
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
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AU2013251494A
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English (en)
Inventor
Akin Akinc
Brian Bettencourt
Klaus CHARISSE
Donald Foster
Satyanarayana Kuchimanchi
Martin Maier
Muthiah Manoharan
Stuart Milstein
Kallanthottathil G. Rajeev
Alfica Sehgal
Ivanka Toudjarska
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Alnylam Pharmaceuticals Inc
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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
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AU2013251494A1 publication Critical patent/AU2013251494A1/en
Priority to AU2018200469A priority Critical patent/AU2018200469B2/en
Priority to AU2020202705A priority patent/AU2020202705B2/en
Priority to AU2022279381A priority patent/AU2022279381B2/en
Priority to AU2025259924A priority patent/AU2025259924A1/en
Abandoned legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
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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
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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3523—Allyl
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2013251494A 2012-04-26 2013-04-25 Serpinc1 iRNA compositions and methods of use thereof Abandoned AU2013251494A1 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
AU2018200469A AU2018200469B2 (en) 2012-04-26 2018-01-19 Serpinc1 irna compositions and methods of use thereof
AU2020202705A AU2020202705B2 (en) 2012-04-26 2020-04-22 Serpinc1 irna compositions and methods of use thereof
AU2022279381A AU2022279381B2 (en) 2012-04-26 2022-11-28 Serpinc1 irna compositions and methods of use thereof
AU2025259924A AU2025259924A1 (en) 2012-04-26 2025-10-30 Serpinc1 irna compositions and methods of use thereof

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201261638952P 2012-04-26 2012-04-26
US61/638,952 2012-04-26
US201261669249P 2012-07-09 2012-07-09
US61/669,249 2012-07-09
US201261734573P 2012-12-07 2012-12-07
US61/734,573 2012-12-07
US13/837,129 US9127274B2 (en) 2012-04-26 2013-03-15 Serpinc1 iRNA compositions and methods of use thereof
US13/837,129 2013-03-15
PCT/US2013/038218 WO2013163430A2 (en) 2012-04-26 2013-04-25 Serpinc1 irna compositions and methods of use thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2018200469A Division AU2018200469B2 (en) 2012-04-26 2018-01-19 Serpinc1 irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
AU2013251494A1 true AU2013251494A1 (en) 2014-10-30

Family

ID=49484030

Family Applications (5)

Application Number Title Priority Date Filing Date
AU2013251494A Abandoned AU2013251494A1 (en) 2012-04-26 2013-04-25 Serpinc1 iRNA compositions and methods of use thereof
AU2018200469A Active AU2018200469B2 (en) 2012-04-26 2018-01-19 Serpinc1 irna compositions and methods of use thereof
AU2020202705A Active AU2020202705B2 (en) 2012-04-26 2020-04-22 Serpinc1 irna compositions and methods of use thereof
AU2022279381A Active AU2022279381B2 (en) 2012-04-26 2022-11-28 Serpinc1 irna compositions and methods of use thereof
AU2025259924A Pending AU2025259924A1 (en) 2012-04-26 2025-10-30 Serpinc1 irna compositions and methods of use thereof

Family Applications After (4)

Application Number Title Priority Date Filing Date
AU2018200469A Active AU2018200469B2 (en) 2012-04-26 2018-01-19 Serpinc1 irna compositions and methods of use thereof
AU2020202705A Active AU2020202705B2 (en) 2012-04-26 2020-04-22 Serpinc1 irna compositions and methods of use thereof
AU2022279381A Active AU2022279381B2 (en) 2012-04-26 2022-11-28 Serpinc1 irna compositions and methods of use thereof
AU2025259924A Pending AU2025259924A1 (en) 2012-04-26 2025-10-30 Serpinc1 irna compositions and methods of use thereof

Country Status (24)

Country Link
US (6) US9127274B2 (enExample)
EP (2) EP2841443B1 (enExample)
JP (4) JP6466832B2 (enExample)
KR (5) KR102254380B1 (enExample)
CN (4) CN110295167A (enExample)
AR (1) AR090869A1 (enExample)
AU (5) AU2013251494A1 (enExample)
CA (1) CA2869922A1 (enExample)
CL (1) CL2016000188A1 (enExample)
CO (1) CO7240361A2 (enExample)
ES (1) ES2934152T3 (enExample)
FI (1) FI2841443T3 (enExample)
HR (1) HRP20221498T1 (enExample)
HU (1) HUE060846T2 (enExample)
IL (4) IL234941B (enExample)
LT (1) LT2841443T (enExample)
MX (2) MX365419B (enExample)
PL (1) PL2841443T3 (enExample)
PT (1) PT2841443T (enExample)
RS (1) RS63838B1 (enExample)
RU (1) RU2691580C2 (enExample)
SI (1) SI2841443T1 (enExample)
WO (1) WO2013163430A2 (enExample)
ZA (2) ZA201407293B (enExample)

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