CN103917647B - 用于神经保护的方法和组合物 - Google Patents

用于神经保护的方法和组合物 Download PDF

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Publication number
CN103917647B
CN103917647B CN201280054213.1A CN201280054213A CN103917647B CN 103917647 B CN103917647 B CN 103917647B CN 201280054213 A CN201280054213 A CN 201280054213A CN 103917647 B CN103917647 B CN 103917647B
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seq
nucleotide
ear
dsrna
disease
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CN103917647A (zh
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埃琳娜·费因斯坦
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Quark Pharmaceuticals Inc
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Quark Pharmaceuticals Inc
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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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/08—Drugs for disorders of the alimentary tract or the digestive system for nausea, cinetosis or vertigo; Antiemetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • 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)
    • C12Y304/22—Cysteine endopeptidases (3.4.22)
    • C12Y304/22017—Calpain (3.4.22.17)
    • 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)
    • C12Y304/22—Cysteine endopeptidases (3.4.22)
    • C12Y304/22055—Caspase-2 (3.4.22.55)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
    • C12Y306/05002—Small monomeric GTPase (3.6.5.2)
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/30—Special therapeutic applications
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Otolaryngology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CN201280054213.1A 2011-11-03 2012-11-01 用于神经保护的方法和组合物 Expired - Fee Related CN103917647B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161554982P 2011-11-03 2011-11-03
US61/554,982 2011-11-03
US201261663627P 2012-06-25 2012-06-25
US61/663,627 2012-06-25
PCT/US2012/062894 WO2013067076A2 (en) 2011-11-03 2012-11-01 Methods and compositions for neuroprotection

Publications (2)

Publication Number Publication Date
CN103917647A CN103917647A (zh) 2014-07-09
CN103917647B true CN103917647B (zh) 2020-07-10

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US (2) US9422560B2 (https=)
EP (1) EP2773758B1 (https=)
JP (1) JP6118331B2 (https=)
KR (1) KR101970034B1 (https=)
CN (1) CN103917647B (https=)
AU (1) AU2012332517B9 (https=)
BR (1) BR112014010769B1 (https=)
CA (1) CA2851296C (https=)
IL (1) IL232085B (https=)
MX (1) MX356814B (https=)
SG (1) SG11201401648RA (https=)
WO (1) WO2013067076A2 (https=)
ZA (1) ZA201403281B (https=)

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SG10201804960RA (en) * 2013-12-12 2018-07-30 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
SG11202002940QA (en) 2017-11-01 2020-04-29 Alnylam Pharmaceuticals Inc Complement component c3 irna compositions and methods of use thereof
WO2021081026A1 (en) 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
WO2021237344A1 (en) * 2020-05-25 2021-12-02 Wilfrid Laurier University Compositions and methods of inducing rnai or type i ifn in ifn competent cells and uses thereof
CN118302525A (zh) 2021-10-29 2024-07-05 阿尔尼拉姆医药品有限公司 补体因子B（CFB）iRNA组合物及其使用方法

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CN103917647A (zh) 2014-07-09
AU2012332517B2 (en) 2017-04-06
JP6118331B2 (ja) 2017-04-19
ZA201403281B (en) 2015-08-26
CA2851296C (en) 2020-08-25
EP2773758B1 (en) 2017-06-07
EP2773758A2 (en) 2014-09-10
KR101970034B1 (ko) 2019-04-17
US20150031746A1 (en) 2015-01-29
US20160362695A1 (en) 2016-12-15
BR112014010769A2 (pt) 2018-08-07
CA2851296A1 (en) 2013-05-10
MX356814B (es) 2018-06-13
US9422560B2 (en) 2016-08-23
AU2012332517B9 (en) 2017-08-10
US9738896B2 (en) 2017-08-22
KR20140091683A (ko) 2014-07-22
JP2015501634A (ja) 2015-01-19
IL232085A0 (en) 2014-05-28
IL232085B (en) 2018-04-30
MX2014005424A (es) 2014-10-17
SG11201401648RA (en) 2014-05-29
WO2013067076A3 (en) 2013-07-04
AU2012332517A1 (en) 2014-04-24
WO2013067076A2 (en) 2013-05-10
BR112014010769B1 (pt) 2021-08-03

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