JP6983752B2 - スーパーオキシドディスムターゼ１（ｓｏｄ１）を標的とする核酸分子 - Google Patents

スーパーオキシドディスムターゼ１（ｓｏｄ１）を標的とする核酸分子 Download PDF

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JP6983752B2
JP6983752B2 JP2018500697A JP2018500697A JP6983752B2 JP 6983752 B2 JP6983752 B2 JP 6983752B2 JP 2018500697 A JP2018500697 A JP 2018500697A JP 2018500697 A JP2018500697 A JP 2018500697A JP 6983752 B2 JP6983752 B2 JP 6983752B2
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seq
nucleic acid
acid molecule
nucleotides
modified
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JP2018519835A5 (OSRAM
JP2018519835A (ja
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カルディア，ジェームス
バーン，マイケル
ブルック，カレン，ジー．
パヴコ，パメラ，エー．
リバティーン，リン
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Phio Pharmaceuticals Corp
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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
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • 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]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2018500697A 2015-07-06 2016-07-06 スーパーオキシドディスムターゼ１（ｓｏｄ１）を標的とする核酸分子 Expired - Fee Related JP6983752B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562189050P 2015-07-06 2015-07-06
US62/189,050 2015-07-06
PCT/US2016/041095 WO2017007813A1 (en) 2015-07-06 2016-07-06 Nucleic acid molecules targeting superoxide dismutase 1 (sod1)

Publications (3)

Publication Number Publication Date
JP2018519835A JP2018519835A (ja) 2018-07-26
JP2018519835A5 JP2018519835A5 (OSRAM) 2019-08-15
JP6983752B2 true JP6983752B2 (ja) 2021-12-17

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JP2018500697A Expired - Fee Related JP6983752B2 (ja) 2015-07-06 2016-07-06 スーパーオキシドディスムターゼ１（ｓｏｄ１）を標的とする核酸分子

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US (1) US11001845B2 (OSRAM)
EP (2) EP3319614B1 (OSRAM)
JP (1) JP6983752B2 (OSRAM)
KR (1) KR20180026739A (OSRAM)
CN (1) CN108135923B (OSRAM)
CA (1) CA2991598A1 (OSRAM)
WO (1) WO2017007813A1 (OSRAM)

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CN104975020B (zh) 2008-02-11 2020-01-17 菲奥医药公司 经修饰的RNAi多核苷酸及其用途
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WO2010059226A2 (en) 2008-11-19 2010-05-27 Rxi Pharmaceuticals Corporation Inhibition of map4k4 through rnai
WO2010090762A1 (en) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
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CA2794187C (en) 2010-03-24 2020-07-14 Rxi Pharmaceuticals Corporation Rna interference in ocular indications
KR20180044433A (ko) 2010-03-24 2018-05-02 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 ｒｎａ 간섭
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
WO2015084897A2 (en) 2013-12-02 2015-06-11 Mirimmune, Llc Immunotherapy of cancer
SI3126499T1 (sl) 2014-04-01 2020-09-30 Biogen Ma Inc. Sestave za moduliranje izražanja SOD-1
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
US10900039B2 (en) 2014-09-05 2021-01-26 Phio Pharmaceuticals Corp. Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1
US10808247B2 (en) 2015-07-06 2020-10-20 Phio Pharmaceuticals Corp. Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
KR20180026739A (ko) 2015-07-06 2018-03-13 알엑스아이 파마슈티칼스 코포레이션 슈퍼옥시드 디스뮤타제 1 (sod1)을 표적화하는 핵산 분자
EP3365446A4 (en) 2015-10-19 2019-06-26 Phio Pharmaceuticals Corp. SMALL SELF-LEANING NUCLEIC ACID COMPOUNDS TURNED AGAINST LONG NON-CODING RNA
AU2020378414A1 (en) * 2019-11-06 2022-05-26 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
WO2022174000A2 (en) * 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US20250297258A1 (en) * 2021-07-07 2025-09-25 Ractigen Therapeutics Oligonucleotide-based delivery vehicle for oligonucleotides agents and methods of use thereof
WO2025045130A1 (zh) * 2023-08-29 2025-03-06 石药集团中奇制药技术（石家庄）有限公司 一种抑制sod1基因表达的dsrna分子及其应用

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