CN108135923B - 靶向超氧化物歧化酶1（sod1）的核酸分子 - Google Patents

靶向超氧化物歧化酶1（sod1）的核酸分子 Download PDF

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CN108135923B
CN108135923B CN201680051666.7A CN201680051666A CN108135923B CN 108135923 B CN108135923 B CN 108135923B CN 201680051666 A CN201680051666 A CN 201680051666A CN 108135923 B CN108135923 B CN 108135923B
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nucleic acid
acid molecule
stranded nucleic
strand comprises
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CN108135923A (zh
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詹姆斯·卡迪亚
迈克尔·伯恩
卡伦·G·布洛克
帕梅拉·A·帕夫科
林恩·利伯蒂娜
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Phio Pharmaceuticals Corp
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201680051666.7A 2015-07-06 2016-07-06 靶向超氧化物歧化酶1（sod1）的核酸分子 Expired - Fee Related CN108135923B (zh)

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)

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CN108135923A CN108135923A (zh) 2018-06-08
CN108135923B true CN108135923B (zh) 2021-03-02

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

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JP5697993B2 (ja) 2008-02-11 2015-04-08 アールエックスアイ ファーマシューティカルズ コーポレーション 修飾ＲＮＡｉポリヌクレオチドおよびその使用
CA2753338A1 (en) 2008-09-22 2010-03-25 Rxi Pharmaceuticals Corporation Neutral nanotransporters
US9074211B2 (en) 2008-11-19 2015-07-07 Rxi Pharmaceuticals Corporation Inhibition of MAP4K4 through RNAI
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
EP2542058B1 (en) 2010-03-02 2018-10-10 RXi Pharmaceuticals Corporation Effective sensitizing dose of a gelled immunomodulating topical composition
EP3578183B1 (en) 2010-03-24 2021-09-08 Phio Pharmaceuticals Corp. Rna interference in ocular indications
AU2011232365A1 (en) 2010-03-24 2012-10-25 Rxi Pharmaceuticals Corporation RNA interference in dermal and fibrotic indications
RU2615143C2 (ru) 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
JP6772062B2 (ja) 2013-12-02 2020-10-21 フィオ ファーマシューティカルズ コーポレーションＰｈｉｏ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐ． 癌の免疫療法
SG11201608109TA (en) 2014-04-01 2016-10-28 Ionis Pharmaceuticals Inc Compositions for modulating sod-1 expression
EP3137119B1 (en) 2014-04-28 2020-07-01 Phio Pharmaceuticals Corp. Methods for treating cancer using a nucleic acid targeting mdm2
KR102506169B1 (ko) 2014-09-05 2023-03-08 피오 파마슈티칼스 코프. Tyr 또는 mmp1을 표적화하는 핵산을 사용한 노화 및 피부 장애의 치료 방법
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
CA2991598A1 (en) 2015-07-06 2017-01-12 Rxi Pharmaceuticals Corporation Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
WO2017070151A1 (en) 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
MX2022005220A (es) * 2019-11-06 2022-08-11 Alnylam Pharmaceuticals Inc Suministro extrahepático.
EP4291654A2 (en) * 2021-02-12 2023-12-20 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

Citations (3)

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WO2009102427A2 (en) * 2008-02-11 2009-08-20 Rxi Pharmaceuticals Corp. Modified rnai polynucleotides and uses thereof
CN102405286A (zh) * 2008-09-22 2012-04-04 阿克赛医药公司 减小大小的自递送RNAi化合物
CN103380145A (zh) * 2010-12-17 2013-10-30 生物控股有限公司 人类抗-sod1抗体

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