JP2015502365A - オリゴヌクレオチドのイン−ビボ送達 - Google Patents

オリゴヌクレオチドのイン−ビボ送達 Download PDF

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Publication number
JP2015502365A
JP2015502365A JP2014547389A JP2014547389A JP2015502365A JP 2015502365 A JP2015502365 A JP 2015502365A JP 2014547389 A JP2014547389 A JP 2014547389A JP 2014547389 A JP2014547389 A JP 2014547389A JP 2015502365 A JP2015502365 A JP 2015502365A
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oligonucleotide
nucleic acid
hes
mirna
rna
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JP2015502365A5 (OSRAM
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パッカード ビバリー
パッカード ビバリー
コモリヤ アキラ
コモリヤ アキラ
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オンコイミューニン，インコーポレイティド
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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
    • A61K31/7084—Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/11—Antisense
    • 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]
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3517—Marker; Tag
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Rheumatology (AREA)
  • Pain & Pain Management (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Dermatology (AREA)
  • Neurology (AREA)
JP2014547389A 2011-12-12 2012-12-12 オリゴヌクレオチドのイン−ビボ送達 Pending JP2015502365A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161630446P 2011-12-12 2011-12-12
US61/630,446 2011-12-12
PCT/US2012/069294 WO2013090457A2 (en) 2011-12-12 2012-12-12 In vivo delivery of oligonucleotides

Related Child Applications (1)

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JP2019017110A Division JP2019073548A (ja) 2011-12-12 2019-02-01 オリゴヌクレオチドのイン−ビボ送達

Publications (2)

Publication Number Publication Date
JP2015502365A true JP2015502365A (ja) 2015-01-22
JP2015502365A5 JP2015502365A5 (OSRAM) 2017-06-15

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JP2014547389A Pending JP2015502365A (ja) 2011-12-12 2012-12-12 オリゴヌクレオチドのイン−ビボ送達
JP2019017110A Pending JP2019073548A (ja) 2011-12-12 2019-02-01 オリゴヌクレオチドのイン−ビボ送達

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Country Status (6)

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US (4) US10557136B2 (OSRAM)
EP (1) EP2790736B1 (OSRAM)
JP (2) JP2015502365A (OSRAM)
CA (1) CA2895077A1 (OSRAM)
DK (1) DK2790736T3 (OSRAM)
WO (1) WO2013090457A2 (OSRAM)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521753A (ja) * 2013-06-12 2016-07-25 オンコイミューニン，インコーポレイティド オリゴヌクレオチドの全身性イン−ビボ送達
JPWO2021020412A1 (OSRAM) * 2019-07-30 2021-02-04
JP2022524218A (ja) * 2019-04-03 2022-04-28 ブリストル－マイヤーズ スクイブ カンパニー Ａｎｇｐｔｌ２アンチセンスオリゴヌクレオチドおよびその使用
JP2023509979A (ja) * 2020-01-11 2023-03-10 オンコイミューニン，インコーポレイティド オリゴヌクレオチドの標的化及び局所インビボ送達

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WO2012083005A2 (en) 2010-12-15 2012-06-21 Miragen Therapeutics Microrna inhibitors comprising locked nucleotides
MX347361B (es) 2011-07-19 2017-04-12 Wave Life Sciences Ltd Metodos para la sintesis de acidos nucleicos funcionalizados.
KR20140091688A (ko) 2011-10-06 2014-07-22 미라젠 세러퓨틱스 인코포레이티드 마이크로rna 조절에 의한 전신 에너지 항상성의 제어
CA2895077A1 (en) * 2011-12-12 2013-06-20 Beverly Packard In vivo delivery of oligonucleotides
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RU2674600C2 (ru) * 2012-07-03 2018-12-11 Просенса Текнолоджиз Б.В. Олигонуклеотид для лечения пациентов с мышечной дистрофией
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US10947543B2 (en) * 2013-03-13 2021-03-16 Yale University Interferon production using short RNA duplexes
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JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
CN106068325B (zh) 2014-01-16 2021-07-09 波涛生命科学有限公司 手性设计
US10119140B2 (en) 2014-04-18 2018-11-06 Board Of Regents Of The Nevada System Of Higher Education On Behalf Of The University Of Nevada, Reno Methods for enhancing or decreasing the levels of MIR124 and MIR29 in subjects with muscular dystrophy
PL3143123T3 (pl) * 2014-05-16 2020-07-13 The Trustees Of The University Of Pennsylvania Indukcja regeneracji serca za pomocą mikroRNA
SG11201705907XA (en) 2015-01-20 2017-08-30 Miragen Therapeutics Inc Mir-92 inhibitors and uses thereof
JP7511326B2 (ja) * 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
WO2018065390A1 (en) * 2016-10-04 2018-04-12 Vib Vzw Means and methods to treat inflammatory diseases
WO2019246450A1 (en) 2018-06-20 2019-12-26 Yale University Rig-i agonists and treatments using same
WO2020181130A2 (en) * 2019-03-05 2020-09-10 MiRagen Therapeutics, Inc. Polynucleotide conjugates and uses thereof
US11786545B2 (en) 2020-12-09 2023-10-17 Yale University Compositions and methods for treating SARS-CoV-2 infection
US11649457B2 (en) 2020-12-09 2023-05-16 Yale University Methods for treating SARS-CoV-2 infection
WO2022232223A1 (en) * 2021-04-28 2022-11-03 The Johns Hopkins University Treating cancer
CN113975397B (zh) * 2021-10-26 2023-08-22 中南大学湘雅三医院 一种基因/小分子化合物纳米递药系统及其制备方法与应用

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