JP2018515479A - 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法 - Google Patents

核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法 Download PDF

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JP2018515479A
JP2018515479A JP2017556986A JP2017556986A JP2018515479A JP 2018515479 A JP2018515479 A JP 2018515479A JP 2017556986 A JP2017556986 A JP 2017556986A JP 2017556986 A JP2017556986 A JP 2017556986A JP 2018515479 A JP2018515479 A JP 2018515479A
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composition
carbohydrate
nucleic acid
poly
cationic polymer
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JP2018515479A5 (enExample
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ホックバーグ，アブラハム
ガルーラ，ジェニファー
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Yissum Research Development Co of Hebrew University of Jerusalem
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Yissum Research Development Co of Hebrew University of Jerusalem
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Priority to JP2021079039A priority Critical patent/JP2021130671A/ja
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    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • A61K48/0041—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 the non-active part being polymeric
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • 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
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Dermatology (AREA)
  • Manufacturing & Machinery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2017556986A 2015-05-05 2016-05-05 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法 Pending JP2018515479A (ja)

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JP2021079039A JP2021130671A (ja) 2015-05-05 2021-05-07 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法

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IL23864315 2015-05-05
IL238643 2015-05-05
PCT/IL2016/050477 WO2016178233A1 (en) 2015-05-05 2016-05-05 Nucleic acid-cationic polymer compositions and methods of making and using the same

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JP2018515479A true JP2018515479A (ja) 2018-06-14
JP2018515479A5 JP2018515479A5 (enExample) 2019-04-25

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JP2021079039A Pending JP2021130671A (ja) 2015-05-05 2021-05-07 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法

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US (2) US20180154023A1 (enExample)
EP (1) EP3291799A1 (enExample)
JP (2) JP2018515479A (enExample)
KR (1) KR20180015145A (enExample)
CN (1) CN107735079A (enExample)
AU (1) AU2016258326A1 (enExample)
BR (1) BR112017023788A2 (enExample)
CA (1) CA2984879A1 (enExample)
IL (1) IL255254A0 (enExample)
MX (1) MX2017014060A (enExample)
PH (1) PH12017502216A1 (enExample)
RU (1) RU2017140065A (enExample)
SG (2) SG11201708760PA (enExample)
WO (1) WO2016178233A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021130671A (ja) * 2015-05-05 2021-09-09 イッサム リサーチ ディベロップメント カンパニー オブ ザ ヘブライ ユニバーシティー オブ エルサレム リミテッドYissum Research Development Company Of The Hebrew Universty Of Jerusalem Ltd. 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法
JP2023523314A (ja) * 2020-04-27 2023-06-02 ジュノー セラピューティクス インコーポレイテッド ポリエチレンイミン－デオキシリボ核酸複合体のサイズおよび活性の安定化

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DE102017003004A1 (de) 2017-03-23 2018-09-27 Friedrich-Schiller-Universität Jena Kationische Polymere mit D-Fructose-Substituenten
US10849921B2 (en) * 2017-05-17 2020-12-01 Bioncotech Therapeutics Sl Pharmaceutical composition comprising particles comprising a complex of a double-stranded polyribonucleotide and a polyalkyleneimine
EP3924463A1 (en) 2019-02-11 2021-12-22 Excellgene SA Novel eukaryotic cell transfection systems and related methods
CN111363761A (zh) * 2020-03-17 2020-07-03 苏州吉恒基因科技有限公司 利用阳离子多聚体dna复合物促进aav介导的基因表达方法
TW202228727A (zh) * 2020-10-01 2022-08-01 德商拜恩迪克公司 適用於治療之微脂體ｒｎａ調配物之製備及儲存
WO2022249180A1 (en) 2021-05-25 2022-12-01 Ramot At Tel-Aviv University Ltd. Compositions comprising extracellular vesicles and an active agent and uses thereof
CN113501889A (zh) * 2021-07-06 2021-10-15 郑州大学 一种三七多糖阳离子衍生物的制备方法及其应用
CN116549626A (zh) * 2022-01-27 2023-08-08 深圳瑞吉生物科技有限公司 一种载核酸脂质纳米颗粒冻干制剂及其制备方法与应用
CN114762731B (zh) * 2022-05-16 2024-08-23 常州大学 一种延长基因药物在温和条件下储存时间的方法
CN116036043A (zh) * 2023-01-10 2023-05-02 深圳市乐土生物医药有限公司 一种核酸药物制剂、冻干粉及制备方法

Citations (1)

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JP2010535800A (ja) * 2007-08-06 2010-11-25 エーゲン、インコーポレイテッド 核酸−リポポリマー組成物

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US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US6087164A (en) * 1997-10-03 2000-07-11 Yissum Research Development Company Of The Hebrew University Of Jerusalem Methods and compositions for inducing tumor-specific cytotoxicity
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WO2007007317A1 (en) 2005-07-07 2007-01-18 Yissum Research Development Company Of The Hebrew University Of Jerusalem Nucleic acid agents for downregulating h19, and methods of using same
JP2009508516A (ja) 2005-09-22 2009-03-05 イッサム リサーチ ディベロプメント カンパニー オブ ザ ヘブリュー ユニバーシティ オブ エルサレム 核酸構築物、薬剤組成物、及び癌治療のためのその使用方法
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021130671A (ja) * 2015-05-05 2021-09-09 イッサム リサーチ ディベロップメント カンパニー オブ ザ ヘブライ ユニバーシティー オブ エルサレム リミテッドYissum Research Development Company Of The Hebrew Universty Of Jerusalem Ltd. 核酸−カチオン性ポリマー組成物、並びにその作製方法及び使用方法
JP2023523314A (ja) * 2020-04-27 2023-06-02 ジュノー セラピューティクス インコーポレイテッド ポリエチレンイミン－デオキシリボ核酸複合体のサイズおよび活性の安定化

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IL255254A0 (en) 2017-12-31
EP3291799A1 (en) 2018-03-14
US20200138977A1 (en) 2020-05-07
US20180154023A1 (en) 2018-06-07
RU2017140065A (ru) 2019-06-05
BR112017023788A2 (pt) 2018-10-16
KR20180015145A (ko) 2018-02-12
CN107735079A (zh) 2018-02-23
MX2017014060A (es) 2018-07-06
CA2984879A1 (en) 2016-11-10
JP2021130671A (ja) 2021-09-09
AU2016258326A1 (en) 2017-11-23
WO2016178233A1 (en) 2016-11-10
RU2017140065A3 (enExample) 2019-09-27
SG11201708760PA (en) 2017-11-29
PH12017502216A1 (en) 2018-06-11
SG10201909209RA (en) 2019-11-28

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