KR20190104392A - 분자의 막투과 전달을 위한 화합물 및 방법 - Google Patents

분자의 막투과 전달을 위한 화합물 및 방법 Download PDF

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KR20190104392A
KR20190104392A KR1020197023505A KR20197023505A KR20190104392A KR 20190104392 A KR20190104392 A KR 20190104392A KR 1020197023505 A KR1020197023505 A KR 1020197023505A KR 20197023505 A KR20197023505 A KR 20197023505A KR 20190104392 A KR20190104392 A KR 20190104392A
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formula
conjugate
compound
sirna
group
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Korean (ko)
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일란 지브
요셉 듀브로브스키
하기트 그림베르크
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아포센스 엘티디.
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/554Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J41/00Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0072Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the A ring of the steroid being aromatic
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07JSTEROIDS
    • C07J51/00Normal steroids with unmodified cyclopenta(a)hydrophenanthrene skeleton not provided for in groups C07J1/00 - C07J43/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Botany (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Steroid Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020197023505A 2017-01-09 2018-01-09 분자의 막투과 전달을 위한 화합물 및 방법 Withdrawn KR20190104392A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762443822P 2017-01-09 2017-01-09
US62/443,822 2017-01-09
PCT/IL2018/050031 WO2018127927A1 (en) 2017-01-09 2018-01-09 Compounds and methods for trans-membrane delivery of molecules

Publications (1)

Publication Number Publication Date
KR20190104392A true KR20190104392A (ko) 2019-09-09

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KR1020197023505A Withdrawn KR20190104392A (ko) 2017-01-09 2018-01-09 분자의 막투과 전달을 위한 화합물 및 방법

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Country Link
US (1) US11230710B2 (https=)
EP (1) EP3565603B1 (https=)
JP (1) JP7348064B2 (https=)
KR (1) KR20190104392A (https=)
CN (1) CN110402152B (https=)
AU (1) AU2018205898B2 (https=)
CA (1) CA3049640A1 (https=)
EA (1) EA201991661A1 (https=)
IL (1) IL267919B2 (https=)
WO (1) WO2018127927A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11318206B2 (en) 2014-03-28 2022-05-03 Aposense Ltd Compounds and methods for trans-membrane delivery of molecules
WO2018127927A1 (en) 2017-01-09 2018-07-12 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
US10195286B2 (en) * 2017-07-04 2019-02-05 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
WO2019130319A1 (en) * 2018-01-01 2019-07-04 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
WO2020044349A1 (en) * 2018-08-30 2020-03-05 Aposense Ltd. Compounds, cojugates and compositions for use in the methods for trans-membrane delivery of molecules
WO2023022055A1 (ja) * 2021-08-17 2023-02-23 国立大学法人東海国立大学機構 修飾ポリヌクレオチド
WO2025096244A1 (en) * 2023-10-30 2025-05-08 Olfera Corporation Olfactory delivery scaffolds and methods for making and using same

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997040679A1 (en) 1996-05-01 1997-11-06 Imarx Pharmaceutical Corp. Methods for delivering compounds into a cell
US6090800A (en) 1997-05-06 2000-07-18 Imarx Pharmaceutical Corp. Lipid soluble steroid prodrugs
WO1998050041A1 (en) 1997-05-06 1998-11-12 Imarx Pharmaceutical Corp. Novel prodrugs comprising fluorinated amphiphiles
FR2846969A1 (fr) 2002-11-08 2004-05-14 Salles Jean Pierre Nouveaux vecteurs moleculaires amphiphiles fluorocarbones a usage biomedical et medical
SE527131C2 (sv) 2004-02-13 2005-12-27 Innoventus Project Ab Steroider för cancerbehandling
WO2006006156A2 (en) 2004-07-08 2006-01-19 Nst Neurosurvival Technologies Ltd Compounds/methods for detection of medical disorders
KR20070053214A (ko) * 2004-08-26 2007-05-23 니콜라스 피라말 인디아 리미티드 신규 생분해성 링커를 함유하는 프로드럭
NZ554176A (en) 2004-10-01 2010-04-30 Prometic Biosciences Inc Medium-chain length fatty alcohols as stimulators of hematopoiesis
RU2408605C2 (ru) 2005-02-18 2011-01-10 Анджиокем Инк. Полипептид, способный преодолевать гематоэнцефалический барьер, и его конъюгат
AU2007299705B2 (en) 2006-09-22 2012-09-06 Dharmacon, Inc. Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2972694C (en) 2008-05-13 2020-02-11 University Of Washington Diblock copolymers and polynucleotide complexes thereof for delivery into cells
WO2009155335A2 (en) 2008-06-19 2009-12-23 University Of Maryland, Baltimore Conjugates of 19f mr imaging tracers and chemotherapeutic agents for drug quantification and drug dose individualization
EP2342340A1 (en) 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
US20120035362A1 (en) 2010-08-03 2012-02-09 Berry And Associates, Inc. Phosphoramidite derivatives of folic acid
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
CA2876860A1 (en) 2012-05-30 2013-12-05 Baylor College Of Medicine Supercoiled minivectors as a tool for dna repair, alteration and replacement
BR112015008365A2 (pt) 2012-10-16 2017-07-04 Endocyte Inc composto da fórmula b-l(d)x, ou um sal farmaceuticamente aceitável do mesmo, composição farmacêutica, uso de um composto, composição de forma de dosagem unitária ou dose unitária, composição para tratar um câncer em um paciente, e método para tratar um câncer em um paciente
WO2014127052A1 (en) 2013-02-13 2014-08-21 Zondlo Neal Perfluoro-tert-butyl hydroxyproline
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US9889202B2 (en) 2014-03-28 2018-02-13 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
US9993563B2 (en) 2014-03-28 2018-06-12 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
CA2944141C (en) * 2014-03-28 2023-03-28 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
US20160016855A1 (en) * 2014-07-21 2016-01-21 Benxin Wu Fabrication of carbon nanotube-nonoxide structural ceramic nanocomposites through laser sintering
WO2018127927A1 (en) 2017-01-09 2018-07-12 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
US10195286B2 (en) 2017-07-04 2019-02-05 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules

Also Published As

Publication number Publication date
EP3565603B1 (en) 2022-12-07
IL267919A (en) 2019-09-26
AU2018205898B2 (en) 2020-12-10
IL267919B2 (en) 2024-03-01
AU2018205898A1 (en) 2019-08-22
WO2018127927A1 (en) 2018-07-12
US20200392490A1 (en) 2020-12-17
CA3049640A1 (en) 2018-07-12
US11230710B2 (en) 2022-01-25
EA201991661A1 (ru) 2019-12-30
EP3565603A4 (en) 2020-09-09
CN110402152B (zh) 2024-03-19
JP2020514299A (ja) 2020-05-21
JP7348064B2 (ja) 2023-09-20
EP3565603A1 (en) 2019-11-13
IL267919B1 (en) 2023-11-01
CN110402152A (zh) 2019-11-01

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PA0105 International application

Patent event date: 20190809

Patent event code: PA01051R01D

Comment text: International Patent Application

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PC1203 Withdrawal of no request for examination