CN111246855A - 用于分子的跨膜递送的化合物和方法 - Google Patents

用于分子的跨膜递送的化合物和方法 Download PDF

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Publication number
CN111246855A
CN111246855A CN201880057472.7A CN201880057472A CN111246855A CN 111246855 A CN111246855 A CN 111246855A CN 201880057472 A CN201880057472 A CN 201880057472A CN 111246855 A CN111246855 A CN 111246855A
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formula
set forth
solvates
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CN201880057472.7A
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English (en)
Chinese (zh)
Inventor
伊兰·齐夫
约瑟夫·杜布罗夫斯基
哈吉特·格林贝格
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Aposense Ltd
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Aposense Ltd
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Priority claimed from US15/641,251 external-priority patent/US20190125884A1/en
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Publication of CN111246855A publication Critical patent/CN111246855A/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/554—Medicinal 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
    • 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
    • A61K48/0033—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 non-polymeric
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J41/00—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
    • C07J41/0033—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
    • C07J41/0072—Normal 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J43/00—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton
    • C07J43/003—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton not condensed
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J51/00—Normal steroids with unmodified cyclopenta(a)hydrophenanthrene skeleton not provided for in groups C07J1/00 - C07J43/00
    • 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
    • C12N2310/11—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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Botany (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
CN201880057472.7A 2017-07-04 2018-07-02 用于分子的跨膜递送的化合物和方法 Pending CN111246855A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US15/641,251 2017-07-04
US15/641,251 US20190125884A1 (en) 2017-07-04 2017-07-04 Compounds and methods for trans-membrane delivery of molecules
US201715662665A 2017-07-28 2017-07-28
US15/662,665 2017-07-28
US201715679192A 2017-08-17 2017-08-17
US15/679,192 2017-08-17
US15/691,821 2017-08-31
US15/691,821 US10195286B2 (en) 2017-07-04 2017-08-31 Compounds and methods for trans-membrane delivery of molecules
PCT/IL2018/050714 WO2019008574A1 (en) 2017-07-04 2018-07-02 COMPOUNDS AND METHODS FOR TRANSMEMBRANE ADMINISTRATION OF MOLECULES

Publications (1)

Publication Number Publication Date
CN111246855A true CN111246855A (zh) 2020-06-05

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US (1) US10195286B2 (enExample)
EP (1) EP3648773A4 (enExample)
JP (1) JP7191869B2 (enExample)
CN (1) CN111246855A (enExample)
AU (1) AU2018295899B2 (enExample)
CA (1) CA3068165A1 (enExample)
IL (1) IL271788A (enExample)
WO (1) WO2019008574A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111615404A (zh) * 2018-01-01 2020-09-01 艾博森斯有限公司 用于分子的跨膜递送的化合物和方法

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20190104392A (ko) 2017-01-09 2019-09-09 아포센스 엘티디. 분자의 막투과 전달을 위한 화합물 및 방법
WO2019039403A1 (ja) 2017-08-22 2019-02-28 国立大学法人名古屋大学 修飾ポリヌクレオチド
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
US20220396830A1 (en) * 2021-05-13 2022-12-15 Promega Corporation Bioluminescent detection of dna synthesis
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

Citations (3)

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US20120035362A1 (en) * 2010-08-03 2012-02-09 Berry And Associates, Inc. Phosphoramidite derivatives of folic acid
US20130178512A1 (en) * 2007-12-04 2013-07-11 Alnylam Pharmaceuticals, Inc Carbohydrate conjugates as delivery agents for oligonucleotides
WO2017029664A1 (en) * 2015-08-20 2017-02-23 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1622572B1 (en) 2003-04-30 2017-12-20 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
MX2016012716A (es) 2014-03-28 2017-08-16 Aposense Ltd Compuestos y metodos para suministro trans-membrana de moleculas.
KR20190104392A (ko) * 2017-01-09 2019-09-09 아포센스 엘티디. 분자의 막투과 전달을 위한 화합물 및 방법

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130178512A1 (en) * 2007-12-04 2013-07-11 Alnylam Pharmaceuticals, Inc Carbohydrate conjugates as delivery agents for oligonucleotides
US20120035362A1 (en) * 2010-08-03 2012-02-09 Berry And Associates, Inc. Phosphoramidite derivatives of folic acid
US20170100486A1 (en) * 2014-03-28 2017-04-13 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
WO2017029664A1 (en) * 2015-08-20 2017-02-23 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules

Non-Patent Citations (1)

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Title
BHATIA.D，等: "DNA Conjugates:Improved Triplex Formation with 5-Amido(7-Deoxycholic Acid)-dU incorporated Oligonucleotides", 《BIOORGANIC & MEDICINAL CHEMISTRY LETTERS》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111615404A (zh) * 2018-01-01 2020-09-01 艾博森斯有限公司 用于分子的跨膜递送的化合物和方法
US12337036B2 (en) 2018-01-01 2025-06-24 Aposense Ltd Compounds and methods for trans-membrane delivery of molecules

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Publication number Publication date
IL271788A (en) 2020-02-27
CA3068165A1 (en) 2019-01-10
US10195286B2 (en) 2019-02-05
US20190008976A1 (en) 2019-01-10
EP3648773A4 (en) 2021-06-09
JP7191869B2 (ja) 2022-12-19
AU2018295899A1 (en) 2020-02-20
WO2019008574A1 (en) 2019-01-10
AU2018295899B2 (en) 2024-03-21
EP3648773A1 (en) 2020-05-13
JP2020525502A (ja) 2020-08-27

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