WO2000074646A3 - Neue liposomale vektorkomplexe und deren verwendung für die gentherapie - Google Patents

Neue liposomale vektorkomplexe und deren verwendung für die gentherapie Download PDF

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Publication number
WO2000074646A3
WO2000074646A3 PCT/EP2000/004678 EP0004678W WO0074646A3 WO 2000074646 A3 WO2000074646 A3 WO 2000074646A3 EP 0004678 W EP0004678 W EP 0004678W WO 0074646 A3 WO0074646 A3 WO 0074646A3
Authority
WO
WIPO (PCT)
Prior art keywords
lysosomolytic
lysosomotropic
gene therapy
liposomal vector
novel liposomal
Prior art date
Application number
PCT/EP2000/004678
Other languages
English (en)
French (fr)
Other versions
WO2000074646A2 (de
Inventor
Sabine Bruesselbach
Kristina Mueller
Alfred Fahr
Original Assignee
Aventis Pharma Gmbh
Sabine Bruesselbach
Kristina Mueller
Alfred Fahr
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Aventis Pharma Gmbh, Sabine Bruesselbach, Kristina Mueller, Alfred Fahr filed Critical Aventis Pharma Gmbh
Priority to JP2001501183A priority Critical patent/JP2003501373A/ja
Priority to EP00929548A priority patent/EP1187929A2/de
Priority to AU47583/00A priority patent/AU4758300A/en
Priority to CA002375854A priority patent/CA2375854A1/en
Publication of WO2000074646A2 publication Critical patent/WO2000074646A2/de
Publication of WO2000074646A3 publication Critical patent/WO2000074646A3/de

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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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • 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
    • A61K47/69—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/555—Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511—Organic adjuvants
    • A61K2039/55555—Liposomes; Vesicles, e.g. nanoparticles; Spheres, e.g. nanospheres; Polymers

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Dispersion Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Die Erfindung bezieht sich auf einen neuen liposomalen Vektorkomplex enthaltend folgende Komponenten: a) eine Nukleinsäuresequenz beliebiger Länge; b) einen kationischen Träger, welcher die Komponente a) kondensiert und lysosomolytisch und/oder lysosomotrop ist; c) Lipide und Phospholipide, welche ein Liposom bilden; d) optional einen Liganden, welcher eine Bindungsstelle für eine Zielzelle hat; und e) optional eine fusogene Substanz, welche die lysosomolytische und/oder lysosomotrope Funktion der Komponente b) ersetzen kann; wobei bei Vorhandensein einer fusogenen Substanz (e), der kationische Träger (b) nicht lysosomolytisch und/oder lysosomotrop sein muß, seine Herstellung und Verwendung.
PCT/EP2000/004678 1999-06-02 2000-05-23 Neue liposomale vektorkomplexe und deren verwendung für die gentherapie WO2000074646A2 (de)

Priority Applications (4)

Application Number Priority Date Filing Date Title
JP2001501183A JP2003501373A (ja) 1999-06-02 2000-05-23 新規なリポソームベクター複合体および遺伝子療法でのそれらの使用
EP00929548A EP1187929A2 (de) 1999-06-02 2000-05-23 Neue liposomale vektorkomplexe und deren verwendung für die gentherapie
AU47583/00A AU4758300A (en) 1999-06-02 2000-05-23 Novel liposomal vector complexes and their use in gene therapy
CA002375854A CA2375854A1 (en) 1999-06-02 2000-05-23 Novel liposomal vector complexes and their use in gene therapy

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE19925143.6 1999-06-02
DE19925143A DE19925143A1 (de) 1999-06-02 1999-06-02 Neue liposomale Vektorkomplexe und deren Verwendung für die Gentherapie

Publications (2)

Publication Number Publication Date
WO2000074646A2 WO2000074646A2 (de) 2000-12-14
WO2000074646A3 true WO2000074646A3 (de) 2001-08-09

Family

ID=7909923

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2000/004678 WO2000074646A2 (de) 1999-06-02 2000-05-23 Neue liposomale vektorkomplexe und deren verwendung für die gentherapie

Country Status (6)

Country Link
EP (1) EP1187929A2 (de)
JP (1) JP2003501373A (de)
AU (1) AU4758300A (de)
CA (1) CA2375854A1 (de)
DE (1) DE19925143A1 (de)
WO (1) WO2000074646A2 (de)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT103865A (pt) * 2007-10-25 2009-05-15 Univ De Coimbra Nano-transportadores de base lipídica para entrega direccionada de vectores virais e processo para a sua produção
CN101270168B (zh) * 2008-05-13 2011-06-22 中国药科大学 透明质酸接枝聚乙烯亚胺共聚物、制备方法及其作为基因载体的应用
WO2012006101A2 (en) 2010-06-28 2012-01-12 The General Hospital Corporation Blood substitutes and uses thereof
JP2020500020A (ja) 2016-11-14 2020-01-09 ノバルティス アーゲー 融合誘導性タンパク質ｍｉｎｉｏｎに関連する組成物、方法、および治療上の使用

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997012051A1 (fr) * 1995-09-28 1997-04-03 Rhone-Poulenc Rorer S.A. Composition pharmaceutique utile pour la transfection d'acides nucleiques et ses utilisations
EP0784984A2 (de) * 1996-01-17 1997-07-23 F. Hoffmann-La Roche Ag Transfektion kompetenter Molekülen
WO1998049350A1 (en) * 1997-04-30 1998-11-05 Regents Of The University Of Minnesota In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes
WO1999051629A2 (de) * 1998-04-07 1999-10-14 Roche Diagnostics Gmbh New compounds
WO2000003683A2 (en) * 1998-07-20 2000-01-27 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
US6051429A (en) * 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6051429A (en) * 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
WO1997012051A1 (fr) * 1995-09-28 1997-04-03 Rhone-Poulenc Rorer S.A. Composition pharmaceutique utile pour la transfection d'acides nucleiques et ses utilisations
EP0784984A2 (de) * 1996-01-17 1997-07-23 F. Hoffmann-La Roche Ag Transfektion kompetenter Molekülen
WO1998049350A1 (en) * 1997-04-30 1998-11-05 Regents Of The University Of Minnesota In vivo use of recombinagenic oligonucleobases to correct genetic lesions in hepatocytes
WO1999051629A2 (de) * 1998-04-07 1999-10-14 Roche Diagnostics Gmbh New compounds
WO2000003683A2 (en) * 1998-07-20 2000-01-27 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes

Non-Patent Citations (9)

* Cited by examiner, † Cited by third party
Title
BANDYOPADHYAY P ET AL: "Liposome-encapsulation of polyethylenimine-compacted DNA for gene delivery to liver cells.", PHARMACEUTICAL RESEARCH (NEW YORK), vol. 14, no. 11 SUPPL., November 1997 (1997-11-01), & Annual Meeting of the American Association of Pharmaceutical Scientists; Boston, MA, USA; November 2-6, 1997, pages S60, Zusammenfassung Nr. 1190,, XP000982344, ISSN: 0724-8741 *
BANDYOPADHYAY PARAMITA ET AL: "Enhanced gene transfer into HuH-7 cells and primary rat hepatocytes using targeted liposomes and polyethylenimine.", BIOTECHNIQUES, vol. 25, no. 2, August 1998 (1998-08-01), pages 282, 284, 286 - 292, XP002161156, ISSN: 0736-6205 *
BANDYOPADHYAY PARAMITA ET AL: "Nucleotide exchange in genomic DNA of rat hepatocytes using RNA/DNA oligonucleotides: Targeted delivery of liposomes and polyethyleneimine to the asialoglycoprotein receptor.", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 274, no. 15, 9 April 1999 (1999-04-09), pages 10163 - 10172, XP002161157, ISSN: 0021-9258 *
GAGNE LUCIE ET AL: "Gene delivery to hepatocytes using serum-stable, targeted anionic liposomes.", JOURNAL OF LIPOSOME RESEARCH, vol. 8, no. 1, February 1998 (1998-02-01), & Sixth Liposome Research Days Conference; Les Embiez, France; May 28-31, 1998, pages 57 - 58, XP002161161, ISSN: 0898-2104 *
HARBOTTLE RICHARD P ET AL: "An RGD-oligolysine peptide: A prototype construct for integrin-mediated gene delivery.", HUMAN GENE THERAPY, vol. 9, no. 7, 1 May 1998 (1998-05-01), pages 1037 - 1047, XP000982341, ISSN: 1043-0342 *
KUROHANE K ET AL: "Inhibitory effect of liposomes modified with a structural mimetic of Arg-Gly-Asp on tumor metastasis.", JOURNAL OF LIPOSOME RESEARCH, vol. 8, no. 1, February 1998 (1998-02-01), & Sixth Liposome Research Days Conference; Les Embiez, France; May 28-31, 1998, pages 74, XP002161160, ISSN: 0898-2104 *
LAKKARAJU A ET AL: "Protection from neuronal excitotoxicity by anionic liposomes encapsulating unmodified p53 antisense oligodeoxyribonucleotides.", JOURNAL OF LIPOSOME RESEARCH, vol. 8, no. 1, February 1998 (1998-02-01), & Sixth Liposome Research Days Conference; Les Embiez, France; May 28-31, 1998, pages 74 - 75, XP002161159, ISSN: 0898-2104 *
SANDIG M ET AL: "The homophilic binding site of the neural cell adhesion molecule NCAM is directly involved in promoting neurite outgrowth from cultured neural retinal cells", JOURNAL OF BIOLOGICAL CHEMISTRY,US,AMERICAN SOCIETY OF BIOLOGICAL CHEMISTS, BALTIMORE, MD, vol. 269, no. 20, 20 May 1994 (1994-05-20), pages 14841 - 14848, XP002104481, ISSN: 0021-9258 *
TURNER C ET AL: "Characterisation of peptide condensed DNA/anionic liposome gene therapy vectors.", JOURNAL OF LIPOSOME RESEARCH, vol. 8, no. 1, February 1998 (1998-02-01), & Sixth Liposome Research Days Conference; Les Embiez, France; May 28-31, 1998, pages 114 - 115, XP002161158, ISSN: 0898-2104 *

Also Published As

Publication number Publication date
EP1187929A2 (de) 2002-03-20
AU4758300A (en) 2000-12-28
CA2375854A1 (en) 2000-12-14
WO2000074646A2 (de) 2000-12-14
DE19925143A1 (de) 2000-12-07
JP2003501373A (ja) 2003-01-14

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