WO2001019400A3 - Composition pharmaceutique sous forme d'un complexe acide nucleique-lipide, sa preparation et son utilisation en therapie genique - Google Patents

Composition pharmaceutique sous forme d'un complexe acide nucleique-lipide, sa preparation et son utilisation en therapie genique Download PDF

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Publication number
WO2001019400A3
WO2001019400A3 PCT/EP2000/008996 EP0008996W WO0119400A3 WO 2001019400 A3 WO2001019400 A3 WO 2001019400A3 EP 0008996 W EP0008996 W EP 0008996W WO 0119400 A3 WO0119400 A3 WO 0119400A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
pharmaceutical composition
gene therapy
production
acid lipid
Prior art date
Application number
PCT/EP2000/008996
Other languages
German (de)
English (en)
Other versions
WO2001019400A2 (fr
Inventor
Jens Schletter
Original Assignee
Cardiogene Gentherapeutische S
Jens Schletter
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 Cardiogene Gentherapeutische S, Jens Schletter filed Critical Cardiogene Gentherapeutische S
Priority to EP00967678A priority Critical patent/EP1216027A2/fr
Priority to AU16388/01A priority patent/AU1638801A/en
Publication of WO2001019400A2 publication Critical patent/WO2001019400A2/fr
Publication of WO2001019400A3 publication Critical patent/WO2001019400A3/fr

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Classifications

    • 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
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids

Abstract

Composition pharmaceutique sous forme d'un complexe acide nucléique-lipide, qui contient au moins un lipide cationique, au moins un lipide non cationique, au moins un acide nucléique codant pour une protéine destinée au traitement de maladies des vaisseaux, en particulier une protéine à propriétés vasodilatatrices et/ou angiogènes, et éventuellement des substances auxiliaires et/ou des adjuvants appropriés. Le lipide cationique (KL) contient un groupe dérivé du cholestérol auquel est relié au moins un groupe amino cationique choisi parmi des groupes amino primaire, secondaire et tertiaire et/ou un sel d'ammonium quaternaire, par l'intermédiaire d'un groupe de liaison, choisi parmi des carboxamides et des carbamoyles, et d'un bras d'espacement constitué par un groupe alkyle linéaire ou ramifié ayant 1 à 20 atomes de carbone. La taille du complexe acide nucléique-lipide est de l'ordre d'env. 300 à 800 nm. La présente invention concerne également la préparation de ladite composition pharmaceutique et son utilisation en thérapie génique.
PCT/EP2000/008996 1999-09-15 2000-09-14 Composition pharmaceutique sous forme d'un complexe acide nucleique-lipide, sa preparation et son utilisation en therapie genique WO2001019400A2 (fr)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP00967678A EP1216027A2 (fr) 1999-09-15 2000-09-14 Composition pharmaceutique sous forme d'un complexe acide nucleique-lipide, sa preparation et son utilisation en therapie genique
AU16388/01A AU1638801A (en) 1999-09-15 2000-09-14 Pharmaceutical composition in the form of a nucleic acid lipid complex, the production thereof and its use in gene therapy

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE1999144262 DE19944262A1 (de) 1999-09-15 1999-09-15 Pharmazeutische Zusammensetzung in Form eines Nukleinsäure-Lipid-Komplexes, ihre Herstellung und Verwendung in der Gentherapie
DE19944262.2 1999-09-15

Publications (2)

Publication Number Publication Date
WO2001019400A2 WO2001019400A2 (fr) 2001-03-22
WO2001019400A3 true WO2001019400A3 (fr) 2002-02-14

Family

ID=7922150

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2000/008996 WO2001019400A2 (fr) 1999-09-15 2000-09-14 Composition pharmaceutique sous forme d'un complexe acide nucleique-lipide, sa preparation et son utilisation en therapie genique

Country Status (4)

Country Link
EP (1) EP1216027A2 (fr)
AU (1) AU1638801A (fr)
DE (1) DE19944262A1 (fr)
WO (1) WO2001019400A2 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1512393A1 (fr) * 2003-09-08 2005-03-09 BOEHRINGER INGELHEIM PHARMA GMBH & CO. KG Procédé pour la production de liposomes et de lipoplexes homogènes

Citations (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996018372A2 (fr) * 1994-12-09 1996-06-20 Genzyme Corporation Amphiphiles cationiques et plasmides destines a la liberation intracellulaire de molecules therapeutiques
WO1996020208A2 (fr) * 1994-12-28 1996-07-04 Max-Delbrück-Centrum für Molekulare Medizin Nouveau derive de cholesterol pour le transfert genique liposomal
WO1996024333A1 (fr) * 1995-02-10 1996-08-15 California Institute Of Technology Constructions de lipides specifiques aux tissus du muscle lisse de la paroi vasculaire
WO1996027393A1 (fr) * 1995-03-07 1996-09-12 University Of Pittsburgh Formulation de poudre seche pour therapie genique
WO1996034109A1 (fr) * 1995-04-25 1996-10-31 Vical Incorporated Formulations en ampoule unidose de complexes adn/lipides
WO1996040265A1 (fr) * 1995-06-07 1996-12-19 The Regents Of The University Of California Stabilisation de complexes de polynucleotides
WO1996040964A2 (fr) * 1995-06-07 1996-12-19 Inex Pharmaceuticals Corporation Particules d'acides nucleiques et de lipides preparees au moyen d'un intermediaire de complexe hydrophobe d'acides nucleiques et de lipides et utilisation pour transferer des genes
WO1996040067A1 (fr) * 1995-06-07 1996-12-19 Aronex Pharmaceuticals, Inc. Sel d'acide de lipide cationique de 3 beta [n-(n',n'-dimethylaminoethane)-carbamyle]cholesterol
US5658565A (en) * 1994-06-24 1997-08-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Inducible nitric oxide synthase gene for treatment of disease
WO1998044934A1 (fr) * 1997-04-07 1998-10-15 Rutgers, The State University Of New Jersey Therapie genique de carcinomes fondee sur la cytokine et le cisplatine
EP0891780A2 (fr) * 1997-07-11 1999-01-20 Cardiogene Gentherapeutische Systeme AG Systèmes de transfection, leur préparation et application en thérapie génétique

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE4411402A1 (de) * 1994-03-31 1995-10-05 Juergen Schrader DNA-Expressionsvektoren zur Verwendung in der gentherapeutischen Behandlung von Gefäßerkrankungen
US5811406A (en) * 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
PT941066E (pt) * 1996-08-26 2004-03-31 Transgene Sa Complexos de lipido cationico - acido nucleico

Patent Citations (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5658565A (en) * 1994-06-24 1997-08-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Inducible nitric oxide synthase gene for treatment of disease
WO1996018372A2 (fr) * 1994-12-09 1996-06-20 Genzyme Corporation Amphiphiles cationiques et plasmides destines a la liberation intracellulaire de molecules therapeutiques
WO1996020208A2 (fr) * 1994-12-28 1996-07-04 Max-Delbrück-Centrum für Molekulare Medizin Nouveau derive de cholesterol pour le transfert genique liposomal
WO1996024333A1 (fr) * 1995-02-10 1996-08-15 California Institute Of Technology Constructions de lipides specifiques aux tissus du muscle lisse de la paroi vasculaire
WO1996027393A1 (fr) * 1995-03-07 1996-09-12 University Of Pittsburgh Formulation de poudre seche pour therapie genique
WO1996034109A1 (fr) * 1995-04-25 1996-10-31 Vical Incorporated Formulations en ampoule unidose de complexes adn/lipides
WO1996040265A1 (fr) * 1995-06-07 1996-12-19 The Regents Of The University Of California Stabilisation de complexes de polynucleotides
WO1996040964A2 (fr) * 1995-06-07 1996-12-19 Inex Pharmaceuticals Corporation Particules d'acides nucleiques et de lipides preparees au moyen d'un intermediaire de complexe hydrophobe d'acides nucleiques et de lipides et utilisation pour transferer des genes
WO1996040067A1 (fr) * 1995-06-07 1996-12-19 Aronex Pharmaceuticals, Inc. Sel d'acide de lipide cationique de 3 beta [n-(n',n'-dimethylaminoethane)-carbamyle]cholesterol
WO1998044934A1 (fr) * 1997-04-07 1998-10-15 Rutgers, The State University Of New Jersey Therapie genique de carcinomes fondee sur la cytokine et le cisplatine
EP0891780A2 (fr) * 1997-07-11 1999-01-20 Cardiogene Gentherapeutische Systeme AG Systèmes de transfection, leur préparation et application en thérapie génétique

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ANCHORDOQUY, THOMAS J. ET AL: "Maintenance of transfection rates and physical characterization of lipid/ DNA complexes after freeze-drying and rehydration", ARCH. BIOCHEM. BIOPHYS. (1997), 348(1), 199-206, XP000992258 *
GAO X ET AL: "A NOVEL CATIONIC LIPOSOME REAGENT FOR EFFICIENT TRANSFECTION OF MAMMALIAN CELLS", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS,US,ACADEMIC PRESS INC. ORLANDO, FL, vol. 179, no. 1, 30 August 1991 (1991-08-30), pages 280 - 285, XP000572654, ISSN: 0006-291X *
NABEL, ELIZABETH G. ET AL: "Methods for liposome-mediated gene transfer to the arterial wall", GENE THER. PROTOC. (1997), 127-133. EDITOR(S): ROBBINS, PAUL D. PUBLISHER: HUMANA, TOTOWA, N. J., XP000992423 *

Also Published As

Publication number Publication date
WO2001019400A2 (fr) 2001-03-22
EP1216027A2 (fr) 2002-06-26
DE19944262A1 (de) 2001-03-29
AU1638801A (en) 2001-04-17

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