WO2001019400A3 - Pharmaceutical composition in the form of a nucleic acid lipid complex, the production thereof and its use in gene therapy - Google Patents

Pharmaceutical composition in the form of a nucleic acid lipid complex, the production thereof and its use in gene therapy 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)
French (fr)
Other versions
WO2001019400A2 (en
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/en
Priority to AU16388/01A priority patent/AU1638801A/en
Publication of WO2001019400A2 publication Critical patent/WO2001019400A2/en
Publication of WO2001019400A3 publication Critical patent/WO2001019400A3/en

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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

The invention relates to a pharmaceutical composition in the form of a nucleic acid lipid complex containing at least one cationic lipid, at least one non-cationic lipid, at least one nucleic acid that codes for a protein used for treating vascular diseases, especially a protein having vasodilatory and/or angiogenic properties, and optionally containing appropriate auxiliary and/or addition agents. According to the invention, the cationic lipid (KL) contains a group derived from cholesterol on which at least one cationic amino group selected from a primary, secondary, tertiary amino group and/or from a quaternary ammonium salt is bound via a connecting group selected from carboxamides and carbamoyls, and via a spacer comprised of a linear or branched alkyl group with 1 to 20 carbon atoms. In addition, the size of the nucleic acid lipid complexes ranges from approximately 300 to 800 nm. The invention also relates to the production of the pharmaceutical composition and to its use in gene therapy.
PCT/EP2000/008996 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 WO2001019400A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP00967678A EP1216027A2 (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
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 (en) 1999-09-15 1999-09-15 Pharmaceutical composition in the form of a nucleic acid-lipid complex, its production and use in gene therapy
DE19944262.2 1999-09-15

Publications (2)

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

Family

ID=7922150

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2000/008996 WO2001019400A2 (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

Country Status (4)

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

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1512393A1 (en) * 2003-09-08 2005-03-09 BOEHRINGER INGELHEIM PHARMA GMBH & CO. KG Process for the production of homogeneous liposomes and lipoplexes

Citations (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996018372A2 (en) * 1994-12-09 1996-06-20 Genzyme Corporation Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules
WO1996020208A2 (en) * 1994-12-28 1996-07-04 Max-Delbrück-Centrum für Molekulare Medizin New cholesterol derivative for liposomal gene transfer
WO1996024333A1 (en) * 1995-02-10 1996-08-15 California Institute Of Technology Lipid constructs for targeting to vascular smooth muscle tissue
WO1996027393A1 (en) * 1995-03-07 1996-09-12 University Of Pittsburgh A dry powder formulation for gene therapy
WO1996034109A1 (en) * 1995-04-25 1996-10-31 Vical Incorporated Single-vial formulations of dna/lipid complexes
WO1996040067A1 (en) * 1995-06-07 1996-12-19 Aronex Pharmaceuticals, Inc. Cationic lipid acid salt of 3 beta [n-(n',n'-dimethylaminoethane)-carbamoyl] cholesterol
WO1996040964A2 (en) * 1995-06-07 1996-12-19 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
WO1996040265A1 (en) * 1995-06-07 1996-12-19 The Regents Of The University Of California Stabilization of polynucleotide complexes
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 (en) * 1997-04-07 1998-10-15 Rutgers, The State University Of New Jersey Cytokine and cisplatin-based gene therapy of carcinomas
EP0891780A2 (en) * 1997-07-11 1999-01-20 Cardiogene Gentherapeutische Systeme AG Transfection systems, their preparation and application in Gentherapy

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE4411402A1 (en) * 1994-03-31 1995-10-05 Juergen Schrader DNA expression vectors for use in gene therapy treatment of vascular diseases
US5811406A (en) * 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
CA2264140A1 (en) * 1996-08-26 1998-03-05 Transgene S.A. Cationic lipid-nucleic acid complexes

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 (en) * 1994-12-09 1996-06-20 Genzyme Corporation Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules
WO1996020208A2 (en) * 1994-12-28 1996-07-04 Max-Delbrück-Centrum für Molekulare Medizin New cholesterol derivative for liposomal gene transfer
WO1996024333A1 (en) * 1995-02-10 1996-08-15 California Institute Of Technology Lipid constructs for targeting to vascular smooth muscle tissue
WO1996027393A1 (en) * 1995-03-07 1996-09-12 University Of Pittsburgh A dry powder formulation for gene therapy
WO1996034109A1 (en) * 1995-04-25 1996-10-31 Vical Incorporated Single-vial formulations of dna/lipid complexes
WO1996040067A1 (en) * 1995-06-07 1996-12-19 Aronex Pharmaceuticals, Inc. Cationic lipid acid salt of 3 beta [n-(n',n'-dimethylaminoethane)-carbamoyl] cholesterol
WO1996040964A2 (en) * 1995-06-07 1996-12-19 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
WO1996040265A1 (en) * 1995-06-07 1996-12-19 The Regents Of The University Of California Stabilization of polynucleotide complexes
WO1998044934A1 (en) * 1997-04-07 1998-10-15 Rutgers, The State University Of New Jersey Cytokine and cisplatin-based gene therapy of carcinomas
EP0891780A2 (en) * 1997-07-11 1999-01-20 Cardiogene Gentherapeutische Systeme AG Transfection systems, their preparation and application in Gentherapy

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 (en) 2001-03-22
DE19944262A1 (en) 2001-03-29
AU1638801A (en) 2001-04-17
EP1216027A2 (en) 2002-06-26

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