WO2001000241A3 - Novel vector complexes and their use in gene therapy - Google Patents

Novel vector complexes and their use in gene therapy Download PDF

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Publication number
WO2001000241A3
WO2001000241A3 PCT/EP2000/005371 EP0005371W WO0100241A3 WO 2001000241 A3 WO2001000241 A3 WO 2001000241A3 EP 0005371 W EP0005371 W EP 0005371W WO 0100241 A3 WO0100241 A3 WO 0100241A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene therapy
component
novel vector
vector complexes
relates
Prior art date
Application number
PCT/EP2000/005371
Other languages
German (de)
French (fr)
Other versions
WO2001000241A2 (en
Inventor
Hans-Harald Sedlacek
Sabine Bruesselbach
Thomas Kissel
Rolf Mueller
Original Assignee
Aventis Pharma Gmbh
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 filed Critical Aventis Pharma Gmbh
Priority to EP00947840A priority Critical patent/EP1196618A2/en
Priority to AU61496/00A priority patent/AU6149600A/en
Priority to JP2001505948A priority patent/JP2003503362A/en
Publication of WO2001000241A2 publication Critical patent/WO2001000241A2/en
Publication of WO2001000241A3 publication Critical patent/WO2001000241A3/en

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Classifications

    • 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

Abstract

The invention relates to a vector complex, containing the following components: a) a nucleic acid sequence of any length; b) a cationic carrier; c) a polymer which has more than 3 cationic charges and more than 3 anionic charges; and d) optionally a ligand which bonds with component a) or component b) or component c) and has at the same time a binding site for a target cell. The invention also relates to the production of said vector complex and the use thereof in the prophylaxis and treatment of diseases.
PCT/EP2000/005371 1999-06-24 2000-06-10 Novel vector complexes and their use in gene therapy WO2001000241A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP00947840A EP1196618A2 (en) 1999-06-24 2000-06-10 Novel vector complexes and their use in gene therapy
AU61496/00A AU6149600A (en) 1999-06-24 2000-06-10 Novel vector complexes and their use in gene therapy
JP2001505948A JP2003503362A (en) 1999-06-24 2000-06-10 Novel vector conjugates and their use for gene therapy

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE1999129104 DE19929104A1 (en) 1999-06-24 1999-06-24 Vector complex, useful for gene therapy of e.g. tumors, comprises nucleic acid, cationic carrier, charged polymer and targeting ligand
DE19929104.7 1999-06-24

Publications (2)

Publication Number Publication Date
WO2001000241A2 WO2001000241A2 (en) 2001-01-04
WO2001000241A3 true WO2001000241A3 (en) 2001-08-09

Family

ID=7912489

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2000/005371 WO2001000241A2 (en) 1999-06-24 2000-06-10 Novel vector complexes and their use in gene therapy

Country Status (5)

Country Link
EP (1) EP1196618A2 (en)
JP (1) JP2003503362A (en)
AU (1) AU6149600A (en)
DE (1) DE19929104A1 (en)
WO (1) WO2001000241A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060141019A1 (en) * 2002-11-15 2006-06-29 Toshiya Kai Liposome
JP2005298486A (en) * 2004-03-15 2005-10-27 Nipro Corp Liposome-containing medicinal composition for treating cancer

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4847240A (en) * 1978-01-16 1989-07-11 The Trustees Of Boston University Method of effecting cellular uptake of molecules
US5354844A (en) * 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5574142A (en) * 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
WO1997012051A1 (en) * 1995-09-28 1997-04-03 Rhone-Poulenc Rorer S.A. Pharmaceutical composition useful for nucleic acid transfection, and use thereof
WO1998006869A1 (en) * 1996-08-14 1998-02-19 The Children's Hospital Of Philadelphia Non-viral vehicles for use in gene transfer
EP0846772A1 (en) * 1996-11-29 1998-06-10 Hoechst Aktiengesellschaft Multifunctional ligand system for cell-specific transfer of nucleic acid
US5972707A (en) * 1994-06-27 1999-10-26 The Johns Hopkins University Gene delivery system

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4119590A (en) * 1975-05-22 1978-10-10 Toyo Soda Manufacturing Co., Ltd. Process for producing a polyion complex having a nucleic acid base
US5908777A (en) * 1995-06-23 1999-06-01 University Of Pittsburgh Lipidic vector for nucleic acid delivery
DE19605279A1 (en) * 1996-02-13 1997-08-14 Hoechst Ag Target cell-specific vectors for the introduction of genes into cells, drugs containing such vectors and their use
GB9623051D0 (en) * 1996-11-06 1997-01-08 Schacht Etienne H Delivery of DNA to target cells in biological systems

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4847240A (en) * 1978-01-16 1989-07-11 The Trustees Of Boston University Method of effecting cellular uptake of molecules
US5354844A (en) * 1989-03-16 1994-10-11 Boehringer Ingelheim International Gmbh Protein-polycation conjugates
US5792645A (en) * 1989-03-16 1998-08-11 Boehringer Ingelheim International Gmbh Protein-polycation nucleic acid complexes and methods of use
US5574142A (en) * 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US5972707A (en) * 1994-06-27 1999-10-26 The Johns Hopkins University Gene delivery system
WO1997012051A1 (en) * 1995-09-28 1997-04-03 Rhone-Poulenc Rorer S.A. Pharmaceutical composition useful for nucleic acid transfection, and use thereof
WO1998006869A1 (en) * 1996-08-14 1998-02-19 The Children's Hospital Of Philadelphia Non-viral vehicles for use in gene transfer
EP0846772A1 (en) * 1996-11-29 1998-06-10 Hoechst Aktiengesellschaft Multifunctional ligand system for cell-specific transfer of nucleic acid

Non-Patent Citations (11)

* Cited by examiner, † Cited by third party
Title
BONFILS E ET AL: "DRUG TARGETING: SYNTHESIS AND ENDOCYTOSIS OF OLIGONUCLEOTIDE- NEOGLYCOPROTEIN CONJUGATES", NUCLEIC ACIDS RESEARCH,GB,OXFORD UNIVERSITY PRESS, SURREY, vol. 20, no. 17, 11 September 1992 (1992-09-11), pages 4621 - 4629, XP000461068, ISSN: 0305-1048 *
DASH P R ET AL: "Factors affecting blood clearance and in vivo distribution of polyelectrolyte complexes for gene delivery.", GENE THERAPY, vol. 6, no. 4, April 1999 (1999-04-01), pages 643 - 650, XP000982444, ISSN: 0969-7128 *
GOTTSCHALK S ET AL: "A NOVEL DNA-PEPTIDE COMPLEX FOR EFFICIENT GENE TRANSFER AND EXPRESSION IN MAMMALIAN CELLS", GENE THERAPY,GB,MACMILLAN PRESS LTD., BASINGSTOKE, vol. 3, no. 5, 1 May 1996 (1996-05-01), pages 448 - 457, XP000577712, ISSN: 0969-7128 *
GUPTA P K ET AL: "ALBUMIN MICROSPHERES II: APPLICATIONS IN DRUG DELIVERY", JOURNAL OF MICROENCAPSULATION,GB,TAYLOR AND FRANCIS INC. LONDON, vol. 6, no. 4, 1 October 1989 (1989-10-01), pages 463 - 472, XP000086853, ISSN: 0265-2048 *
HAN JUNGHEE ET AL: "Receptor-mediated gene transfer to cells of hepatic origin by galactosylated albumin-polylysine complexes.", BIOLOGICAL & PHARMACEUTICAL BULLETIN, vol. 22, no. 8, August 1999 (1999-08-01), pages 836 - 840, XP000979353, ISSN: 0918-6158 *
HAN JUNGHEE ET AL: "Receptor-mediated gene transfer to cells of hepatic origin by galactosylated albumin-polylysine conjugates.", PHARMACEUTICAL RESEARCH (NEW YORK), vol. 14, no. 11 SUPPL., November 1997 (1997-11-01), & Annual Meeting of the American Association of Pharmaceutical Scientists; Boston, US; November 2-6, 1997, pages S55, Zusammenfassung Nummer 1171,, XP000982439, ISSN: 0724-8741 *
INTERNATIONAL SYMPOSIUM ON CONTROLLED RELEASE BIOACTIVE MATERIALS, 20 June 1999 (1999-06-20) - 25 June 1999 (1999-06-25), BOSTON, US *
KINSEY B. ET AL: "Polmethyleneimine conjugated to macroaggregated albumin mediates high level transfection in vitro and in vivo", PROCEEDINGS OF THE CONTROLLED RELEASE SOCIETY, 1999, NO. 26, PAGE(S) 619-620, XP000982539 *
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 *
SHEN W -C ET AL: "CONJUGATION OF POLY-L-LYSINE TO ALBUMIN AND HORSERADISH PEROXIDASE:A NOVEL METHOD OF ENHANCING THE CELLULAR UPTAKE OF PROTEINS", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES,NATIONAL ACADEMY OF SCIENCE, WASHINGTON, DC,US, vol. 75, no. 4, April 1978 (1978-04-01), pages 1872 - 1876, XP000953474, ISSN: 0027-8424 *
WU C H ET AL: "TARGETING GENES: DELIVERY AND PERSISTENT EXPRESSION OF A FOREIGN GENE DRIVEN BY MAMMALIAN REGULATORY ELEMENTS IN VIVO", JOURNAL OF BIOLOGICAL CHEMISTRY,US,AMERICAN SOCIETY OF BIOLOGICAL CHEMISTS, BALTIMORE, MD, vol. 264, no. 29, 15 October 1989 (1989-10-15), pages 16985 - 16987, XP002030967, ISSN: 0021-9258 *

Also Published As

Publication number Publication date
AU6149600A (en) 2001-01-31
EP1196618A2 (en) 2002-04-17
WO2001000241A2 (en) 2001-01-04
JP2003503362A (en) 2003-01-28
DE19929104A1 (en) 2000-12-28

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