EP1689230A4 - Compositions and methods for systemic nucleic acid sequence delivery - Google Patents

Compositions and methods for systemic nucleic acid sequence delivery

Info

Publication number
EP1689230A4
EP1689230A4 EP04810994A EP04810994A EP1689230A4 EP 1689230 A4 EP1689230 A4 EP 1689230A4 EP 04810994 A EP04810994 A EP 04810994A EP 04810994 A EP04810994 A EP 04810994A EP 1689230 A4 EP1689230 A4 EP 1689230A4
Authority
EP
European Patent Office
Prior art keywords
compositions
methods
nucleic acid
acid sequence
sequence delivery
Prior art date
Legal status (The legal status 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 status listed.)
Withdrawn
Application number
EP04810994A
Other languages
German (de)
French (fr)
Other versions
EP1689230A2 (en
Inventor
Jeffrey S Chamberlain
Paul Gregorevic
Michael J Blankinship
James M Allen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Washington
Original Assignee
University of Washington
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 University of Washington filed Critical University of Washington
Priority claimed from PCT/US2004/038060 external-priority patent/WO2005049850A2/en
Publication of EP1689230A2 publication Critical patent/EP1689230A2/en
Publication of EP1689230A4 publication Critical patent/EP1689230A4/en
Withdrawn legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/10—Peptides having 12 to 20 amino acids
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
EP04810994A 2003-11-14 2004-11-15 Compositions and methods for systemic nucleic acid sequence delivery Withdrawn EP1689230A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US52046003P 2003-11-14 2003-11-14
PCT/US2004/038060 WO2005049850A2 (en) 2003-11-14 2004-11-15 Compositions and methods for systemic nucleic acid sequence delivery

Publications (2)

Publication Number Publication Date
EP1689230A2 EP1689230A2 (en) 2006-08-16
EP1689230A4 true EP1689230A4 (en) 2009-09-16

Family

ID=36649334

Family Applications (1)

Application Number Title Priority Date Filing Date
EP04810994A Withdrawn EP1689230A4 (en) 2003-11-14 2004-11-15 Compositions and methods for systemic nucleic acid sequence delivery

Country Status (1)

Country Link
EP (1) EP1689230A4 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999055379A1 (en) * 1998-04-30 1999-11-04 Mirus Corporation A process of delivering a polynucleotide to a cell via the vascular system
WO2002063025A2 (en) * 2001-02-06 2002-08-15 Avigen, Inc. Muscle-directed gene therapy with aav-1 and aav-6 vectors
WO2002088347A2 (en) * 2001-04-25 2002-11-07 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Aav helper plasmids for helper virus-free packaging and pseudo typification of aav vectors

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999055379A1 (en) * 1998-04-30 1999-11-04 Mirus Corporation A process of delivering a polynucleotide to a cell via the vascular system
WO2002063025A2 (en) * 2001-02-06 2002-08-15 Avigen, Inc. Muscle-directed gene therapy with aav-1 and aav-6 vectors
WO2002088347A2 (en) * 2001-04-25 2002-11-07 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Aav helper plasmids for helper virus-free packaging and pseudo typification of aav vectors

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
GREELISH J P ET AL: "STABLE RESTORATION OF THE SARCOGLYCAN COMPLEX IN DYSTROPHIC MUSCLE PERFUSED WITH HISTAMINE AND A RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR", NATURE MEDICINE, NATURE PUBLISHING GROUP, NEW YORK, NY, US, vol. 5, no. 4, 1 April 1999 (1999-04-01), pages 439 - 443, XP002920731, ISSN: 1078-8956 *
GREGOREVIC P ET AL: "Systemic delivery of genes to striated muscles using adeno-associated viral vectors", NATURE MEDICINE, NATURE PUBLISHING GROUP, NEW YORK, NY, US, vol. 10, no. 8, 1 August 2004 (2004-08-01), pages 828 - 834, XP002314366, ISSN: 1078-8956 *
TOI M ET AL: "Vascular endothelial growth factor: its prognostic, predictive, and therapeutic implications", LANCET ONCOLOGY, LANCET PUBLISHING GROUP, LONDON, GB, vol. 2, no. 11, 1 November 2001 (2001-11-01), pages 667 - 673, XP004811782, ISSN: 1470-2045 *
XIAO ET AL: "EFFICIENT LONG-TERM GENE TRANSFER INTO MUSCLE TISSUE OF IMMUNOCOMPETENT MICE BY ADENO-ASSOCIATED VIRUS VECTOR", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US, vol. 70, no. 11, 1 November 1996 (1996-11-01), pages 8098 - 8108, XP002082892, ISSN: 0022-538X *

Also Published As

Publication number Publication date
EP1689230A2 (en) 2006-08-16

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Legal Events

Date Code Title Description
PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

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Effective date: 20060614

AK Designated contracting states

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Designated state(s): AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IS IT LI LU MC NL PL PT RO SE SI SK TR

AX Request for extension of the european patent

Extension state: AL HR LT LV MK YU

RIN1 Information on inventor provided before grant (corrected)

Inventor name: ALLEN, JAMES, M.

Inventor name: BLANKINSHIP, MICHAEL, J.

Inventor name: GREGOREVIC, PAUL

Inventor name: CHAMBERLAIN, JEFFREY, S.

RIC1 Information provided on ipc code assigned before grant

Ipc: A61K 31/70 20060101AFI20071211BHEP

A4 Supplementary search report drawn up and despatched

Effective date: 20090818

RIC1 Information provided on ipc code assigned before grant

Ipc: A61K 48/00 20060101AFI20090812BHEP

17Q First examination report despatched

Effective date: 20100705

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18D Application deemed to be withdrawn

Effective date: 20121023