EP1651239A4 - Intravascular delivery of non-viral nucleic acid - Google Patents

Intravascular delivery of non-viral nucleic acid

Info

Publication number
EP1651239A4
EP1651239A4 EP03814414A EP03814414A EP1651239A4 EP 1651239 A4 EP1651239 A4 EP 1651239A4 EP 03814414 A EP03814414 A EP 03814414A EP 03814414 A EP03814414 A EP 03814414A EP 1651239 A4 EP1651239 A4 EP 1651239A4
Authority
EP
European Patent Office
Prior art keywords
cell
nucleic acid
viral nucleic
intravascular delivery
polynucleotide
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Withdrawn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
EP03814414A
Other languages
German (de)
French (fr)
Other versions
EP1651239A1 (en
Inventor
Jon A Wolff
James E Hagstrom
Vladimir G Budker
David B Rozema
Sean D Monahan
Paul M Slattum
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.)
Arrowhead Madison Inc
Original Assignee
Mirus Bio Corp
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
Priority claimed from US10/600,290 external-priority patent/US7148205B2/en
Priority claimed from US10/600,098 external-priority patent/US20030216347A1/en
Application filed by Mirus Bio Corp filed Critical Mirus Bio Corp
Publication of EP1651239A1 publication Critical patent/EP1651239A1/en
Publication of EP1651239A4 publication Critical patent/EP1651239A4/en
Withdrawn legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Disclosed is a process for transfecting genetic material into a mammalian cell to alter endogenous properties of the cell. The process comprises designing a polynucleotide for transfection. Then the polynucleotide is inserted into a mammalian vessel such as a tail vein or artery. Prior to insertion, subsequent to insertion, or concurrent with insertion the permeability of the vessel is increased thereby the genetic material is delivered to the parenchymal cell altering endogenous properties of the cell.
EP03814414A 2003-06-20 2003-07-22 Intravascular delivery of non-viral nucleic acid Withdrawn EP1651239A4 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US10/600,290 US7148205B2 (en) 1995-12-13 2003-06-20 Intravascular delivery of non-viral nucleic acid
US10/600,098 US20030216347A1 (en) 1999-02-26 2003-06-20 Intravascular delivery of non-viral nucleic acid
PCT/US2003/022817 WO2005004879A1 (en) 2003-06-20 2003-07-22 Intravascular delivery of non-viral nucleic acid

Publications (2)

Publication Number Publication Date
EP1651239A1 EP1651239A1 (en) 2006-05-03
EP1651239A4 true EP1651239A4 (en) 2006-12-20

Family

ID=34068491

Family Applications (1)

Application Number Title Priority Date Filing Date
EP03814414A Withdrawn EP1651239A4 (en) 2003-06-20 2003-07-22 Intravascular delivery of non-viral nucleic acid

Country Status (2)

Country Link
EP (1) EP1651239A4 (en)
WO (1) WO2005004879A1 (en)

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000050617A1 (en) * 1999-02-26 2000-08-31 Mirus Corporation Intravascular delivery of non-viral nucleic acid
US20010004636A1 (en) * 1995-12-13 2001-06-21 Sean D. Monahan Intravascular delivery of non-viral nucleic acid
US6265387B1 (en) * 1995-10-11 2001-07-24 Mirus, Inc. Process of delivering naked DNA into a hepatocyte via bile duct
US20020132788A1 (en) * 2000-11-06 2002-09-19 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
US20020137707A1 (en) * 1997-12-30 2002-09-26 Monahan Sean D. Intravascular delivery of non-viral nucleic acid
US20030092180A1 (en) * 2001-08-27 2003-05-15 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
WO2003080794A2 (en) * 2001-08-27 2003-10-02 Mirus Corporation Inhibition of rna function by delivery of inhibitors to animal cells

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5658892A (en) * 1993-01-15 1997-08-19 The General Hospital Corporation Compound delivery using high-pressure impulse transients
US5399352A (en) * 1993-04-14 1995-03-21 Emory University Device for local drug delivery and methods for using the same
US6177403B1 (en) * 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6265387B1 (en) * 1995-10-11 2001-07-24 Mirus, Inc. Process of delivering naked DNA into a hepatocyte via bile duct
US20010004636A1 (en) * 1995-12-13 2001-06-21 Sean D. Monahan Intravascular delivery of non-viral nucleic acid
US20020137707A1 (en) * 1997-12-30 2002-09-26 Monahan Sean D. Intravascular delivery of non-viral nucleic acid
WO2000050617A1 (en) * 1999-02-26 2000-08-31 Mirus Corporation Intravascular delivery of non-viral nucleic acid
US20020132788A1 (en) * 2000-11-06 2002-09-19 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
US20030092180A1 (en) * 2001-08-27 2003-05-15 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
WO2003080794A2 (en) * 2001-08-27 2003-10-02 Mirus Corporation Inhibition of rna function by delivery of inhibitors to animal cells

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BUDKER V ET AL: "THE EFFICIENT EXPRESSION OF INTRAVASCULARLY DELIVERED DNA IN RAT MUSCLE", GENE THERAPY, MACMILLAN PRESS LTD., BASINGSTOKE, GB, vol. 5, no. 2, February 1998 (1998-02-01), pages 272 - 276, XP002921644, ISSN: 0969-7128 *
GLASSPOOL-MALONE JILL ET AL: "Marked enhancement of direct respiratory tissue transfection by aurintricarboxylic acid", 1 July 1999, HUMAN GENE THERAPY, VOL. 10, NR. 10, PAGE(S) 1703-1713, ISSN: 1043-0342, XP002404913 *
See also references of WO2005004879A1 *

Also Published As

Publication number Publication date
EP1651239A1 (en) 2006-05-03
WO2005004879A1 (en) 2005-01-20

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Legal Events

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

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Owner name: MIRUS BIO CORPORATION

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Ipc: A61K 48/00 20060101AFI20061031BHEP

A4 Supplementary search report drawn up and despatched

Effective date: 20061120

RIN1 Information on inventor provided before grant (corrected)

Inventor name: ROZEMA, DAVID, B.

Inventor name: WOLFF, JON, A.

Inventor name: SLATTUM, PAUL, M.

Inventor name: MONAHAN, SEAN, D.

Inventor name: BUDKER, VLADIMIR, G.

Inventor name: HAGSTROM, JAMES, E.

RIN1 Information on inventor provided before grant (corrected)

Inventor name: HAGSTROM, JAMES, E.

Inventor name: ROZEMA, DAVID, B.

Inventor name: MONAHAN, SEAN, D.

Inventor name: WOLFF, JON, A.

Inventor name: BUDKER, VLADIMIR, G.

Inventor name: SLATTUM, PAUL, M.

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