WO2004007721A1 - Conjugue de liberation de sequences d'arn double brin et methodes associees - Google Patents

Conjugue de liberation de sequences d'arn double brin et methodes associees Download PDF

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Publication number
WO2004007721A1
WO2004007721A1 PCT/IB2003/002828 IB0302828W WO2004007721A1 WO 2004007721 A1 WO2004007721 A1 WO 2004007721A1 IB 0302828 W IB0302828 W IB 0302828W WO 2004007721 A1 WO2004007721 A1 WO 2004007721A1
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WO
WIPO (PCT)
Prior art keywords
double
conjugate
stranded rna
cells
sirna
Prior art date
Application number
PCT/IB2003/002828
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English (en)
Inventor
Roger Michael Eccles
Alexandra Muratovska
Original Assignee
University Of Otago
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 Otago filed Critical University Of Otago
Priority to AU2003281078A priority Critical patent/AU2003281078A1/en
Publication of WO2004007721A1 publication Critical patent/WO2004007721A1/fr

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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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

Abstract

L'invention porte sur un conjugué de formule: X-Y-Z, dans laquelle: X est un ARN double brin, de préférence une séquence d'ARN court interférant (siRNA); Y est une liaison ou une molécule de liaison liant X à Y par covalence; et Z est un peptide membranaire perméant. L'invention porte également sur un procédé de libération d'ARN double brin dans le cytoplasme d'une cellule consistant à administrer à la cellule une dose efficace dudit conjugué pour provoquer la libération de l'ARN double brin du peptide membranaire perméant dans le cytoplasme.
PCT/IB2003/002828 2002-07-17 2003-07-17 Conjugue de liberation de sequences d'arn double brin et methodes associees WO2004007721A1 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2003281078A AU2003281078A1 (en) 2002-07-17 2003-07-17 Conjugate for double-stranded rna sequence releasing and methods thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US39609302P 2002-07-17 2002-07-17
US60/396,093 2002-07-17

Publications (1)

Publication Number Publication Date
WO2004007721A1 true WO2004007721A1 (fr) 2004-01-22

Family

ID=30115969

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2003/002828 WO2004007721A1 (fr) 2002-07-17 2003-07-17 Conjugue de liberation de sequences d'arn double brin et methodes associees

Country Status (2)

Country Link
AU (1) AU2003281078A1 (fr)
WO (1) WO2004007721A1 (fr)

Cited By (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005117991A2 (fr) * 2004-05-04 2005-12-15 Nastech Pharmaceutical Company Inc. Compositions et procedes pour l'amelioration de l'administration d'acides nucleiques dans des cellules et pour la modification de l'expression de genes cibles dans des cellules
EP1657306A1 (fr) * 2004-11-16 2006-05-17 Qiagen GmbH Silençage genique médiée au moyen d'un hybride ADN sens et ARN anisens couplé à des peptides pour faciliter leur absorption par les cellules
EP1984399A2 (fr) * 2006-02-10 2008-10-29 The Regents of the University of California Livraison transductrice d'arnsi par liaison arn double brin de domaines de fusion au ptd/cpps
WO2010086597A1 (fr) * 2009-01-27 2010-08-05 Trojan Technologies Ltd. Introduction d'acides nucléiques à l'aide de peptides pénétrant dans les cellules
US8299236B2 (en) 2004-05-04 2012-10-30 Marina Biotech, Inc. Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US9260493B2 (en) 2009-05-07 2016-02-16 The Regents Of The University Of California Transducible delivery of nucleic acids using modified dsRNA binding domains
US9950001B2 (en) 2012-08-20 2018-04-24 The Regents Of The University Of California Polynucleotides having bioreversible groups
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
US11981703B2 (en) 2017-08-17 2024-05-14 Sirius Therapeutics, Inc. Polynucleotide constructs

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999005302A1 (fr) * 1997-07-24 1999-02-04 The Perkin-Elmer Corporation Conjugues de peptides transporteurs et analogues d'acides nucleiques ainsi que leur utilisation

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999005302A1 (fr) * 1997-07-24 1999-02-04 The Perkin-Elmer Corporation Conjugues de peptides transporteurs et analogues d'acides nucleiques ainsi que leur utilisation

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ASTRIAB-FISHER ANNA ET AL: "Conjugates of antisense oligonucleotides with the Tat and Antennapedia cell-penetrating peptides: Effects on cellular uptake, binding to target sequences, and biologic actions", PHARMACEUTICAL RESEARCH (NEW YORK), vol. 19, no. 6, June 2002 (2002-06-01), pages 744 - 754, XP002259727, ISSN: 0724-8741 *
LINDGREN M M ET AL: "Cell-penetrating peptides", TRENDS IN PHARMACOLOGICAL SCIENCES, ELSEVIER TRENDS JOURNAL, CAMBRIDGE, GB, vol. 21, no. 3, March 2000 (2000-03-01), pages 99 - 103, XP004202572, ISSN: 0165-6147 *
NORMAND NADIA ET AL: "Particle formation by a conserved domain of the herpes simplex virus protein VP22 facilitating protein and nucleic acid delivery", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 276, no. 18, 4 May 2001 (2001-05-04), pages 15042 - 15050, XP002259726, ISSN: 0021-9258 *
SCHERR M ET AL: "GENE SILENCING MEDIATED BY SMALL INTERFERING RNAS IN MAMMALIAN CELLS", CURRENT MEDICINAL CHEMISTRY, BENTHAM SCIENCE PUBLISHERS BV, BE, vol. 10, no. 3, February 2003 (2003-02-01), pages 245 - 256, XP009019025, ISSN: 0929-8673 *
SCHWARTZ J ET AL: "PEPTIDE-MEDIATED CELLULAR DELIVERY", CURRENT OPINION IN MOLECULAR THERAPEUTICS, CURRENT DRUGS, LONDON,, GB, vol. 2, no. 2, April 2000 (2000-04-01), pages 162 - 167, XP001037249, ISSN: 1464-8431 *

Cited By (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005117991A2 (fr) * 2004-05-04 2005-12-15 Nastech Pharmaceutical Company Inc. Compositions et procedes pour l'amelioration de l'administration d'acides nucleiques dans des cellules et pour la modification de l'expression de genes cibles dans des cellules
US8299236B2 (en) 2004-05-04 2012-10-30 Marina Biotech, Inc. Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
WO2005117991A3 (fr) * 2004-05-04 2007-01-18 Nastech Pharm Co Compositions et procedes pour l'amelioration de l'administration d'acides nucleiques dans des cellules et pour la modification de l'expression de genes cibles dans des cellules
WO2006053683A3 (fr) * 2004-11-16 2006-08-03 Qiagen Gmbh Silençage de genes utilisant des constructions hybrides d'adn sens et d'arn antisens couplees a des peptides facilitant l'apport dans les cellules
WO2006053683A2 (fr) * 2004-11-16 2006-05-26 Qiagen Gmbh Silençage de genes utilisant des constructions hybrides d'adn sens et d'arn antisens couplees a des peptides facilitant l'apport dans les cellules
EP1657306A1 (fr) * 2004-11-16 2006-05-17 Qiagen GmbH Silençage genique médiée au moyen d'un hybride ADN sens et ARN anisens couplé à des peptides pour faciliter leur absorption par les cellules
EP1984399A2 (fr) * 2006-02-10 2008-10-29 The Regents of the University of California Livraison transductrice d'arnsi par liaison arn double brin de domaines de fusion au ptd/cpps
EP1984399A4 (fr) * 2006-02-10 2010-03-03 Univ California Livraison transductrice d'arnsi par liaison arn double brin de domaines de fusion au ptd/cpps
US8273867B2 (en) 2006-02-10 2012-09-25 The Regents Of The University Of California Transducible delivery of siRNA by dsRNA binding domain fusions to PTD/CPPS
WO2010086597A1 (fr) * 2009-01-27 2010-08-05 Trojan Technologies Ltd. Introduction d'acides nucléiques à l'aide de peptides pénétrant dans les cellules
US9260493B2 (en) 2009-05-07 2016-02-16 The Regents Of The University Of California Transducible delivery of nucleic acids using modified dsRNA binding domains
US9950001B2 (en) 2012-08-20 2018-04-24 The Regents Of The University Of California Polynucleotides having bioreversible groups
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
US11981703B2 (en) 2017-08-17 2024-05-14 Sirius Therapeutics, Inc. Polynucleotide constructs

Also Published As

Publication number Publication date
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