WO2007030619A3 - Pharmaceutical compositions for delivery of ribonucleic acid to a cell - Google Patents

Pharmaceutical compositions for delivery of ribonucleic acid to a cell Download PDF

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Publication number
WO2007030619A3
WO2007030619A3 PCT/US2006/034859 US2006034859W WO2007030619A3 WO 2007030619 A3 WO2007030619 A3 WO 2007030619A3 US 2006034859 W US2006034859 W US 2006034859W WO 2007030619 A3 WO2007030619 A3 WO 2007030619A3
Authority
WO
WIPO (PCT)
Prior art keywords
delivery
cell
pharmaceutical compositions
ribonucleic acid
dsrna
Prior art date
Application number
PCT/US2006/034859
Other languages
French (fr)
Other versions
WO2007030619A2 (en
Inventor
Kunyuan Cui
Michael E Houston Jr
Lishan Chen
Sasha J Mayer
Yuching Chen
Original Assignee
Nastech Pharm Co
Kunyuan Cui
Michael E Houston Jr
Lishan Chen
Sasha J Mayer
Yuching Chen
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 US11/223,699 external-priority patent/US20060035815A1/en
Application filed by Nastech Pharm Co, Kunyuan Cui, Michael E Houston Jr, Lishan Chen, Sasha J Mayer, Yuching Chen filed Critical Nastech Pharm Co
Priority to CA002621694A priority Critical patent/CA2621694A1/en
Priority to EP06803119A priority patent/EP1934359A2/en
Priority to JP2008530199A priority patent/JP2009507852A/en
Priority to AU2006287481A priority patent/AU2006287481A1/en
Publication of WO2007030619A2 publication Critical patent/WO2007030619A2/en
Publication of WO2007030619A3 publication Critical patent/WO2007030619A3/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1136Non-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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)

Abstract

A composition, method for causing uptake in animal cells of double stranded RNA (dsRNA) and reduction of a target mRNA and a use of a mixture for the production of a medicament for the treatment for Tumor Necrosis Factor-alpha (TNF-α) associated inflammatory condition(s) in an animal subject comprising a polynucleotide delivery-enhancing polypeptide and a dsRNA, wherein the polynucleotide delivery-enhancing polypeptide is amphipathic and comprises nucleic acid binding properties are described.
PCT/US2006/034859 2005-09-08 2006-09-08 Pharmaceutical compositions for delivery of ribonucleic acid to a cell WO2007030619A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA002621694A CA2621694A1 (en) 2005-09-08 2006-09-08 Pharmaceutical compositions for delivery of ribonucleic acid to a cell
EP06803119A EP1934359A2 (en) 2005-09-08 2006-09-08 Pharmaceutical compositions for delivery of ribonucleic acid to a cell
JP2008530199A JP2009507852A (en) 2005-09-08 2006-09-08 Pharmaceutical composition for delivery of ribonucleic acid to cells
AU2006287481A AU2006287481A1 (en) 2005-09-08 2006-09-08 Pharmaceutical compositions for delivery of ribonucleic acid to a cell

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US11/223,699 2005-09-08
US11/223,699 US20060035815A1 (en) 2004-05-04 2005-09-08 Pharmaceutical compositions for delivery of ribonucleic acid to a cell
US72721605P 2005-10-14 2005-10-14
US60/727,216 2005-10-14
US73366405P 2005-11-04 2005-11-04
US60/733,664 2005-11-04

Publications (2)

Publication Number Publication Date
WO2007030619A2 WO2007030619A2 (en) 2007-03-15
WO2007030619A3 true WO2007030619A3 (en) 2007-10-04

Family

ID=37836462

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2006/034859 WO2007030619A2 (en) 2005-09-08 2006-09-08 Pharmaceutical compositions for delivery of ribonucleic acid to a cell

Country Status (5)

Country Link
EP (1) EP1934359A2 (en)
JP (1) JP2009507852A (en)
AU (1) AU2006287481A1 (en)
CA (1) CA2621694A1 (en)
WO (1) WO2007030619A2 (en)

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8921314B2 (en) 2008-10-15 2014-12-30 Angiochem, Inc. Conjugates of GLP-1 agonists and uses thereof
US8969310B2 (en) 2005-07-15 2015-03-03 Angiochem Inc. Potentiation of anticancer agents
US9161988B2 (en) 2009-07-02 2015-10-20 Angiochem Inc. Multimeric peptide conjugates and uses thereof
US9173891B2 (en) 2009-04-20 2015-11-03 Angiochem, Inc. Treatment of ovarian cancer using an anticancer agent conjugated to an angiopep-2 analog
US9221867B2 (en) 2003-01-06 2015-12-29 Angiochem Inc. Method for transporting a compound across the blood-brain barrier
US9365634B2 (en) 2007-05-29 2016-06-14 Angiochem Inc. Aprotinin-like polypeptides for delivering agents conjugated thereto to tissues

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060040882A1 (en) * 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
CA2597958C (en) 2005-02-18 2019-01-15 Angiochem Inc. Molecules for transporting a compound across the blood-brain barrier
EP2056845B1 (en) 2006-08-08 2017-10-11 Rheinische Friedrich-Wilhelms-Universität Bonn Structure and use of 5' phosphate oligonucleotides
US20080076701A1 (en) * 2006-08-18 2008-03-27 Nastech Pharmaceutical Company Inc. Dicer substrate rna peptide conjugates and methods for rna therapeutics
MX2010006925A (en) * 2007-12-20 2011-05-02 Angiochem Inc Polypeptide-nucleic acid conjugates and uses thereof.
US9040492B2 (en) 2008-03-31 2015-05-26 National Institute Of Advanced Industrial Science And Technology Double-stranded lipid-modified RNA having high RNA interference effect
JP5689413B2 (en) 2008-05-21 2015-03-25 ライニッシュ フリードリッヒ−ウィルヘルムズ−ユニバーシタット ボン 5 'triphosphate oligonucleotide having blunt ends and uses thereof
CN102307904A (en) 2008-12-05 2012-01-04 安吉奥开米公司 Conjugates of neurotensin or neurotensin analogs and uses thereof
EP2508530A1 (en) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification of triphosphorylated oligonucleotides using capture tags
WO2013103899A2 (en) * 2012-01-06 2013-07-11 H. Lee Moffitt Cancer Center And Research Institute Phosphorylation of histones and uses thereof
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
ES2826827T3 (en) 2015-06-15 2021-05-19 Angiochem Inc Methods for the treatment of leptomeningeal carcinomatosis
CN114259460B (en) * 2020-09-16 2024-03-15 苏州大学 Hydrogel composition based on immunoadjuvant and application thereof
CN114632161B (en) * 2020-12-16 2024-02-06 中国人民解放军军事科学院军事医学研究院 Application of tumor necrosis factor-alpha as nucleic acid gene medicine in-vivo delivery carrier

Citations (5)

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US5679559A (en) * 1996-07-03 1997-10-21 University Of Utah Research Foundation Cationic polymer and lipoprotein-containing system for gene delivery
WO2001081370A2 (en) * 2000-04-24 2001-11-01 Novartis Ag Histone h2a-derived peptides useful in gene delivery
WO2004087931A1 (en) * 2003-04-03 2004-10-14 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US20050147993A1 (en) * 2003-10-24 2005-07-07 Shaharyar Khan Methods and compositions for delivering polynucleotides
WO2005117991A2 (en) * 2004-05-04 2005-12-15 Nastech Pharmaceutical Company Inc. Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells

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US6589503B1 (en) * 1998-06-20 2003-07-08 Washington University Membrane-permeant peptide complexes for medical imaging, diagnostics, and pharmaceutical therapy
AU2003217550A1 (en) * 2002-02-20 2003-09-09 Ribozyme Pharmaceuticals, Incorporated RNA INTERFERENCE MEDIATED INHIBITION OF TNF AND TNF RECEPTOR SUPERFAMILY GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

Patent Citations (5)

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Publication number Priority date Publication date Assignee Title
US5679559A (en) * 1996-07-03 1997-10-21 University Of Utah Research Foundation Cationic polymer and lipoprotein-containing system for gene delivery
WO2001081370A2 (en) * 2000-04-24 2001-11-01 Novartis Ag Histone h2a-derived peptides useful in gene delivery
WO2004087931A1 (en) * 2003-04-03 2004-10-14 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US20050147993A1 (en) * 2003-10-24 2005-07-07 Shaharyar Khan Methods and compositions for delivering polynucleotides
WO2005117991A2 (en) * 2004-05-04 2005-12-15 Nastech Pharmaceutical Company Inc. Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells

Non-Patent Citations (5)

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Title
DATABASE GENESEQ E.B.I. Hinxton U.K.; 14 February 2002 (2002-02-14), BALICKI D AND BEUTLER E: "Histone 2A-derived gene delivery peptide #15", XP002431354, Database accession no. AAU70895 *
JOHNSON P.H.: "Development of Therapeutic siRNAs against TNF-alpha and Novel Peptide Delivery Agents for the Treatment of Rheumatoid Arthritis", 13 June 2005 (2005-06-13), XP002431337, Retrieved from the Internet <URL:http://www.nastech.com/pdf/RNAi_Conference%20Presentation_CHI_061305.pdf> [retrieved on 20070425] *
PUEBLA I ET AL: "A RECOMBINANT H1 HISTONE-BASED SYSTEM FOR EFFICIENT DELIVERY OF NUCLEIC ACIDS", JOURNAL OF BIOTECHNOLOGY, ELSEVIER SCIENCE PUBLISHERS, AMSTERDAM, NL, vol. 105, no. 3, 2003, pages 215 - 226, XP009057520, ISSN: 0168-1656 *
SIMEONI F ET AL: "Insight into the mechanism of the peptide-based gene delivery system MPG: implications for delivery of siRNA into mammalian cells", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 31, no. 11, 13 March 2003 (2003-03-13), pages 2717 - 2724, XP002984580, ISSN: 0305-1048 *
WITKOWSKA RENATA T ET AL: "Peptide-mediated delivery of siRNA via noncovalent complexes and covalent conjugates", June 2005 (2005-06-01), 19TH AMERICAN PEPTIDE SYMPOSIUM; SAN DIEGO, CA, USA; JUNE 18 -23, 2005, XP002431336, Retrieved from the Internet <URL:http://www.nastech.com/pdf/nastech_APS_062005.pdf> [retrieved on 20070425] *

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9221867B2 (en) 2003-01-06 2015-12-29 Angiochem Inc. Method for transporting a compound across the blood-brain barrier
US8969310B2 (en) 2005-07-15 2015-03-03 Angiochem Inc. Potentiation of anticancer agents
US9365634B2 (en) 2007-05-29 2016-06-14 Angiochem Inc. Aprotinin-like polypeptides for delivering agents conjugated thereto to tissues
US8921314B2 (en) 2008-10-15 2014-12-30 Angiochem, Inc. Conjugates of GLP-1 agonists and uses thereof
US9173891B2 (en) 2009-04-20 2015-11-03 Angiochem, Inc. Treatment of ovarian cancer using an anticancer agent conjugated to an angiopep-2 analog
US9161988B2 (en) 2009-07-02 2015-10-20 Angiochem Inc. Multimeric peptide conjugates and uses thereof

Also Published As

Publication number Publication date
WO2007030619A2 (en) 2007-03-15
EP1934359A2 (en) 2008-06-25
JP2009507852A (en) 2009-02-26
AU2006287481A1 (en) 2007-03-15
CA2621694A1 (en) 2007-03-15

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