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EP1086116A4 - COMPOSITIONS FOR PULMONARY DELIVERY OF NUCLEIC ACIDS AND METHODS - Google Patents

COMPOSITIONS FOR PULMONARY DELIVERY OF NUCLEIC ACIDS AND METHODS

Info

Publication number
EP1086116A4
EP1086116A4 EP99923263A EP99923263A EP1086116A4 EP 1086116 A4 EP1086116 A4 EP 1086116A4 EP 99923263 A EP99923263 A EP 99923263A EP 99923263 A EP99923263 A EP 99923263A EP 1086116 A4 EP1086116 A4 EP 1086116A4
Authority
EP
European Patent Office
Prior art keywords
compositions
methods
pulmonary delivery
nucleic acids
animal
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Withdrawn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
EP99923263A
Other languages
German (de)
French (fr)
Other versions
EP1086116A1 (en
Inventor
Clarence Frank Bennett
David J Ecker
Phillip Dan Cook
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.)
Ionis Pharmaceuticals Inc
Original Assignee
Isis Pharmaceuticals Inc
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 Isis Pharmaceuticals Inc filed Critical Isis Pharmaceuticals Inc
Publication of EP1086116A1 publication Critical patent/EP1086116A1/en
Publication of EP1086116A4 publication Critical patent/EP1086116A4/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Withdrawn legal-status Critical Current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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • 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
    • C12N15/1137—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 against enzymes
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

Landscapes

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

Abstract

The present invention relates to compositions and methods for the pulmonary delivery of nucleic acids, particularly oligonucleotides. In one preferred embodiment, the compositions and methods of the invention are utilized to effect the pulmonary delivery of an antisense oligonucleotide to an animal in order to modulate the expression of a gene in the animal for investigative, therapeutic or prophylactic purposes.
EP99923263A 1998-05-21 1999-05-20 COMPOSITIONS FOR PULMONARY DELIVERY OF NUCLEIC ACIDS AND METHODS Withdrawn EP1086116A4 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US8358598A 1998-05-21 1998-05-21
US83585 1998-05-21
PCT/US1999/011214 WO1999060010A1 (en) 1998-05-21 1999-05-20 Compositions and methods for the pulmonary delivery of nucleic acids

Publications (2)

Publication Number Publication Date
EP1086116A1 EP1086116A1 (en) 2001-03-28
EP1086116A4 true EP1086116A4 (en) 2004-03-17

Family

ID=22179298

Family Applications (1)

Application Number Title Priority Date Filing Date
EP99923263A Withdrawn EP1086116A4 (en) 1998-05-21 1999-05-20 COMPOSITIONS FOR PULMONARY DELIVERY OF NUCLEIC ACIDS AND METHODS

Country Status (3)

Country Link
EP (1) EP1086116A4 (en)
AU (1) AU4007899A (en)
WO (1) WO1999060010A1 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5955443A (en) * 1998-03-19 1999-09-21 Isis Pharmaceuticals Inc. Antisense modulation of PECAM-1
US6197584B1 (en) * 1998-05-01 2001-03-06 Isis Pharmaceuticals, Inc. Antisense modulation of CD40 expression
US10745701B2 (en) 2007-06-28 2020-08-18 The Trustees Of Princeton University Methods of identifying and treating poor-prognosis cancers
US20090324596A1 (en) 2008-06-30 2009-12-31 The Trustees Of Princeton University Methods of identifying and treating poor-prognosis cancers
ES2641290T3 (en) 2007-11-20 2017-11-08 Ionis Pharmaceuticals, Inc CD40 expression modulation

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993019203A1 (en) * 1992-03-16 1993-09-30 Isis Pharmaceuticals, Inc. Oligonucleotide modulation of protein kinase c
WO1999011778A1 (en) * 1997-09-02 1999-03-11 University Of Sheffield Antisense treatment of pulmonary hypertension
WO2000020645A1 (en) * 1998-10-05 2000-04-13 Isis Pharmaceuticals, Inc. ANTISENSE OLIGONUCLEOTIDE MODULATION OF TUMOR NECROSIS FACTOR-α (TNF-α) EXPRESSION
WO2000050050A1 (en) * 1999-02-23 2000-08-31 Isis Pharmaceuticals, Inc. Multiparticulate formulation

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5591623A (en) * 1990-08-14 1997-01-07 Isis Pharmaceuticals, Inc. Oligonucleotide modulation of cell adhesion
US5858784A (en) * 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993019203A1 (en) * 1992-03-16 1993-09-30 Isis Pharmaceuticals, Inc. Oligonucleotide modulation of protein kinase c
WO1999011778A1 (en) * 1997-09-02 1999-03-11 University Of Sheffield Antisense treatment of pulmonary hypertension
WO2000020645A1 (en) * 1998-10-05 2000-04-13 Isis Pharmaceuticals, Inc. ANTISENSE OLIGONUCLEOTIDE MODULATION OF TUMOR NECROSIS FACTOR-α (TNF-α) EXPRESSION
WO2000050050A1 (en) * 1999-02-23 2000-08-31 Isis Pharmaceuticals, Inc. Multiparticulate formulation

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
LEVESQUE L. ET AL.: "Antisense oligonucleotides targeting human Protein Kinase C-alpha inhibit phorbol ester-induced reduction of Bradykinin-evoked Calcium mobilization in A549 cells", MOLECULAR PHARMACOLOGY, vol. 51, 1997, pages 209 - 216, XP002247729 *
MCKAY R.A. ET AL.: "Characterization of a potent and specific class of antisense oligonucleotide inhibitor of human Protein Kinase C-alpha expression", THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 274, no. 3, 15 January 1999 (1999-01-15), pages 1715 - 1722, XP002247728 *
NICKLIN P.L. ET AL.: "Pulmonary bioavailability of a phosphorothioate oligonucleotide (CGP 64128A): comparison with other delivery routes", PHARMACEUTICAL RESEARCH, vol. 15, no. 4, April 1998 (1998-04-01), pages 583 - 591, XP008019675 *
NYCE J.W. AND METZGER W.J.: "DNA antisense therapy for asthma in an animal model", NATURE, vol. 385, 20 February 1997 (1997-02-20), pages 721 - 725, XP000891493, ISSN: 0028-0836 *
PHAN S.H.: "New strategies for treatment of pulmonary fibrosis", THORAX, vol. 50, no. 4, 1 April 1995 (1995-04-01), pages 415 - 421, XP000601085, ISSN: 0040-6376 *
ROJANASAKUL Y. ET AL.: "Antisense inhibition of Silica-induced Tumor Necrosis Factor in alveolar macrophages", THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 272, no. 7, 14 February 1997 (1997-02-14), pages 3910 - 3914, XP002247730 *
See also references of WO9960010A1 *

Also Published As

Publication number Publication date
WO1999060010A1 (en) 1999-11-25
AU4007899A (en) 1999-12-06
EP1086116A1 (en) 2001-03-28

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