WO2001066149A3 - Nucleic acid formulations for gene delivery and methods of use - Google Patents

Nucleic acid formulations for gene delivery and methods of use Download PDF

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Publication number
WO2001066149A3
WO2001066149A3 PCT/US2001/006953 US0106953W WO0166149A3 WO 2001066149 A3 WO2001066149 A3 WO 2001066149A3 US 0106953 W US0106953 W US 0106953W WO 0166149 A3 WO0166149 A3 WO 0166149A3
Authority
WO
WIPO (PCT)
Prior art keywords
poly
acid
nucleic acid
gene delivery
methods
Prior art date
Application number
PCT/US2001/006953
Other languages
French (fr)
Other versions
WO2001066149A2 (en
Inventor
Jason G Fewell
Fiona Maclaughlin
Louis C Smith
Francois Nicol
Alain Rolland
Original Assignee
Valentis Inc
Jason G Fewell
Fiona Maclaughlin
Louis C Smith
Francois Nicol
Alain Rolland
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 Valentis Inc, Jason G Fewell, Fiona Maclaughlin, Louis C Smith, Francois Nicol, Alain Rolland filed Critical Valentis Inc
Priority to BR0108962-5A priority Critical patent/BR0108962A/en
Priority to EP01918339A priority patent/EP1259265B1/en
Priority to DK01918339.1T priority patent/DK1259265T3/en
Priority to JP2001564801A priority patent/JP4987205B2/en
Priority to CA2401327A priority patent/CA2401327C/en
Priority to AT01918339T priority patent/ATE511400T1/en
Priority to AU2001245427A priority patent/AU2001245427A1/en
Publication of WO2001066149A2 publication Critical patent/WO2001066149A2/en
Publication of WO2001066149A3 publication Critical patent/WO2001066149A3/en
Priority to US10/234,406 priority patent/US7173116B2/en
Priority to US11/561,847 priority patent/US7491537B2/en

Links

Classifications

    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1816—Erythropoietin [EPO]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/21—Interferons [IFN]
    • A61K38/212—IFN-alpha
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination

Abstract

A nucleic acid formulation for use in gene delivery comprising a nucleic acid and an anionic polymer is disclosed. Examples of the anionic polymer includes anionic amino acid polymer or poly-amino acid (such as poly-L-glutamic acid, poly-D-glutamic acid, poly-L-aspartic acid, poly-D-aspartic acid), poly-acrylic acid, polynucleotides, poly galacturonic acid, and poly vinyl sulfate.
PCT/US2001/006953 2000-03-03 2001-03-02 Nucleic acid formulations for gene delivery and methods of use WO2001066149A2 (en)

Priority Applications (9)

Application Number Priority Date Filing Date Title
BR0108962-5A BR0108962A (en) 2000-03-03 2001-03-02 Nucleic acid formulations for gene distribution and methods of use
EP01918339A EP1259265B1 (en) 2000-03-03 2001-03-02 Nucleic acid formulations for gene delivery
DK01918339.1T DK1259265T3 (en) 2000-03-03 2001-03-02 Nucleic acid formulations for delivery of genes
JP2001564801A JP4987205B2 (en) 2000-03-03 2001-03-02 Nucleic acid preparations for gene delivery and methods of use
CA2401327A CA2401327C (en) 2000-03-03 2001-03-02 Nucleic acid formulations comprising poly-amino acids for gene delivery and methods of use
AT01918339T ATE511400T1 (en) 2000-03-03 2001-03-02 NUCLEIC ACID FORMULATIONS FOR GENE ADMINISTRATION
AU2001245427A AU2001245427A1 (en) 2000-03-03 2001-03-02 Nucleic acid formulations for gene delivery and methods of use
US10/234,406 US7173116B2 (en) 2000-03-03 2002-09-03 Nucleic acid formulations for gene delivery and methods of use
US11/561,847 US7491537B2 (en) 2000-03-03 2006-11-20 Nucleic acid formulations for gene delivery and methods of use

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US18723600P 2000-03-03 2000-03-03
US60/187,236 2000-03-03
US26175101P 2001-01-16 2001-01-16
US60/261,751 2001-01-16

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US10/234,406 Continuation US7173116B2 (en) 2000-03-03 2002-09-03 Nucleic acid formulations for gene delivery and methods of use

Publications (2)

Publication Number Publication Date
WO2001066149A2 WO2001066149A2 (en) 2001-09-13
WO2001066149A3 true WO2001066149A3 (en) 2002-03-14

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2001/006953 WO2001066149A2 (en) 2000-03-03 2001-03-02 Nucleic acid formulations for gene delivery and methods of use

Country Status (9)

Country Link
US (2) US7173116B2 (en)
EP (1) EP1259265B1 (en)
JP (1) JP4987205B2 (en)
AT (1) ATE511400T1 (en)
AU (1) AU2001245427A1 (en)
BR (1) BR0108962A (en)
CA (1) CA2401327C (en)
DK (1) DK1259265T3 (en)
WO (1) WO2001066149A2 (en)

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