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1. EP0832271 - LIPID-NUCLEIC ACID PARTICLES PREPARED VIA A HYDROPHOBIC LIPID-NUCLEIC ACID COMPLEX INTERMEDIATE AND USE FOR GENE TRANSFER

Office European Patent Office
Application Number 96922432
Application Date 06.06.1996
Publication Number 0832271
Publication Date 01.04.1998
Publication Kind B8
IPC
C12N 15/09
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
A61K 9/127
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Liposomes
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C12N 15/88
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
88using microencapsulation, e.g. using liposome vesicle
CPC
C12N 15/88
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
88using microencapsulation, e.g. using ; amphiphile; liposome vesicle
A61K 9/1272
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Liposomes
1271Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
1272with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
A61K 47/6911
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
69the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
6905the form being a colloid or an emulsion
6911the form being a liposome
Applicants INEX PHARMACEUTICALS CORP
Inventors WHEELER JEFFERY J
BALLY MARCEL B
ZHANG YUAN-PENG
REIMER DOROTHY L
HOPE MICHAEL
CULLIS PIETER R
SCHERRER PETER
Designated States
Priority Data 08484282 07.06.1995 US
08485458 07.06.1995 US
9609949 06.06.1996 US
Title
(DE) HERSTELLUNG VON LIPID-NUKLEINSÄURE PARTIKELN DUCH EIN HYDROPHOBISCHE LIPID-NUKLEINSÄUREE KOMPLEXE ZWISCHENPRODUKT UND ZUR VERWENDUNG IN DER GENTRANSFER
(EN) LIPID-NUCLEIC ACID PARTICLES PREPARED VIA A HYDROPHOBIC LIPID-NUCLEIC ACID COMPLEX INTERMEDIATE AND USE FOR GENE TRANSFER
(FR) PARTICULES D'ACIDES NUCLEIQUES ET DE LIPIDES PREPAREES AU MOYEN D'UN INTERMEDIAIRE DE COMPLEXE HYDROPHOBE D'ACIDES NUCLEIQUES ET DE LIPIDES ET UTILISATION POUR TRANSFERER DES GENES
Abstract
(EN)
Novel lipid-nucleic acid particulate complexes which are useful for $i(in vitro) or $i(in vivo) gene transfer are described. The particles can be formed using either detergent dialysis methods or methods which utilize organic solvents. Upon removal of a solubilizing component (i.e, detergent or an organic solvent) the lipid-nucleic acid complexes form particles wherein the nucleic acid is serum-stable and is protected from degradation. The particles thus formed have access to extravascular sites and target cell populations and are suitable for the therapeutic delivery of nucleic acids.

(FR)
L'invention concerne de nouveaux complexes d'acides nucléiques et de lipides, utiles pour effectuer des transferts de gènes $i(in vitro) et $i(in vivo). On peut obtenir ces particules au moyen de procédés de dialyse à détergent ou de procédés utilisant des solvants organiques. Lorsqu'on supprime un constituant de solubilisation (c'est-à-dire, un détergent ou un solvant organique), les complexes d'acides nucléiques et de lipides forment des particules, l'acide nucléique présentant une stabilité au sérum et étant protégé de la dégradation. Ces particules peuvent alors accéder aux sites extravasculaires, ainsi qu'aux populations de cellules cible et sont appropriées pour l'administration thérapeutique d'acides nucléiques.