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1. WO1996040962 - CATIONIC LIPID:DNA COMPLEXES FOR GENE TARGETING

Publication Number WO/1996/040962
Publication Date 19.12.1996
International Application No. PCT/US1996/009526
International Filing Date 07.06.1996
Chapter 2 Demand Filed 27.12.1996
IPC
A61K 9/127 2006.01
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 38/17 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
A61K 47/48 2006.01
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
48the non-active ingredient being chemically bound to the active ingredient, e.g. polymer drug conjugates
A61K 48/00 2006.01
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 2006.01
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
A61K 38/1709
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
1703from vertebrates
1709from mammals
A61K 47/54
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
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
A61K 47/542
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
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
542Carboxylic acids, e.g. a fatty acid or an amino acid
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
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
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
Applicants
  • MEGABIOS CORPORATION [US]/[US] (AllExceptUS)
  • GORMAN, Cori, M. [US]/[US] (UsOnly)
  • McCLARRINON, Molly [US]/[US] (UsOnly)
Inventors
  • GORMAN, Cori, M.
  • McCLARRINON, Molly
Agents
  • NOONAN, Kevin, E.
Priority Data
08/485,00507.06.1995US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CATIONIC LIPID:DNA COMPLEXES FOR GENE TARGETING
(FR) COMPLEXES LIPIDE CATIONIQUE/ADN POUR CIBLAGE GENIQUE
Abstract
(EN)
The invention herein describes pharmaceutical compositions and methods for targeted delivery of functional genes into cells and tissues $i(in vivo). The invention discloses DNA:lipid complexes, methods of making such complexes and methods of using such complexes for facilitating the targeted delivery and entry of recombinant expression constructs into cells and tissues $i(in vivo), and particularly delivery of such recombinant expression constructs into cells and tissues $i(in vivo), and particularly delivery of such recombinant expression constructs by intravenous, intraperitoneal or direct injection. The delivery vehicle for the targeting of the recombinant construct encoding the gene of interest, is composed of a mixture of a cationic imidazolinium lipid of formula (I) wherein R and R1 mean a C11-29 aliphatic hydrocarbyl group, together with neutral lipids (e.g. DOPE, cholesterol).
(FR)
La présente invention concerne des compositions et des procédés pharmaceutiques permettant un apport ciblé in vivo de gènes fonctionnels dans des cellules et des tissus. En l'occurrence, l'invention concerne des complexes ADN/lipide, des procédés de production de tels complexes, ainsi que des procédés d'utilisation de ces complexes pour faciliter l'apport et l'introduction ciblés de produits d'assemblage d'expression recombinés in vivo dans des cellules et tissus, et notamment l'apport de tels produits d'assemblage d'expression recombinés par injection intraveineuse, intrapéritonéale ou directe. Le véhicule d'apport du produit d'assemblage recombiné codant le gène d'intérêt se compose d'un mélange d'un lipide d'imidazolinium cationique de formule (I), dans laquelle R et R1 représentent un groupe hydrocarbyle aliphatique C11-29, avec des lipides neutres (par exemple, DOPE, cholestérol).
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