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1. WO2020089342 - COMPOSITIONS FOR TRANSFECTING mRNA INTO A CELL AND THEIR APPLICATIONS

Publication Number WO/2020/089342
Publication Date 07.05.2020
International Application No. PCT/EP2019/079742
International Filing Date 30.10.2019
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 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
A61K 31/7105 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 31/713 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
713Double-stranded nucleic acids or oligonucleotides
C12N 15/90 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
90Stable introduction of foreign DNA into chromosome
CPC
A61K 31/7105
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 31/713
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
713Double-stranded nucleic acids or oligonucleotides
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
C12N 15/907
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
90Stable introduction of foreign DNA into chromosome
902using homologous recombination
907in mammalian cells
Applicants
  • POLYPLUS TRANSFECTION [FR]/[FR]
Inventors
  • STOCK, Fabrice
  • TOUSSAINT MOREAU, Valérie
  • ERBACHER, Patrick
Agents
  • GUTMANN, Ernest
  • DESAIX, Anne
  • ALMOND-MARTIN, Carol
  • DERAMBURE, Christian
  • JOLLY, Christophe
  • LE QUERÉ, Hervé
  • LEJEUNE, Anne
  • PERNOT, Pierre
  • PICHAT, Thierry
  • POUCHAIN, Delphine
  • ROBERT, Mathias
  • SELLIN, Carole
Priority Data
18306417.930.10.2018EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS FOR TRANSFECTING mRNA INTO A CELL AND THEIR APPLICATIONS
(FR) COMPOSITIONS POUR TRANSFECTER UN ARNM DANS UNE CELLULE ET LEURS APPLICATIONS
Abstract
(EN)
The present invention relates to compositions for transfecting a messenger RNA (m RNA) into a cell and their applications. The present invention is directed to a composition for transfecting a mRNA into a cell comprising a mRNA, at least one neutral lipid and a cationic lipid of formula (I), wherein R1 R2, R3, R4 and R5, (CH2)n and A- are as defined in the description. The present invention also relates to uses of said composition and to a method for in vitro transfection of live cells.
(FR)
La présente invention concerne des compositions destinées à transfecter un ARN messager (ARNm) dans une cellule et leurs applications. La présente invention porte sur une composition permettant de transfecter un ARNm dans une cellule comprenant un ARNm, au moins un lipide neutre et un lipide cationique de formule (I), dans laquelle R1 R2, R3, R4 et R5, (CH2)n et A- sont tels que définis dans la description. L'invention se rapporte également à des utilisations desdites compositions et à un procédé de transfection in vitro de cellules vivantes.
Also published as
Latest bibliographic data on file with the International Bureau