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1. WO2019048645 - STABILIZED CEBPA SARNA COMPOSITIONS AND METHODS OF USE

Publication Number WO/2019/048645
Publication Date 14.03.2019
International Application No. PCT/EP2018/074211
International Filing Date 07.09.2018
IPC
C12N 15/113 2010.01
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
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
CPC
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 47/549
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
549Sugars, nucleosides, nucleotides or nucleic acids
C12N 15/113
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
11DNA or RNA fragments; Modified forms thereof
113Non-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
C12N 15/85
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
C12N 2310/10
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
2310Structure or type of the nucleic acid
10Type of nucleic acid
C12N 2310/113
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
2310Structure or type of the nucleic acid
10Type of nucleic acid
11Antisense
113targeting other non-coding nucleic acids, e.g. antagomirs
Applicants
  • MINA THERAPEUTICS LIMITED [GB]/[GB]
Inventors
  • HUBER, Hans E.
  • BLAKEY, David
  • VOUTILA, Jon
  • REEBYE, Vikash
Agents
  • CHAPMAN, Desmond Mark
  • JACKSON, Richard Eric
  • COCKERTON, Bruce Roger
  • MARSHALL, Cameron John
  • GOODFELLOW, Hugh Robin
  • TUNSTALL, Christopher Stephen
  • KIRSCH, Susan Edith
  • SMALL, Gary James
  • WARNER, James Alexander
  • BRUNNER, John Michael Owen
  • CHAPMAN, Desmond Mark
  • WISE, Daniel Joseph
  • OATES, Edward Christopher
  • MCGUIRE, Gillian Margaret
  • MORRIS, Claire Louise
  • COX, Jennifer Jane
  • GEORGIOU, Matthew
  • HOLLAND, David Christopher
  • BETTRIDGE, Paul Sebastian
  • DRAGOSAVAC, Susannah Marie
  • PREISSNER, Tanja Stephanie
  • LEATHLEY, Anna Elisabeth
  • FISHER, John William
  • HUSBAND, Benjamin
  • DUFFIELD, Stephen
  • MACLEOD, Ian Roger
  • HOGGETT, Christopher James
  • GRABENSTEIN, Jens
  • MICHELL, Stephen Robert
  • DARBY, Richard John
  • BARRY, Isobel Amy
  • MCKINLEY, Elizabeth Anne
  • BIRCH, Matthew John
  • THEISEN, Andreas Robert
Priority Data
62/555,95108.09.2017US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) STABILIZED CEBPA SARNA COMPOSITIONS AND METHODS OF USE
(FR) COMPOSITIONS STABILISÉES DE PETITS ARN ACTIVATEURS (PARNA) DE CEBPA ET PROCÉDÉS D'UTILISATION
Abstract
(EN)
The invention relates to modified oligonucleotides, e.g., saRNAs useful in upregulating the expression of a target gene and therapeutic compositions comprising such oligonucleotides. Methods of using the oligonucleotides and the therapeutic compositions are also provided.
(FR)
L'invention concerne des oligonucléotides modifiés, par exemple, des pARNa utiles pour la régulation à la hausse de l'expression d'un gène cible et des compositions thérapeutiques contenant ces oligonucléotides. L'invention concerne également des procédés d'utilisation des oligonucléotides et des compositions thérapeutiques les contenant.
Also published as
Latest bibliographic data on file with the International Bureau