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1. WO2015140330 - IMPROVED SMALL INTERFERING RIBONUCLEIC ACID MOLECULES

Publication Number WO/2015/140330
Publication Date 24.09.2015
International Application No. PCT/EP2015/056012
International Filing Date 20.03.2015
IPC
C12N 15/11 2006.1
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
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
C12N 15/111
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
111General methods applicable to biologically active non-coding 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 2310/111
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
111spanning the whole gene, or a large part of it
C12N 2310/14
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2310Structure or type of the nucleic acid
10Type of nucleic acid
14interfering N.A.
C12N 2320/50
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
2320Applications; Uses
50Methods for regulating/modulating their activity
Applicants
  • VARGHESE, Oommen [SE]/[SE]
  • OOMMEN, Oommen [SE]/[SE]
Inventors
  • VARGHESE, Oommen
  • OOMMEN, Oommen
Agents
  • BOULT WADE TENNANT
Priority Data
1450312-220.03.2014SE
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) IMPROVED SMALL INTERFERING RIBONUCLEIC ACID MOLECULES
(FR) PETITES MOLÉCULES D'ACIDE RIBONUCLÉIQUE D'INTERFÉRENCE AMÉLIORÉES
Abstract
(EN) Double-stranded siRNA molecules (100, 200, 300) are provided comprising a sense RNA strand (110, 210, 310) and an antisense RNA strand (120, 220, 320). The sense strand (110, 210, 310) has a 3'-overhang (115, 215, 315) of from four to eight nucleotides, of which at least one nucleotide is a non-ribonucleotide. The present double-stranded siRNA molecules (100, 200, 300) have beneficial properties in terms of carrier-free cellular uptake, endosomal escape and increased knockdown activity against target genes. Related compositions, cells, uses and methods are also provided.
(FR) Cette invention concerne des molécules de siARN (100, 200, 300) comprenant un brin ARN sens (110, 210, 310) et un brin ARN antisens (120, 220, 320). Le brin sens (110, 210, 310) comporte une extension en 3' (115, 215, 315) de quatre à huit nucléotides, dont un au moins de type non-ribonucléotide. Les présentes molécules siARN double brin (100, 200, 300) possèdent des propriétés bénéfiques en termes d'absorption cellulaire sans support, d'échappement endosomal et d'activité d'inactivation accrue contre des gènes cibles. Des compositions, des cellules, des utilisations et des procédés associés sont en outre décrits.
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