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1. WO2021067744 - CHEMICAL MODIFICATIONS OF SMALL INTERFERING RNA WITH MINIMAL FLUORINE CONTENT

Publication Number WO/2021/067744
Publication Date 08.04.2021
International Application No. PCT/US2020/053999
International Filing Date 02.10.2020
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
CPC
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 2310/14
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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 2310/322
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
30Chemical structure
32of the sugar
3222'-R Modification
C12N 2310/344
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2310Structure or type of the nucleic acid
30Chemical structure
34Spatial arrangement of the modifications
344Position-specific modifications, e.g. on every purine, at the 3'-end
C12N 2310/3515
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2310Structure or type of the nucleic acid
30Chemical structure
35Nature of the modification
351Conjugate
3515Lipophilic moiety, e.g. cholesterol
C12N 2310/531
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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2310Structure or type of the nucleic acid
50Physical structure
53partially self-complementary or closed
531Stem-loop; Hairpin
Applicants
  • DICERNA PHARMACEUTICALS, INC. [US]/[US]
Inventors
  • WANG, Weimin
  • NAZEF, Naim
Agents
  • REID, Andrea L., C.
  • OH, Euk Charlie
  • DAVIS, Chad E.
  • NIHAN, Danielle M.
  • ARICO, Joseph W.
  • WANG, Gang
  • FLEMING, Paul R.
  • STEVENSON, Christian
  • MANAS, Michael
  • MACKLIN, Todd
Priority Data
62/909,27802.10.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CHEMICAL MODIFICATIONS OF SMALL INTERFERING RNA WITH MINIMAL FLUORINE CONTENT
(FR) MODIFICATIONS CHIMIQUES DE PETITS ARN INTERFÉRENTS AVEC UNE TENEUR MINIMALE EN FLUOR
Abstract
(EN)
The present invention provides oligonucleotides comprising 2'-O-methyl (2'-OMe) and 2'-deoxy-2'-fluoro (2'-F) modifications, compositions thereof, and methods of use for reducing the expression or activity of a gene.
(FR)
La présente invention concerne des oligonucléotides comprenant des modifications 2'-O-méthyl (2'-OMe) et 2'-désoxy-2'-fluoro (2'-F), des compositions de ceux-ci et des procédés d'utilisation pour réduire l'expression ou l'activité d'un gène.
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