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1. WO2020136125 - ANTISENSE OLIGONUCLEOTIDES TARGETING CARD9

Publication Number WO/2020/136125
Publication Date 02.07.2020
International Application No. PCT/EP2019/086725
International Filing Date 20.12.2019
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 31/712 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
712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
A61K 31/7125 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
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
C07H 21/00 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
CPC
A61K 31/712
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
712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
A61K 31/7125
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
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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 2310/11
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
C12N 2310/315
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
31of the backbone
315Phosphorothioates
C12N 2310/3231
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
323modified ring structure
3231having an additional ring, e.g. LNA, ENA
Applicants
  • BOEHRINGER INGELHEIM INTERNATIONAL GMBH [DE]/[DE]
  • F. HOFFMANN-LA ROCHE AG [CH]/[CH] (AllExceptUS)
  • HOFFMANN-LA ROCHE, INC. [US]/[US] (US)
Inventors
  • FINE, Jay
  • MBOW, Mouhamadou a/k/a Lamine
  • WAHLE, Joe Adam
  • SHEN, Fei
  • KLEIN, Elliott Sanford
  • SAI, Kristina Mary
  • HAGEDORN, Peter
  • MOELHART HOEG, Anja
Agents
  • ALTMANN STÖSSEL DICK PATENTANWÄLTE PARTG MBB
Priority Data
62/784,28521.12.2018US
62/832,20710.04.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) ANTISENSE OLIGONUCLEOTIDES TARGETING CARD9
(FR) OLIGONUCLÉOTIDES ANTISENS CIBLANT LA CARD9
Abstract
(EN)
The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to CARD9 pre-mRNA intron and exon sequences, which are capable of inhibiting the expression of CARD9 protein. Inhibition of CARD9 expression is beneficial for a range of medical disorders including inflammatory bowel disease, pancreatitis, IgA nephropathy, primary sclerosing cholangitis, cardiovascular disease, cancer and diabetes.
(FR)
La présente invention concerne des oligonucléotides LNA antisens (oligomères) complémentaires à des séquences d'introns et d'exon pré-ARNm de CARD9, qui sont aptes à inhiber l'expression de la protéine CARD9. L'inhibition de l'expression de CARD9 est bénéfique pour une gamme de troubles médicaux comprenant la maladie intestinale inflammatoire, la pancréatite, la néphropathie à IgA, l'angiocholite sclérosante primitive, la maladie cardiovasculaire, le cancer et le diabète.
Latest bibliographic data on file with the International Bureau