Processing

Please wait...

PATENTSCOPE will be unavailable a few hours for maintenance reason on Saturday 31.10.2020 at 7:00 AM CET
Settings

Settings

Goto Application

1. WO2007147143 - RNA INTERFERENCE MEDIATED INHIBITION OF 11 BETA-HYDROXYSTEROID DEHYDROGENASE-1 (11 BETA-HSD-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)

Publication Number WO/2007/147143
Publication Date 21.12.2007
International Application No. PCT/US2007/071388
International Filing Date 15.06.2007
IPC
C12N 15/11 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
11DNA or RNA fragments; Modified forms thereof
C12P 19/34 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
19Preparation of compounds containing saccharide radicals
26Preparation of nitrogen-containing carbohydrates
28N-glycosides
30Nucleotides
34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
C07H 21/02 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
02with ribosyl as saccharide radical
C07H 21/04 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
04with deoxyribosyl as saccharide radical
A01N 43/04 2006.01
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
NPRESERVATION OF BODIES OF HUMANS OR ANIMALS OR PLANTS OR PARTS THEREOF; BIOCIDES, e.g. AS DISINFECTANTS, AS PESTICIDES OR AS HERBICIDES; PEST REPELLANTS OR ATTRACTANTS; PLANT GROWTH REGULATORS
43Biocides, pest repellants or attractants, or plant growth regulators containing heterocyclic compounds
02having rings with one or more oxygen or sulfur atoms as the only ring hetero atom
04with one hetero atom
CPC
C12N 15/1131
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
1131against viruses
C12N 15/1137
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
1137against enzymes
C12N 2310/14
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
14interfering N.A.
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 2310/317
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
317with an inverted bond, e.g. a cap structure
C12N 2310/321
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
3212'-O-R Modification
Applicants
  • SIRNA THERAPEUTICS, INC. [US]/[US] (AllExceptUS)
  • MCSWIGGEN, James [US]/[US] (UsOnly)
  • VARGEESE, Chandra [US]/[US] (UsOnly)
  • JADHAV, Vasant [IN]/[US] (UsOnly)
Inventors
  • MCSWIGGEN, James
  • VARGEESE, Chandra
  • JADHAV, Vasant
Agents
  • SINGER, Christopher, P.
Priority Data
11/455,20516.06.2006US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) RNA INTERFERENCE MEDIATED INHIBITION OF 11 BETA-HYDROXYSTEROID DEHYDROGENASE-1 (11 BETA-HSD-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
(FR) INHIBITION MÉDIÉE PAR L'INTERFÉRENCE D'ARN DE L'EXPRESSION DU GÈNE DE LA 11-BÊTA-HYDROXYSTÉROÏDE DÉSHYDROGÉNASE 1 (11-BÊTA-HSD-1) EN UTILISANT UN ACIDE NUCLÉIQUE INTERFÉRENT COURT (ANSI)
Abstract
(EN)
The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate 11 beta-HSD-1 gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce metabolic diseases traits and conditions, including but not limited to diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity, hypercholesterolemia, cardiovascular disease and/or other disease states, conditions, or traits associated with 11 beta-HSD-1 gene expression or activity in a subject or organism.
(FR)
La présente invention concerne des composés, des compositions et des procédés d'étude, de diagnostic et de traitement de traits, maladies et états qui réagissent à la modulation de l'expression et/ou de l'activité du gène de la 11-bêta-hydroxystéroïde déshydrogénase 1 (11-bêta-HSD-1). L'invention concerne également des composés, des compositions et des procédés relatifs à des traits, maladies et états qui réagissent à la modulation de l'expression et/ou de l'activité de gènes impliqués dans les mécanismes d'expression du gène de la 11-bêta-hydroxystéroïde déshydrogénase 1 (11-bêta-HSD-1) ou dans d'autres processus cellulaires qui médient la maintenance ou le développement de ces traits, maladies et états. Spécifiquement, l'invention concerne des molécules d'acides nucléiques double brin, comprenant de petites molécules d'acides nucléiques telles que des molécules d'acide nucléique interférent court (ANsi), d'ARN interférent court (ARNsi), d'ARN double brin (ARNdb), de micro-ARN (ARNmi) et d'ARN en épingle à cheveux court (ARNsh) capables de médier l'interférence d'un ARN (iARN) contre l'expression du gène de la 11-bêta-hydroxystéroïde déshydrogénase 1 (11-bêta-HSD-1), y compris des cocktails de ces petites molécules d'acides nucléiques et des formulations de nanoparticules lipidiques (LNP) de ces petites molécules d'acides nucléiques. L'invention concerne également de petites molécules d'acides nucléiques tels que l'ANsi, l'ARNsi et autres, capables d'inhiber la fonction de molécules d'ARN endogènes telles que le micro-ARN (ARNmi) endogène (par ex. des inhibiteurs d'ARNmi) ou un ARN interférent court (ARNsi) endogène (par ex. des inhibiteurs d'ARNsi) ou capables d'inhiber la fonction du complexe RISC (par ex. des inhibiteurs de RISC), pour moduler l'expression du gène de la 11-bêta-HSD-1 en interférant avec la fonction régulatrice de ces ARN endogènes ou de protéines associées à ces ARN endogènes (par ex. RISC), y compris des cocktails de ces petites molécules d'acides nucléiques et des formulations de nanoparticules lipidiques (LNP) de ces petites molécules d'acides nucléiques. Ces petites molécules d'acides nucléiques sont utiles par exemple pour préparer des compositions destinées à prévenir, inhiber ou réduire des traits, maladies et états métaboliques, y compris non limitativement le diabète (par ex. le diabète de type I et/ou de type II), la résistance à l'insuline, l'obésité, l'hypercholestérolémie, la maladie cardiovasculaire et/ou d'autres pathologies, états ou traits associés à l'expression ou à l'activité du gène de la 11-bêta-HSD-1 chez un sujet ou dans un organisme.
Latest bibliographic data on file with the International Bureau