WO2015051135A3 - Compositions organiques destinées au traitement de maladies associées à l'hepcidine - Google Patents

Compositions organiques destinées au traitement de maladies associées à l'hepcidine Download PDF

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Publication number
WO2015051135A3
WO2015051135A3 PCT/US2014/058851 US2014058851W WO2015051135A3 WO 2015051135 A3 WO2015051135 A3 WO 2015051135A3 US 2014058851 W US2014058851 W US 2014058851W WO 2015051135 A3 WO2015051135 A3 WO 2015051135A3
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WO
WIPO (PCT)
Prior art keywords
anemia
disease
hepcidin
iron
related diseases
Prior art date
Application number
PCT/US2014/058851
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English (en)
Other versions
WO2015051135A2 (fr
Inventor
Jeremy Lee BARYZA
Marcel Blommers
Todd Borland
Cesar Fernandez
Tom GEBUHR
Erin GENO
Alvar GOSSERT
Paulette Greenidge
Dieter Huesken
Juerg Hunziker
Francois Jean-Charles Natt
Anup Patnaik
Andrew Patterson
Jean-Michel Rene RONDEAU
Jonathan Solomon
Jan Weiler
Meicheng ZHU
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Novartis Ag
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Publication date
Application filed by Novartis Ag filed Critical Novartis Ag
Publication of WO2015051135A2 publication Critical patent/WO2015051135A2/fr
Publication of WO2015051135A3 publication Critical patent/WO2015051135A3/fr

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1136—Non-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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

Abstract

L'invention concerne des méthodes de traitement de maladies associées à l'hepcidine, telles que l'anémie, l'érythropoïèse ferriprive, l'hypoferrémie, un apport alimentaire en fer réduit, la séquestration du fer, l'anémie inflammatoire (AI) (appelée parfois anémie des maladies chroniques ou AMC), l'athérosclérose, le diabète, ainsi que les troubles neurodégénératifs multiples, tels que la maladie d'Alzheimer, la maladie de Parkinson et l'ataxie de Friedrich, l'insuffisance cardiaque, la néphropathie chronique, le syndrome d'anémie cardio-rénale, les infections, la perte sanguine, l'hémolyse, la carence en vitamine B12 ou en folates, l'hyperparathyroïdie, les hémoglobinopathies et les malignités, le cancer, le SIDA, la chirurgie, la croissance chétive et la chute de cheveux, au moyen d'une quantité thérapeutiquement efficace d'un agent ARNi sur l'hepcidine.
PCT/US2014/058851 2013-10-04 2014-10-02 Compositions organiques destinées au traitement de maladies associées à l'hepcidine WO2015051135A2 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361886753P 2013-10-04 2013-10-04
US61/886,753 2013-10-04
US201461930681P 2014-01-23 2014-01-23
US61/930,681 2014-01-23

Publications (2)

Publication Number Publication Date
WO2015051135A2 WO2015051135A2 (fr) 2015-04-09
WO2015051135A3 true WO2015051135A3 (fr) 2015-09-03

Family

ID=51795773

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2014/058851 WO2015051135A2 (fr) 2013-10-04 2014-10-02 Compositions organiques destinées au traitement de maladies associées à l'hepcidine

Country Status (1)

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WO (1) WO2015051135A2 (fr)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015050871A2 (fr) 2013-10-04 2015-04-09 Novartis Ag Composés organiques destinés au traitement du virus de l'hépatite b
EP3052627B1 (fr) 2013-10-04 2018-08-22 Novartis AG Formats inédits pour composés organiques utilisables en interférence arn
CA3188691A1 (fr) 2013-10-04 2015-04-09 Novartis Ag Extremites 3'pour agents arni destines a etre utilises dans l'interference arn

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WO2007120883A2 (fr) * 2006-04-12 2007-10-25 Isis Pharmaceuticals, Inc. Compositions et leurs utilisations sur l'hepcidine
WO2008036933A2 (fr) * 2006-09-21 2008-03-27 Alnylam Pharmaceuticals, Inc. Compositions et procédés servant à inhiber l'expression du gène hamp
US8084600B2 (en) * 2006-05-04 2011-12-27 Novartis Ag Short interfering ribonucleic acid (siRNA) with improved pharmacological properties
WO2012177921A2 (fr) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc Compositions et procédés d'inhibition de l'expression du peptide antimicrobien hepcidine (hamp) ou du gène lié à hamp

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US6977244B2 (en) 1996-10-04 2005-12-20 Board Of Regents, The University Of Texas Systems Inhibition of Bcl-2 protein expression by liposomal antisense oligodeoxynucleotides
US5891467A (en) 1997-01-31 1999-04-06 Depotech Corporation Method for utilizing neutral lipids to modify in vivo release from multivesicular liposomes
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DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
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US8084600B2 (en) * 2006-05-04 2011-12-27 Novartis Ag Short interfering ribonucleic acid (siRNA) with improved pharmacological properties
US8404831B2 (en) * 2006-05-04 2013-03-26 Novartis Ag Short interfering ribonucleic acid (siRNA) for oral administration
WO2008036933A2 (fr) * 2006-09-21 2008-03-27 Alnylam Pharmaceuticals, Inc. Compositions et procédés servant à inhiber l'expression du gène hamp
WO2012177921A2 (fr) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc Compositions et procédés d'inhibition de l'expression du peptide antimicrobien hepcidine (hamp) ou du gène lié à hamp

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