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WO2015051135A3 - Organic compositions to treat hepcidin-related diseases - Google Patents

Organic compositions to treat hepcidin-related diseases 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
Authority
WO
WIPO (PCT)
Prior art keywords
anemia
disease
hepcidin
iron
related diseases
Prior art date
Application number
PCT/US2014/058851
Other languages
French (fr)
Other versions
WO2015051135A2 (en
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
Original Assignee
Novartis Ag
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Novartis Ag filed Critical Novartis Ag
Publication of WO2015051135A2 publication Critical patent/WO2015051135A2/en
Publication of WO2015051135A3 publication Critical patent/WO2015051135A3/en

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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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Endocrinology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present disclosure relates to methods of treating Hepcidin-related diseases such as anemia, iron-deficient erythropoiesis, hypoferremia, impaired dietary iron uptake, iron sequestration, anemia of inflammation (AI) (sometimes designated anemia of chronic disease or ACD), atherosclerosis, diabetes, and multiple neurodegenerative disorders such as Alzheimers disease, Parkinsons disease and Friedrichs ataxia, heart failure, chronic kidney disease, cardiorenal-anemia syndrome, infection, blood loss, hemolysis, vitamin B12 or folate deficiency, hyperparathyroidism, hemoglobinopathies and malignancies, cancer, AIDS, surgery, runted growth, and hair loss, using a therapeutically effective amount of a RNAi agent to Hepcidin.
PCT/US2014/058851 2013-10-04 2014-10-02 Organic compositions to treat hepcidin-related diseases WO2015051135A2 (en)

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 (en) 2015-04-09
WO2015051135A3 true WO2015051135A3 (en) 2015-09-03

Family

ID=51795773

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2014/058851 WO2015051135A2 (en) 2013-10-04 2014-10-02 Organic compositions to treat hepcidin-related diseases

Country Status (1)

Country Link
WO (1) WO2015051135A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015051045A2 (en) 2013-10-04 2015-04-09 Novartis Ag 3'END CAPS FOR RNAi AGENTS FOR USE IN RNA INTERFERENCE
US9988627B2 (en) 2013-10-04 2018-06-05 Novartis Ag Formats for organic compounds for use in RNA interference
WO2015050871A2 (en) 2013-10-04 2015-04-09 Novartis Ag Organic compounds to treat hepatitis b virus
CN115703826A (en) * 2021-08-03 2023-02-17 浙江大学 Hepcidin modified body and application thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007120883A2 (en) * 2006-04-12 2007-10-25 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hepcidin
WO2008036933A2 (en) * 2006-09-21 2008-03-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the hamp gene
US8084600B2 (en) * 2006-05-04 2011-12-27 Novartis Ag Short interfering ribonucleic acid (siRNA) with improved pharmacological properties
WO2012177921A2 (en) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc Compositions and methods for inhibiting hepcidin antimicrobial peptide (hamp) or hamp-related gene expression

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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
WO2000044914A1 (en) 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna
DE19956568A1 (en) 1999-01-30 2000-08-17 Roland Kreutzer Method and medicament for inhibiting the expression of a given gene
IL151781A0 (en) 2000-03-16 2003-04-10 Genetica Inc Methods and compositions for rna interference
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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007120883A2 (en) * 2006-04-12 2007-10-25 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hepcidin
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 (en) * 2006-09-21 2008-03-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the hamp gene
WO2012177921A2 (en) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc Compositions and methods for inhibiting hepcidin antimicrobial peptide (hamp) or hamp-related gene expression

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
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G. BARTOLOMEI ET AL: "Modulation of hepatitis C virus replication by iron and hepcidin in Huh7 hepatocytes", JOURNAL OF GENERAL VIROLOGY, vol. 92, no. 9, 18 May 2011 (2011-05-18), pages 2072 - 2081, XP055042420, ISSN: 0022-1317, DOI: 10.1099/vir.0.032706-0 *
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