AU2019222767A1 - Oligonucleotide therapy for Wilson disease - Google Patents

Oligonucleotide therapy for Wilson disease Download PDF

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Publication number
AU2019222767A1
AU2019222767A1 AU2019222767A AU2019222767A AU2019222767A1 AU 2019222767 A1 AU2019222767 A1 AU 2019222767A1 AU 2019222767 A AU2019222767 A AU 2019222767A AU 2019222767 A AU2019222767 A AU 2019222767A AU 2019222767 A1 AU2019222767 A1 AU 2019222767A1
Authority
AU
Australia
Prior art keywords
antisense oligonucleotide
atp7b
seq
exon
sequence
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2019222767A
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English (en)
Inventor
Daniele MERICO
Matthew O'hara
Frank SCHMITGES
Erno Wienholds
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Deep Genomics Inc
Original Assignee
Deep Genomics Inc
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 Deep Genomics Inc filed Critical Deep Genomics Inc
Publication of AU2019222767A1 publication Critical patent/AU2019222767A1/en
Abandoned legal-status Critical Current

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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
    • C12N15/1137—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 enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03004—Cu2+-exporting ATPase (3.6.3.4)

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2019222767A 2018-02-14 2019-02-14 Oligonucleotide therapy for Wilson disease Abandoned AU2019222767A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862630565P 2018-02-14 2018-02-14
US62/630,565 2018-02-14
PCT/US2019/018076 WO2019161105A1 (en) 2018-02-14 2019-02-14 Oligonucleotide therapy for wilson disease

Publications (1)

Publication Number Publication Date
AU2019222767A1 true AU2019222767A1 (en) 2020-08-27

Family

ID=67619598

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019222767A Abandoned AU2019222767A1 (en) 2018-02-14 2019-02-14 Oligonucleotide therapy for Wilson disease

Country Status (15)

Country Link
US (1) US11578327B2 (https=)
EP (1) EP3752614A4 (https=)
JP (1) JP2021513851A (https=)
KR (1) KR20200120675A (https=)
CN (1) CN112041439A (https=)
AU (1) AU2019222767A1 (https=)
BR (1) BR112020016524A2 (https=)
CA (1) CA3090517A1 (https=)
CL (1) CL2020002100A1 (https=)
CO (1) CO2020010226A2 (https=)
IL (1) IL276701A (https=)
MX (1) MX2020008533A (https=)
PH (1) PH12020551219A1 (https=)
SG (1) SG11202007644PA (https=)
WO (1) WO2019161105A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease
CN117925624B (zh) * 2024-03-22 2024-05-31 上海凌医生物科技有限公司 一种金属响应调控元件

Family Cites Families (36)

* Cited by examiner, † Cited by third party
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US5578493A (en) * 1993-09-01 1996-11-26 The Trustees Of Columbia University In The City Of New York Wilson's disease gene
CA2108927C (en) 1993-09-21 2008-09-02 Peter Bull Wilson disease gene
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US20040101854A1 (en) * 2002-11-21 2004-05-27 Isis Pharmaceuticals Inc. Modulation of BCL2-associated athanogene expression
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20030180953A1 (en) 2000-12-29 2003-09-25 Elitra Pharmaceuticals, Inc. Gene disruption methodologies for drug target discovery
US20040126761A1 (en) * 2002-12-10 2004-07-01 Isis Pharmaceuticals Inc. Modulation of alpha-methylacyl-CoA racemase expression
WO2004035819A2 (en) 2002-10-21 2004-04-29 Exiqon A/S Oligonucleotide analogues for detecting and analyzing nucleic acids
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
CN1934251A (zh) * 2004-01-29 2007-03-21 塞尔卓姆股份公司 使用atp7a－调控剂治疗神经变性疾病
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
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JP4841279B2 (ja) 2006-03-23 2011-12-21 一般財団法人 化学物質評価研究機構 被検物質の発がん性予測方法
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Also Published As

Publication number Publication date
SG11202007644PA (en) 2020-09-29
PH12020551219A1 (en) 2021-04-19
MX2020008533A (es) 2020-11-06
US11578327B2 (en) 2023-02-14
CA3090517A1 (en) 2019-08-22
EP3752614A4 (en) 2021-11-10
CL2020002100A1 (es) 2021-01-29
IL276701A (en) 2020-09-30
WO2019161105A1 (en) 2019-08-22
CN112041439A (zh) 2020-12-04
US20210032629A1 (en) 2021-02-04
CO2020010226A2 (es) 2020-12-10
JP2021513851A (ja) 2021-06-03
EP3752614A1 (en) 2020-12-23
BR112020016524A2 (pt) 2021-09-28
KR20200120675A (ko) 2020-10-21

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