SG11202007644PA - Oligonucleotide therapy for wilson disease - Google Patents

Oligonucleotide therapy for wilson disease

Info

Publication number
SG11202007644PA
SG11202007644PA SG11202007644PA SG11202007644PA SG11202007644PA SG 11202007644P A SG11202007644P A SG 11202007644PA SG 11202007644P A SG11202007644P A SG 11202007644PA SG 11202007644P A SG11202007644P A SG 11202007644PA SG 11202007644P A SG11202007644P A SG 11202007644PA
Authority
SG
Singapore
Prior art keywords
wilson disease
oligonucleotide therapy
oligonucleotide
therapy
wilson
Prior art date
Application number
SG11202007644PA
Inventor
Daniele Merico
Erno Wienholds
Frank Schmitges
Matthew O'hara
Original Assignee
Deep Genomics Incorporated
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 Incorporated filed Critical Deep Genomics Incorporated
Publication of SG11202007644PA publication Critical patent/SG11202007644PA/en

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Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y306/00Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03004Cu2+-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)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (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)
SG11202007644PA 2018-02-14 2019-02-14 Oligonucleotide therapy for wilson disease SG11202007644PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862630565P 2018-02-14 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
SG11202007644PA true SG11202007644PA (en) 2020-09-29

Family

ID=67619598

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202007644PA SG11202007644PA (en) 2018-02-14 2019-02-14 Oligonucleotide therapy for wilson disease

Country Status (14)

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

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117925624B (en) * 2024-03-22 2024-05-31 上海凌医生物科技有限公司 Metal response regulating element

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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
EP0862439A4 (en) 1995-11-22 2001-01-10 O Paul O P Ts Ligands to enhance cellular uptake of biomolecules
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
AU2003273778A1 (en) 2002-10-21 2004-05-04 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
EP1718741A2 (en) * 2004-01-29 2006-11-08 Cellzome Ag Treatment of neurodegenerative diseases by the use of atp7a
WO2005116204A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
EP2322650A1 (en) 2004-05-14 2011-05-18 Rosetta Genomics Ltd MicroRNAs and uses thereof
US20070048756A1 (en) 2005-04-18 2007-03-01 Affymetrix, Inc. Methods for whole genome association studies
EP1783645A1 (en) * 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
US8178503B2 (en) 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
JP4841279B2 (en) 2006-03-23 2011-12-21 一般財団法人 化学物質評価研究機構 Method for predicting carcinogenicity of test substance
EP4074344A1 (en) 2007-12-04 2022-10-19 Arbutus Biopharma Corporation Targeting lipids
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US20160258005A1 (en) * 2013-07-02 2016-09-08 The Trustees Of The University Of Pennsylvania Methods for rapid ribonucleic acid fluorescence in situ hybridization
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CN105986015B (en) * 2015-02-05 2020-10-23 大连晶泰生物技术有限公司 Method and kit for detecting one or more target sequences of multiple samples based on high-throughput sequencing
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Also Published As

Publication number Publication date
JP2021513851A (en) 2021-06-03
US11578327B2 (en) 2023-02-14
CL2020002100A1 (en) 2021-01-29
CN112041439A (en) 2020-12-04
AU2019222767A1 (en) 2020-08-27
IL276701A (en) 2020-09-30
BR112020016524A2 (en) 2021-09-28
EP3752614A1 (en) 2020-12-23
EP3752614A4 (en) 2021-11-10
WO2019161105A1 (en) 2019-08-22
CA3090517A1 (en) 2019-08-22
CO2020010226A2 (en) 2020-12-10
KR20200120675A (en) 2020-10-21
US20210032629A1 (en) 2021-02-04
MX2020008533A (en) 2020-11-06

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