CA3090517A1 - Oligonucleotide therapy for wilson disease - Google Patents

Oligonucleotide therapy for wilson disease Download PDF

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Publication number
CA3090517A1
CA3090517A1 CA3090517A CA3090517A CA3090517A1 CA 3090517 A1 CA3090517 A1 CA 3090517A1 CA 3090517 A CA3090517 A CA 3090517A CA 3090517 A CA3090517 A CA 3090517A CA 3090517 A1 CA3090517 A1 CA 3090517A1
Authority
CA
Canada
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.)
Pending
Application number
CA3090517A
Other languages
English (en)
French (fr)
Inventor
Frank SCHMITGES
Daniele MERICO
Erno Wienholds
Matthew O'hara
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 CA3090517A1 publication Critical patent/CA3090517A1/en
Pending 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)
CA3090517A 2018-02-14 2019-02-14 Oligonucleotide therapy for wilson disease Pending CA3090517A1 (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
CA3090517A1 true CA3090517A1 (en) 2019-08-22

Family

ID=67619598

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3090517A Pending CA3090517A1 (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
AU1039397A (en) 1995-11-22 1997-06-27 Johns Hopkins University, The 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
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
CA2566519C (en) 2004-05-14 2020-04-21 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 (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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CA2860676A1 (en) * 2012-01-09 2013-07-18 Novartis Ag Organic compositions to treat beta-catenin-related diseases
WO2014036301A1 (en) * 2012-08-30 2014-03-06 Isis Pharmaceuticals, Inc. Modulation of copper related diseases by ctr1
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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
AU2014346658A1 (en) 2013-11-06 2016-06-02 Solstice Biologics, Ltd. Polynucleotide constructs having disulfide groups
JP2017522046A (ja) 2014-06-06 2017-08-10 ソルスティス バイオロジクス，リミティッド 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物
WO2016004043A1 (en) 2014-06-30 2016-01-07 Blend Therapeutics, Inc. Targeted conjugates and particles and formulations thereof
JP2017533721A (ja) 2014-11-14 2017-11-16 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． タンパク質の調節のための化合物及び方法
EP3247366A4 (en) 2015-01-21 2018-10-31 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
CN105986015B (zh) * 2015-02-05 2020-10-23 大连晶泰生物技术有限公司 一种基于高通量测序的多样本的一个或多个靶序列的检测方法和试剂盒
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AU2016365828A1 (en) 2015-12-08 2018-07-05 Solstice Biologics, Ltd. Polynucleotide constructs having an auxiliary moiety non-bioreversibly linked to an internucleoside phosphate or phosphorothioate
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Also Published As

Publication number Publication date
SG11202007644PA (en) 2020-09-29
PH12020551219A1 (en) 2021-04-19
AU2019222767A1 (en) 2020-08-27
MX2020008533A (es) 2020-11-06
US11578327B2 (en) 2023-02-14
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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