AU2016231067B2 - Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy - Google Patents

Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy Download PDF

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Publication number
AU2016231067B2
AU2016231067B2 AU2016231067A AU2016231067A AU2016231067B2 AU 2016231067 B2 AU2016231067 B2 AU 2016231067B2 AU 2016231067 A AU2016231067 A AU 2016231067A AU 2016231067 A AU2016231067 A AU 2016231067A AU 2016231067 B2 AU2016231067 B2 AU 2016231067B2
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Australia
Prior art keywords
exon
oligoribonucleotide
antisense
mrna
oligonucleotide
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Ceased
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AU2016231067A
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AU2016231067A1 (en
Inventor
Wouter BEUMER
Vera BRINKS
Elisabeth Marlene Haisma
Marko POTMAN
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Wings Therapeutics Inc
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Wings Therapeutics Inc
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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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • C12N2310/32—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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/3521—Methyl
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2016231067A 2015-03-11 2016-03-11 Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy Ceased AU2016231067B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1504124.7 2015-03-11
GBGB1504124.7A GB201504124D0 (en) 2015-03-11 2015-03-11 Oligonucleotides
PCT/EP2016/055360 WO2016142538A1 (en) 2015-03-11 2016-03-11 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy

Publications (2)

Publication Number Publication Date
AU2016231067A1 AU2016231067A1 (en) 2017-10-26
AU2016231067B2 true AU2016231067B2 (en) 2022-04-21

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AU2016231067A Ceased AU2016231067B2 (en) 2015-03-11 2016-03-11 Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy

Country Status (20)

Country Link
US (2) US10563198B2 (https=)
EP (2) EP3268474B1 (https=)
JP (2) JP6929783B2 (https=)
KR (1) KR20170120617A (https=)
CN (1) CN107567498B (https=)
AU (1) AU2016231067B2 (https=)
BR (1) BR112017019225A2 (https=)
CA (1) CA2978375A1 (https=)
DK (1) DK3268474T3 (https=)
EA (1) EA035732B1 (https=)
ES (1) ES2803651T3 (https=)
GB (1) GB201504124D0 (https=)
HR (1) HRP20200944T1 (https=)
HU (1) HUE050898T2 (https=)
IL (1) IL254173B (https=)
MX (2) MX384959B (https=)
PL (1) PL3268474T3 (https=)
PT (1) PT3268474T (https=)
SI (1) SI3268474T1 (https=)
WO (1) WO2016142538A1 (https=)

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US10849917B2 (en) * 2015-06-01 2020-12-01 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type VII collagen
US12227743B2 (en) 2018-01-25 2025-02-18 Osaka University Pharmaceutical composition for treatment of diseases associated with upregulated periostin expression or periostin splice variant switching
GB201902735D0 (en) 2019-02-28 2019-04-17 Proqr Therapeutics Ii Bv Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa
WO2021113270A1 (en) 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing
WO2022122900A1 (en) * 2020-12-10 2022-06-16 INSERM (Institut National de la Santé et de la Recherche Médicale) Substances and methods for treating dystrophic epidermolysis bullosa
EP4609882A1 (en) * 2022-10-12 2025-09-03 CPC Corporation Gene introduction method, gene therapy method, and tissue regeneration method
CN116555349A (zh) * 2023-01-09 2023-08-08 中吉智药(南京)生物技术有限公司 一种腺相关病毒载体及其构建方法与应用
CN118001404B (zh) * 2024-02-06 2025-02-25 四川大学华西医院 抑制C17orf67基因表达试剂在制备治疗获得性大疱表皮松解症药物中的用途

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Also Published As

Publication number Publication date
EA201791986A1 (ru) 2018-01-31
US10563198B2 (en) 2020-02-18
WO2016142538A1 (en) 2016-09-15
JP2018509903A (ja) 2018-04-12
PT3268474T (pt) 2020-06-17
SI3268474T1 (sl) 2020-07-31
IL254173A0 (en) 2017-10-31
AU2016231067A1 (en) 2017-10-26
HUE050898T2 (hu) 2021-01-28
EP3763816A1 (en) 2021-01-13
IL254173B (en) 2022-03-01
KR20170120617A (ko) 2017-10-31
GB201504124D0 (en) 2015-04-22
EA035732B1 (ru) 2020-07-31
CN107567498B (zh) 2021-09-21
MX2017011599A (es) 2018-06-15
US11352626B2 (en) 2022-06-07
EP3268474A1 (en) 2018-01-17
JP2022000020A (ja) 2022-01-04
US20200399638A1 (en) 2020-12-24
ES2803651T3 (es) 2021-01-28
JP6929783B2 (ja) 2021-09-01
JP7188810B2 (ja) 2022-12-13
EP3268474B1 (en) 2020-05-27
HRP20200944T1 (hr) 2020-09-18
CN107567498A (zh) 2018-01-09
MX384959B (es) 2025-03-14
NZ736083A (en) 2021-08-27
PL3268474T3 (pl) 2020-08-24
BR112017019225A2 (pt) 2018-05-08
CA2978375A1 (en) 2016-09-15
MX2021009135A (es) 2021-09-10
DK3268474T3 (da) 2020-06-15
US20180216106A1 (en) 2018-08-02

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