HUE050898T2 - Oligonukleotidok, amelyek megfelelnek a COL7A1 73-as exonnak, epidermolysis bullosa terápiához - Google Patents

Oligonukleotidok, amelyek megfelelnek a COL7A1 73-as exonnak, epidermolysis bullosa terápiához

Info

Publication number
HUE050898T2
HUE050898T2 HUE16709465A HUE16709465A HUE050898T2 HU E050898 T2 HUE050898 T2 HU E050898T2 HU E16709465 A HUE16709465 A HU E16709465A HU E16709465 A HUE16709465 A HU E16709465A HU E050898 T2 HUE050898 T2 HU E050898T2
Authority
HU
Hungary
Prior art keywords
col7a1
exon
therapy
epidermolysis bullosa
oligonucleotides corresponding
Prior art date
Application number
HUE16709465A
Other languages
English (en)
Hungarian (hu)
Inventor
Elisabeth Haisma
Marko Potman
Wouter Beumer
Vera Brinks
Original Assignee
Wings Therapeutics 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 Wings Therapeutics Inc filed Critical Wings Therapeutics Inc
Publication of HUE050898T2 publication Critical patent/HUE050898T2/hu

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    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • 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
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • 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
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/1138Non-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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • 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
    • C12N2310/3521Methyl
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration 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)
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  • 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)
HUE16709465A 2015-03-11 2016-03-11 Oligonukleotidok, amelyek megfelelnek a COL7A1 73-as exonnak, epidermolysis bullosa terápiához HUE050898T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1504124.7A GB201504124D0 (en) 2015-03-11 2015-03-11 Oligonucleotides

Publications (1)

Publication Number Publication Date
HUE050898T2 true HUE050898T2 (hu) 2021-01-28

Family

ID=52998751

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE16709465A HUE050898T2 (hu) 2015-03-11 2016-03-11 Oligonukleotidok, amelyek megfelelnek a COL7A1 73-as exonnak, epidermolysis bullosa terápiához

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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AU2808397A (en) 1996-04-26 1997-11-19 Case Western Reserve University Skin regeneration using mesenchymal stem cells
JP2002080662A (ja) 2000-09-08 2002-03-19 Jsr Corp ゴム組成物
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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
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
AU2016231067B2 (en) 2022-04-21
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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