PT3268474T - Oligonucleótidos compatíveis com o exão 73 de col7a1 para terapia de epidermólise bolhosa - Google Patents

Oligonucleótidos compatíveis com o exão 73 de col7a1 para terapia de epidermólise bolhosa

Info

Publication number
PT3268474T
PT3268474T PT167094655T PT16709465T PT3268474T PT 3268474 T PT3268474 T PT 3268474T PT 167094655 T PT167094655 T PT 167094655T PT 16709465 T PT16709465 T PT 16709465T PT 3268474 T PT3268474 T PT 3268474T
Authority
PT
Portugal
Prior art keywords
therapy
epidermolysis bullosa
col7a1 exon
oligonucleotides matching
oligonucleotides
Prior art date
Application number
PT167094655T
Other languages
English (en)
Portuguese (pt)
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 PT3268474T publication Critical patent/PT3268474T/pt

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    • 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
    • C12N15/10Processes 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
    • 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
    • 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
    • C12N15/1138Non-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
    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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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
    • CCHEMISTRY; METALLURGY
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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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
  • 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)
PT167094655T 2015-03-11 2016-03-11 Oligonucleótidos compatíveis com o exão 73 de col7a1 para terapia de epidermólise bolhosa PT3268474T (pt)

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
PT3268474T true PT3268474T (pt) 2020-06-17

Family

ID=52998751

Family Applications (1)

Application Number Title Priority Date Filing Date
PT167094655T PT3268474T (pt) 2015-03-11 2016-03-11 Oligonucleótidos compatíveis com o exão 73 de col7a1 para terapia de epidermólise bolhosa

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
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
CA2790034A1 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
WO2003053369A1 (en) 2001-12-19 2003-07-03 Alwyn Company, Inc. Allantoin-containing preparations for administration as gels and aerosols
US20040096833A1 (en) 2002-11-16 2004-05-20 Isis Pharmaceuticals Inc. Modulation of FBP-interacting repressor expression
US20040092464A1 (en) 2002-11-11 2004-05-13 Isis Pharmaceuticals Inc. Modulation of mitogen-activated protein kinase kinase kinase 11 expression
DE10238298A1 (de) 2002-08-21 2004-03-04 Beiersdorf Ag Verwendung von Antisense-Oligonucleotiden zur Behandlung von degenerativen Hauterscheinungen
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
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WO2005086768A2 (en) 2004-03-11 2005-09-22 Albert Einstein College Of Medicine Of Yeshiva University Enhanced production of functional proteins from defective genes
ATE498685T1 (de) 2004-06-28 2011-03-15 Univ Western Australia Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon
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PT2161038E (pt) 2006-01-26 2014-03-10 Isis Pharmaceuticals Inc Composições e suas utilizações dirigidas à huntingtina
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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
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
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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