IL254173B - Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy - Google Patents

Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy

Info

Publication number
IL254173B
IL254173B IL254173A IL25417317A IL254173B IL 254173 B IL254173 B IL 254173B IL 254173 A IL254173 A IL 254173A IL 25417317 A IL25417317 A IL 25417317A IL 254173 B IL254173 B IL 254173B
Authority
IL
Israel
Prior art keywords
col7a1
blistering
exon
skin
treatment
Prior art date
Application number
IL254173A
Other languages
English (en)
Hebrew (he)
Other versions
IL254173A0 (en
Original Assignee
Proqr Therapeutics Ii Bv
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 Proqr Therapeutics Ii Bv, Wings Therapeutics Inc filed Critical Proqr Therapeutics Ii Bv
Publication of IL254173A0 publication Critical patent/IL254173A0/en
Publication of IL254173B publication Critical patent/IL254173B/en

Links

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    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • 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
    • 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
    • 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
    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/34Spatial 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
    • 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
    • C12N2310/3521Methyl
    • 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

Landscapes

  • 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)
IL254173A 2015-03-11 2017-08-27 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy IL254173B (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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
IL254173A0 IL254173A0 (en) 2017-10-31
IL254173B true IL254173B (en) 2022-03-01

Family

ID=52998751

Family Applications (1)

Application Number Title Priority Date Filing Date
IL254173A IL254173B (en) 2015-03-11 2017-08-27 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy

Country Status (20)

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1253335A1 (zh) * 2015-06-01 2019-06-14 Sarepta Therapeutics, Inc. 在vii型胶原蛋白中反义诱导外显子的排除
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
JP2023554180A (ja) * 2020-12-10 2023-12-26 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ジストロフィー性表皮水疱症を治療するための物質および方法。
JPWO2024080311A1 (enExample) * 2022-10-12 2024-04-18
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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US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
WO1997041208A1 (en) 1996-04-26 1997-11-06 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
EP2221376B1 (en) 2001-06-21 2012-11-21 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
AU2002366799A1 (en) 2001-12-19 2003-07-09 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
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
US8765169B2 (en) 2004-02-13 2014-07-01 Smith & Nephew, Inc. Wound healing profile
WO2005086768A2 (en) 2004-03-11 2005-09-22 Albert Einstein College Of Medicine Of Yeshiva University Enhanced production of functional proteins from defective genes
HRP20110352T1 (hr) 2004-06-28 2011-06-30 The University Of Western Australia Antisens olinukleotidi za induciranje zaobilaženja egzona i postupci njihove upotrebe
GB0505202D0 (en) 2005-03-14 2005-04-20 Intercytex Ltd Skin equivalent culture
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PT2161038E (pt) 2006-01-26 2014-03-10 Isis Pharmaceuticals Inc Composições e suas utilizações dirigidas à huntingtina
EP1857548A1 (en) * 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
PL2049664T3 (pl) 2006-08-11 2012-02-29 Biomarin Tech Bv Jednoniciowe oligonukleotydy komplementarne do powtarzalnych elementów do leczenia chorób genetycznych związanych z niestabilnością powtórzeń DNA
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HK1253335A1 (zh) 2015-06-01 2019-06-14 Sarepta Therapeutics, Inc. 在vii型胶原蛋白中反义诱导外显子的排除
SI3307277T1 (sl) 2015-06-15 2021-02-26 Tirmed Pharma Ab Enoverižni oligonukleotidi za uporabo pri zdravljenju motenj kože
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Also Published As

Publication number Publication date
CA2978375A1 (en) 2016-09-15
US20180216106A1 (en) 2018-08-02
CN107567498B (zh) 2021-09-21
WO2016142538A1 (en) 2016-09-15
HUE050898T2 (hu) 2021-01-28
JP6929783B2 (ja) 2021-09-01
EA035732B1 (ru) 2020-07-31
NZ736083A (en) 2021-08-27
ES2803651T3 (es) 2021-01-28
JP2022000020A (ja) 2022-01-04
US11352626B2 (en) 2022-06-07
JP2018509903A (ja) 2018-04-12
MX384959B (es) 2025-03-14
EP3268474A1 (en) 2018-01-17
IL254173A0 (en) 2017-10-31
PL3268474T3 (pl) 2020-08-24
AU2016231067B2 (en) 2022-04-21
JP7188810B2 (ja) 2022-12-13
CN107567498A (zh) 2018-01-09
AU2016231067A1 (en) 2017-10-26
MX2021009135A (es) 2021-09-10
SI3268474T1 (sl) 2020-07-31
EP3763816A1 (en) 2021-01-13
KR20170120617A (ko) 2017-10-31
HRP20200944T1 (hr) 2020-09-18
DK3268474T3 (da) 2020-06-15
PT3268474T (pt) 2020-06-17
MX2017011599A (es) 2018-06-15
US20200399638A1 (en) 2020-12-24
EA201791986A1 (ru) 2018-01-31
GB201504124D0 (en) 2015-04-22
BR112017019225A2 (pt) 2018-05-08
EP3268474B1 (en) 2020-05-27
US10563198B2 (en) 2020-02-18

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