CA2978375A1 - 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
CA2978375A1
CA2978375A1 CA2978375A CA2978375A CA2978375A1 CA 2978375 A1 CA2978375 A1 CA 2978375A1 CA 2978375 A CA2978375 A CA 2978375A CA 2978375 A CA2978375 A CA 2978375A CA 2978375 A1 CA2978375 A1 CA 2978375A1
Authority
CA
Canada
Prior art keywords
exon
mrna
oligoribonucleotide
oligonucleotide
antisense
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
CA2978375A
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English (en)
French (fr)
Inventor
Elisabeth Marlene Haisma
Marko POTMAN
Wouter BEUMER
Vera BRINKS
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.)
Wings Therapeutics Inc
Original Assignee
ProQR Therapeutics II BV
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Filing date
Publication date
Application filed by ProQR Therapeutics II BV filed Critical ProQR Therapeutics II BV
Publication of CA2978375A1 publication Critical patent/CA2978375A1/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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • 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
    • 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
    • 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
    • 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)
CA2978375A 2015-03-11 2016-03-11 Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy Pending CA2978375A1 (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 (1)

Publication Number Publication Date
CA2978375A1 true CA2978375A1 (en) 2016-09-15

Family

ID=52998751

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2978375A Pending CA2978375A1 (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=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
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
AU2005215213C1 (en) 2004-02-13 2011-04-28 Smith & Nephew Orthopaedics Ag Pharmaceutical composition comprising a fibrin matrix and dermal fibroblasts for the treatment of wounds
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
GB0505202D0 (en) 2005-03-14 2005-04-20 Intercytex Ltd Skin equivalent culture
KR101083047B1 (ko) 2005-12-16 2011-11-16 주식회사 엠씨티티 간엽줄기세포의 배양방법 및 간엽줄기세포를 포함하는 피부결함의 치료용 조성물
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
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
CA2717251A1 (en) 2007-03-07 2008-09-12 Aarhus Universitet Pig model for breast cancer, mitochondria related protein folding disorders and/or epidermolysis bullosa simplex
US8077297B2 (en) 2008-06-30 2011-12-13 Nellcor Puritan Bennett Ireland Methods and systems for discriminating bands in scalograms
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
WO2012018257A1 (en) 2010-08-05 2012-02-09 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
EP2672977A1 (en) * 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
WO2013033436A1 (en) * 2011-09-01 2013-03-07 University Of Iowa Research Foundation Oligonucleotide-based probes for detection of bacterial nucleases
WO2013052965A2 (en) * 2011-10-06 2013-04-11 Miragen Therapeutics Control of whole body energy homeostasis by microrna regulation
EP3279324B1 (en) * 2011-10-11 2021-02-17 Institut National De La Sante Et De La Recherche Medicale (Inserm) Exon skipping therapy for dystrophic epidermolysis bullosa
CA2866563A1 (en) 2012-03-07 2013-09-12 Fibrocell Technologies, Inc. Topical dermal formulations and methods of personalized treatment of skin
US20150141320A1 (en) 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
JP6571075B2 (ja) 2013-07-08 2019-09-04 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 アンチセンスオリゴヌクレオチド介在性エクソンスキッピングを、それを必要とする対象の網膜において行うための方法
US10016466B2 (en) 2014-11-17 2018-07-10 Rev Pharma Corp Topical medicament for skin and mucosal injuries associated with Epidermolisis bullosa
US10849917B2 (en) 2015-06-01 2020-12-01 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type VII collagen
US10626401B2 (en) 2015-06-15 2020-04-21 Tirmed Pharma Ab Single-stranded oligonucleotides for use in the medical treatment of skin disorders
WO2017078526A2 (en) 2015-11-05 2017-05-11 Rijksuniversiteit Groningen Antisense oligonucleotide-mediated exon skipping as systemic therapy for recessive dystrophic epidermolysis bullosa (rdeb).

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
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
MX2021009135A (es) 2021-09-10
DK3268474T3 (da) 2020-06-15
US20180216106A1 (en) 2018-08-02

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