SI3268474T1 - Oligonukleotidi, ki ustrezajo COL7A1 ekson 73 za zdravljenje bulozne epidermolize - Google Patents
Oligonukleotidi, ki ustrezajo COL7A1 ekson 73 za zdravljenje bulozne epidermolizeInfo
- Publication number
- SI3268474T1 SI3268474T1 SI201630794T SI201630794T SI3268474T1 SI 3268474 T1 SI3268474 T1 SI 3268474T1 SI 201630794 T SI201630794 T SI 201630794T SI 201630794 T SI201630794 T SI 201630794T SI 3268474 T1 SI3268474 T1 SI 3268474T1
- Authority
- SI
- Slovenia
- Prior art keywords
- treatment
- oligonucleotides corresponding
- bullous epidermolysis
- col7a1 exon
- col7a1
- Prior art date
Links
Classifications
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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
-
- 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
- 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
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)
Applications Claiming Priority (3)
| 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 |
| EP16709465.5A EP3268474B1 (en) | 2015-03-11 | 2016-03-11 | Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SI3268474T1 true SI3268474T1 (sl) | 2020-07-31 |
Family
ID=52998751
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SI201630794T SI3268474T1 (sl) | 2015-03-11 | 2016-03-11 | Oligonukleotidi, ki ustrezajo COL7A1 ekson 73 za zdravljenje bulozne epidermolize |
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)
| 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 |
Family Cites Families (35)
| 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 |
| 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 |
| WO2007069813A1 (en) | 2005-12-16 | 2007-06-21 | Modern Cell & Tissue Technologies Inc. | Methods for culturing mesenchymal stem cell and compositions comprising mesenchymal stem cell for reparing skin defects |
| 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 |
| EP2134848A2 (en) | 2007-03-07 | 2009-12-23 | 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 |
| EP2601294B1 (en) | 2010-08-05 | 2018-11-28 | Academisch Ziekenhuis Leiden h.o.d.n. LUMC | Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins |
| US9078911B2 (en) * | 2011-02-08 | 2015-07-14 | The Charlotte-Mecklenburg Hospital Authority | Antisense oligonucleotides |
| US9603949B2 (en) * | 2011-09-01 | 2017-03-28 | University Of Iowa Research Foundation | Oligonucleotide-based probes for detection of bacterial nucleases |
| JP6177243B2 (ja) * | 2011-10-06 | 2017-08-09 | ミラゲン セラピューティクス， インコーポレイテッド | マイクロｒｎａの調節による全身エネルギーホメオスタシスの制御 |
| WO2013053819A1 (en) | 2011-10-11 | 2013-04-18 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Exon skipping therapy for dystrophic epidermolysis bullosa |
| KR20150009521A (ko) | 2012-03-07 | 2015-01-26 | 파이브로셀 테크놀로지스, 인코퍼레이티드 | 국소피부제제 및 개인맞춤형 피부치료방법 |
| WO2013173637A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| EP3702461A1 (en) | 2013-07-08 | 2020-09-02 | Institut National De La Sante Et De La Recherche Medicale - Inserm | Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof |
| PT3220924T (pt) | 2014-11-17 | 2022-01-12 | Rev Pharma Corp | Medicamento tópico para lesões da pele e mucosas associadas à epidermólise bolhosa |
| 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 |
| WO2017078526A2 (en) | 2015-11-05 | 2017-05-11 | Rijksuniversiteit Groningen | Antisense oligonucleotide-mediated exon skipping as systemic therapy for recessive dystrophic epidermolysis bullosa (rdeb). |
-
2015
- 2015-03-11 GB GBGB1504124.7A patent/GB201504124D0/en not_active Ceased
-
2016
- 2016-03-11 WO PCT/EP2016/055360 patent/WO2016142538A1/en not_active Ceased
- 2016-03-11 DK DK16709465.5T patent/DK3268474T3/da active
- 2016-03-11 EA EA201791986A patent/EA035732B1/ru unknown
- 2016-03-11 PL PL16709465T patent/PL3268474T3/pl unknown
- 2016-03-11 KR KR1020177025038A patent/KR20170120617A/ko not_active Abandoned
- 2016-03-11 CA CA2978375A patent/CA2978375A1/en active Pending
- 2016-03-11 CN CN201680015142.2A patent/CN107567498B/zh not_active Expired - Fee Related
- 2016-03-11 ES ES16709465T patent/ES2803651T3/es active Active
- 2016-03-11 HR HRP20200944TT patent/HRP20200944T1/hr unknown
- 2016-03-11 JP JP2017547468A patent/JP6929783B2/ja not_active Expired - Fee Related
- 2016-03-11 HU HUE16709465A patent/HUE050898T2/hu unknown
- 2016-03-11 EP EP16709465.5A patent/EP3268474B1/en active Active
- 2016-03-11 US US15/554,420 patent/US10563198B2/en active Active
- 2016-03-11 AU AU2016231067A patent/AU2016231067B2/en not_active Ceased
- 2016-03-11 BR BR112017019225-0A patent/BR112017019225A2/pt not_active Application Discontinuation
- 2016-03-11 SI SI201630794T patent/SI3268474T1/sl unknown
- 2016-03-11 EP EP20175344.9A patent/EP3763816A1/en not_active Withdrawn
- 2016-03-11 MX MX2017011599A patent/MX384959B/es unknown
- 2016-03-11 PT PT167094655T patent/PT3268474T/pt unknown
-
2017
- 2017-08-27 IL IL254173A patent/IL254173B/en unknown
- 2017-09-08 MX MX2021009135A patent/MX2021009135A/es unknown
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2020
- 2020-01-17 US US16/746,403 patent/US11352626B2/en active Active
-
2021
- 2021-08-11 JP JP2021131294A patent/JP7188810B2/ja active Active
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