MX394185B - Oligonucleotidos antisentido para tratar epidermolisis bullosa distrofica. - Google Patents

Oligonucleotidos antisentido para tratar epidermolisis bullosa distrofica.

Info

Publication number
MX394185B
MX394185B MX2017014855A MX2017014855A MX394185B MX 394185 B MX394185 B MX 394185B MX 2017014855 A MX2017014855 A MX 2017014855A MX 2017014855 A MX2017014855 A MX 2017014855A MX 394185 B MX394185 B MX 394185B
Authority
MX
Mexico
Prior art keywords
treatment
antisense oligonucleotides
epidermolysis bullosa
dystrophic epidermolysis
inclusion
Prior art date
Application number
MX2017014855A
Other languages
English (en)
Other versions
MX2017014855A (es
Inventor
Elisabeth Marlene Haisma
Gerardus Johannes Platenburg
Marko Potman
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
Priority claimed from GBGB1508733.1A external-priority patent/GB201508733D0/en
Priority claimed from GBGB1516505.3A external-priority patent/GB201516505D0/en
Application filed by Wings Therapeutics Inc filed Critical Wings Therapeutics Inc
Publication of MX2017014855A publication Critical patent/MX2017014855A/es
Publication of MX394185B publication Critical patent/MX394185B/es

Links

Classifications

    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • C12N2310/32—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Dermatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)

Abstract

La presente invención se refiere a un oligonucleótido antisentido (AON) capaz de prevenir o reducir la inclusión de exón 80 en un m RNA de COL7A1 humano, cuando dicho mRNA es producido mediante empalme a partir de un pre-mRNA en una célula, en donde el AON comprende una secuencia de nucleótidos seleccionada del grupo que consiste en las SEQ ID NOs: 8, 26, 31, y 32.
MX2017014855A 2015-05-21 2016-05-20 Oligonucleotidos antisentido para tratar epidermolisis bullosa distrofica. MX394185B (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1508733.1A GB201508733D0 (en) 2015-05-21 2015-05-21 Antisense oligonucleotides
GBGB1516505.3A GB201516505D0 (en) 2015-09-17 2015-09-17 Antisense oligonucleotides
PCT/EP2016/061495 WO2016185041A1 (en) 2015-05-21 2016-05-20 Antisense oligonucleotides to treat dystrophic epidermolysis bullosa

Publications (2)

Publication Number Publication Date
MX2017014855A MX2017014855A (es) 2018-04-20
MX394185B true MX394185B (es) 2025-03-24

Family

ID=56026883

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2017014855A MX394185B (es) 2015-05-21 2016-05-20 Oligonucleotidos antisentido para tratar epidermolisis bullosa distrofica.

Country Status (12)

Country Link
US (2) US10370660B2 (es)
EP (1) EP3298144A1 (es)
JP (2) JP6871175B2 (es)
KR (1) KR20180012255A (es)
CN (1) CN107667174B (es)
AU (1) AU2016265457B2 (es)
CA (1) CA2986642A1 (es)
EA (1) EA036393B1 (es)
HK (1) HK1245826A1 (es)
IL (1) IL255180B2 (es)
MX (1) MX394185B (es)
WO (1) WO2016185041A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20180012255A (ko) 2015-05-21 2018-02-05 프로큐알 테라퓨틱스 Ⅱ 비.브이. 이영양성 수포성 표피박리증 치료를 위한 안티센스 올리고뉴클레오타이드
MA50829A (fr) * 2015-06-01 2018-04-11 Sarepta Therapeutics Inc Exclusion d'exon induite pat technologie antisens dans le collagène de type vii
GB201902735D0 (en) 2019-02-28 2019-04-17 Proqr Therapeutics Ii Bv Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa
JP7727628B2 (ja) 2019-12-03 2025-08-21 アラマー バイオサイエンシーズ， インク． 核酸結合免疫サンドイッチアッセイ（ｎｕｌｉｓａ）
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 (12)

* 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
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US20040023378A1 (en) * 2002-07-31 2004-02-05 Isis Pharmaceuticals Inc. Antisense modulation of KIAA1531 protein expression
EP1857548A1 (en) * 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
PL2203173T3 (pl) * 2007-10-26 2016-06-30 Academisch Ziekenhuis Leiden Środki i sposoby przeciwdziałania zaburzeniom mięśni
CN102695797B (zh) * 2009-06-16 2018-05-25 库尔纳公司 通过抑制针对胶原基因的天然反义转录物来治疗胶原基因相关的疾病
EP2766480B1 (en) * 2011-10-11 2018-03-14 INSERM - Institut National de la Santé et de la Recherche Médicale Exon skipping therapy for dystrophic epidermolysis bullosa
US9463244B2 (en) 2013-03-15 2016-10-11 Massachusetts Institute Of Technology Compositions and methods for nucleic acid delivery
KR20180012255A (ko) 2015-05-21 2018-02-05 프로큐알 테라퓨틱스 Ⅱ 비.브이. 이영양성 수포성 표피박리증 치료를 위한 안티센스 올리고뉴클레오타이드
MA50829A (fr) * 2015-06-01 2018-04-11 Sarepta Therapeutics Inc Exclusion d'exon induite pat technologie antisens dans le collagène de type vii
HRP20202066T1 (hr) 2015-06-15 2021-04-30 Tirmed Pharma Ab Jednolančani oligonukleotidi za uporabu u medicinskom liječenju poremećaja kože

Also Published As

Publication number Publication date
CA2986642A1 (en) 2016-11-24
EA036393B1 (ru) 2020-11-05
CN107667174B (zh) 2021-10-26
HK1245826A1 (zh) 2018-08-31
IL255180B2 (en) 2023-11-01
AU2016265457A1 (en) 2017-12-07
IL255180B1 (en) 2023-07-01
IL255180A0 (en) 2017-12-31
US20180245078A1 (en) 2018-08-30
EP3298144A1 (en) 2018-03-28
MX2017014855A (es) 2018-04-20
NZ737592A (en) 2021-08-27
KR20180012255A (ko) 2018-02-05
JP2018515122A (ja) 2018-06-14
JP2021112197A (ja) 2021-08-05
CN107667174A (zh) 2018-02-06
US11104900B2 (en) 2021-08-31
JP6871175B2 (ja) 2021-05-12
WO2016185041A1 (en) 2016-11-24
EA201792406A1 (ru) 2018-04-30
JP7188804B2 (ja) 2022-12-13
US20200024601A1 (en) 2020-01-23
AU2016265457B2 (en) 2022-06-09
US10370660B2 (en) 2019-08-06

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