HK1245826A1 - 用於治療營養不良性大疱性表皮鬆解症的反義寡核苷酸 - Google Patents

用於治療營養不良性大疱性表皮鬆解症的反義寡核苷酸

Info

Publication number
HK1245826A1
HK1245826A1 HK18105119.5A HK18105119A HK1245826A1 HK 1245826 A1 HK1245826 A1 HK 1245826A1 HK 18105119 A HK18105119 A HK 18105119A HK 1245826 A1 HK1245826 A1 HK 1245826A1
Authority
HK
Hong Kong
Prior art keywords
antisense oligonucleotides
epidermolysis bullosa
dystrophic epidermolysis
treat
treat dystrophic
Prior art date
Application number
HK18105119.5A
Other languages
English (en)
Inventor
Elisabeth Marlene Haisma
Marko Potman
Gerardus Johannes Platenburg
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 HK1245826A1 publication Critical patent/HK1245826A1/zh

Links

Classifications

    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • 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
    • C12N2310/32Chemical 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
HK18105119.5A 2015-05-21 2018-04-19 用於治療營養不良性大疱性表皮鬆解症的反義寡核苷酸 HK1245826A1 (zh)

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 (1)

Publication Number Publication Date
HK1245826A1 true HK1245826A1 (zh) 2018-08-31

Family

ID=56026883

Family Applications (1)

Application Number Title Priority Date Filing Date
HK18105119.5A HK1245826A1 (zh) 2015-05-21 2018-04-19 用於治療營養不良性大疱性表皮鬆解症的反義寡核苷酸

Country Status (12)

Country Link
US (2) US10370660B2 (zh)
EP (1) EP3298144A1 (zh)
JP (2) JP6871175B2 (zh)
KR (1) KR20180012255A (zh)
CN (1) CN107667174B (zh)
AU (1) AU2016265457B2 (zh)
CA (1) CA2986642A1 (zh)
EA (1) EA036393B1 (zh)
HK (1) HK1245826A1 (zh)
IL (1) IL255180B2 (zh)
MX (1) MX2017014855A (zh)
WO (1) WO2016185041A1 (zh)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6871175B2 (ja) 2015-05-21 2021-05-12 ウィングス セラピューティクス,インコーポレイテッド ジストロフィー型表皮水疱症を処置するためのアンチセンスオリゴヌクレオチド
WO2016196670A1 (en) 2015-06-01 2016-12-08 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type vii collagen
GB201902735D0 (en) 2019-02-28 2019-04-17 Proqr Therapeutics Ii Bv Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa

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
AU2008317566B2 (en) * 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
JP5944311B2 (ja) * 2009-06-16 2016-07-05 クルナ・インコーポレーテッド コラーゲン遺伝子に対する天然アンチセンス転写物の抑制によるコラーゲン遺伝子関連疾患の治療
US9340783B2 (en) * 2011-10-11 2016-05-17 Inserm (Institut National De La Sante Et De La Recherche Medicale 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
JP6871175B2 (ja) 2015-05-21 2021-05-12 ウィングス セラピューティクス,インコーポレイテッド ジストロフィー型表皮水疱症を処置するためのアンチセンスオリゴヌクレオチド
WO2016196670A1 (en) * 2015-06-01 2016-12-08 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type vii collagen
CA2989371C (en) 2015-06-15 2024-02-06 Anna-Lena SPETZ HOLMGREN Single-stranded oligonucleotides for use in the medical treatment of skin disorders

Also Published As

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

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