HK1160169A1 - Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna (dmd) mrna 45 - Google Patents

Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna (dmd) mrna 45

Info

Publication number
HK1160169A1
HK1160169A1 HK12100495.6A HK12100495A HK1160169A1 HK 1160169 A1 HK1160169 A1 HK 1160169A1 HK 12100495 A HK12100495 A HK 12100495A HK 1160169 A1 HK1160169 A1 HK 1160169A1
Authority
HK
Hong Kong
Prior art keywords
mrna
dmd
exon
methods
muscular dystrophy
Prior art date
Application number
HK12100495.6A
Other languages
English (en)
Chinese (zh)
Inventor
Josephus Johannes De Kimpe
Gerardus Johannes Platenburg
Judith Christina Theodora Van Deutekom
Annemieke Aartsma-Rus
Garrit-Jan Boudewijn Van Ommen
Original Assignee
Prosensa Technologies Bv
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=40873329&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HK1160169(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from PCT/NL2008/050673 external-priority patent/WO2009054725A2/en
Application filed by Prosensa Technologies Bv filed Critical Prosensa Technologies Bv
Publication of HK1160169A1 publication Critical patent/HK1160169A1/xx

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
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
HK12100495.6A 2008-10-27 2012-01-16 Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna (dmd) mrna 45 HK1160169A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PCT/NL2008/050673 WO2009054725A2 (en) 2007-10-26 2008-10-27 Means and methods for counteracting muscle disorders
PCT/NL2009/050006 WO2010050801A1 (en) 2008-10-27 2009-01-13 Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna

Publications (1)

Publication Number Publication Date
HK1160169A1 true HK1160169A1 (en) 2012-08-10

Family

ID=40873329

Family Applications (1)

Application Number Title Priority Date Filing Date
HK12100495.6A HK1160169A1 (en) 2008-10-27 2012-01-16 Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna (dmd) mrna 45

Country Status (15)

Country Link
EP (4) EP2344637B2 (xx)
JP (2) JP5786109B2 (xx)
AU (1) AU2009310557B2 (xx)
CA (3) CA2741629C (xx)
CY (1) CY1116305T1 (xx)
DK (2) DK2344637T4 (xx)
ES (2) ES2562658T3 (xx)
HK (1) HK1160169A1 (xx)
HR (1) HRP20160078T1 (xx)
IL (2) IL212509A (xx)
NZ (1) NZ592498A (xx)
PL (1) PL2607484T3 (xx)
PT (2) PT2344637E (xx)
SI (1) SI2607484T1 (xx)
WO (2) WO2010050801A1 (xx)

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Also Published As

Publication number Publication date
AU2009310557A8 (en) 2011-09-29
ES2562658T3 (es) 2016-03-07
JP2012506697A (ja) 2012-03-22
AU2009310557B2 (en) 2014-09-11
EP3400948B1 (en) 2022-11-02
CY1116305T1 (el) 2017-02-08
EP2344637A1 (en) 2011-07-20
SI2607484T1 (sl) 2016-11-30
EP2607484A1 (en) 2013-06-26
WO2010050802A3 (en) 2010-08-12
DK2344637T4 (en) 2018-04-23
IL212508A0 (en) 2011-06-30
IL212509A0 (en) 2011-06-30
CA2741629C (en) 2022-07-05
EP2349287B1 (en) 2018-08-15
EP2344637B2 (en) 2018-02-28
WO2010050801A1 (en) 2010-05-06
AU2009310557A1 (en) 2010-05-06
JP5905260B2 (ja) 2016-04-20
ES2532634T3 (es) 2015-03-30
AU2009310558A8 (en) 2015-01-22
AU2009310558B2 (en) 2014-09-04
DK2344637T3 (en) 2015-02-02
DK2607484T3 (en) 2016-03-07
HRP20160078T1 (hr) 2016-04-08
CA2741793C (en) 2018-10-30
EP2607484B1 (en) 2016-01-06
NZ592498A (en) 2012-05-25
ES2532634T5 (es) 2018-04-30
IL212509A (en) 2016-04-21
EP3400948A1 (en) 2018-11-14
AU2009310558A1 (en) 2010-05-06
IL212508A (en) 2013-11-28
EP2344637B1 (en) 2014-12-24
CA3017539A1 (en) 2010-05-06
CA2741793A1 (en) 2010-05-06
WO2010050802A2 (en) 2010-05-06
PT2607484E (pt) 2016-03-09
EP2349287A2 (en) 2011-08-03
PL2607484T3 (pl) 2016-06-30
JP5786109B2 (ja) 2015-09-30
JP2012506698A (ja) 2012-03-22
CA2741629A1 (en) 2010-05-06
PT2344637E (pt) 2015-03-23

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