CA2740328A1 - Multiple exon skipping compositions for dmd - Google Patents

Multiple exon skipping compositions for dmd Download PDF

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Publication number
CA2740328A1
CA2740328A1 CA2740328A CA2740328A CA2740328A1 CA 2740328 A1 CA2740328 A1 CA 2740328A1 CA 2740328 A CA2740328 A CA 2740328A CA 2740328 A CA2740328 A CA 2740328A CA 2740328 A1 CA2740328 A1 CA 2740328A1
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Prior art keywords
dmd
seq
exon
nos
group
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CA2740328A
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French (fr)
Inventor
Peter Sazani
Ryszard Kole
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Sarepta Therapeutics Inc
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AVI Biopharma Inc
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=41510869&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CA2740328(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by AVI Biopharma Inc filed Critical AVI Biopharma Inc
Priority to CA3066050A priority Critical patent/CA3066050A1/en
Publication of CA2740328A1 publication Critical patent/CA2740328A1/en
Pending legal-status Critical Current

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    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
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    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA2740328A 2008-10-24 2009-10-23 Multiple exon skipping compositions for dmd Pending CA2740328A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3066050A CA3066050A1 (en) 2008-10-24 2009-10-23 Multiple exon skipping compositions for dmd

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US10841608P 2008-10-24 2008-10-24
US61/108,416 2008-10-24
PCT/US2009/061960 WO2010048586A1 (en) 2008-10-24 2009-10-23 Multiple exon skipping compositions for dmd

Related Child Applications (1)

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CA3066050A Division CA3066050A1 (en) 2008-10-24 2009-10-23 Multiple exon skipping compositions for dmd

Publications (1)

Publication Number Publication Date
CA2740328A1 true CA2740328A1 (en) 2010-04-29

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ID=41510869

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CA2740328A Pending CA2740328A1 (en) 2008-10-24 2009-10-23 Multiple exon skipping compositions for dmd
CA3066050A Pending CA3066050A1 (en) 2008-10-24 2009-10-23 Multiple exon skipping compositions for dmd

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3066050A Pending CA3066050A1 (en) 2008-10-24 2009-10-23 Multiple exon skipping compositions for dmd

Country Status (22)

Country Link
US (15) US8871918B2 (enExample)
EP (7) EP2762567B1 (enExample)
JP (10) JP5864257B2 (enExample)
KR (6) KR20180118828A (enExample)
CN (4) CN102203253B (enExample)
AU (1) AU2009308167B2 (enExample)
BR (3) BR122020021368B1 (enExample)
CA (2) CA2740328A1 (enExample)
CY (1) CY1122369T1 (enExample)
DK (1) DK3133160T3 (enExample)
ES (2) ES2714787T3 (enExample)
HR (1) HRP20190387T1 (enExample)
HU (1) HUE042979T2 (enExample)
IL (6) IL304042A (enExample)
LT (1) LT3133160T (enExample)
PL (1) PL3133160T3 (enExample)
PT (1) PT3133160T (enExample)
SI (1) SI3133160T1 (enExample)
SM (1) SMT201900159T1 (enExample)
TR (1) TR201902952T4 (enExample)
TW (7) TWI488965B (enExample)
WO (1) WO2010048586A1 (enExample)

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