CA2776651C - Tricyclo-phosphorothioate dna - Google Patents

Tricyclo-phosphorothioate dna Download PDF

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Publication number
CA2776651C
CA2776651C CA2776651A CA2776651A CA2776651C CA 2776651 C CA2776651 C CA 2776651C CA 2776651 A CA2776651 A CA 2776651A CA 2776651 A CA2776651 A CA 2776651A CA 2776651 C CA2776651 C CA 2776651C
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CA
Canada
Prior art keywords
nucleic acid
acid molecule
dna
tricyclo
treatment
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA2776651A
Other languages
English (en)
French (fr)
Other versions
CA2776651A1 (en
Inventor
Christian Leumann
Luis Garcia
Thomas Voit
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Universitaet Bern
Centre National de la Recherche Scientifique CNRS
Universite Pierre et Marie Curie
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Original Assignee
Universitaet Bern
Centre National de la Recherche Scientifique CNRS
Universite Pierre et Marie Curie
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
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
Application filed by Universitaet Bern, Centre National de la Recherche Scientifique CNRS, Universite Pierre et Marie Curie, Institut National de la Sante et de la Recherche Medicale INSERM, Association Institut de Myologie filed Critical Universitaet Bern
Priority to JP2014535113A priority Critical patent/JP6181653B2/ja
Priority to PCT/EP2012/070349 priority patent/WO2013053928A1/en
Priority to AU2012322903A priority patent/AU2012322903B2/en
Priority to CA2851970A priority patent/CA2851970A1/en
Priority to US14/351,733 priority patent/US9738891B2/en
Priority to BR112014009066A priority patent/BR112014009066B8/pt
Priority to CN201280057688.6A priority patent/CN104245935B/zh
Priority to EP12770503.6A priority patent/EP2766479B1/en
Priority to ES12770503.6T priority patent/ES2651216T3/es
Publication of CA2776651A1 publication Critical patent/CA2776651A1/en
Priority to IL231983A priority patent/IL231983B/en
Priority to IN3463DEN2014 priority patent/IN2014DN03463A/en
Application granted granted Critical
Publication of CA2776651C publication Critical patent/CA2776651C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • C12N15/11—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Cardiology (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
CA2776651A 2011-10-13 2012-04-27 Tricyclo-phosphorothioate dna Active CA2776651C (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
CN201280057688.6A CN104245935B (zh) 2011-10-13 2012-10-12 三环硫代磷酸酯dna
AU2012322903A AU2012322903B2 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna
CA2851970A CA2851970A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna
US14/351,733 US9738891B2 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate DNA
BR112014009066A BR112014009066B8 (pt) 2011-10-13 2012-10-12 molécula de ácido nucleico, método para a sintetização de uma molécula de dna de triciclo-fosforotioato e composição farmacêutica
EP12770503.6A EP2766479B1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna
JP2014535113A JP6181653B2 (ja) 2011-10-13 2012-10-12 トリシクロ−ホスホロチオエートｄｎａ
ES12770503.6T ES2651216T3 (es) 2011-10-13 2012-10-12 Triciclo-ADN fosforotioato
PCT/EP2012/070349 WO2013053928A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna
IL231983A IL231983B (en) 2011-10-13 2014-04-07 Dna containing tricyclo-phosphorothioate
IN3463DEN2014 IN2014DN03463A (enExample) 2011-10-13 2014-04-30

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201161546942P 2011-10-13 2011-10-13
EP11185129.1A EP2581448B1 (en) 2011-10-13 2011-10-13 Tricyclo-phosphorothioate DNA
EP11185129.1 2011-10-13
US61/546.942 2011-10-13

Publications (2)

Publication Number Publication Date
CA2776651A1 CA2776651A1 (en) 2013-04-13
CA2776651C true CA2776651C (en) 2021-06-08

Family

ID=44799824

Family Applications (2)

Application Number Title Priority Date Filing Date
CA2776651A Active CA2776651C (en) 2011-10-13 2012-04-27 Tricyclo-phosphorothioate dna
CA2851970A Abandoned CA2851970A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2851970A Abandoned CA2851970A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna

Country Status (14)

Country Link
US (1) US9738891B2 (enExample)
EP (2) EP2581448B1 (enExample)
JP (1) JP6181653B2 (enExample)
CN (1) CN104245935B (enExample)
AU (1) AU2012322903B2 (enExample)
BR (1) BR112014009066B8 (enExample)
CA (2) CA2776651C (enExample)
DK (1) DK2581448T3 (enExample)
ES (2) ES2535654T3 (enExample)
IL (1) IL231983B (enExample)
IN (1) IN2014DN03463A (enExample)
PL (1) PL2581448T3 (enExample)
PT (1) PT2581448E (enExample)
WO (1) WO2013053928A1 (enExample)

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Also Published As

Publication number Publication date
BR112014009066B8 (pt) 2021-02-23
CN104245935A (zh) 2014-12-24
PT2581448E (pt) 2015-05-21
ES2651216T3 (es) 2018-01-25
EP2581448B1 (en) 2015-01-28
EP2581448A1 (en) 2013-04-17
BR112014009066B1 (pt) 2020-08-25
BR112014009066A2 (pt) 2017-04-18
CA2851970A1 (en) 2013-04-18
JP6181653B2 (ja) 2017-08-16
ES2535654T3 (es) 2015-05-13
IN2014DN03463A (enExample) 2015-06-05
WO2013053928A1 (en) 2013-04-18
JP2015501144A (ja) 2015-01-15
PL2581448T3 (pl) 2015-08-31
DK2581448T3 (en) 2015-04-27
CA2776651A1 (en) 2013-04-13
IL231983A0 (en) 2014-05-28
AU2012322903A1 (en) 2014-05-29
IL231983B (en) 2018-04-30
HK1186205A1 (en) 2014-03-07
CN104245935B (zh) 2018-05-08
EP2766479A1 (en) 2014-08-20
US9738891B2 (en) 2017-08-22
BR112014009066A8 (pt) 2018-01-09
US20140296323A1 (en) 2014-10-02
AU2012322903B2 (en) 2017-09-14
EP2766479B1 (en) 2017-09-13

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