CA2967367C - Compositions and methods of treating amyotrophic lateral sclerosis (als) - Google Patents

Compositions and methods of treating amyotrophic lateral sclerosis (als) Download PDF

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Publication number
CA2967367C
CA2967367C CA2967367A CA2967367A CA2967367C CA 2967367 C CA2967367 C CA 2967367C CA 2967367 A CA2967367 A CA 2967367A CA 2967367 A CA2967367 A CA 2967367A CA 2967367 C CA2967367 C CA 2967367C
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Prior art keywords
sod1
nucleotides
sirna
aav
expression
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CA2967367A
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French (fr)
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CA2967367A1 (en
Inventor
Dinah Wen-Yee Sah
Jinzhao Hou
Mathieu E. NONNENMACHER
Pengcheng ZHOU
Markus Hossbach
Jochen DECKERT
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Voyager Therapeutics Inc
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Voyager Therapeutics Inc
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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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • 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
    • C12N15/1137Non-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 against enzymes
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12Y115/00Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001Superoxide dismutase (1.15.1.1)
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Biotechnology (AREA)
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  • Medicinal Chemistry (AREA)
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  • Plant Pathology (AREA)
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  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA2967367A 2014-11-14 2015-11-13 Compositions and methods of treating amyotrophic lateral sclerosis (als) Active CA2967367C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA3193811A CA3193811A1 (en) 2014-11-14 2015-11-13 Compositions and methods of treating amyotrophic lateral sclerosis (als)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201462079588P 2014-11-14 2014-11-14
US62/079,588 2014-11-14
US201562211992P 2015-08-31 2015-08-31
US62/211,992 2015-08-31
US201562234466P 2015-09-29 2015-09-29
US62/234,466 2015-09-29
PCT/US2015/060562 WO2016077687A1 (en) 2014-11-14 2015-11-13 Compositions and methods of treating amyotrophic lateral sclerosis (als)

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA3193811A Division CA3193811A1 (en) 2014-11-14 2015-11-13 Compositions and methods of treating amyotrophic lateral sclerosis (als)

Publications (2)

Publication Number Publication Date
CA2967367A1 CA2967367A1 (en) 2016-05-19
CA2967367C true CA2967367C (en) 2023-05-16

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CA2967367A Active CA2967367C (en) 2014-11-14 2015-11-13 Compositions and methods of treating amyotrophic lateral sclerosis (als)
CA3193811A Pending CA3193811A1 (en) 2014-11-14 2015-11-13 Compositions and methods of treating amyotrophic lateral sclerosis (als)

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CA3193811A Pending CA3193811A1 (en) 2014-11-14 2015-11-13 Compositions and methods of treating amyotrophic lateral sclerosis (als)

Country Status (14)

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US (5) US10597660B2 (enExample)
EP (1) EP3218484A4 (enExample)
JP (4) JP2017535266A (enExample)
KR (2) KR102599909B1 (enExample)
CN (2) CN114717264A (enExample)
AU (2) AU2015346162B2 (enExample)
CA (2) CA2967367C (enExample)
HK (1) HK1244299A1 (enExample)
IL (3) IL292999A (enExample)
MX (2) MX2017006216A (enExample)
RU (2) RU2020108189A (enExample)
SG (1) SG11201703281RA (enExample)
WO (1) WO2016077687A1 (enExample)
ZA (1) ZA201702991B (enExample)

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