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DK3039146T3 - Produkter og fremgangsmåder til behandling af amyotrofisk lateral sklerose - Google Patents

Produkter og fremgangsmåder til behandling af amyotrofisk lateral sklerose Download PDF

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Publication number
DK3039146T3
DK3039146T3 DK14761771.6T DK14761771T DK3039146T3 DK 3039146 T3 DK3039146 T3 DK 3039146T3 DK 14761771 T DK14761771 T DK 14761771T DK 3039146 T3 DK3039146 T3 DK 3039146T3
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Prior art keywords
amyotrofic
products
treatment
methods
lateral sclerosis
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Application number
DK14761771.6T
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English (en)
Inventor
Brian K Kaspar
Kevin Foust
Don W Cleveland
Original Assignee
Res Institute At Nationwide Children's Hospital
Ludwig Inst For Cancer Res Ltd
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Publication of DK3039146T3 publication Critical patent/DK3039146T3/da

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    • 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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P25/28—Drugs 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
DK14761771.6T 2013-08-27 2014-08-26 Produkter og fremgangsmåder til behandling af amyotrofisk lateral sklerose DK3039146T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361870585P 2013-08-27 2013-08-27
PCT/US2014/052753 WO2015031392A1 (en) 2013-08-27 2014-08-26 Products and methods for treatment of amyotrophic lateral sclerosis

Publications (1)

Publication Number Publication Date
DK3039146T3 true DK3039146T3 (da) 2020-08-10

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Family Applications (2)

Application Number Title Priority Date Filing Date
DK20165345.8T DK3702466T5 (da) 2013-08-27 2014-08-26 Produkter og fremgangsmåder til behandling af amyotrofisk lateral sclerose
DK14761771.6T DK3039146T3 (da) 2013-08-27 2014-08-26 Produkter og fremgangsmåder til behandling af amyotrofisk lateral sklerose

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DK20165345.8T DK3702466T5 (da) 2013-08-27 2014-08-26 Produkter og fremgangsmåder til behandling af amyotrofisk lateral sclerose

Country Status (15)

Country Link
US (5) US20160272976A1 (da)
EP (3) EP3702466B1 (da)
CA (1) CA2922500A1 (da)
CY (1) CY1123272T1 (da)
DK (2) DK3702466T5 (da)
ES (2) ES2799437T3 (da)
FI (1) FI3702466T3 (da)
HR (2) HRP20230015T1 (da)
HU (2) HUE061629T2 (da)
LT (2) LT3039146T (da)
PL (1) PL3702466T3 (da)
PT (2) PT3702466T (da)
RS (2) RS60434B1 (da)
SI (2) SI3039146T1 (da)
WO (1) WO2015031392A1 (da)

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PL3702466T3 (pl) 2023-04-24
LT3702466T (lt) 2023-02-27
LT3039146T (lt) 2020-07-27
EP3702466A1 (en) 2020-09-02
CA2922500A1 (en) 2015-03-05
SI3039146T1 (sl) 2020-10-30
EP4219727A3 (en) 2023-09-06
HUE050546T2 (hu) 2020-12-28
WO2015031392A1 (en) 2015-03-05
CY1123272T1 (el) 2021-12-31
US20250154511A1 (en) 2025-05-15
PT3039146T (pt) 2020-08-11
HUE061629T2 (hu) 2023-07-28
PT3702466T (pt) 2023-01-24
EP4219727A2 (en) 2023-08-02
WO2015031392A9 (en) 2015-06-18
ES2799437T3 (es) 2020-12-17
HRP20200862T1 (hr) 2020-08-21
EP3039146A1 (en) 2016-07-06
US20160272976A1 (en) 2016-09-22
DK3702466T3 (da) 2023-03-20
EP3039146B1 (en) 2020-05-27
US20240344071A1 (en) 2024-10-17
DK3702466T5 (da) 2024-08-26
FI3702466T3 (fi) 2023-03-23
US20210108209A1 (en) 2021-04-15
US20190144868A1 (en) 2019-05-16
RS60434B1 (sr) 2020-07-31
HRP20230015T1 (hr) 2023-02-17
US10793861B2 (en) 2020-10-06
ES2936220T3 (es) 2023-03-15
RS63942B1 (sr) 2023-02-28
SI3702466T1 (sl) 2023-04-28

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