DK3757214T3 - Sammensætninger til modulering af sod-1-ekspression - Google Patents

Sammensætninger til modulering af sod-1-ekspression Download PDF

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Publication number
DK3757214T3
DK3757214T3 DK20176637.5T DK20176637T DK3757214T3 DK 3757214 T3 DK3757214 T3 DK 3757214T3 DK 20176637 T DK20176637 T DK 20176637T DK 3757214 T3 DK3757214 T3 DK 3757214T3
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Denmark
Prior art keywords
moduling
sod
expression
compositions
moduling sod
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DK20176637.5T
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English (en)
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Eric E Swayze
Tracy Cole
Holly Kordasiewicz
Susan M Freier
Thomas P Condon
Edward Wancewicz
Trisha Lockhart
Timothy Vickers
Priyam Singh
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Biogen Ma 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
    • C12N15/11DNA 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
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    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N2310/31Chemical structure of the backbone
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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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
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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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
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    • C12YENZYMES
    • 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)
DK20176637.5T 2014-04-01 2015-04-01 Sammensætninger til modulering af sod-1-ekspression DK3757214T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461973803P 2014-04-01 2014-04-01
EP15773965.7A EP3126499B1 (en) 2014-04-01 2015-04-01 Compositions for modulating sod-1 expression

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DK3757214T3 true DK3757214T3 (da) 2022-07-04

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DK20176637.5T DK3757214T3 (da) 2014-04-01 2015-04-01 Sammensætninger til modulering af sod-1-ekspression
DK15773965.7T DK3126499T3 (da) 2014-04-01 2015-04-01 Sammensætninger til modulering af sod-1-ekspression

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US (3) US10385341B2 (da)
EP (3) EP3126499B1 (da)
JP (4) JP6622214B2 (da)
KR (4) KR20210097225A (da)
CN (2) CN111440798A (da)
AU (3) AU2015240761B2 (da)
BR (1) BR112016021848B1 (da)
CA (1) CA2942394A1 (da)
CL (3) CL2016002509A1 (da)
CY (1) CY1123288T1 (da)
DK (2) DK3757214T3 (da)
ES (2) ES2818236T3 (da)
HR (2) HRP20220798T1 (da)
HU (2) HUE050704T2 (da)
IL (2) IL247714B (da)
LT (2) LT3126499T (da)
MX (2) MX2016012922A (da)
MY (2) MY175665A (da)
NZ (1) NZ724100A (da)
PL (2) PL3126499T3 (da)
PT (2) PT3126499T (da)
RS (2) RS60707B1 (da)
RU (1) RU2704619C2 (da)
SG (3) SG10201906520RA (da)
SI (2) SI3126499T1 (da)
WO (1) WO2015153800A2 (da)

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