AU2015240761B2 - Compositions for modulating SOD-1 expression - Google Patents

Compositions for modulating SOD-1 expression Download PDF

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Publication number
AU2015240761B2
AU2015240761B2 AU2015240761A AU2015240761A AU2015240761B2 AU 2015240761 B2 AU2015240761 B2 AU 2015240761B2 AU 2015240761 A AU2015240761 A AU 2015240761A AU 2015240761 A AU2015240761 A AU 2015240761A AU 2015240761 B2 AU2015240761 B2 AU 2015240761B2
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Prior art keywords
antisense compound
sod
isis
pharmaceutically acceptable
certain embodiments
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AU2015240761A1 (en
Inventor
Tracy COLE
Thomas P. Condon
Susan M. Freier
Holly Kordasiewicz
Trisha Lockhart
Priyam SINGH
Eric E. Swayze
Timothy Vickers
Edward Wancewicz
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Biogen MA Inc
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Biogen MA Inc
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Publication of AU2015240761B2 publication Critical patent/AU2015240761B2/en
Priority to AU2019268063A priority Critical patent/AU2019268063A1/en
Priority to AU2022200002A priority patent/AU2022200002A1/en
Priority to AU2024202423A priority patent/AU2024202423A1/en
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    • C—CHEMISTRY; METALLURGY
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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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • 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)
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2015240761A 2014-04-01 2015-04-01 Compositions for modulating SOD-1 expression Active AU2015240761B2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU2019268063A AU2019268063A1 (en) 2014-04-01 2019-11-19 Compositions for modulating sod-1 expression
AU2022200002A AU2022200002A1 (en) 2014-04-01 2022-01-03 Compositions for modulating sod-1 expression
AU2024202423A AU2024202423A1 (en) 2014-04-01 2024-04-12 Compositions for modulating sod-1 expression

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461973803P 2014-04-01 2014-04-01
US61/973,803 2014-04-01
PCT/US2015/023934 WO2015153800A2 (en) 2014-04-01 2015-04-01 Compositions for modulating sod-1 expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2019268063A Division AU2019268063A1 (en) 2014-04-01 2019-11-19 Compositions for modulating sod-1 expression

Publications (2)

Publication Number Publication Date
AU2015240761A1 AU2015240761A1 (en) 2016-09-22
AU2015240761B2 true AU2015240761B2 (en) 2019-09-12

Family

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Family Applications (4)

Application Number Title Priority Date Filing Date
AU2015240761A Active AU2015240761B2 (en) 2014-04-01 2015-04-01 Compositions for modulating SOD-1 expression
AU2019268063A Abandoned AU2019268063A1 (en) 2014-04-01 2019-11-19 Compositions for modulating sod-1 expression
AU2022200002A Abandoned AU2022200002A1 (en) 2014-04-01 2022-01-03 Compositions for modulating sod-1 expression
AU2024202423A Pending AU2024202423A1 (en) 2014-04-01 2024-04-12 Compositions for modulating sod-1 expression

Family Applications After (3)

Application Number Title Priority Date Filing Date
AU2019268063A Abandoned AU2019268063A1 (en) 2014-04-01 2019-11-19 Compositions for modulating sod-1 expression
AU2022200002A Abandoned AU2022200002A1 (en) 2014-04-01 2022-01-03 Compositions for modulating sod-1 expression
AU2024202423A Pending AU2024202423A1 (en) 2014-04-01 2024-04-12 Compositions for modulating sod-1 expression

Country Status (30)

Country Link
US (3) US10385341B2 (enExample)
EP (3) EP3126499B1 (enExample)
JP (5) JP6622214B2 (enExample)
KR (6) KR20250113519A (enExample)
CN (2) CN106459972B (enExample)
AU (4) AU2015240761B2 (enExample)
BR (1) BR112016021848B1 (enExample)
CA (1) CA2942394A1 (enExample)
CL (3) CL2016002509A1 (enExample)
CY (1) CY1123288T1 (enExample)
DK (2) DK3757214T3 (enExample)
ES (2) ES2926869T3 (enExample)
FI (1) FIC20240035I1 (enExample)
FR (1) FR24C1039I2 (enExample)
HR (2) HRP20220798T1 (enExample)
HU (3) HUE059109T2 (enExample)
IL (2) IL247714B (enExample)
LT (3) LT3757214T (enExample)
MX (2) MX373908B (enExample)
MY (2) MY192634A (enExample)
NL (1) NL301293I2 (enExample)
NZ (1) NZ724100A (enExample)
PL (2) PL3126499T3 (enExample)
PT (2) PT3757214T (enExample)
RS (2) RS60707B1 (enExample)
RU (1) RU2704619C2 (enExample)
SG (3) SG10201906520RA (enExample)
SI (2) SI3126499T1 (enExample)
SM (2) SMT202200479T1 (enExample)
WO (1) WO2015153800A2 (enExample)

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JP7455831B2 (ja) * 2018-11-21 2024-03-26 アイオーニス ファーマシューティカルズ， インコーポレーテッド プリオン発現を低減するための化合物及び方法
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EP4430191A2 (en) * 2021-11-08 2024-09-18 University Of Massachusetts Oligonucleotides for sod1 modulation
CN114369130B (zh) * 2021-12-28 2023-10-03 杭州天龙药业有限公司 修饰的硫代寡核苷酸及其应用
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CN120202298A (zh) * 2023-08-29 2025-06-24 石药集团中奇制药技术（石家庄）有限公司 一种抑制sod1基因表达的dsRNA分子及其应用
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