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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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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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
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    • 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
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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
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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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    • C12N2310/32—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
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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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  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (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

ID=54241435

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) EP4137573A3 (enExample)
JP (5) JP6622214B2 (enExample)
KR (6) KR20230085222A (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) ES2818236T3 (enExample)
FI (1) FIC20240035I1 (enExample)
FR (1) FR24C1039I2 (enExample)
HR (2) HRP20201078T1 (enExample)
HU (3) HUE059109T2 (enExample)
IL (2) IL247714B (enExample)
LT (3) LT3126499T (enExample)
MX (2) MX373908B (enExample)
MY (2) MY192634A (enExample)
NL (1) NL301293I2 (enExample)
NZ (1) NZ724100A (enExample)
PL (2) PL3757214T3 (enExample)
PT (2) PT3757214T (enExample)
RS (2) RS63487B1 (enExample)
RU (1) RU2704619C2 (enExample)
SG (3) SG10201906520RA (enExample)
SI (2) SI3757214T1 (enExample)
SM (2) SMT202200479T1 (enExample)
WO (1) WO2015153800A2 (enExample)

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WO2018218219A1 (en) * 2017-05-26 2018-11-29 University Of Miami Determining onset of amyotrophic lateral sclerosis
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JP2025522433A (ja) 2022-06-15 2025-07-15 アローヘッド ファーマシューティカルズ インコーポレイテッド スーパーオキシドジスムターゼ１（ＳＯＤ１）の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用の方法
KR20240038469A (ko) 2022-09-16 2024-03-25 김정훈 사용자 콘텐츠 기반 홍보 서비스 제공 장치 및 방법
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