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PE20211242A1 - Moduladores de la expresion dnm2 - Google Patents

Moduladores de la expresion dnm2

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Publication number
PE20211242A1
PE20211242A1 PE2020000957A PE2020000957A PE20211242A1 PE 20211242 A1 PE20211242 A1 PE 20211242A1 PE 2020000957 A PE2020000957 A PE 2020000957A PE 2020000957 A PE2020000957 A PE 2020000957A PE 20211242 A1 PE20211242 A1 PE 20211242A1
Authority
PE
Peru
Prior art keywords
intron
dnm2
nucleobase sequence
seq
contiguous nucleobases
Prior art date
Application number
PE2020000957A
Other languages
English (en)
Inventor
Susan M Freier
Huynh-Hoa Bui
Susan F Murray
Brett P Monia
Shuling Guo
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of PE20211242A1 publication Critical patent/PE20211242A1/es

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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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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
    • C12N2310/323—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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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Microbiology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invencion se refiere a un compuesto que comprende un oligonucleotido modificado de 14 a 30 nucleosidos unidos en longitud que comprende : a) una secuencia de nucleobase que comprende 14 nucleobases contiguas de la SEQ ID NO:2879; b) una secuencia de nucleobase que comprende 14 nucleobases contiguas 100% complementarias a una porcion de igual longitud de las nucleobases 87,359-90,915; 90,968-97,263; 83,573-87,287; 3,404-44,737;97,378-104,979; 81,061-81,199; o 116,048-116,903 de la SEQ ID NO: 1, c) una secuencia de nucleobase complementaria a un intron 12, intron 13, intron 11, intron 1, intron 14, exon 10, o el 3'-UTR de un ARNm humano de DNM2 o ARNm humano de DNM2 , d) una secuencia de nucleobase que comprende al menos 12, al menos 13, al menos 14, o al menos nucleobases contiguas de acuerdo con cualquiera de las secuencias de nucleobase de las SEQ ID NOs: 7-3134 , tambien comprende una composicion farmaceutica y debido a que inhiben la expresion de DNM2 son utiles para el tratamiento de miopatia centronuclear, distrofia muscular de Duchenne, enfermedad de Charcot-Marie Tooth.
PE2020000957A 2018-01-15 2019-01-15 Moduladores de la expresion dnm2 PE20211242A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862617411P 2018-01-15 2018-01-15
PCT/US2019/013689 WO2019140452A1 (en) 2018-01-15 2019-01-15 Modulators of dnm2 expression

Publications (1)

Publication Number Publication Date
PE20211242A1 true PE20211242A1 (es) 2021-07-09

Family

ID=65324590

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2020000957A PE20211242A1 (es) 2018-01-15 2019-01-15 Moduladores de la expresion dnm2

Country Status (18)

Country Link
US (2) US20200392510A1 (es)
EP (1) EP3740575A1 (es)
JP (1) JP2021511072A (es)
KR (1) KR20200113214A (es)
CN (1) CN111902537A (es)
AU (1) AU2019206731A1 (es)
BR (1) BR112020014425A2 (es)
CA (1) CA3088522A1 (es)
CL (1) CL2020001851A1 (es)
CO (1) CO2020008769A2 (es)
IL (1) IL276007A (es)
MA (1) MA51645A (es)
MX (1) MX2020007369A (es)
PE (1) PE20211242A1 (es)
RU (1) RU2020127049A (es)
SG (1) SG11202006711UA (es)
TW (1) TW201934129A (es)
WO (1) WO2019140452A1 (es)

Families Citing this family (7)

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US12312585B2 (en) * 2020-02-14 2025-05-27 University Of Massachusetts Oligonucleotides targeting frataxin and related methods
US12275941B2 (en) 2021-04-15 2025-04-15 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of dynamin-1 variants
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
EP4311546A1 (en) 2022-07-26 2024-01-31 Dynacure Combination therapy for myopathies
CN121263524A (zh) * 2023-05-04 2026-01-02 豪夫迈·罗氏有限公司 能够上调葡糖脑苷脂酶表达的寡核苷酸
WO2025085732A1 (en) * 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Humanized dnm2 mouse model generation, characterization and methods of use
WO2025085810A2 (en) * 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies

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