PL3976791T3 - Związki i sposoby do zmniejszania ekspresji fus - Google Patents

Związki i sposoby do zmniejszania ekspresji fus

Info

Publication number
PL3976791T3
PL3976791T3 PL20815459.1T PL20815459T PL3976791T3 PL 3976791 T3 PL3976791 T3 PL 3976791T3 PL 20815459 T PL20815459 T PL 20815459T PL 3976791 T3 PL3976791 T3 PL 3976791T3
Authority
PL
Poland
Prior art keywords
compounds
methods
reducing
fus expression
expression
Prior art date
Application number
PL20815459.1T
Other languages
English (en)
Inventor
Huynh-Hoa Bui
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 PL3976791T3 publication Critical patent/PL3976791T3/pl

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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 ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
PL20815459.1T 2019-05-28 2020-05-28 Związki i sposoby do zmniejszania ekspresji fus PL3976791T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962853616P 2019-05-28 2019-05-28
PCT/US2020/034907 WO2020243292A1 (en) 2019-05-28 2020-05-28 Compounds and methods for reducing fus expression

Publications (1)

Publication Number Publication Date
PL3976791T3 true PL3976791T3 (pl) 2025-08-04

Family

ID=73553907

Family Applications (1)

Application Number Title Priority Date Filing Date
PL20815459.1T PL3976791T3 (pl) 2019-05-28 2020-05-28 Związki i sposoby do zmniejszania ekspresji fus

Country Status (13)

Country Link
US (1) US20220243203A1 (pl)
EP (2) EP4556565A3 (pl)
DK (1) DK3976791T3 (pl)
ES (1) ES3033104T3 (pl)
FI (1) FI3976791T3 (pl)
HR (1) HRP20250468T1 (pl)
HU (1) HUE071962T2 (pl)
LT (1) LT3976791T (pl)
PL (1) PL3976791T3 (pl)
PT (1) PT3976791T (pl)
SI (1) SI3976791T1 (pl)
TW (1) TW202111123A (pl)
WO (1) WO2020243292A1 (pl)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025507278A (ja) * 2022-02-02 2025-03-18 ペロン インスティテュート フォー ニューロロジカル アンド トランスレーショナル サイエンス リミテッド Ｆｕｓ関連疾患を処置するための組成物および方法
EP4473111A4 (en) * 2022-02-02 2026-08-12 Perron Inst For Neurological And Translational Science Limited COMPOSITIONS AND METHODS OF TREATMENT OF DISEASES ASSOCIATED WITH VARIANTS OF PATHOGENIC FUS
US20260132401A1 (en) * 2022-05-06 2026-05-14 Ionis Pharmaceuticals, Inc. RNAi AGENTS FOR MODULATING SNCA

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Also Published As

Publication number Publication date
EP3976791A1 (en) 2022-04-06
ES3033104T3 (en) 2025-07-30
LT3976791T (lt) 2025-07-25
EP4556565A3 (en) 2026-08-05
TW202111123A (zh) 2021-03-16
DK3976791T3 (da) 2025-05-19
HRP20250468T1 (hr) 2025-07-18
PT3976791T (pt) 2025-06-26
US20220243203A1 (en) 2022-08-04
WO2020243292A1 (en) 2020-12-03
SI3976791T1 (sl) 2025-06-30
EP3976791A4 (en) 2023-10-11
FI3976791T3 (fi) 2025-06-10
EP3976791B1 (en) 2025-04-09
EP4556565A2 (en) 2025-05-21
HUE071962T2 (hu) 2025-10-28

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