PL3947684T3 - Związki i sposoby modulacji ube3a-ats - Google Patents

Związki i sposoby modulacji ube3a-ats

Info

Publication number
PL3947684T3
PL3947684T3 PL20784141.2T PL20784141T PL3947684T3 PL 3947684 T3 PL3947684 T3 PL 3947684T3 PL 20784141 T PL20784141 T PL 20784141T PL 3947684 T3 PL3947684 T3 PL 3947684T3
Authority
PL
Poland
Prior art keywords
ube3a
ats
modulating
compounds
methods
Prior art date
Application number
PL20784141.2T
Other languages
English (en)
Inventor
Susan M. Freier
Huynh-Hoa Bui
Paymaan JAFAR-NEJAD
Frank Rigo
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 PL3947684T3 publication Critical patent/PL3947684T3/pl

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    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/113Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • 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
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL20784141.2T 2019-03-29 2020-03-27 Związki i sposoby modulacji ube3a-ats PL3947684T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962826521P 2019-03-29 2019-03-29
US201962877765P 2019-07-23 2019-07-23
PCT/US2020/025110 WO2020205463A1 (en) 2019-03-29 2020-03-27 Compounds and methods for modulating ube3a-ats

Publications (1)

Publication Number Publication Date
PL3947684T3 true PL3947684T3 (pl) 2025-07-21

Family

ID=72666233

Family Applications (1)

Application Number Title Priority Date Filing Date
PL20784141.2T PL3947684T3 (pl) 2019-03-29 2020-03-27 Związki i sposoby modulacji ube3a-ats

Country Status (32)

Country Link
US (3) US11261446B2 (pl)
EP (2) EP3947684B1 (pl)
JP (2) JP7550165B2 (pl)
KR (1) KR20210144822A (pl)
CN (2) CN113728104B (pl)
AU (1) AU2020253821B2 (pl)
BR (1) BR112021019427A2 (pl)
CA (1) CA3135180A1 (pl)
CL (1) CL2021002515A1 (pl)
CO (1) CO2021012796A2 (pl)
CR (1) CR20210538A (pl)
DK (1) DK3947684T3 (pl)
ES (1) ES3025960T3 (pl)
FI (1) FI3947684T3 (pl)
HR (1) HRP20250596T1 (pl)
HU (1) HUE071494T2 (pl)
IL (1) IL286681A (pl)
JO (1) JOP20210261A1 (pl)
LT (1) LT3947684T (pl)
MD (1) MD3947684T2 (pl)
MX (1) MX2021011916A (pl)
NZ (1) NZ780663A (pl)
PE (1) PE20220284A1 (pl)
PH (1) PH12021500044A1 (pl)
PL (1) PL3947684T3 (pl)
PT (1) PT3947684T (pl)
SG (1) SG11202110679YA (pl)
SI (1) SI3947684T1 (pl)
TW (2) TWI874376B (pl)
UA (1) UA128437C2 (pl)
UY (1) UY38626A (pl)
WO (1) WO2020205463A1 (pl)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2809199T3 (es) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
EP3374498A1 (en) 2015-11-12 2018-09-19 H. Hoffnabb-La Roche Ag Standardized neuronal cell assays from primate species
PL3947684T3 (pl) 2019-03-29 2025-07-21 Ionis Pharmaceuticals, Inc. Związki i sposoby modulacji ube3a-ats
WO2022178160A1 (en) * 2021-02-17 2022-08-25 Q-State Biosciences, Inc. Ube3a antisense therapeutics
KR20250068540A (ko) * 2023-11-08 2025-05-16 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
TW202536178A (zh) * 2023-11-09 2025-09-16 美商Ionis製藥公司 用於調節ube3a-ats之方法
US20250353873A1 (en) * 2024-05-17 2025-11-20 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating apoe expression

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