WO2023164656A3 - Compounds and methods for modulating atn1 expression - Google Patents

Compounds and methods for modulating atn1 expression Download PDF

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Publication number
WO2023164656A3
WO2023164656A3 PCT/US2023/063280 US2023063280W WO2023164656A3 WO 2023164656 A3 WO2023164656 A3 WO 2023164656A3 US 2023063280 W US2023063280 W US 2023063280W WO 2023164656 A3 WO2023164656 A3 WO 2023164656A3
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WIPO (PCT)
Prior art keywords
atn1
methods
compounds
modulating
expression
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PCT/US2023/063280
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French (fr)
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WO2023164656A2 (en
Inventor
Hunyh-Hoa BUI
Susan M. Freier
Tracy A. COLE
Holly Kordasiewicz
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Ionis Pharmaceuticals, Inc.
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Application filed by Ionis Pharmaceuticals, Inc. filed Critical Ionis Pharmaceuticals, Inc.
Publication of WO2023164656A2 publication Critical patent/WO2023164656A2/en
Publication of WO2023164656A3 publication Critical patent/WO2023164656A3/en

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    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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 ===---===

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  • 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)

Abstract

Provided arc compounds, pharmaceutical compositions, and methods of use for reducing the amount or activity of ATN1 RNA in a cell or subject, and in certain instances reducing the amount of atrophin-1 protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a poly glutamine (polyQ) disease or disorder associated with ATN1.
PCT/US2023/063280 2022-02-25 2023-02-24 Compounds and methods for modulating atn1 expression WO2023164656A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263313948P 2022-02-25 2022-02-25
US63/313,948 2022-02-25

Publications (2)

Publication Number Publication Date
WO2023164656A2 WO2023164656A2 (en) 2023-08-31
WO2023164656A3 true WO2023164656A3 (en) 2023-09-28

Family

ID=87766780

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/063280 WO2023164656A2 (en) 2022-02-25 2023-02-24 Compounds and methods for modulating atn1 expression

Country Status (1)

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WO (1) WO2023164656A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200149043A1 (en) * 2012-04-23 2020-05-14 Biomarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
WO2021153747A1 (en) * 2020-01-31 2021-08-05 株式会社三和化学研究所 Antisense oligonucleotide of atn1
WO2021174031A2 (en) * 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating splicing of pre-mrna

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200149043A1 (en) * 2012-04-23 2020-05-14 Biomarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
WO2021153747A1 (en) * 2020-01-31 2021-08-05 株式会社三和化学研究所 Antisense oligonucleotide of atn1
WO2021174031A2 (en) * 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating splicing of pre-mrna

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DATABASE NUCLEOTIDE ANONYMOUS : "PREDICTED: Mirounga leonina atrophin 1 (ATN1), transcript variant X2, mRNA", XP093096276, retrieved from NCBI *

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Publication number Publication date
WO2023164656A2 (en) 2023-08-31

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