WO2023164656A3 - Compounds and methods for modulating atn1 expression - Google Patents
Compounds and methods for modulating atn1 expression Download PDFInfo
- 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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- WO
- WIPO (PCT)
- Prior art keywords
- atn1
- methods
- compounds
- modulating
- expression
- Prior art date
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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
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- 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
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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
- 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)
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- 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.
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)
Country | Link |
---|---|
WO (1) | WO2023164656A2 (en) |
Citations (3)
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 |
-
2023
- 2023-02-24 WO PCT/US2023/063280 patent/WO2023164656A2/en unknown
Patent Citations (3)
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)
Title |
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DATABASE NUCLEOTIDE ANONYMOUS : "PREDICTED: Mirounga leonina atrophin 1 (ATN1), transcript variant X2, mRNA", XP093096276, retrieved from NCBI * |
Also Published As
Publication number | Publication date |
---|---|
WO2023164656A2 (en) | 2023-08-31 |
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