JP2024520205A - Ｕｎｃ１３ａの発現を調節するための化合物 - Google Patents

Ｕｎｃ１３ａの発現を調節するための化合物 Download PDF

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Publication number
JP2024520205A
JP2024520205A JP2023571857A JP2023571857A JP2024520205A JP 2024520205 A JP2024520205 A JP 2024520205A JP 2023571857 A JP2023571857 A JP 2023571857A JP 2023571857 A JP2023571857 A JP 2023571857A JP 2024520205 A JP2024520205 A JP 2024520205A
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Prior art keywords
oligomeric compound
modified
certain embodiments
modified oligonucleotide
sugar moiety
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JP2023571857A
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English (en)
Japanese (ja)
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JP2024520205A5 (https=
JPWO2022246251A5 (https=
Inventor
バラス，ルーベン・イー
リン，カー・ユン・カレン
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of JP2024520205A publication Critical patent/JP2024520205A/ja
Publication of JP2024520205A5 publication Critical patent/JP2024520205A5/ja
Publication of JPWO2022246251A5 publication Critical patent/JPWO2022246251A5/ja
Pending legal-status Critical Current

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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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • C12N2310/32—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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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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)
  • Medicinal Preparation (AREA)
JP2023571857A 2021-05-21 2022-05-20 Ｕｎｃ１３ａの発現を調節するための化合物 Pending JP2024520205A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163191905P 2021-05-21 2021-05-21
US63/191,905 2021-05-21
PCT/US2022/030335 WO2022246251A2 (en) 2021-05-21 2022-05-20 Compounds for modulating unc13a expression

Publications (3)

Publication Number Publication Date
JP2024520205A true JP2024520205A (ja) 2024-05-22
JP2024520205A5 JP2024520205A5 (https=) 2025-05-26
JPWO2022246251A5 JPWO2022246251A5 (https=) 2025-05-26

Family

ID=84140868

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023571857A Pending JP2024520205A (ja) 2021-05-21 2022-05-20 Ｕｎｃ１３ａの発現を調節するための化合物

Country Status (4)

Country Link
US (1) US20240301415A1 (https=)
EP (1) EP4341406A4 (https=)
JP (1) JP2024520205A (https=)
WO (1) WO2022246251A2 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
CA3227115A1 (en) * 2021-07-21 2023-01-26 Wen-Hsuan Chang Unc13a antisense oligonucleotides
GB202117758D0 (en) * 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
CN118974254A (zh) * 2021-12-21 2024-11-15 豪夫迈·罗氏有限公司 靶向unc13a的反义寡核苷酸
AU2023356317A1 (en) * 2022-10-05 2025-05-15 Trace Neuroscience, Inc. Unc13a antisense oligonucleotides and uses thereof
WO2024178223A1 (en) * 2023-02-24 2024-08-29 Northwestern University Antisense oligonucleotides for preventing unc13a misplicing
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
AU2024299328A1 (en) 2023-07-21 2026-01-22 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
US20250263702A1 (en) 2024-02-19 2025-08-21 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting cideb and related methods
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2026080323A1 (en) * 2024-10-09 2026-04-16 Quralis Corporation Treatment of neurological diseases using modulators of unc13a gene transcripts

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08510130A (ja) * 1993-05-11 1996-10-29 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
WO2022122872A1 (en) * 2020-12-09 2022-06-16 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060008800A1 (en) * 2001-11-05 2006-01-12 Christian Rosenmund Unc-13 in the modulation of neurotransmission and secretion events
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
EP1752536A4 (en) * 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
US20110195848A1 (en) * 2010-01-08 2011-08-11 Roopra Avtar S Gene expression and breast cancer
US10517889B2 (en) * 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08510130A (ja) * 1993-05-11 1996-10-29 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 異常スプライシングを阻害するアンチセンスオリゴヌクレオチドと同物質の利用法
WO2022122872A1 (en) * 2020-12-09 2022-06-16 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ANNA-LEIGH BROWN ET AL.: ""Common ALS/FTD risk variants in UNC13Aexacerbate its cryptic splicing and loss upon TDP-43 misloca", BIORXIV, JPN6026007603, 4 April 2021 (2021-04-04), ISSN: 0005804909 *

Also Published As

Publication number Publication date
WO2022246251A2 (en) 2022-11-24
EP4341406A4 (en) 2026-03-25
US20240301415A1 (en) 2024-09-12
EP4341406A2 (en) 2024-03-27
WO2022246251A3 (en) 2022-12-29

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