JP2023544162A - Ｃｈｍｐ７を調節するための化合物 - Google Patents

Ｃｈｍｐ７を調節するための化合物 Download PDF

Info

Publication number
JP2023544162A
JP2023544162A JP2023520049A JP2023520049A JP2023544162A JP 2023544162 A JP2023544162 A JP 2023544162A JP 2023520049 A JP2023520049 A JP 2023520049A JP 2023520049 A JP2023520049 A JP 2023520049A JP 2023544162 A JP2023544162 A JP 2023544162A
Authority
JP
Japan
Prior art keywords
modified
certain embodiments
oligomeric compound
oligomeric
modified oligonucleotide
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2023520049A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023544162A5 (enExample
Inventor
ブイ，フィン－ホア
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
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 JP2023544162A publication Critical patent/JP2023544162A/ja
Publication of JP2023544162A5 publication Critical patent/JP2023544162A5/ja
Pending legal-status Critical Current

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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 ===---===
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023520049A 2020-10-01 2021-10-01 Ｃｈｍｐ７を調節するための化合物 Pending JP2023544162A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063086576P 2020-10-01 2020-10-01
US63/086,576 2020-10-01
US202063119448P 2020-11-30 2020-11-30
US63/119,448 2020-11-30
US202163139715P 2021-01-20 2021-01-20
US63/139,715 2021-01-20
PCT/US2021/053107 WO2022072787A1 (en) 2020-10-01 2021-10-01 Compounds for modulating chmp7

Publications (2)

Publication Number Publication Date
JP2023544162A true JP2023544162A (ja) 2023-10-20
JP2023544162A5 JP2023544162A5 (enExample) 2024-10-07

Family

ID=80950932

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023520049A Pending JP2023544162A (ja) 2020-10-01 2021-10-01 Ｃｈｍｐ７を調節するための化合物

Country Status (4)

Country Link
US (1) US20240002852A1 (enExample)
EP (1) EP4222261A4 (enExample)
JP (1) JP2023544162A (enExample)
WO (1) WO2022072787A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023549160A (ja) * 2020-11-10 2023-11-22 ザ・ジョンズ・ホプキンス・ユニバーシティ 神経変性疾患の治療のために神経細胞におけるｃｈｍｐ７発現を阻害するための方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2563921B1 (en) * 2010-04-30 2016-11-23 Cellectis Method for modulating double-strand break-induced homologous recombination
WO2012122015A2 (en) * 2011-03-04 2012-09-13 Immune Disease Institute, Inc. Selective inhibitors of tumor-initiating cells
IL300258A (en) * 2020-08-07 2023-03-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating SCN2A

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
J. CELL BIOL. 2020 VOL. 219 NO. 5 E201908179, vol. Vol.219, No.5, e201908179, JPN6025036774, April 2020 (2020-04-01), pages 1 - 12, ISSN: 0005680043 *
J. CELL BIOL., vol. 218, no. 7, JPN6025036775, 2019, pages 2136 - 2149, ISSN: 0005874645 *
ONCOGENESIS, vol. 8, no. 29, JPN6025036776, 2019, pages 1 - 14, ISSN: 0005680042 *

Also Published As

Publication number Publication date
WO2022072787A1 (en) 2022-04-07
EP4222261A4 (en) 2025-10-15
US20240002852A1 (en) 2024-01-04
EP4222261A1 (en) 2023-08-09

Similar Documents

Publication Publication Date Title
JP7557469B2 (ja) Ａｐｐの発現を低減するための化合物及び方法
JP7557378B2 (ja) Ｓｔｍｎ２発現を増加させるための化合物及び方法
JP7550165B2 (ja) Ｕｂｅ３ａ－ａｔｓを調節するための化合物及び方法
JP7110485B2 (ja) Ｐｎｐｌａ３発現のモジュレーター
JP2024520205A (ja) Ｕｎｃ１３ａの発現を調節するための化合物
WO2023064707A1 (en) Compounds and methods for reducing tau expression
JP2024504377A (ja) Ｄｕｘ４の発現を低減するための化合物及び方法
JP7834712B2 (ja) Ｓｃｎ２ａを調節する化合物および方法
JP7820374B2 (ja) アンジオテンシノーゲン発現を調節するための化合物及び方法
JP2023536472A (ja) Ａｐｐの発現を低減するための化合物及び方法
JP2024506351A (ja) Ｐｌｎの発現を低減するための化合物及び方法
JP2023543215A (ja) Ａｐｏｅの発現を低減するための化合物及び方法
JP2024540537A (ja) プログラニュリン発現を調節するための化合物及び方法
JP2024523363A (ja) Ｉｆｎａｒ１の発現を低減するための化合物及び方法
JP2026062868A (ja) Ｐｌｐ１を調節するための化合物及び方法
JP2024505226A (ja) ハンチンチンを調節するための化合物及び方法
JP2023530072A (ja) Ｐｍｐ２２を調節するための化合物及び方法
WO2024064854A2 (en) Compounds and methods for reducing mecp2 expression
JP2024540079A (ja) Ｐｓｄ３発現を低減させるための化合物及び方法
EP4222261A1 (en) Compounds for modulating chmp7
JP2025500438A (ja) Ｐｃｄｈ１９発現を低減するための化合物及び方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240927

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240927

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250903

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20251106

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260227

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20260602