JP2025510339A - オリゴマー化合物を使用してｓｃｎ２ａ関連障害を治療する方法 - Google Patents

オリゴマー化合物を使用してｓｃｎ２ａ関連障害を治療する方法 Download PDF

Info

Publication number
JP2025510339A
JP2025510339A JP2024557582A JP2024557582A JP2025510339A JP 2025510339 A JP2025510339 A JP 2025510339A JP 2024557582 A JP2024557582 A JP 2024557582A JP 2024557582 A JP2024557582 A JP 2024557582A JP 2025510339 A JP2025510339 A JP 2025510339A
Authority
JP
Japan
Prior art keywords
certain embodiments
modified
oligomeric compound
nucleobase
seq
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
JP2024557582A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025510339A5 (enExample
Inventor
マイケル エス． オールダム，
スティーブン ペトロー，
Original Assignee
プラクシス プレシジョン メディシンズ， インコーポレイテッド
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 プラクシス プレシジョン メディシンズ， インコーポレイテッド filed Critical プラクシス プレシジョン メディシンズ， インコーポレイテッド
Publication of JP2025510339A publication Critical patent/JP2025510339A/ja
Publication of JP2025510339A5 publication Critical patent/JP2025510339A5/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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • C12N2310/32—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
    • C12N2310/34—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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pain & Pain Management (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Hydrogenated Pyridines (AREA)
JP2024557582A 2022-03-28 2023-03-28 オリゴマー化合物を使用してｓｃｎ２ａ関連障害を治療する方法 Pending JP2025510339A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263324531P 2022-03-28 2022-03-28
US63/324,531 2022-03-28
US202263335152P 2022-04-26 2022-04-26
US63/335,152 2022-04-26
PCT/US2023/065074 WO2023192885A2 (en) 2022-03-28 2023-03-28 Methods of using oligomeric compounds to treat scn2a-related disorders

Publications (2)

Publication Number Publication Date
JP2025510339A true JP2025510339A (ja) 2025-04-14
JP2025510339A5 JP2025510339A5 (enExample) 2026-04-06

Family

ID=88203426

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024557582A Pending JP2025510339A (ja) 2022-03-28 2023-03-28 オリゴマー化合物を使用してｓｃｎ２ａ関連障害を治療する方法

Country Status (6)

Country Link
US (1) US20250179503A1 (enExample)
EP (1) EP4499835A2 (enExample)
JP (1) JP2025510339A (enExample)
AU (1) AU2023244348A1 (enExample)
CA (1) CA3246834A1 (enExample)
WO (1) WO2023192885A2 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024343201A1 (en) * 2023-09-15 2026-03-26 Praxis Precision Medicines, Inc. Elsunersen / prax-222 for use in the treatment of scn2a-related disorders

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8138201B2 (en) * 2007-07-11 2012-03-20 Medicinova, Inc. Treatment of progressive neurodegenerative disease with ibudilast
IL297642A (en) * 2020-04-29 2022-12-01 Praxis Prec Medicines Inc Methods for using t-type calcium channel modulators
IL300258A (en) * 2020-08-07 2023-03-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating SCN2A

Also Published As

Publication number Publication date
WO2023192885A2 (en) 2023-10-05
EP4499835A2 (en) 2025-02-05
US20250179503A1 (en) 2025-06-05
AU2023244348A1 (en) 2024-10-10
CA3246834A1 (en) 2023-10-05
WO2023192885A3 (en) 2024-01-11

Similar Documents

Publication Publication Date Title
JP7477569B2 (ja) タウ発現を低減するための化合物及び方法
JP7557469B2 (ja) Ａｐｐの発現を低減するための化合物及び方法
JP2025121956A (ja) Ｌｒｒｋ２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
TWI874376B (zh) 用於調節ube3a-ats 之化合物及方法
JP2024520205A (ja) Ｕｎｃ１３ａの発現を調節するための化合物
JP7446443B2 (ja) Ｓｍｎ２を調節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
JP7564817B2 (ja) ｋｃｎｔ１発現を低減するための化合物及び方法
JP7511563B2 (ja) Ｃｌｎ３の発現を調節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
JP2026062868A (ja) Ｐｌｐ１を調節するための化合物及び方法
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
JP2025510339A (ja) オリゴマー化合物を使用してｓｃｎ２ａ関連障害を治療する方法
EP4660307A2 (en) Compounds and methods for modulating gfap
JP2026506010A (ja) Ａｐｐ発現を低減するための化合物及び方法
JP2023533153A (ja) Ｋｃｎｔ１の発現を低減するための化合物及び方法
HK40129490A (en) Compounds and methods for modulating ube3a-ats
HK40107010A (en) Compounds and methods for reducing tau expression
JP2025500438A (ja) Ｐｃｄｈ１９発現を低減するための化合物及び方法
HK40067280B (en) Compounds and methods for modulating ube3a-ats
HK40067280A (en) Compounds and methods for modulating ube3a-ats
EA048039B1 (ru) Соединения и способы модулирования smn2
EA043298B1 (ru) Соединения и способы модулирования ube3a-ats

Legal Events

Date Code Title Description
A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20241212

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260327

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20260327