JP2024540537A - 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 - Google Patents

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 Download PDF

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Publication number
JP2024540537A
JP2024540537A JP2024529579A JP2024529579A JP2024540537A JP 2024540537 A JP2024540537 A JP 2024540537A JP 2024529579 A JP2024529579 A JP 2024529579A JP 2024529579 A JP2024529579 A JP 2024529579A JP 2024540537 A JP2024540537 A JP 2024540537A
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Japan
Prior art keywords
modified
oligomeric compound
modified oligonucleotide
certain embodiments
sugar moiety
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Pending
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JP2024529579A
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English (en)
Japanese (ja)
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JPWO2023092057A5 (https=
JP2024540537A5 (https=
Inventor
ブイ，フィン・ホア
ジャファー・ネジャド，ペイマン
グエン，アンドリュー・トゥアン・ドゥク
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of JP2024540537A publication Critical patent/JP2024540537A/ja
Publication of JPWO2023092057A5 publication Critical patent/JPWO2023092057A5/ja
Publication of JP2024540537A5 publication Critical patent/JP2024540537A5/ja
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • C12N2310/33—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024529579A 2021-11-18 2022-11-18 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 Pending JP2024540537A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163281057P 2021-11-18 2021-11-18
US63/281,057 2021-11-18
PCT/US2022/080113 WO2023092057A1 (en) 2021-11-18 2022-11-18 Compounds and methods for modulating progranulin expression

Publications (3)

Publication Number Publication Date
JP2024540537A true JP2024540537A (ja) 2024-10-31
JPWO2023092057A5 JPWO2023092057A5 (https=) 2025-11-06
JP2024540537A5 JP2024540537A5 (https=) 2025-11-06

Family

ID=86397858

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024529579A Pending JP2024540537A (ja) 2021-11-18 2022-11-18 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

Country Status (4)

Country Link
US (1) US20250109396A1 (https=)
EP (1) EP4433591A1 (https=)
JP (1) JP2024540537A (https=)
WO (1) WO2023092057A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20260051070A (ko) 2023-08-10 2026-04-15 하네스 테라퓨틱스 리미티드 기능적 핵산
WO2025104638A1 (en) * 2023-11-17 2025-05-22 Universita' Degli Studi Di Brescia Antisense oligonucleotides for modulation of progranulin splicing
GB202401412D0 (en) 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2520935A3 (en) * 2006-08-09 2013-02-13 Homestead Clinical Corporation Organ-specific proteins and methods of their use
CN106967799A (zh) * 2017-03-24 2017-07-21 杭州艾迪康医学检验中心有限公司 检测btk基因突变的方法、寡核苷酸及其应用
US12595479B2 (en) * 2019-03-20 2026-04-07 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
TW202208628A (zh) * 2020-05-13 2022-03-01 瑞士商赫孚孟拉羅股份公司 靶向顆粒蛋白前體之寡核苷酸促效劑

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Publication number Publication date
WO2023092057A1 (en) 2023-05-25
US20250109396A1 (en) 2025-04-03
EP4433591A1 (en) 2024-09-25

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