JP2024525647A - シグナルで活性化可能な核酸複合体 - Google Patents

シグナルで活性化可能な核酸複合体 Download PDF

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JP2024525647A
JP2024525647A JP2024500651A JP2024500651A JP2024525647A JP 2024525647 A JP2024525647 A JP 2024525647A JP 2024500651 A JP2024500651 A JP 2024500651A JP 2024500651 A JP2024500651 A JP 2024500651A JP 2024525647 A JP2024525647 A JP 2024525647A
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nucleic acid
acid strand
strand
acid complex
nucleosides
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JP2024525647A5 (https=
JPWO2023283550A5 (https=
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ハン、スー－ピン
ダフ、ロバート
シェラー、リサ
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スウィッチ・セラピューティクス・インコーポレイテッド
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Publication of JP2024525647A5 publication Critical patent/JP2024525647A5/ja
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2024500651A 2021-07-06 2022-07-05 シグナルで活性化可能な核酸複合体 Pending JP2024525647A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163218852P 2021-07-06 2021-07-06
US63/218,852 2021-07-06
PCT/US2022/073430 WO2023283550A1 (en) 2021-07-06 2022-07-05 Signal activatable nucleic acid complexes

Publications (3)

Publication Number Publication Date
JP2024525647A true JP2024525647A (ja) 2024-07-12
JP2024525647A5 JP2024525647A5 (https=) 2025-07-14
JPWO2023283550A5 JPWO2023283550A5 (https=) 2025-07-14

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ID=84802044

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JP2024500651A Pending JP2024525647A (ja) 2021-07-06 2022-07-05 シグナルで活性化可能な核酸複合体

Country Status (9)

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US (1) US20250257353A1 (https=)
EP (1) EP4367245A4 (https=)
JP (1) JP2024525647A (https=)
KR (1) KR20240095155A (https=)
CN (1) CN117916376A (https=)
AU (1) AU2022308843A1 (https=)
CA (1) CA3224950A1 (https=)
IL (1) IL309943A (https=)
WO (1) WO2023283550A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019014656A1 (en) 2017-07-14 2019-01-17 Han Si Ping METALLIC OLIGONUCLEOTIDE JONCTIONS FOR THE ADMINISTRATION OF THERAPEUTIC AGENTS
EP3665281A4 (en) 2017-08-10 2021-05-05 City of Hope CONDITIONAL SIRNA AND ITS USE IN THE TREATMENT OF CARDIAC HYERTROPHY
CN113166750B (zh) 2018-08-10 2025-02-18 希望之城 可编程的条件性sirna及其用途
CN116478410B (zh) * 2023-06-20 2023-09-12 觅投克(北京)生物医学技术有限公司 一种菊糖修饰的聚乙烯亚胺衍生物及其制备方法和应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6696285B1 (en) * 1998-08-25 2004-02-24 Lucent Technolgies Inc. Nanomachines fueled by nucleic acid strand exchange
US9029524B2 (en) * 2007-12-10 2015-05-12 California Institute Of Technology Signal activated RNA interference
US20100112556A1 (en) * 2008-11-03 2010-05-06 Sampson Jeffrey R Method for sample analysis using q probes
US8710199B2 (en) * 2010-06-23 2014-04-29 California Institute Of Technology Signal activated molecular delivery
US9725715B2 (en) * 2011-06-23 2017-08-08 California Institute Of Technology Signal activatable constructs and related components compositions methods and systems
WO2013142735A1 (en) * 2012-03-21 2013-09-26 California Institute Of Technology Targeting domain and related signal activated molecular delivery
US9856472B2 (en) * 2013-07-01 2018-01-02 California Institute Of Technology Small conditional RNAs
WO2019033083A1 (en) * 2017-08-10 2019-02-14 City Of Hope CONDITIONAL RNAIDS AND THEIR USES IN THE TREATMENT OF ACUTE MYELOID LEUKEMIA
EP3665281A4 (en) * 2017-08-10 2021-05-05 City of Hope CONDITIONAL SIRNA AND ITS USE IN THE TREATMENT OF CARDIAC HYERTROPHY
CN113166750B (zh) * 2018-08-10 2025-02-18 希望之城 可编程的条件性sirna及其用途

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Publication number Publication date
EP4367245A4 (en) 2025-07-02
WO2023283550A1 (en) 2023-01-12
IL309943A (en) 2024-03-01
CA3224950A1 (en) 2023-01-12
EP4367245A1 (en) 2024-05-15
CN117916376A (zh) 2024-04-19
KR20240095155A (ko) 2024-06-25
AU2022308843A1 (en) 2024-02-01
US20250257353A1 (en) 2025-08-14

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