JP2024525646A - 条件付きで活性化可能な核酸複合体 - Google Patents

条件付きで活性化可能な核酸複合体 Download PDF

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JP2024525646A
JP2024525646A JP2024500649A JP2024500649A JP2024525646A JP 2024525646 A JP2024525646 A JP 2024525646A JP 2024500649 A JP2024500649 A JP 2024500649A JP 2024500649 A JP2024500649 A JP 2024500649A JP 2024525646 A JP2024525646 A JP 2024525646A
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nucleic acid
strand
acid strand
acid complex
aspects
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JP2024525646A5 (https=
JPWO2023283546A5 (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 JP2024525646A5 publication Critical patent/JP2024525646A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • 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
    • 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
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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]
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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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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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
    • C12N2310/30—Chemical structure
    • 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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    • C12N2310/33—Chemical structure of the base
    • C12N2310/331—Universal or degenerate base
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

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  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
JP2024500649A 2021-07-06 2022-07-05 条件付きで活性化可能な核酸複合体 Pending JP2024525646A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163218833P 2021-07-06 2021-07-06
US63/218,833 2021-07-06
PCT/US2022/073426 WO2023283546A1 (en) 2021-07-06 2022-07-05 Conditionally activatable nucleic acid complexes

Publications (3)

Publication Number Publication Date
JP2024525646A true JP2024525646A (ja) 2024-07-12
JP2024525646A5 JP2024525646A5 (https=) 2025-07-14
JPWO2023283546A5 JPWO2023283546A5 (https=) 2025-07-14

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ID=84802039

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JP2024500649A Pending JP2024525646A (ja) 2021-07-06 2022-07-05 条件付きで活性化可能な核酸複合体

Country Status (9)

Country Link
US (1) US20250188453A1 (https=)
EP (1) EP4367126A4 (https=)
JP (1) JP2024525646A (https=)
KR (1) KR20240095154A (https=)
CN (1) CN118202048A (https=)
AU (1) AU2022308051A1 (https=)
CA (1) CA3224942A1 (https=)
IL (1) IL309942A (https=)
WO (1) WO2023283546A1 (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
WO2026006174A1 (en) * 2024-06-24 2026-01-02 Arizona Board Of Regents On Behalf Of Arizona State University Compositions and methods related to removal of transmembrane sensors

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2019014656A1 (en) * 2017-07-14 2019-01-17 Han Si Ping METALLIC OLIGONUCLEOTIDE JONCTIONS FOR THE ADMINISTRATION OF THERAPEUTIC AGENTS
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
US11834659B2 (en) * 2019-09-26 2023-12-05 Massachusetts Institute Of Technology Trans-activated functional RNA by strand displacement and uses thereof

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Publication number Publication date
KR20240095154A (ko) 2024-06-25
EP4367126A1 (en) 2024-05-15
CA3224942A1 (en) 2023-01-12
US20250188453A1 (en) 2025-06-12
AU2022308051A9 (en) 2024-02-22
IL309942A (en) 2024-03-01
WO2023283546A1 (en) 2023-01-12
AU2022308051A1 (en) 2024-01-25
EP4367126A4 (en) 2025-07-02
CN118202048A (zh) 2024-06-14

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