JP2024523702A - オリゴヌクレオチド剤用オリゴヌクレオチドベースの送達担体およびその使用方法 - Google Patents

オリゴヌクレオチド剤用オリゴヌクレオチドベースの送達担体およびその使用方法 Download PDF

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JP2024523702A
JP2024523702A JP2024500176A JP2024500176A JP2024523702A JP 2024523702 A JP2024523702 A JP 2024523702A JP 2024500176 A JP2024500176 A JP 2024500176A JP 2024500176 A JP2024500176 A JP 2024500176A JP 2024523702 A JP2024523702 A JP 2024523702A
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seq
oligonucleotide
nucleotide sequence
oligonucleotide agent
stranded
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ムーリン カン
ロンチョン リ
ロバート エフ プレイス
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ラクティゲン セラピューティクス
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    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/1137—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 enzymes
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3517—Marker; Tag
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024500176A 2021-07-07 2022-07-06 オリゴヌクレオチド剤用オリゴヌクレオチドベースの送達担体およびその使用方法 Pending JP2024523702A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
CN2021105081 2021-07-07
CNPCT/CN2021/105081 2021-07-07
CNPCT/CN2022/091076 2022-05-06
CN2022091076 2022-05-06
PCT/CN2022/104037 WO2023280190A1 (en) 2021-07-07 2022-07-06 Oligonucleotide-based delivery vehicle for oligonucleotides agents and methods of use thereof

Publications (2)

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JP2024523702A true JP2024523702A (ja) 2024-06-28
JP2024523702A5 JP2024523702A5 (https=) 2025-07-10

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JP2024500176A Pending JP2024523702A (ja) 2021-07-07 2022-07-06 オリゴヌクレオチド剤用オリゴヌクレオチドベースの送達担体およびその使用方法

Country Status (9)

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US (1) US20250297258A1 (https=)
EP (1) EP4367244A4 (https=)
JP (1) JP2024523702A (https=)
KR (1) KR20240043822A (https=)
CN (1) CN117916374A (https=)
AU (1) AU2022308123A1 (https=)
CA (1) CA3225998A1 (https=)
TW (1) TW202321449A (https=)
WO (1) WO2023280190A1 (https=)

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JP2025522433A (ja) 2022-06-15 2025-07-15 アローヘッド ファーマシューティカルズ インコーポレイテッド スーパーオキシドジスムターゼ１（ＳＯＤ１）の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用の方法
KR20250148653A (ko) * 2023-02-17 2025-10-14 아이오니스 파마수티컬즈, 인코포레이티드 App 발현을 감소시키기 위한 화합물 및 방법
KR20250155023A (ko) * 2023-02-24 2025-10-29 락티젠 세러퓨틱스 Sod1을 표적으로 하는 올리고뉴클레오타이드
WO2024199445A1 (en) * 2023-03-30 2024-10-03 Ractigen Therapeutics Vehicles for delivering oligonucleotides and methods of use thereof
EP4728073A1 (en) * 2023-06-16 2026-04-22 Ractigen Therapeutics Oligonucleotide formulation
CN117402096A (zh) * 2023-10-20 2024-01-16 康羽生命科学技术(苏州)有限公司 一种辅氨酸衍生物的合成方法
WO2025119303A1 (en) * 2023-12-07 2025-06-12 Ractigen Therapeutics Abasic modification compound, gene modulating agents and the use thereof
WO2025126153A2 (en) * 2023-12-14 2025-06-19 Aviadobio Ltd. Compositions and methods for treating sod1-mediated neurological diseases

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CA2715289C (en) * 2008-02-11 2019-12-24 Rxi Pharmaceuticals Corporation Modified rnai polynucleotides and uses thereof
EP2342340A1 (en) * 2008-09-22 2011-07-13 Rxi Pharmaceuticals Corporation Rna interference in skin indications
EP2362729A1 (en) * 2008-11-17 2011-09-07 Enzon Pharmaceuticals, Inc. Releasable conjugates for nucleic acids delivery systems
US9745574B2 (en) * 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
EP2756845B1 (en) * 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
RU2615143C2 (ru) * 2010-03-24 2017-04-04 Адвирна Самодоставляющие PHKi соединения уменьшенного размера
CA2871089A1 (en) * 2011-04-20 2012-10-26 Smith Holdings, Llc Methods and compositions for modulating gene expression using components that self assemble in cells and produce rnai activity
EP3004347B1 (en) * 2013-05-30 2018-09-26 National University Corporation Tokyo Medical and Dental University Double-stranded agents for delivering therapeutic oligonucleotides
EP3862005A1 (en) * 2015-07-06 2021-08-11 Phio Pharmaceuticals Corp. Nucleic acid molecules targeting superoxide dismutase 1 (sod1)
US11260134B2 (en) * 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
SG10201609048RA (en) * 2016-10-28 2018-05-30 Agency Science Tech & Res Antisense oligonucleotides
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CN113614232A (zh) * 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚

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Publication number Publication date
US20250297258A1 (en) 2025-09-25
AU2022308123A1 (en) 2024-02-22
EP4367244A4 (en) 2025-07-09
CA3225998A1 (en) 2023-01-12
TW202321449A (zh) 2023-06-01
EP4367244A1 (en) 2024-05-15
KR20240043822A (ko) 2024-04-03
CN117916374A (zh) 2024-04-19
WO2023280190A1 (en) 2023-01-12

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