KR20240043822A - 올리고뉴클레오티드 제제를 위한 올리고뉴클레오티드 기반 전달체 및 이의 사용 방법 - Google Patents

올리고뉴클레오티드 제제를 위한 올리고뉴클레오티드 기반 전달체 및 이의 사용 방법 Download PDF

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KR20240043822A
KR20240043822A KR1020247004696A KR20247004696A KR20240043822A KR 20240043822 A KR20240043822 A KR 20240043822A KR 1020247004696 A KR1020247004696 A KR 1020247004696A KR 20247004696 A KR20247004696 A KR 20247004696A KR 20240043822 A KR20240043822 A KR 20240043822A
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seq
oligonucleotide
nucleotide sequence
stranded oligonucleotide
preparation
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Korean (ko)
Inventor
무림 강
롱쳉 리
로버트 플레이스
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락티젠 세러퓨틱스
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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
    • 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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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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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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    • C12N2310/30—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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
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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
    • C12N2310/3517—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)
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  • 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)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
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  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020247004696A 2021-07-07 2022-07-06 올리고뉴클레오티드 제제를 위한 올리고뉴클레오티드 기반 전달체 및 이의 사용 방법 Pending KR20240043822A (ko)

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 (1)

Publication Number Publication Date
KR20240043822A true KR20240043822A (ko) 2024-04-03

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KR1020247004696A Pending KR20240043822A (ko) 2021-07-07 2022-07-06 올리고뉴클레오티드 제제를 위한 올리고뉴클레오티드 기반 전달체 및 이의 사용 방법

Country Status (9)

Country Link
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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Publication number Priority date Publication date Assignee Title
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
US20210002640A1 (en) * 2018-02-28 2021-01-07 Exicure, Inc. Liposomal spherical nucleic acid (sna) constructs for survival of motor neuron (sma) inhibitors
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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US20250297258A1 (en) 2025-09-25
AU2022308123A1 (en) 2024-02-22
JP2024523702A (ja) 2024-06-28
EP4367244A4 (en) 2025-07-09
CA3225998A1 (en) 2023-01-12
TW202321449A (zh) 2023-06-01
EP4367244A1 (en) 2024-05-15
CN117916374A (zh) 2024-04-19
WO2023280190A1 (en) 2023-01-12

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