KR20260027260A - Snp를 표적화하는 변형된 올리고뉴클레오티드 - Google Patents

Snp를 표적화하는 변형된 올리고뉴클레오티드

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Publication number
KR20260027260A
KR20260027260A KR1020267001731A KR20267001731A KR20260027260A KR 20260027260 A KR20260027260 A KR 20260027260A KR 1020267001731 A KR1020267001731 A KR 1020267001731A KR 20267001731 A KR20267001731 A KR 20267001731A KR 20260027260 A KR20260027260 A KR 20260027260A
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South Korea
Prior art keywords
nucleotide
rna
nucleotides
snp
strand
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Pending
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KR1020267001731A
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English (en)
Korean (ko)
Inventor
아나스타샤 크보로바
줄리아 알터만
페이스 콘로이
이디스 피스터
닐 아로닌
켄 야마다
Original Assignee
유니버시티 오브 매사추세츠
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Publication of KR20260027260A publication Critical patent/KR20260027260A/ko
Pending legal-status Critical Current

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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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/312—Phosphonates
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020267001731A 2018-08-10 2019-08-09 Snp를 표적화하는 변형된 올리고뉴클레오티드 Pending KR20260027260A (ko)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201862717287P 2018-08-10 2018-08-10
US62/717,287 2018-08-10
US201962825429P 2019-03-28 2019-03-28
US62/825,429 2019-03-28
KR1020217006987A KR102916447B1 (ko) 2018-08-10 2019-08-09 Snp를 표적화하는 변형된 올리고뉴클레오티드
PCT/US2019/046013 WO2020033899A1 (en) 2018-08-10 2019-08-09 Modified oligonucleotides targeting snps

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020217006987A Division KR102916447B1 (ko) 2018-08-10 2019-08-09 Snp를 표적화하는 변형된 올리고뉴클레오티드

Publications (1)

Publication Number Publication Date
KR20260027260A true KR20260027260A (ko) 2026-02-27

Family

ID=69414459

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020267001731A Pending KR20260027260A (ko) 2018-08-10 2019-08-09 Snp를 표적화하는 변형된 올리고뉴클레오티드
KR1020217006987A Active KR102916447B1 (ko) 2018-08-10 2019-08-09 Snp를 표적화하는 변형된 올리고뉴클레오티드

Family Applications After (1)

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KR1020217006987A Active KR102916447B1 (ko) 2018-08-10 2019-08-09 Snp를 표적화하는 변형된 올리고뉴클레오티드

Country Status (12)

Country Link
US (2) US11827882B2 (https=)
EP (1) EP3833763A4 (https=)
JP (2) JP7618229B2 (https=)
KR (2) KR20260027260A (https=)
CN (1) CN112805383A (https=)
AU (2) AU2019316640B2 (https=)
BR (1) BR112021002440A2 (https=)
CA (1) CA3109133A1 (https=)
IL (1) IL280724A (https=)
MX (2) MX2021001590A (https=)
SG (1) SG11202101288TA (https=)
WO (1) WO2020033899A1 (https=)

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US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
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US10478503B2 (en) 2016-01-31 2019-11-19 University Of Massachusetts Branched oligonucleotides
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
WO2020033899A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
US11820985B2 (en) 2019-03-26 2023-11-21 University Of Massachusetts Modified oligonucleotides with increased stability
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
WO2021041247A1 (en) * 2019-08-23 2021-03-04 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
US20210363524A1 (en) * 2020-03-18 2021-11-25 University Of Massachusetts Oligonucleotides for snca modulation
JP7837560B2 (ja) 2020-03-26 2026-03-31 ユニバーシティー オブ マサチューセッツ 安定性の向上を有する修飾オリゴヌクレオチドの合成
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
EP4158028A4 (en) * 2020-05-28 2024-10-02 University Of Massachusetts OLIGONUCLEOTIDES FOR THE MODULATION OF SARS-COV-2
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
CN117677699A (zh) 2021-06-23 2024-03-08 马萨诸塞大学 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物
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CN120020252A (zh) * 2023-11-17 2025-05-20 纳肽得(青岛)生物医药有限公司 抑制LPA表达的siRNA及其用途

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