KR20250160515A - Pcsk9 또는 angptl3의 유전자 편집, 이를 이용한 조성물 및 질병의 치료에 사용하는 방법 - Google Patents

Pcsk9 또는 angptl3의 유전자 편집, 이를 이용한 조성물 및 질병의 치료에 사용하는 방법

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Publication number
KR20250160515A
KR20250160515A KR1020257035760A KR20257035760A KR20250160515A KR 20250160515 A KR20250160515 A KR 20250160515A KR 1020257035760 A KR1020257035760 A KR 1020257035760A KR 20257035760 A KR20257035760 A KR 20257035760A KR 20250160515 A KR20250160515 A KR 20250160515A
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South Korea
Prior art keywords
represent
sequence
uridine
adenosine
naturally occurring
Prior art date
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Pending
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KR1020257035760A
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English (en)
Korean (ko)
Inventor
앤드류 엠 벨링거
칼란토타틸 지 라지브
캐롤라인 레이스
제이미 데니지오
하리하란 자야람
소우미아 아이어
알메이다 핀토 가르시아 사라 크리스티나 데
쿠이 왕
알렉산드라 채드윅
크리스토퍼 쳉
리차드 글렌 리
엘렌 로데
Original Assignee
버브 테라퓨틱스, 인크.
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Application filed by 버브 테라퓨틱스, 인크. filed Critical 버브 테라퓨틱스, 인크.
Publication of KR20250160515A publication Critical patent/KR20250160515A/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
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    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • 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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
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KR1020257035760A 2021-09-22 2022-09-22 Pcsk9 또는 angptl3의 유전자 편집, 이를 이용한 조성물 및 질병의 치료에 사용하는 방법 Pending KR20250160515A (ko)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US202163247236P 2021-09-22 2021-09-22
US63/247,236 2021-09-22
US202163255333P 2021-10-13 2021-10-13
US63/255,333 2021-10-13
US202263389679P 2022-07-15 2022-07-15
US63/389,679 2022-07-15
KR1020247013385A KR20240099200A (ko) 2021-09-22 2022-09-22 Pcsk9 또는 angptl3의 유전자 편집, 이를 이용한 조성물 및 질병의 치료에 사용하는 방법
PCT/US2022/044453 WO2023049299A2 (en) 2021-09-22 2022-09-22 Gene editing of pcsk9 or angptl3 and compositions and methods of using same for treatment of disease

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020247013385A Division KR20240099200A (ko) 2021-09-22 2022-09-22 Pcsk9 또는 angptl3의 유전자 편집, 이를 이용한 조성물 및 질병의 치료에 사용하는 방법

Publications (1)

Publication Number Publication Date
KR20250160515A true KR20250160515A (ko) 2025-11-13

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KR1020257035760A Pending KR20250160515A (ko) 2021-09-22 2022-09-22 Pcsk9 또는 angptl3의 유전자 편집, 이를 이용한 조성물 및 질병의 치료에 사용하는 방법
KR1020247013385A Pending KR20240099200A (ko) 2021-09-22 2022-09-22 Pcsk9 또는 angptl3의 유전자 편집, 이를 이용한 조성물 및 질병의 치료에 사용하는 방법

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KR1020247013385A Pending KR20240099200A (ko) 2021-09-22 2022-09-22 Pcsk9 또는 angptl3의 유전자 편집, 이를 이용한 조성물 및 질병의 치료에 사용하는 방법

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US (1) US20240390520A1 (cg-RX-API-DMAC7.html)
EP (1) EP4405470A4 (cg-RX-API-DMAC7.html)
JP (2) JP2024534575A (cg-RX-API-DMAC7.html)
KR (2) KR20250160515A (cg-RX-API-DMAC7.html)
AU (1) AU2022349449A1 (cg-RX-API-DMAC7.html)
CA (1) CA3232742A1 (cg-RX-API-DMAC7.html)
GB (4) GB2702733A (cg-RX-API-DMAC7.html)
IL (1) IL311607A (cg-RX-API-DMAC7.html)
MX (1) MX2024003570A (cg-RX-API-DMAC7.html)
WO (1) WO2023049299A2 (cg-RX-API-DMAC7.html)

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WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
JP7646552B2 (ja) 2019-01-31 2025-03-17 ビーム セラピューティクス インク． 低減された非標的脱アミノ化を有する核酸塩基エディターおよび核酸塩基エディターの特徴づけのためのアッセイ
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
WO2025137465A1 (en) * 2023-12-21 2025-06-26 Beam Therapeutics Inc. Base editors with modified editing windows
WO2025167914A1 (zh) * 2024-02-07 2025-08-14 益杰立科新加坡有限公司 通过表观编辑多靶点治疗疾病的方法
WO2026143185A1 (en) 2024-12-23 2026-07-02 Ensoma Aps Engineered rna-guided dna-binding polypeptides

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