KR102802134B1 - 신규한 omni-50 crispr 뉴클레아제 - Google Patents

신규한 omni-50 crispr 뉴클레아제 Download PDF

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KR102802134B1
KR102802134B1 KR1020217037743A KR20217037743A KR102802134B1 KR 102802134 B1 KR102802134 B1 KR 102802134B1 KR 1020217037743 A KR1020217037743 A KR 1020217037743A KR 20217037743 A KR20217037743 A KR 20217037743A KR 102802134 B1 KR102802134 B1 KR 102802134B1
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데이비드 바람
리오 이즈하
아사엘 헤르만
리앗 록카
나다브 마르바크-바
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에멘도바이오 인코포레이티드
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    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
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    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
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    • C12N2310/00—Structure or type of the nucleic acid
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KR1020217037743A 2019-04-30 2020-04-30 신규한 omni-50 crispr 뉴클레아제 Active KR102802134B1 (ko)

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KR1020257013727A KR20250060327A (ko) 2019-04-30 2020-04-30 신규한 omni-50 crispr 뉴클레아제

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US201962841046P 2019-04-30 2019-04-30
US62/841,046 2019-04-30
US201962897806P 2019-09-09 2019-09-09
US62/897,806 2019-09-09
US201962931630P 2019-11-06 2019-11-06
US62/931,630 2019-11-06
US202062959672P 2020-01-10 2020-01-10
US62/959,672 2020-01-10
US202062991285P 2020-03-18 2020-03-18
US62/991,285 2020-03-18
PCT/US2020/030782 WO2020223514A2 (en) 2019-04-30 2020-04-30 Novel omni-50 crispr nuclease

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CN (1) CN114127274A (enExample)
AU (2) AU2020266587B2 (enExample)
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CA (1) CA3135759A1 (enExample)
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Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
JP2023531384A (ja) 2020-06-04 2023-07-24 エメンドバイオ・インコーポレイテッド 新規なｏｍｎｉ－５９、６１、６７、７６、７９、８０、８１及び８２クリスパーヌクレアーゼ
CN116670271A (zh) * 2020-11-04 2023-08-29 埃门多生物公司 新型omni-50 crispr核酸酶-rna复合物
WO2022132765A1 (en) * 2020-12-14 2022-06-23 Emendobio Inc. Biallelic knockout of b2m
EP4259159A4 (en) * 2020-12-14 2025-04-09 Emendobio Inc. Biallelic knockout of trac
US12091688B2 (en) 2021-02-08 2024-09-17 Emendobio Inc. OMNI-103 CRISPR nuclease
JP2024506608A (ja) 2021-02-08 2024-02-14 エメンドバイオ・インコーポレイテッド Ｏｍｎｉ－１０３ ｃｒｉｓｐｒヌクレアーゼ
AU2022262623A1 (en) * 2021-04-22 2023-11-30 Emendobio Inc. NOVEL OMNI 117, 140, 150-158, 160-165, 167-177, 180-188, 191-198, 200, 201, 203, 205-209, 211-217, 219, 220, 222, 223, 226, 227, 229, 231-236, 238-245, 247, 250, 254, 256, 257, 260 and 262 CRISPR NUCLEASES
IL310730A (en) * 2021-08-12 2024-04-01 Emendobio Inc Engineered high fidelity omni-50 nuclease variants
CN118556119A (zh) * 2021-11-19 2024-08-27 埃门多生物公司 新型omni crispr核酸酶
CN118647715A (zh) * 2021-12-01 2024-09-13 埃门多生物公司 工程化高活性omni-79核酸酶变体
AU2023308345A1 (en) * 2022-07-15 2025-02-27 Emendobio Inc. Strategies for knock-ins at aplp2 safe harbor sites
WO2024042165A2 (en) * 2022-08-26 2024-02-29 UCB Biopharma SRL Novel rna-guided nucleases and nucleic acid targeting systems comprising such rna-guided nucleases
WO2025043140A1 (en) * 2023-08-23 2025-02-27 Emendobio Inc. Methods and compositions for modifying expression of a mutant transforming growth factor beta induced (tgfbi) allele

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015134812A1 (en) * 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8383604B2 (en) 2008-09-15 2013-02-26 Children's Medical Center Corporation Modulation of BCL11A for treatment of hemoglobinopathies
AU2013266968B2 (en) * 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
WO2017024047A1 (en) 2015-08-03 2017-02-09 Emendobio Inc. Compositions and methods for increasing nuclease induced recombination rate in cells
US12043843B2 (en) 2015-11-04 2024-07-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN105567735A (zh) * 2016-01-05 2016-05-11 华东师范大学 一种凝血因子基因突变的定点修复载体系统及方法

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015134812A1 (en) * 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa

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WO2020223514A3 (en) 2020-12-24
WO2020223514A2 (en) 2020-11-05
JP2024050582A (ja) 2024-04-10
IL287693A (en) 2021-12-01
KR20250060327A (ko) 2025-05-07
AU2020266587B2 (en) 2024-01-04
JP2022531347A (ja) 2022-07-06
BR112021021912A2 (pt) 2022-01-18
EP3963061A4 (en) 2023-02-08
US20220202913A1 (en) 2022-06-30
CN114127274A (zh) 2022-03-01
US11666641B2 (en) 2023-06-06
EP3963061A2 (en) 2022-03-09
US20240226246A9 (en) 2024-07-11
CA3135759A1 (en) 2020-11-05
US20240131121A1 (en) 2024-04-25
AU2020266587A1 (en) 2021-12-16
KR20220012245A (ko) 2022-02-03

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