JP2023540797A - 塩基編集酵素 - Google Patents
塩基編集酵素 Download PDFInfo
- Publication number
- JP2023540797A JP2023540797A JP2023516057A JP2023516057A JP2023540797A JP 2023540797 A JP2023540797 A JP 2023540797A JP 2023516057 A JP2023516057 A JP 2023516057A JP 2023516057 A JP2023516057 A JP 2023516057A JP 2023540797 A JP2023540797 A JP 2023540797A
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- Japan
- Prior art keywords
- endonuclease
- nucleic acid
- seq
- sequence
- engineered
- Prior art date
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- 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
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
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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
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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
- C12N15/70—Vectors or expression systems specially adapted for E. coli
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- 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
- 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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- 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
- C12N15/63—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
- C12N15/86—Viral vectors
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- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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- 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
- C12N15/87—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
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2497—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing N- glycosyl compounds (3.2.2)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/02—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2) hydrolysing N-glycosyl compounds (3.2.2)
- C12Y302/02027—Uracil-DNA glycosylase (3.2.2.27)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Mycology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Graft Or Block Polymers (AREA)
- Dental Preparations (AREA)
- Paints Or Removers (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063077057P | 2020-09-11 | 2020-09-11 | |
| US63/077,057 | 2020-09-11 | ||
| US202163222351P | 2021-07-15 | 2021-07-15 | |
| US63/222,351 | 2021-07-15 | ||
| PCT/US2021/049962 WO2022056324A1 (en) | 2020-09-11 | 2021-09-10 | Base editing enzymes |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023540797A true JP2023540797A (ja) | 2023-09-26 |
| JP2023540797A5 JP2023540797A5 (https=) | 2024-09-13 |
| JPWO2022056324A5 JPWO2022056324A5 (https=) | 2024-09-13 |
Family
ID=80629930
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023516057A Pending JP2023540797A (ja) | 2020-09-11 | 2021-09-10 | 塩基編集酵素 |
Country Status (9)
| Country | Link |
|---|---|
| US (3) | US12286654B2 (https=) |
| EP (1) | EP4211234A4 (https=) |
| JP (1) | JP2023540797A (https=) |
| KR (1) | KR20230062873A (https=) |
| AU (1) | AU2021339819A1 (https=) |
| CA (1) | CA3192224A1 (https=) |
| GB (1) | GB2605340B (https=) |
| MX (1) | MX2023002913A (https=) |
| WO (1) | WO2022056324A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| CN121555430A (zh) | 2018-05-11 | 2026-02-24 | 比姆医疗股份有限公司 | 使用可编程碱基编辑器系统取代病原性氨基酸的方法 |
| KR20210055733A (ko) | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| WO2020168122A1 (en) | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| GB2617658B (en) | 2020-03-06 | 2024-04-17 | Metagenomi Inc | Class II, type V CRISPR systems |
| GB2608292B (en) * | 2020-03-31 | 2024-02-14 | Metagenomi Inc | Class II, type II CRISPR systems |
| CA3192224A1 (en) | 2020-09-11 | 2022-03-17 | Jyun-Liang LIN | Base editing enzymes |
| AU2021347359A1 (en) | 2020-09-25 | 2023-05-18 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| BR112023014719A2 (pt) * | 2021-01-22 | 2023-12-05 | Metagenomi Inc | Novas nucleases quiméricas e manipuladas |
| CA3225082A1 (en) | 2021-08-27 | 2023-03-02 | Brian C. Thomas | Enzymes with ruvc domains |
| AU2022380842A1 (en) * | 2021-11-05 | 2024-05-23 | Metagenomi Therapeutics, Inc. | Base editing enzymes |
| MX2024006257A (es) | 2021-11-24 | 2024-07-29 | Metagenomi Inc | Sistemas de endonucleasas. |
| CN121241133A (zh) * | 2023-03-31 | 2025-12-30 | 阿伯生物技术公司 | Crispr核酸酶多肽和包含此类crispr核酸酶多肽的基因编辑系统 |
| WO2025003344A1 (en) * | 2023-06-28 | 2025-01-02 | Alia Therapeutics Srl | Type ii cas proteins and applications thereof |
| WO2025106894A1 (en) * | 2023-11-15 | 2025-05-22 | Profluent Bio Inc. | Adenine deaminases and compositions, systems, and methods thereof |
| WO2025240372A1 (en) * | 2024-05-13 | 2025-11-20 | Metagenomi, Inc. | Compositions and methods for editing t cells |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018179578A1 (ja) * | 2017-03-30 | 2018-10-04 | 国立大学法人京都大学 | ゲノム編集によるエクソンスキッピング誘導方法 |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102547316B1 (ko) * | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| US11192929B2 (en) | 2016-12-08 | 2021-12-07 | Regents Of The University Of Minnesota | Site-specific DNA base editing using modified APOBEC enzymes |
| EP3572525A4 (en) | 2017-01-17 | 2020-09-30 | Institute for Basic Science | PROCEDURE FOR IDENTIFYING AN OFF-TARGET BASIC EDITING SITE BY BREAKING A SINGLE DNA STRAND |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| US9982279B1 (en) * | 2017-06-23 | 2018-05-29 | Inscripta, Inc. | Nucleic acid-guided nucleases |
| AU2018290843B2 (en) * | 2017-06-26 | 2025-04-24 | Massachusetts Institute Of Technology | CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing |
| JP2021506251A (ja) * | 2017-12-14 | 2021-02-22 | クリスパー セラピューティクス アーゲー | 新規rnaプログラム可能エンドヌクレアーゼ系、ならびにゲノム編集および他の適用におけるその使用 |
| US11946040B2 (en) * | 2019-02-04 | 2024-04-02 | The General Hospital Corporation | Adenine DNA base editor variants with reduced off-target RNA editing |
| US20220298494A1 (en) | 2019-02-14 | 2022-09-22 | Metagenomi Ip Technologies, Llc | Enzymes with ruvc domains |
| AU2020223370B2 (en) | 2019-02-14 | 2023-04-20 | Metagenomi Therapeutics, Inc. | Enzymes with RuvC domains |
| WO2021097118A1 (en) | 2019-11-12 | 2021-05-20 | The Broad Institute, Inc. | Small type ii cas proteins and methods of use thereof |
| GB2608292B (en) * | 2020-03-31 | 2024-02-14 | Metagenomi Inc | Class II, type II CRISPR systems |
| GB2700205B (en) | 2020-09-11 | 2026-04-15 | Metagenomi Therapeutics Inc | Base editing enzymes |
| CA3192224A1 (en) | 2020-09-11 | 2022-03-17 | Jyun-Liang LIN | Base editing enzymes |
| AU2022380842A1 (en) * | 2021-11-05 | 2024-05-23 | Metagenomi Therapeutics, Inc. | Base editing enzymes |
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2021
- 2021-09-10 CA CA3192224A patent/CA3192224A1/en active Pending
- 2021-09-10 GB GB2209534.3A patent/GB2605340B/en active Active
- 2021-09-10 MX MX2023002913A patent/MX2023002913A/es unknown
- 2021-09-10 JP JP2023516057A patent/JP2023540797A/ja active Pending
- 2021-09-10 EP EP21867718.5A patent/EP4211234A4/en active Pending
- 2021-09-10 AU AU2021339819A patent/AU2021339819A1/en active Pending
- 2021-09-10 KR KR1020237012244A patent/KR20230062873A/ko active Pending
- 2021-09-10 WO PCT/US2021/049962 patent/WO2022056324A1/en not_active Ceased
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2022
- 2022-06-15 US US17/841,082 patent/US12286654B2/en active Active
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2023
- 2023-03-07 US US18/180,009 patent/US20230348876A1/en active Pending
- 2023-05-31 US US18/326,440 patent/US20230374482A1/en active Pending
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018179578A1 (ja) * | 2017-03-30 | 2018-10-04 | 国立大学法人京都大学 | ゲノム編集によるエクソンスキッピング誘導方法 |
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| EXPERIMENTAL & MOLECULAR MEDICINE, vol. 51:130, JPN6025041383, 2019, pages 1 - 11, ISSN: 0005705350 * |
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| EP4211234A1 (en) | 2023-07-19 |
| GB2605340A8 (en) | 2024-02-21 |
| US20230374482A1 (en) | 2023-11-23 |
| GB2605340A (en) | 2022-09-28 |
| AU2021339819A1 (en) | 2023-05-25 |
| CA3192224A1 (en) | 2022-03-17 |
| WO2022056324A1 (en) | 2022-03-17 |
| US20220364067A1 (en) | 2022-11-17 |
| GB2605340B (en) | 2024-12-18 |
| MX2023002913A (es) | 2023-06-01 |
| EP4211234A4 (en) | 2025-04-30 |
| KR20230062873A (ko) | 2023-05-09 |
| US20230348876A1 (en) | 2023-11-02 |
| GB202209534D0 (en) | 2022-08-10 |
| US12286654B2 (en) | 2025-04-29 |
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