GB2616584B - Engineered class 2 type V CRISPR systems - Google Patents
Engineered class 2 type V CRISPR systemsInfo
- Publication number
- GB2616584B GB2616584B GB2309876.7A GB202309876A GB2616584B GB 2616584 B GB2616584 B GB 2616584B GB 202309876 A GB202309876 A GB 202309876A GB 2616584 B GB2616584 B GB 2616584B
- Authority
- GB
- United Kingdom
- Prior art keywords
- type
- crispr systems
- engineered class
- engineered
- class
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
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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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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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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
- C12N15/11—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
- 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
- C12N15/11—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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- 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
- C12N15/63—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
- 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
- C12N15/63—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
- 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
- C12N15/87—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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- 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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
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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
- 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
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Virology (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063121196P | 2020-12-03 | 2020-12-03 | |
| US202163162346P | 2021-03-17 | 2021-03-17 | |
| US202163208855P | 2021-06-09 | 2021-06-09 | |
| PCT/US2021/061673 WO2022120095A1 (en) | 2020-12-03 | 2021-12-02 | Engineered class 2 type v crispr systems |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| GB202309876D0 GB202309876D0 (en) | 2023-08-16 |
| GB2616584A GB2616584A (en) | 2023-09-13 |
| GB2616584B true GB2616584B (en) | 2026-03-04 |
Family
ID=80050633
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| GB2309876.7A Active GB2616584B (en) | 2020-12-03 | 2021-12-02 | Engineered class 2 type V CRISPR systems |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US20240026385A1 (https=) |
| EP (1) | EP4256054A1 (https=) |
| JP (1) | JP2023552374A (https=) |
| KR (1) | KR20230128289A (https=) |
| AU (1) | AU2021392719B2 (https=) |
| CA (1) | CA3201258A1 (https=) |
| CL (2) | CL2023001594A1 (https=) |
| GB (1) | GB2616584B (https=) |
| MX (1) | MX2023006566A (https=) |
| PE (1) | PE20231178A1 (https=) |
| WO (1) | WO2022120095A1 (https=) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3980533A1 (en) | 2019-06-07 | 2022-04-13 | Scribe Therapeutics Inc. | Engineered casx systems |
| WO2021050601A1 (en) | 2019-09-09 | 2021-03-18 | Scribe Therapeutics Inc. | Compositions and methods for use in immunotherapy |
| WO2021113763A1 (en) | 2019-12-06 | 2021-06-10 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of rhodopsin |
| KR20230128289A (ko) | 2020-12-03 | 2023-09-04 | 스크라이브 테라퓨틱스 인크. | 조작된 클래스 2 유형 v crispr 시스템 |
| WO2022261149A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| GB2625500B (en) | 2021-09-21 | 2026-04-01 | Scribe Therapeutics Inc | Engineered CasX repressor systems |
| WO2023235818A2 (en) | 2022-06-02 | 2023-12-07 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| WO2023235888A2 (en) | 2022-06-03 | 2023-12-07 | Scribe Therapeutics Inc. | COMPOSITIONS AND METHODS FOR CpG DEPLETION |
| WO2023240027A1 (en) | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Particle delivery systems |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| TW202413643A (zh) | 2022-06-07 | 2024-04-01 | 美商斯奎柏治療公司 | 用於靶向pcsk9的組合物及方法 |
| WO2023240157A2 (en) | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of dmd |
| US20250361525A1 (en) | 2022-06-08 | 2025-11-27 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| CN116676291B (zh) * | 2022-08-22 | 2024-02-27 | 华中农业大学 | 核酸内切酶Genie scissor及其介导的基因编辑系统 |
| WO2024206565A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Repressor fusion protein systems |
| PE20252746A1 (es) | 2023-03-29 | 2025-12-05 | Scribe Therapeutics Inc | Composiciones y metodos para el direccionamiento a pcsk9 |
| WO2024206676A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of lpa |
| WO2024206620A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Messenger rna encoding casx |
| JP7560818B1 (ja) | 2023-08-25 | 2024-10-03 | 株式会社キュライオ | エンジニアリングされたタンパク質 |
| AU2024332871A1 (en) * | 2023-08-25 | 2026-03-12 | Jichi Medical University | Engineered protein |
| WO2025072293A1 (en) | 2023-09-27 | 2025-04-03 | Scribe Therapeutics Inc. | Optimized mrnas encoding casx proteins |
| WO2025240940A1 (en) | 2024-05-17 | 2025-11-20 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of apolipoprotein c3 |
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| WO2021084533A1 (en) | 2019-10-28 | 2021-05-06 | Targetgene Biotechnologies Ltd | Pam-reduced and pam-abolished cas derivatives compositions and uses thereof in genetic modulation |
| EP4069716A1 (en) * | 2019-12-06 | 2022-10-12 | Scribe Therapeutics Inc. | Particle delivery systems |
| WO2021113763A1 (en) | 2019-12-06 | 2021-06-10 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of rhodopsin |
| EP4069846A1 (en) * | 2019-12-07 | 2022-10-12 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of htt |
| IL294620A (en) | 2020-01-10 | 2022-09-01 | Scribe Therapeutics Inc | Compositions and methods for the targeting of pcsk9 |
| AU2021237633A1 (en) | 2020-03-18 | 2022-10-06 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of C9orf72 |
| WO2022119957A1 (en) | 2020-12-02 | 2022-06-09 | The Regents Of The University Of California | Crispr-cas effector polypeptides and methods of use thereof |
| KR20230128289A (ko) | 2020-12-03 | 2023-09-04 | 스크라이브 테라퓨틱스 인크. | 조작된 클래스 2 유형 v crispr 시스템 |
| AU2021391783A1 (en) | 2020-12-03 | 2023-06-22 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of bcl11a |
| WO2022125843A1 (en) | 2020-12-09 | 2022-06-16 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| CA3212612A1 (en) | 2021-03-26 | 2022-09-29 | Brent E. STEAD | Targeting oncogentic mutations with dual-cleaving endonuclease |
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| GB2625500B (en) | 2021-09-21 | 2026-04-01 | Scribe Therapeutics Inc | Engineered CasX repressor systems |
| EP4405490A2 (en) | 2021-09-23 | 2024-07-31 | Scribe Therapeutics Inc. | Self-inactivating vectors for gene editing |
| WO2023235818A2 (en) | 2022-06-02 | 2023-12-07 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| TW202413643A (zh) | 2022-06-07 | 2024-04-01 | 美商斯奎柏治療公司 | 用於靶向pcsk9的組合物及方法 |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| WO2023240157A2 (en) | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of dmd |
| WO2024206676A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of lpa |
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2021
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- 2021-12-02 JP JP2023533837A patent/JP2023552374A/ja active Pending
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| EP4256054A1 (en) | 2023-10-11 |
| AU2021392719A1 (en) | 2023-06-22 |
| US20250011756A1 (en) | 2025-01-09 |
| CA3201258A1 (en) | 2022-06-09 |
| PE20231178A1 (es) | 2023-08-01 |
| US20240026385A1 (en) | 2024-01-25 |
| CL2023001594A1 (es) | 2024-01-05 |
| US12559743B2 (en) | 2026-02-24 |
| US20230054437A1 (en) | 2023-02-23 |
| MX2023006566A (es) | 2023-08-07 |
| AU2021392719A9 (en) | 2024-10-03 |
| JP2023552374A (ja) | 2023-12-15 |
| GB202309876D0 (en) | 2023-08-16 |
| WO2022120095A1 (en) | 2022-06-09 |
| US20240376462A1 (en) | 2024-11-14 |
| TW202237836A (zh) | 2022-10-01 |
| KR20230128289A (ko) | 2023-09-04 |
| CL2024001727A1 (es) | 2024-10-18 |
| GB2616584A (en) | 2023-09-13 |
| US12163125B2 (en) | 2024-12-10 |
| AU2021392719B2 (en) | 2025-08-14 |
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