JP2024528202A - 遺伝子修飾のための組成物及び方法 - Google Patents
遺伝子修飾のための組成物及び方法 Download PDFInfo
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- JP2024528202A JP2024528202A JP2024506631A JP2024506631A JP2024528202A JP 2024528202 A JP2024528202 A JP 2024528202A JP 2024506631 A JP2024506631 A JP 2024506631A JP 2024506631 A JP2024506631 A JP 2024506631A JP 2024528202 A JP2024528202 A JP 2024528202A
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- grna
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- nuclease
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
-
- 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]
-
- 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
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Developmental Biology & Embryology (AREA)
- Veterinary Medicine (AREA)
- Immunology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Crystallography & Structural Chemistry (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163228548P | 2021-08-02 | 2021-08-02 | |
| US63/228,548 | 2021-08-02 | ||
| US202163229484P | 2021-08-04 | 2021-08-04 | |
| US63/229,484 | 2021-08-04 | ||
| US202263341346P | 2022-05-12 | 2022-05-12 | |
| US63/341,346 | 2022-05-12 | ||
| US202263346819P | 2022-05-27 | 2022-05-27 | |
| US63/346,819 | 2022-05-27 | ||
| PCT/US2022/074423 WO2023015182A1 (en) | 2021-08-02 | 2022-08-02 | Compositions and methods for gene modification |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024528202A true JP2024528202A (ja) | 2024-07-26 |
| JP2024528202A5 JP2024528202A5 (https=) | 2025-08-13 |
Family
ID=83558256
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024506631A Pending JP2024528202A (ja) | 2021-08-02 | 2022-08-02 | 遺伝子修飾のための組成物及び方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20250122534A1 (https=) |
| EP (1) | EP4381062A1 (https=) |
| JP (1) | JP2024528202A (https=) |
| AU (1) | AU2022324093A1 (https=) |
| CA (1) | CA3228272A1 (https=) |
| WO (1) | WO2023015182A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024073751A1 (en) | 2022-09-29 | 2024-04-04 | Vor Biopharma Inc. | Methods and compositions for gene modification and enrichment |
| WO2025030010A1 (en) | 2023-08-01 | 2025-02-06 | Vor Biopharma Inc. | Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| WO2015157070A2 (en) | 2014-04-09 | 2015-10-15 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating cystic fibrosis |
| CA2969619A1 (en) | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
| KR102888521B1 (ko) | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| GB201506509D0 (en) | 2015-04-16 | 2015-06-03 | Univ Wageningen | Nuclease-mediated genome editing |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| CA2996211C (en) | 2015-08-25 | 2024-05-21 | Snooz, Llc | Machine and method for acoustic white noise generation |
| AU2016337525B2 (en) | 2015-10-16 | 2022-01-20 | The Trustees Of Columbia University In The City Of New York | Compositions and methods for inhibition of lineage specific antigens |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| WO2017155407A1 (en) | 2016-03-11 | 2017-09-14 | Wageningen Universiteit | Improved crispr-cpf1 genome editing tool |
| KR20260004568A (ko) | 2016-04-19 | 2026-01-08 | 더 브로드 인스티튜트, 인코퍼레이티드 | 신규한 crispr 효소 및 시스템 |
| US11499168B2 (en) | 2016-04-25 | 2022-11-15 | Universitat Basel | Allele editing and applications thereof |
| US10767175B2 (en) | 2016-06-08 | 2020-09-08 | Agilent Technologies, Inc. | High specificity genome editing using chemically modified guide RNAs |
| AU2017291851B2 (en) * | 2016-07-06 | 2022-10-13 | Cellectis | Sequential gene editing in primary immune cells |
| GB201618507D0 (en) | 2016-11-02 | 2016-12-14 | Stichting Voor De Technische Wetenschappen And Wageningen Univ | Microbial genome editing |
| LT3535392T (lt) | 2016-11-02 | 2024-04-25 | Universität Basel | Imunologiškai atpažįstami ląstelių paviršiaus variantai, skirti naudoti ląstelių terapijoje |
| KR20190082318A (ko) | 2016-11-22 | 2019-07-09 | 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. | Crispr/cpf1 시스템 및 방법 |
| CA3048434A1 (en) | 2016-12-30 | 2018-07-05 | Editas Medicine, Inc. | Synthetic guide molecules, compositions and methods relating thereto |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| EP3724326A1 (en) | 2017-12-11 | 2020-10-21 | Editas Medicine, Inc. | Cpf1-related methods and compositions for gene editing |
| EP3746554A1 (en) * | 2018-01-30 | 2020-12-09 | Editas Medicine, Inc. | Systems and methods for modulating chromosomal rearrangements |
| CN111699254A (zh) * | 2018-02-08 | 2020-09-22 | 齐默尔根公司 | 使用crispr在棒状杆菌属中进行基因组编辑 |
| EA202092044A1 (ru) | 2018-03-14 | 2021-03-03 | Дзе Юнайтед Стейтс Оф Америка, Эз Репрезентед Бай Дзе Секретари, Департмент Оф Хелс Энд Хьюман Сёрвисез | Химерные рецепторы антигена против cd33 и их применения |
| CA3110837A1 (en) | 2018-08-28 | 2020-03-05 | Vor Biopharma Inc. | Genetically engineered hematopoietic stem cells and uses thereof |
| US20200216825A1 (en) | 2019-01-08 | 2020-07-09 | Integrated Dna Technologies, Inc. | CAS12a MUTANT GENES AND POLYPEPTIDES ENCODED BY SAME |
| BR112021014010A2 (pt) | 2019-01-16 | 2021-09-21 | The Trustees Of Columbia University In The City Of New York | Composições e métodos para inibição de antígenos linhagem-específicos |
| CN113811608B (zh) | 2019-02-22 | 2024-10-29 | 合成Dna技术公司 | 毛螺菌科细菌nd2006 cas12a突变型基因和由其编码的多肽 |
| AU2020280103A1 (en) | 2019-05-23 | 2021-12-23 | Vor Biopharma Inc. | Compositions and methods for CD33 modification |
| AU2020338011A1 (en) | 2019-08-28 | 2022-03-10 | Vor Biopharma Inc. | Compositions and methods for CLL1 modification |
| WO2021041977A1 (en) | 2019-08-28 | 2021-03-04 | Vor Biopharma, Inc. | Compositions and methods for cd123 modification |
-
2022
- 2022-08-02 AU AU2022324093A patent/AU2022324093A1/en active Pending
- 2022-08-02 CA CA3228272A patent/CA3228272A1/en active Pending
- 2022-08-02 US US18/293,882 patent/US20250122534A1/en active Pending
- 2022-08-02 JP JP2024506631A patent/JP2024528202A/ja active Pending
- 2022-08-02 WO PCT/US2022/074423 patent/WO2023015182A1/en not_active Ceased
- 2022-08-02 EP EP22783650.9A patent/EP4381062A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20250122534A1 (en) | 2025-04-17 |
| EP4381062A1 (en) | 2024-06-12 |
| AU2022324093A1 (en) | 2024-02-08 |
| WO2023015182A1 (en) | 2023-02-09 |
| CA3228272A1 (en) | 2023-02-09 |
| AU2022324093A2 (en) | 2024-02-15 |
| WO2023015182A9 (en) | 2023-10-19 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20250804 |
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| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20250804 |