JP2024511163A - アデノシンデアミナーゼバリアント及びその使用 - Google Patents
アデノシンデアミナーゼバリアント及びその使用 Download PDFInfo
- Publication number
- JP2024511163A JP2024511163A JP2023558765A JP2023558765A JP2024511163A JP 2024511163 A JP2024511163 A JP 2024511163A JP 2023558765 A JP2023558765 A JP 2023558765A JP 2023558765 A JP2023558765 A JP 2023558765A JP 2024511163 A JP2024511163 A JP 2024511163A
- Authority
- JP
- Japan
- Prior art keywords
- adenosine deaminase
- variant
- amino acid
- editing
- base
- 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.)
- Pending
Links
Images
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
-
- 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]
-
- 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)
-
- 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)
- C12Y305/04005—Cytidine deaminase (3.5.4.5)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Crystallography & Structural Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163166778P | 2021-03-26 | 2021-03-26 | |
| US63/166,778 | 2021-03-26 | ||
| US202163229057P | 2021-08-03 | 2021-08-03 | |
| US63/229,057 | 2021-08-03 | ||
| PCT/US2022/022050 WO2022204574A1 (en) | 2021-03-26 | 2022-03-25 | Adenosine deaminase variants and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024511163A true JP2024511163A (ja) | 2024-03-12 |
| JP2024511163A5 JP2024511163A5 (https=) | 2025-04-01 |
Family
ID=83397962
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023558765A Pending JP2024511163A (ja) | 2021-03-26 | 2022-03-25 | アデノシンデアミナーゼバリアント及びその使用 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20240158775A1 (https=) |
| EP (1) | EP4313118A4 (https=) |
| JP (1) | JP2024511163A (https=) |
| KR (1) | KR20240004276A (https=) |
| AU (1) | AU2022246176A1 (https=) |
| BR (1) | BR112023019539A2 (https=) |
| CA (1) | CA3214494A1 (https=) |
| WO (1) | WO2022204574A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| EP3847254A4 (en) | 2018-09-07 | 2022-08-10 | Beam Therapeutics Inc. | COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM |
| AU2020215730A1 (en) | 2019-01-31 | 2021-07-29 | Beam Therapeutics Inc. | Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors |
| CN120174005A (zh) | 2019-02-13 | 2025-06-20 | 比姆医疗股份有限公司 | 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞 |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| CA3196831A1 (en) | 2020-09-25 | 2022-03-31 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| EP4705444A2 (en) * | 2023-05-04 | 2026-03-11 | Technische Universität Dresden | Highly active crispr base editors obtained through cas-assisted substrate-linked directed evolution (caslide) |
| EP4458963A1 (en) * | 2023-05-04 | 2024-11-06 | Technische Universität Dresden | Highly active crispr base editors obtained through cas-assisted substrate-linked directed evolution (caslide) |
| CN120290534A (zh) * | 2024-01-10 | 2025-07-11 | 上海数因信科智能科技有限公司 | 腺嘌呤脱氨酶、包含其的碱基编辑器融合蛋白和碱基编辑器系统及其应用 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021022043A2 (en) * | 2019-07-30 | 2021-02-04 | Pairwise Plants Services, Inc. | Morphogenic regulators and methods of using the same |
| WO2021050571A1 (en) * | 2019-09-09 | 2021-03-18 | Beam Therapeutics Inc. | Novel nucleobase editors and methods of using same |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112017007765B1 (pt) * | 2014-10-14 | 2023-10-03 | Halozyme, Inc | Composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo |
| AU2020215730A1 (en) * | 2019-01-31 | 2021-07-29 | Beam Therapeutics Inc. | Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors |
| WO2020160517A1 (en) * | 2019-01-31 | 2020-08-06 | Beam Therapeutics Inc. | Nucleobase editors having reduced off-target deamination and methods of using same to modify a nucleobase target sequence |
| WO2021042062A2 (en) * | 2019-08-30 | 2021-03-04 | Joung J Keith | Combinatorial adenine and cytosine dna base editors |
-
2022
- 2022-03-25 EP EP22776767.0A patent/EP4313118A4/en active Pending
- 2022-03-25 JP JP2023558765A patent/JP2024511163A/ja active Pending
- 2022-03-25 KR KR1020237033780A patent/KR20240004276A/ko active Pending
- 2022-03-25 BR BR112023019539A patent/BR112023019539A2/pt unknown
- 2022-03-25 CA CA3214494A patent/CA3214494A1/en active Pending
- 2022-03-25 AU AU2022246176A patent/AU2022246176A1/en active Pending
- 2022-03-25 WO PCT/US2022/022050 patent/WO2022204574A1/en not_active Ceased
-
2023
- 2023-09-26 US US18/474,969 patent/US20240158775A1/en active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021022043A2 (en) * | 2019-07-30 | 2021-02-04 | Pairwise Plants Services, Inc. | Morphogenic regulators and methods of using the same |
| WO2021050571A1 (en) * | 2019-09-09 | 2021-03-18 | Beam Therapeutics Inc. | Novel nucleobase editors and methods of using same |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022204574A1 (en) | 2022-09-29 |
| EP4313118A4 (en) | 2025-06-18 |
| CA3214494A1 (en) | 2022-09-29 |
| KR20240004276A (ko) | 2024-01-11 |
| EP4313118A1 (en) | 2024-02-07 |
| BR112023019539A2 (pt) | 2023-12-05 |
| AU2022246176A1 (en) | 2023-10-05 |
| US20240158775A1 (en) | 2024-05-16 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7717684B2 (ja) | 新規核酸塩基エディター及びその使用方法 | |
| JP2024511163A (ja) | アデノシンデアミナーゼバリアント及びその使用 | |
| JP7679311B2 (ja) | プログラム可能塩基エディターシステムを用いた一塩基多型編集法 | |
| JP7646554B2 (ja) | アルファ-1アンチトリプシン不全を治療するための組成物および方法 | |
| CN114026237B (zh) | 用于治疗1a型糖原贮积病的组成物和方法 | |
| CN114072509A (zh) | 脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法 | |
| CN116497067A (zh) | 治疗血红素病变的组合物和方法 | |
| CA3208612A1 (en) | Recombinant rabies viruses for gene therapy | |
| US20240117345A1 (en) | Compositions and methods for treating transthyretin amyloidosis | |
| CA3198671A1 (en) | Compositions and methods for treating glycogen storage disease type 1a | |
| WO2020231863A1 (en) | Compositions and methods for treating hepatitis b | |
| WO2023086953A1 (en) | Compositions and methods for the treatment of hereditary angioedema (hae) | |
| CN117561074A (zh) | 腺苷脱氨酶变体及其用途 | |
| US20240132868A1 (en) | Compositions and methods for the self-inactivation of base editors | |
| WO2023049935A1 (en) | Compositions and methods for treating hepatitis b virus infection | |
| AU2022343725A1 (en) | Viral guide rna delivery |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20250324 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20250324 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20260106 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20260403 |