CA3129158A1 - Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence - Google Patents
Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence Download PDFInfo
- Publication number
- CA3129158A1 CA3129158A1 CA3129158A CA3129158A CA3129158A1 CA 3129158 A1 CA3129158 A1 CA 3129158A1 CA 3129158 A CA3129158 A CA 3129158A CA 3129158 A CA3129158 A CA 3129158A CA 3129158 A1 CA3129158 A1 CA 3129158A1
- Authority
- CA
- Canada
- Prior art keywords
- fusion protein
- tada
- adenosine deaminase
- domain
- cas9
- 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
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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
- 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
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
-
- 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
- C12N15/11—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)
- C12N9/16—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
- 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
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
- C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
-
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- Crystallography & Structural Chemistry (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Plant Substances (AREA)
Applications Claiming Priority (33)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962805271P | 2019-02-13 | 2019-02-13 | |
| US201962805238P | 2019-02-13 | 2019-02-13 | |
| US201962805277P | 2019-02-13 | 2019-02-13 | |
| US62/805,277 | 2019-02-13 | ||
| US62/805,238 | 2019-02-13 | ||
| US62/805,271 | 2019-02-13 | ||
| US201962852224P | 2019-05-23 | 2019-05-23 | |
| US201962852228P | 2019-05-23 | 2019-05-23 | |
| US62/852,228 | 2019-05-23 | ||
| US62/852,224 | 2019-05-23 | ||
| US201962873140P | 2019-07-11 | 2019-07-11 | |
| US201962873144P | 2019-07-11 | 2019-07-11 | |
| US201962873138P | 2019-07-11 | 2019-07-11 | |
| US62/873,138 | 2019-07-11 | ||
| US62/873,140 | 2019-07-11 | ||
| US62/873,144 | 2019-07-11 | ||
| US201962876354P | 2019-07-19 | 2019-07-19 | |
| US62/876,354 | 2019-07-19 | ||
| US201962888867P | 2019-08-19 | 2019-08-19 | |
| US62/888,867 | 2019-08-19 | ||
| US201962912992P | 2019-10-09 | 2019-10-09 | |
| US62/912,992 | 2019-10-09 | ||
| US201962931747P | 2019-11-06 | 2019-11-06 | |
| US201962931722P | 2019-11-06 | 2019-11-06 | |
| US62/931,722 | 2019-11-06 | ||
| US62/931,747 | 2019-11-06 | ||
| US201962941523P | 2019-11-27 | 2019-11-27 | |
| US201962941569P | 2019-11-27 | 2019-11-27 | |
| US62/941,569 | 2019-11-27 | ||
| US62/941,523 | 2019-11-27 | ||
| US202062966526P | 2020-01-27 | 2020-01-27 | |
| US62/966,526 | 2020-01-27 | ||
| PCT/US2020/018192 WO2020168132A1 (en) | 2019-02-13 | 2020-02-13 | Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3129158A1 true CA3129158A1 (en) | 2020-08-20 |
Family
ID=72043868
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3129158A Pending CA3129158A1 (en) | 2019-02-13 | 2020-02-13 | Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US20220170027A1 (https=) |
| EP (1) | EP3924479A4 (https=) |
| JP (2) | JP7693552B2 (https=) |
| KR (1) | KR20210139265A (https=) |
| CN (1) | CN114072496A (https=) |
| AU (1) | AU2020221366A1 (https=) |
| CA (1) | CA3129158A1 (https=) |
| WO (1) | WO2020168132A1 (https=) |
Families Citing this family (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL3765615T3 (pl) | 2018-03-14 | 2023-11-13 | Arbor Biotechnologies, Inc. | Nowe enzymy i układy crispr ukierunkowane na dna |
| 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 |
| US12522812B2 (en) | 2018-05-16 | 2026-01-13 | Arbor Biotechnologies, Inc. | CRISPR-associated systems and components |
| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| KR20210055733A (ko) | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| 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 |
| KR20210139265A (ko) * | 2019-02-13 | 2021-11-22 | 빔 테라퓨틱스, 인크. | 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기 및 이의 사용 방법 |
| US20220235347A1 (en) | 2019-02-13 | 2022-07-28 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| US12569438B2 (en) | 2019-07-29 | 2026-03-10 | Georgia Tech Research Corporation | Nanomaterials containing constrained lipids and uses thereof |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| US12435035B2 (en) | 2020-01-09 | 2025-10-07 | Guide Therapeutics, Llc | Nanomaterials |
| CA3170326A1 (en) * | 2020-02-13 | 2021-08-19 | Beam Therapeutics Inc. | Compositions and methods for engraftment of base edited cells |
| KR20230009379A (ko) * | 2020-03-31 | 2023-01-17 | 아버 바이오테크놀로지스, 인크. | Cas12i2 변이체 폴리펩타이드를 포함하는 조성물 및 이의 용도 |
| CA3194019A1 (en) * | 2020-09-04 | 2022-03-10 | National University Corporation Kobe University | Miniaturized cytidine deaminase-containing complex for modifying double-stranded dna |
| AU2021347359A1 (en) | 2020-09-25 | 2023-05-18 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| US20240018550A1 (en) * | 2020-12-01 | 2024-01-18 | Iucf-Hyu (Industry-University Cooperation Foundation Hanyang University) | Adenine base editor having increased thymine-cytosine sequence-specific cytosine editing activity, and use thereof |
| WO2022119294A1 (ko) * | 2020-12-01 | 2022-06-09 | 한양대학교 산학협력단 | 사이토신 교정 활성이 제거된 아데닌 염기교정 유전자가위 및 이의 용도 |
| KR20230131863A (ko) | 2021-01-20 | 2023-09-14 | 빔 테라퓨틱스, 인크. | 나노물질 |
| IL303845A (en) | 2021-01-20 | 2023-08-01 | Beam Therapeutics Inc | Nanomaterials include a degradable property |
| CN115704015B (zh) * | 2021-08-12 | 2025-08-12 | 清华大学 | 基于腺嘌呤和胞嘧啶双碱基编辑器的靶向诱变系统 |
| CN115772512B (zh) * | 2021-09-07 | 2025-12-23 | 华东师范大学 | 腺嘌呤脱氨酶、包含其的腺嘌呤碱基编辑器及其应用 |
| WO2023052366A1 (en) | 2021-09-28 | 2023-04-06 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Base editing approaches for the treatment of beta-hemoglobinopathies |
| CN119286831A (zh) * | 2021-10-21 | 2025-01-10 | 复旦大学 | 碱基编辑器及其构建方法与应用 |
| WO2023099591A1 (en) | 2021-12-01 | 2023-06-08 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer |
| EP4452925A1 (en) | 2021-12-20 | 2024-10-30 | Beam Therapeutics Inc. | Nanomaterial comprising diamines |
| EP4452942A1 (en) | 2021-12-20 | 2024-10-30 | Beam Therapeutics Inc. | Nanomaterials comprising disulfides |
| EP4452930A1 (en) | 2021-12-20 | 2024-10-30 | Beam Therapeutics Inc. | Ionizable amine and ester lipids and lipid nanoparticles |
| CA3241488A1 (en) | 2021-12-20 | 2023-06-29 | Beam Therapeutics Inc. | Ionizable amine lipids and lipid nanoparticles |
| WO2023121971A1 (en) | 2021-12-20 | 2023-06-29 | Beam Therapeutics Inc. | Nanomaterials comprising tetravalent lipid compounds |
| US20250161492A1 (en) | 2022-01-25 | 2025-05-22 | Institut National de la Santé et de la Recherche Médicale | Base editing approaches for the treatment of beta-thalassemia |
| WO2023169454A1 (zh) * | 2022-03-08 | 2023-09-14 | 中国科学院遗传与发育生物学研究所 | 腺嘌呤脱氨酶及其在碱基编辑中的用途 |
| CN117925585A (zh) * | 2022-04-07 | 2024-04-26 | 尧唐(上海)生物科技有限公司 | 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途 |
| CN120456933A (zh) | 2022-04-28 | 2025-08-08 | 布罗德研究所股份有限公司 | 编码碱基编辑器的aav载体及其用途 |
| CN114686456B (zh) * | 2022-05-10 | 2023-02-17 | 中山大学 | 基于双分子脱氨酶互补的碱基编辑系统及其应用 |
| EP4522647A1 (en) | 2022-05-10 | 2025-03-19 | Institut National de la Santé et de la Recherche Médicale | Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from beta-thalassemia |
| WO2023217904A1 (en) | 2022-05-10 | 2023-11-16 | Institut National de la Santé et de la Recherche Médicale | Syncitin-1 fusion proteins and uses thereof for cargo delivery into target cells |
| CN119923466A (zh) * | 2022-06-15 | 2025-05-02 | 比姆医疗股份有限公司 | 用于减少补体激活的组合物和方法 |
| EP4543469A2 (en) * | 2022-06-27 | 2025-04-30 | Beam Therapeutics Inc. | Compositions and methods for non-genotoxic cell conditioning |
| WO2024019936A1 (en) | 2022-07-20 | 2024-01-25 | Beam Therapeutics Inc. | Nanomaterials comprising triols |
| EP4558633A1 (en) | 2022-07-22 | 2025-05-28 | Institut National de la Santé et de la Recherche Médicale | Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from beta-thalassemia |
| WO2024086596A1 (en) * | 2022-10-18 | 2024-04-25 | 4M Genomics Inc. | Polypeptide fusions or conjugates for gene editing |
| JP2026506554A (ja) | 2023-02-06 | 2026-02-25 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | 多重塩基編集による遺伝子改変造血幹細胞の濃縮 |
| EP4705461A2 (en) * | 2023-05-03 | 2026-03-11 | Beam Therapeutics Inc. | Compositions and methods for treating stargardt disease |
| WO2024230760A1 (zh) * | 2023-05-09 | 2024-11-14 | 北京齐禾生科生物科技有限公司 | 一种可作用于dna的腺苷脱氨酶及其应用 |
| CN118871578A (zh) | 2023-05-24 | 2024-10-29 | 锐正基因(苏州)有限公司 | 用于碱基编辑的脱氨酶及其变体 |
| WO2025038821A1 (en) * | 2023-08-16 | 2025-02-20 | Chroma Medicine, Inc. | Methods and compositions for multiplex epigenetic editing |
| WO2025059520A1 (en) * | 2023-09-15 | 2025-03-20 | Beam Therapeutics Inc. | Compositions and methods for base editing a phenylalanine hydroxylase polynucleotide |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| 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 |
| CN117568313B (zh) * | 2024-01-15 | 2024-04-26 | 上海贝斯昂科生物科技有限公司 | 基因编辑组合物及其用途 |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025202473A1 (en) * | 2024-03-28 | 2025-10-02 | Revvity Discovery Limited | A nucleic acid deaminase, a base editor and uses thereof |
| US20250332260A1 (en) | 2024-04-10 | 2025-10-30 | Garuda Therapeutics, Inc. | Immune compatible cells for allogeneic cell therapies to cover global, ethnic, or disease-specific populations |
| WO2025228273A1 (zh) * | 2024-04-28 | 2025-11-06 | 广州瑞风生物科技有限公司 | 脱氨酶、单碱基编辑器及其应用 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| CA2982966C (en) * | 2015-04-24 | 2024-02-20 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| WO2017048969A1 (en) * | 2015-09-17 | 2017-03-23 | The Regents Of The University Of California | Variant cas9 polypeptides comprising internal insertions |
| KR102547316B1 (ko) | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| WO2018089664A1 (en) * | 2016-11-11 | 2018-05-17 | The Regents Of The University Of California | Variant rna-guided polypeptides and methods of use |
| EP3625342B1 (en) * | 2017-05-18 | 2022-08-24 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| US11168322B2 (en) * | 2017-06-30 | 2021-11-09 | Arbor Biotechnologies, Inc. | CRISPR RNA targeting enzymes and systems and uses thereof |
| US11946040B2 (en) * | 2019-02-04 | 2024-04-02 | The General Hospital Corporation | Adenine DNA base editor variants with reduced off-target RNA editing |
| US20220235347A1 (en) * | 2019-02-13 | 2022-07-28 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| KR20210139265A (ko) * | 2019-02-13 | 2021-11-22 | 빔 테라퓨틱스, 인크. | 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기 및 이의 사용 방법 |
| KR20210126680A (ko) * | 2019-02-13 | 2021-10-20 | 빔 테라퓨틱스, 인크. | 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법 |
| US20220387622A1 (en) * | 2019-05-21 | 2022-12-08 | Beam Therapeutics Inc. | Methods of editing a single nucleotide polymorphism using programmable base editor systems |
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2020
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- 2020-02-13 EP EP20755422.1A patent/EP3924479A4/en active Pending
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| EP3924479A4 (en) | 2023-01-25 |
| CN114072496A (zh) | 2022-02-18 |
| US20220170027A1 (en) | 2022-06-02 |
| AU2020221366A1 (en) | 2021-08-26 |
| EP3924479A1 (en) | 2021-12-22 |
| US11155803B2 (en) | 2021-10-26 |
| US20210130805A1 (en) | 2021-05-06 |
| JP2022520081A (ja) | 2022-03-28 |
| WO2020168132A1 (en) | 2020-08-20 |
| JP2025143279A (ja) | 2025-10-01 |
| KR20210139265A (ko) | 2021-11-22 |
| JP7693552B2 (ja) | 2025-06-17 |
| WO2020168132A9 (en) | 2020-10-08 |
| US20240132867A1 (en) | 2024-04-25 |
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