JP2022520080A5 - - Google Patents
Info
- Publication number
- JP2022520080A5 JP2022520080A5 JP2021546888A JP2021546888A JP2022520080A5 JP 2022520080 A5 JP2022520080 A5 JP 2022520080A5 JP 2021546888 A JP2021546888 A JP 2021546888A JP 2021546888 A JP2021546888 A JP 2021546888A JP 2022520080 A5 JP2022520080 A5 JP 2022520080A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- gene
- adenosine
- base editor
- 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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024193416A JP2025032080A (ja) | 2019-02-13 | 2024-11-05 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
Applications Claiming Priority (19)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962805271P | 2019-02-13 | 2019-02-13 | |
| US62/805,271 | 2019-02-13 | ||
| US201962850919P | 2019-05-21 | 2019-05-21 | |
| US62/850,919 | 2019-05-21 | ||
| 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 | ||
| US201962873138P | 2019-07-11 | 2019-07-11 | |
| US62/873,138 | 2019-07-11 | ||
| US201962888867P | 2019-08-19 | 2019-08-19 | |
| US62/888,867 | 2019-08-19 | ||
| US201962931722P | 2019-11-06 | 2019-11-06 | |
| US62/931,722 | 2019-11-06 | ||
| US201962941569P | 2019-11-27 | 2019-11-27 | |
| US62/941,569 | 2019-11-27 | ||
| US202062966526P | 2020-01-27 | 2020-01-27 | |
| US62/966,526 | 2020-01-27 | ||
| PCT/US2020/018073 WO2020168051A1 (en) | 2019-02-13 | 2020-02-13 | Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024193416A Division JP2025032080A (ja) | 2019-02-13 | 2024-11-05 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022520080A JP2022520080A (ja) | 2022-03-28 |
| JP2022520080A5 true JP2022520080A5 (enExample) | 2023-02-21 |
| JP7586601B2 JP7586601B2 (ja) | 2024-11-19 |
Family
ID=72045106
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021546888A Active JP7586601B2 (ja) | 2019-02-13 | 2020-02-13 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
| JP2024193416A Pending JP2025032080A (ja) | 2019-02-13 | 2024-11-05 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024193416A Pending JP2025032080A (ja) | 2019-02-13 | 2024-11-05 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230140953A1 (enExample) |
| EP (1) | EP3924484A4 (enExample) |
| JP (2) | JP7586601B2 (enExample) |
| KR (1) | KR20210127206A (enExample) |
| CN (2) | CN119280261A (enExample) |
| AU (1) | AU2020223306A1 (enExample) |
| CA (1) | CA3128876A1 (enExample) |
| WO (1) | WO2020168051A1 (enExample) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2019236210B2 (en) | 2018-03-14 | 2025-07-03 | Arbor Biotechnologies, Inc. | Novel CRISPR DNA targeting enzymes and systems |
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US12522812B2 (en) | 2018-05-16 | 2026-01-13 | Arbor Biotechnologies, Inc. | CRISPR-associated systems and components |
| AU2019336245B2 (en) | 2018-09-07 | 2026-02-26 | Beam Therapeutics Inc. | Compositions and methods for delivering a nucleobase editing system |
| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| CN114040977B (zh) | 2019-01-31 | 2024-09-03 | 比姆医疗股份有限公司 | 非靶脱氨反应减低的核碱基编辑器和用于定性核碱基编辑器的测定 |
| CA3236512C (en) | 2019-02-13 | 2026-02-24 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| AU2020223314A1 (en) | 2019-02-13 | 2021-08-12 | Beam Therapeutics Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| SG11202108346WA (en) | 2019-02-13 | 2021-08-30 | Beam Therapeutics Inc | Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence |
| PH12021552613A1 (en) | 2019-04-15 | 2022-06-27 | Edigene Therapeutics Beijing Inc | METHODS AND COMPOSITIONS FOR EDITING RNAs |
| CN114269919B (zh) | 2019-07-12 | 2023-08-18 | 北京大学 | 采用工程化rna利用内源adar进行靶向的rna编辑 |
| CN115279398B (zh) | 2019-09-27 | 2025-07-04 | 比姆医疗股份有限公司 | 治疗液态癌症的组合物和方法 |
| PH12022551595A1 (en) * | 2019-12-30 | 2023-11-29 | Edigene Therapeutics Beijing Inc | Leaper technology based method for treating mps ih and composition |
| BR112022015969A2 (pt) * | 2020-02-13 | 2022-10-25 | Beam Therapeutics Inc | Composições e métodos para enxerto de células editadas de base |
| WO2022027035A1 (en) * | 2020-07-27 | 2022-02-03 | The Children's Hospital Of Philadelphia | In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders |
| US12576151B2 (en) | 2020-09-25 | 2026-03-17 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| CN116783296A (zh) * | 2020-10-21 | 2023-09-19 | 小利兰·斯坦福大学托管委员会 | 用于募集adar的向导rna的筛选平台 |
| WO2022221699A1 (en) * | 2021-04-16 | 2022-10-20 | Beam Therapeutics, Inc. | Genetic modification of hepatocytes |
| CN117729931A (zh) * | 2021-05-14 | 2024-03-19 | 比姆医疗股份有限公司 | 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法 |
| CA3220738A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| WO2023081070A1 (en) * | 2021-11-02 | 2023-05-11 | University Of Massachusetts | Nme2Cas9 INLAID DOMAIN FUSION PROTEINS |
| WO2023102449A2 (en) * | 2021-12-01 | 2023-06-08 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides |
| CN117187220A (zh) * | 2022-03-08 | 2023-12-08 | 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 |
| 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 |
| JP2025530183A (ja) * | 2022-09-08 | 2025-09-11 | ザ ユニヴァーシティ コート オブ ザ ユニヴァーシティ オブ エディンバラ | レット症候群治療法 |
| CN121152875A (zh) * | 2023-05-09 | 2025-12-16 | 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 |
| WO2024233941A1 (en) * | 2023-05-11 | 2024-11-14 | Massachusetts Eye And Ear Infirmary | Base editing approaches to treat abca4-associated stargardt disease |
| EP4713348A1 (en) * | 2023-05-15 | 2026-03-25 | University of Georgia Research Foundation, Inc. | Doubly constrained peptides as allosteric inhibitors of leucine rich repeat kinase 2 |
| CN116836962B (zh) * | 2023-06-28 | 2024-04-05 | 微光基因(苏州)有限公司 | 工程化的腺苷脱氨酶及碱基编辑器 |
| CN120536418A (zh) * | 2024-02-26 | 2025-08-26 | 尧唐(上海)生物科技有限公司 | 一种脱氨酶变体、包含其的碱基编辑器及其应用 |
| WO2025184515A1 (en) * | 2024-02-29 | 2025-09-04 | Vesigen, Inc. | Arrdc1-mediated microvesicle-based delivery of therapeutic agents to cells and tissues of the eye |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2591356A1 (en) * | 2010-07-09 | 2013-05-15 | Ecole Polytechnique Fédérale de Lausanne (EPFL) | Method for in-vitro monitoring of neuronal disorders and use thereof |
| 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 |
| KR20250103795A (ko) * | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| SG11201903089RA (en) * | 2016-10-14 | 2019-05-30 | Harvard College | Aav delivery of nucleobase editors |
| WO2018213708A1 (en) * | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| CA3064601A1 (en) * | 2017-06-26 | 2019-01-03 | President And Fellows Of Harvard College | Crispr/cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing |
| CN109306361B (zh) * | 2018-02-11 | 2022-06-28 | 华东师范大学 | 一种新的a/t到g/c碱基定点转换的基因编辑系统 |
| CN109295186B (zh) * | 2018-09-30 | 2023-10-03 | 中山大学 | 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用 |
-
2020
- 2020-02-13 CN CN202411269394.0A patent/CN119280261A/zh active Pending
- 2020-02-13 CA CA3128876A patent/CA3128876A1/en active Pending
- 2020-02-13 AU AU2020223306A patent/AU2020223306A1/en active Pending
- 2020-02-13 WO PCT/US2020/018073 patent/WO2020168051A1/en not_active Ceased
- 2020-02-13 KR KR1020217029268A patent/KR20210127206A/ko active Pending
- 2020-02-13 CN CN202080028186.5A patent/CN114040970B/zh active Active
- 2020-02-13 US US17/430,672 patent/US20230140953A1/en active Pending
- 2020-02-13 EP EP20756724.9A patent/EP3924484A4/en active Pending
- 2020-02-13 JP JP2021546888A patent/JP7586601B2/ja active Active
-
2024
- 2024-11-05 JP JP2024193416A patent/JP2025032080A/ja active Pending
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