JP2025032080A5 - - Google Patents
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- Publication number
- JP2025032080A5 JP2025032080A5 JP2024193416A JP2024193416A JP2025032080A5 JP 2025032080 A5 JP2025032080 A5 JP 2025032080A5 JP 2024193416 A JP2024193416 A JP 2024193416A JP 2024193416 A JP2024193416 A JP 2024193416A JP 2025032080 A5 JP2025032080 A5 JP 2025032080A5
- Authority
- JP
- Japan
- Prior art keywords
- gene
- base editor
- regulatory element
- snp
- amino acid
- 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
Applications Claiming Priority (20)
| 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 | ||
| JP2021546888A 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 |
| 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 Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021546888A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025032080A JP2025032080A (ja) | 2025-03-11 |
| JP2025032080A5 true JP2025032080A5 (https=) | 2025-06-16 |
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 Before (1)
| 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 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230140953A1 (https=) |
| EP (1) | EP3924484A4 (https=) |
| JP (2) | JP7586601B2 (https=) |
| KR (1) | KR20210127206A (https=) |
| CN (2) | CN119280261A (https=) |
| AU (1) | AU2020223306A1 (https=) |
| CA (1) | CA3128876A1 (https=) |
| WO (1) | WO2020168051A1 (https=) |
Families Citing this family (29)
| 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 |
| KR20210055733A (ko) | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| EP3850088A4 (en) | 2018-09-07 | 2023-07-19 | Beam Therapeutics, Inc. | Compositions and methods for improving base editing |
| US20220235347A1 (en) | 2019-02-13 | 2022-07-28 | Beam Therapeutics Inc. | Compositions and methods for treating hemoglobinopathies |
| 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 |
| KR102851101B1 (ko) | 2019-04-15 | 2025-08-27 | 에디진 테라퓨틱스(베이징) 인크. | Rna를 편집하기 위한 방법 및 조성물 |
| PE20220300A1 (es) | 2019-07-12 | 2022-03-09 | Univ Beijing | Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente |
| EP4034138A4 (en) | 2019-09-27 | 2024-07-31 | Beam Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS |
| IL294201A (en) * | 2019-12-30 | 2022-08-01 | Edigene Therapeutics Beijing Inc | A method based on leaper technology to treat mps ih and a preparation |
| CA3170326A1 (en) * | 2020-02-13 | 2021-08-19 | Beam Therapeutics Inc. | Compositions and methods for engraftment of base edited cells |
| 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 |
| AU2021347359A1 (en) | 2020-09-25 | 2023-05-18 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| CA3196425A1 (en) * | 2020-10-21 | 2022-04-28 | Jin Billy Li | A screening platform for adar-recruiting guide rnas |
| WO2022221699A1 (en) * | 2021-04-16 | 2022-10-20 | Beam Therapeutics, Inc. | Genetic modification of hepatocytes |
| WO2022241270A2 (en) * | 2021-05-14 | 2022-11-17 | Beam Therapeutics Inc. | Compositions and methods for treating transthyretin amyloidosis |
| WO2022246266A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| EP4426821A1 (en) * | 2021-11-02 | 2024-09-11 | University of Massachusetts | Nme2cas9 inlaid domain fusion proteins |
| EP4441219A2 (en) * | 2021-12-01 | 2024-10-09 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides for rna editing targeting lrrk2 |
| 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 |
| 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 |
| KR20250067157A (ko) * | 2022-09-08 | 2025-05-14 | 더 유니버시티 코트 오브 더 유니버시티 오브 에딘버그 | 레트 증후군 요법 |
| 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 |
| WO2024233941A1 (en) * | 2023-05-11 | 2024-11-14 | Massachusetts Eye And Ear Infirmary | Base editing approaches to treat abca4-associated stargardt disease |
| WO2024238623A1 (en) * | 2023-05-15 | 2024-11-21 | University Of Georgia Research Foundation, Inc. | Doubly constrained peptides as allosteric inhibitors of leucine rich repeat kinase 2 |
| CN117965505A (zh) * | 2023-06-28 | 2024-05-03 | 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 |
|---|---|---|---|---|
| US20130109048A1 (en) * | 2010-07-09 | 2013-05-02 | Ecole Polytechnique Federale De Lausanne (Epfl) | Method for in-vitro monitoring of neuronal disorders and use thereof |
| ES2753391T3 (es) * | 2014-10-14 | 2020-04-08 | Halozyme Inc | Composiciones de adenosina desaminasa 2 (ADA2), variantes de la misma y métodos de uso de las mismas |
| KR102547316B1 (ko) * | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| EP3526320A1 (en) * | 2016-10-14 | 2019-08-21 | President and Fellows of 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 |
| AU2018290843B2 (en) * | 2017-06-26 | 2025-04-24 | Massachusetts Institute Of Technology | 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 AU AU2020223306A patent/AU2020223306A1/en active Pending
- 2020-02-13 CA CA3128876A patent/CA3128876A1/en active Pending
- 2020-02-13 CN CN202411269394.0A patent/CN119280261A/zh active Pending
- 2020-02-13 CN CN202080028186.5A patent/CN114040970B/zh active Active
- 2020-02-13 WO PCT/US2020/018073 patent/WO2020168051A1/en not_active Ceased
- 2020-02-13 EP EP20756724.9A patent/EP3924484A4/en active Pending
- 2020-02-13 JP JP2021546888A patent/JP7586601B2/ja active Active
- 2020-02-13 US US17/430,672 patent/US20230140953A1/en active Pending
- 2020-02-13 KR KR1020217029268A patent/KR20210127206A/ko active Pending
-
2024
- 2024-11-05 JP JP2024193416A patent/JP2025032080A/ja active Pending
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