JP2022519882A5 - - Google Patents
Info
- Publication number
- JP2022519882A5 JP2022519882A5 JP2021546892A JP2021546892A JP2022519882A5 JP 2022519882 A5 JP2022519882 A5 JP 2022519882A5 JP 2021546892 A JP2021546892 A JP 2021546892A JP 2021546892 A JP2021546892 A JP 2021546892A JP 2022519882 A5 JP2022519882 A5 JP 2022519882A5
- Authority
- JP
- Japan
- Prior art keywords
- adenosine deaminase
- modification
- gsd1a
- base editor
- 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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025017262A JP2025087691A (ja) | 2019-02-13 | 2025-02-05 | 糖原病１ａ型を治療するための組成物および方法 |
Applications Claiming Priority (17)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962805271P | 2019-02-13 | 2019-02-13 | |
| US62/805,271 | 2019-02-13 | ||
| US201962852228P | 2019-05-23 | 2019-05-23 | |
| US201962852224P | 2019-05-23 | 2019-05-23 | |
| US62/852,228 | 2019-05-23 | ||
| US62/852,224 | 2019-05-23 | ||
| US201962876354P | 2019-07-19 | 2019-07-19 | |
| US62/876,354 | 2019-07-19 | ||
| US201962912992P | 2019-10-09 | 2019-10-09 | |
| US62/912,992 | 2019-10-09 | ||
| 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/US2020/018124 WO2020168088A1 (en) | 2019-02-13 | 2020-02-13 | Compositions and methods for treating glycogen storage disease type 1a |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025017262A Division JP2025087691A (ja) | 2019-02-13 | 2025-02-05 | 糖原病１ａ型を治療するための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022519882A JP2022519882A (ja) | 2022-03-25 |
| JP2022519882A5 true JP2022519882A5 (https=) | 2023-02-17 |
| JP7631209B2 JP7631209B2 (ja) | 2025-02-18 |
Family
ID=72045644
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021546892A Active JP7631209B2 (ja) | 2019-02-13 | 2020-02-13 | 糖原病１ａ型を治療するための組成物および方法 |
| JP2025017262A Pending JP2025087691A (ja) | 2019-02-13 | 2025-02-05 | 糖原病１ａ型を治療するための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025017262A Pending JP2025087691A (ja) | 2019-02-13 | 2025-02-05 | 糖原病１ａ型を治療するための組成物および方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20220127594A1 (https=) |
| EP (1) | EP3924478A4 (https=) |
| JP (2) | JP7631209B2 (https=) |
| KR (1) | KR20210129108A (https=) |
| CN (2) | CN120138047A (https=) |
| AU (1) | AU2020221355A1 (https=) |
| CA (1) | CA3128886A1 (https=) |
| WO (1) | WO2020168088A1 (https=) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019178427A1 (en) | 2018-03-14 | 2019-09-19 | 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 |
| WO2019222555A1 (en) | 2018-05-16 | 2019-11-21 | Arbor Biotechnologies, Inc. | Novel crispr-associated systems and components |
| 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 |
| WO2020168088A1 (en) * | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| 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 |
| JP2023505234A (ja) * | 2019-12-04 | 2023-02-08 | アーバー バイオテクノロジーズ， インコーポレイテッド | ヌクレアーゼを含む組成物及びその使用 |
| US12416001B2 (en) * | 2020-02-14 | 2025-09-16 | Ohio State Innovation Foundation | Nucleobase editors and methods of use thereof |
| CA3196831A1 (en) | 2020-09-25 | 2022-03-31 | Beam Therapeutics Inc. | Fratricide resistant modified immune cells and methods of using the same |
| CA3198671A1 (en) * | 2020-10-14 | 2022-04-21 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| EP4504947A4 (en) * | 2022-04-07 | 2026-03-25 | Univ Duke | COMPOSITIONS AND METHODS TO PROMOTE LIVER REGENERATION BY GENE EDITING IN METABOLIC LIVER DISEASE |
| 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 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL310721B2 (en) * | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| BR112018008971A2 (pt) * | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| LT3436077T (lt) * | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| WO2018020323A2 (en) * | 2016-07-25 | 2018-02-01 | Crispr Therapeutics Ag | Materials and methods for treatment of fatty acid disorders |
| CN110214183A (zh) * | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| RU2019130504A (ru) * | 2017-02-28 | 2021-03-30 | Вор Байофарма, Инк. | Композиции и способы ингибирования линиеспецифических белков |
| KR102761791B1 (ko) * | 2017-06-26 | 2025-02-05 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법 |
| US10392616B2 (en) * | 2017-06-30 | 2019-08-27 | Arbor Biotechnologies, Inc. | CRISPR RNA targeting enzymes and systems and uses thereof |
| CN111801345A (zh) * | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| KR20250133482A (ko) * | 2018-05-11 | 2025-09-05 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법 |
| WO2020168088A1 (en) * | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| EP4100519A2 (en) * | 2020-02-05 | 2022-12-14 | The Broad Institute, Inc. | Adenine base editors and uses thereof |
-
2020
- 2020-02-13 WO PCT/US2020/018124 patent/WO2020168088A1/en not_active Ceased
- 2020-02-13 CA CA3128886A patent/CA3128886A1/en active Pending
- 2020-02-13 CN CN202510269938.1A patent/CN120138047A/zh active Pending
- 2020-02-13 KR KR1020217029280A patent/KR20210129108A/ko active Pending
- 2020-02-13 US US17/430,274 patent/US20220127594A1/en active Pending
- 2020-02-13 EP EP20754972.6A patent/EP3924478A4/en active Pending
- 2020-02-13 AU AU2020221355A patent/AU2020221355A1/en active Pending
- 2020-02-13 JP JP2021546892A patent/JP7631209B2/ja active Active
- 2020-02-13 CN CN202080028267.5A patent/CN114026237B/zh active Active
-
2025
- 2025-02-05 JP JP2025017262A patent/JP2025087691A/ja active Pending
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