JP2023526007A5 - - Google Patents
Info
- Publication number
- JP2023526007A5 JP2023526007A5 JP2022568520A JP2022568520A JP2023526007A5 JP 2023526007 A5 JP2023526007 A5 JP 2023526007A5 JP 2022568520 A JP2022568520 A JP 2022568520A JP 2022568520 A JP2022568520 A JP 2022568520A JP 2023526007 A5 JP2023526007 A5 JP 2023526007A5
- Authority
- JP
- Japan
- Prior art keywords
- hbg1
- promoter
- gene editing
- hbg2
- eukaryotic cells
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP20305488.7 | 2020-05-13 | ||
| EP20305488 | 2020-05-13 | ||
| PCT/EP2021/062633 WO2021228944A1 (en) | 2020-05-13 | 2021-05-12 | Base editing approaches for the treatment of betahemoglobinopathies |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023526007A JP2023526007A (ja) | 2023-06-20 |
| JP2023526007A5 true JP2023526007A5 (https=) | 2024-05-01 |
Family
ID=71575241
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022568520A Pending JP2023526007A (ja) | 2020-05-13 | 2021-05-12 | β－ヘモグロビン異常症の処置のための塩基編集アプローチ |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20230279438A1 (https=) |
| EP (1) | EP4150081A1 (https=) |
| JP (1) | JP2023526007A (https=) |
| WO (1) | WO2021228944A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2019265019B2 (en) | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| WO2023144104A1 (en) * | 2022-01-25 | 2023-08-03 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Base editing approaches for the treatment of βeta-thalassemia |
| WO2025017033A1 (en) | 2023-07-17 | 2025-01-23 | Institut National de la Santé et de la Recherche Médicale | Prime editing of the -115 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell |
| WO2025017030A1 (en) | 2023-07-17 | 2025-01-23 | Institut National de la Santé et de la Recherche Médicale | Prime editing of the -200 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell |
| WO2025065716A1 (en) * | 2023-09-29 | 2025-04-03 | Yoltech Therapeutics Co., Ltd | Lipid nanoparticle formulations and methods of use thereof |
| WO2025120664A1 (en) * | 2023-12-04 | 2025-06-12 | Christian Medical College | Strategies for precision editing of homologous regions |
| WO2025147212A1 (en) * | 2024-01-05 | 2025-07-10 | Singapore Health Services Pte Ltd | Induction of fetal haemoglobin using paired cas9 nickases |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2476689T3 (en) | 2007-05-10 | 2016-01-18 | Agilent Technologies Inc | Thiocarbonbeskyttende groups for RNA synthesis |
| US20150166969A1 (en) * | 2012-02-24 | 2015-06-18 | Fred Hutchinson Cancer Research Center | Compositions and methods for the treatment of hemoglobinopathies |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| US20140273230A1 (en) | 2013-03-15 | 2014-09-18 | Sigma-Aldrich Co., Llc | Crispr-based genome modification and regulation |
| CN112301024A (zh) | 2013-03-15 | 2021-02-02 | 通用医疗公司 | 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性 |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| KR102907245B1 (ko) * | 2018-03-14 | 2026-01-05 | 에디타스 메디신, 인코포레이티드 | 혈색소병증 치료를 위한 시스템 및 방법 |
| AU2019265019B2 (en) * | 2018-05-11 | 2025-11-06 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US20220033856A1 (en) * | 2018-09-11 | 2022-02-03 | Université de Paris | Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies |
-
2021
- 2021-05-12 US US17/998,603 patent/US20230279438A1/en active Pending
- 2021-05-12 JP JP2022568520A patent/JP2023526007A/ja active Pending
- 2021-05-12 WO PCT/EP2021/062633 patent/WO2021228944A1/en not_active Ceased
- 2021-05-12 EP EP21724321.1A patent/EP4150081A1/en active Pending
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