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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2026028532A JP2026124805A (ja) | 2020-05-13 | 2026-02-25 | 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 |
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2026028532A Division JP2026124805A (ja) | 2020-05-13 | 2026-02-25 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023526007A JP2023526007A (ja) | 2023-06-20 |
| JP2023526007A5 true JP2023526007A5 (enExample) | 2024-05-01 |
| JP7856581B2 JP7856581B2 (ja) | 2026-05-11 |
Family
ID=71575241
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022568520A Active JP7856581B2 (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 |
| JP2026028532A Pending JP2026124805A (ja) | 2020-05-13 | 2026-02-25 | 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2026028532A Pending JP2026124805A (ja) | 2020-05-13 | 2026-02-25 | 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 (enExample) |
| EP (1) | EP4150081A1 (enExample) |
| JP (2) | JP7856581B2 (enExample) |
| WO (1) | WO2021228944A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019217942A1 (en) | 2018-05-11 | 2019-11-14 | 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 |
| 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 |
| EP4747377A1 (en) | 2023-07-17 | 2026-05-27 | 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 |
| CN122341362A (zh) * | 2023-09-29 | 2026-07-03 | 上海尧唐生物科技股份有限公司 | 脂质纳米颗粒制剂及其使用方法 |
| 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 |
| WO2026069358A1 (en) * | 2024-09-24 | 2026-04-02 | Christian Medical College | Base editing methods and compositions |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2476689T3 (pl) | 2007-05-10 | 2016-04-29 | Agilent Technologies Inc | Tiowęglowe grupy zabezpieczające do syntezy RNA |
| EP2836226B1 (en) * | 2012-02-24 | 2017-06-28 | Fred Hutchinson Cancer Research Center | Compositions and methods for the treatment of hemoglobinopathies |
| ES2728782T3 (es) | 2012-05-25 | 2019-10-28 | Univ California | Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| CN105408483A (zh) | 2013-03-15 | 2016-03-16 | 通用医疗公司 | 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向 |
| US20140273230A1 (en) | 2013-03-15 | 2014-09-18 | Sigma-Aldrich Co., Llc | Crispr-based genome modification and regulation |
| IL277234B2 (en) * | 2018-03-14 | 2026-03-01 | Editas Medicine Inc | Systems and methods for treating hemoglobin diseases |
| WO2019217942A1 (en) * | 2018-05-11 | 2019-11-14 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| EP3850094A1 (en) * | 2018-09-11 | 2021-07-21 | INSERM (Institut National de la Santé et de la Recherche Médicale) | 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 EP EP21724321.1A patent/EP4150081A1/en active Pending
- 2021-05-12 JP JP2022568520A patent/JP7856581B2/ja active Active
- 2021-05-12 WO PCT/EP2021/062633 patent/WO2021228944A1/en not_active Ceased
-
2026
- 2026-02-25 JP JP2026028532A patent/JP2026124805A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US12385070B2 (en) | Homology directed repair compositions for the treatment of hemoglobinopathies | |
| Liu et al. | Expanding the CRISPR toolbox in zebrafish for studying development and disease | |
| JP2025026840A5 (enExample) | ||
| Demirci et al. | CRISPR/Cas9 for sickle cell disease: applications, future possibilities, and challenges | |
| AU2018215726B2 (en) | Compositions and methods for the treatment of hemoglobinopathies | |
| RU2650811C2 (ru) | Композиции и способы лечения гемоглобинопатии | |
| KR102796744B1 (ko) | 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물 | |
| JP6873917B2 (ja) | ヌクレアーゼ介在性遺伝子発現調節 | |
| US20190184035A1 (en) | Bcl11a homing endonuclease variants, compositions, and methods of use | |
| US20190345450A1 (en) | Strategies to assess and/or produce cell populations with predictive engraftment potential | |
| JP2019500043A5 (enExample) | ||
| US20220228142A1 (en) | Compositions and methods for editing beta-globin for treatment of hemaglobinopathies | |
| IL308706A (en) | Compositions and methods for the treatment of hemoglobinopathies | |
| Maganti et al. | Persistence of CRISPR/Cas9 gene edited hematopoietic stem cells following transplantation: A systematic review and meta-analysis of preclinical studies | |
| EP3765617A1 (en) | Systems and methods for the treatment of hemoglobinopathies | |
| WO2019173654A2 (en) | Systems and methods for the treatment of hemoglobinopathies | |
| JP2024534720A5 (enExample) | ||
| Katta et al. | Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease | |
| JP2021521838A (ja) | ブルトン型チロシンキナーゼに対するtalenベースのおよびcrispr/casベースのゲノム編集 | |
| Xu et al. | In vivo genome editing of human hematopoietic stem cells for treatment of blood disorders by mRNA delivery | |
| DeWitt et al. | Efficient correction of the sickle mutation in human hematopoietic stem cells using a Cas9 ribonucleoprotein complex | |
| JP2024543966A5 (enExample) | ||
| WO2025189017A1 (en) | Methods for genetically engineering mammalian cells using serial delivery | |
| Jacob | IMPROVING TARGETED GENE EDITING IN HEMATOPOIETIC STEM CELLS FOR CLINICAL TRANSLATION | |
| Howe et al. | Barriers to Effective Genome Editing of Haematopoietic Stem Cells |