JP2023552379A5 - - Google Patents
Info
- Publication number
- JP2023552379A5 JP2023552379A5 JP2023533861A JP2023533861A JP2023552379A5 JP 2023552379 A5 JP2023552379 A5 JP 2023552379A5 JP 2023533861 A JP2023533861 A JP 2023533861A JP 2023533861 A JP2023533861 A JP 2023533861A JP 2023552379 A5 JP2023552379 A5 JP 2023552379A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- grna
- bcl11a
- seq
- protein
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063120885P | 2020-12-03 | 2020-12-03 | |
| US63/120,885 | 2020-12-03 | ||
| PCT/US2021/061672 WO2022120094A2 (en) | 2020-12-03 | 2021-12-02 | Compositions and methods for the targeting of bcl11a |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023552379A JP2023552379A (ja) | 2023-12-15 |
| JP2023552379A5 true JP2023552379A5 (enExample) | 2024-12-06 |
| JP7857026B2 JP7857026B2 (ja) | 2026-05-12 |
Family
ID=81854906
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023533861A Active JP7857026B2 (ja) | 2020-12-03 | 2021-12-02 | Ｂｃｌ１１ａの標的化のための組成物及び方法 |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20240026386A1 (enExample) |
| EP (1) | EP4255502A4 (enExample) |
| JP (1) | JP7857026B2 (enExample) |
| KR (1) | KR20230129230A (enExample) |
| CN (1) | CN116801913A (enExample) |
| AU (1) | AU2021391783A1 (enExample) |
| BR (1) | BR112023010717A2 (enExample) |
| CA (1) | CA3200815A1 (enExample) |
| CL (2) | CL2023001593A1 (enExample) |
| CO (1) | CO2023007900A2 (enExample) |
| GB (1) | GB2616795B (enExample) |
| IL (1) | IL303359A (enExample) |
| MX (1) | MX2023006565A (enExample) |
| PE (1) | PE20231177A1 (enExample) |
| WO (1) | WO2022120094A2 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2021392719B2 (en) | 2020-12-03 | 2025-08-14 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| WO2022261150A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| WO2023235888A2 (en) | 2022-06-03 | 2023-12-07 | Scribe Therapeutics Inc. | COMPOSITIONS AND METHODS FOR CpG DEPLETION |
| EP4536819A1 (en) | 2022-06-07 | 2025-04-16 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| CN119998446A (zh) | 2022-06-07 | 2025-05-13 | 斯克里贝治疗公司 | 用于靶向pcsk9的组合物和方法 |
| WO2023240027A1 (en) | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Particle delivery systems |
| EP4536286A1 (en) | 2022-06-08 | 2025-04-16 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| CN121759434A (zh) * | 2024-09-30 | 2026-03-31 | 尧唐(上海)生物科技有限公司 | 治疗血红蛋白病的组合物及其应用 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2018007987A (es) * | 2015-12-28 | 2019-01-10 | Novartis Ag | Composiciones y metodos para el tratamiento de hemoglobinopatias. |
| JP6974349B2 (ja) * | 2016-04-18 | 2021-12-01 | クリスパー セラピューティクス アクチェンゲゼルシャフト | ヘモグロビン異常症の処置のための材料及び方法 |
| JP2019532644A (ja) | 2016-09-30 | 2019-11-14 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | Ｒｎａ誘導型核酸修飾酵素及びその使用方法 |
| US9963719B1 (en) * | 2016-12-05 | 2018-05-08 | Editas Medicine, Inc. | Systems and methods for one-shot guide RNA (ogRNA) targeting of endogenous and source DNA |
| US20190367924A1 (en) | 2017-02-17 | 2019-12-05 | Temple University - Of The Commonwealth System Of Higher Education | Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5 |
| CN109722414A (zh) * | 2017-10-27 | 2019-05-07 | 博雅辑因（北京）生物科技有限公司 | 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基 |
| US12522811B2 (en) | 2018-05-01 | 2026-01-13 | The Children's Medical Center Corporation | Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9 |
| CA3106035A1 (en) * | 2018-08-07 | 2020-02-13 | The Broad Institute, Inc. | Cas12b enzymes and systems |
| US12551560B2 (en) * | 2019-09-09 | 2026-02-17 | Scribe Therapeutics Inc. | Compositions and methods for use in immunotherapy |
-
2021
- 2021-12-02 EP EP21901487.5A patent/EP4255502A4/en active Pending
- 2021-12-02 WO PCT/US2021/061672 patent/WO2022120094A2/en not_active Ceased
- 2021-12-02 MX MX2023006565A patent/MX2023006565A/es unknown
- 2021-12-02 AU AU2021391783A patent/AU2021391783A1/en active Pending
- 2021-12-02 JP JP2023533861A patent/JP7857026B2/ja active Active
- 2021-12-02 KR KR1020237022391A patent/KR20230129230A/ko active Pending
- 2021-12-02 CA CA3200815A patent/CA3200815A1/en active Pending
- 2021-12-02 US US18/255,172 patent/US20240026386A1/en active Pending
- 2021-12-02 BR BR112023010717A patent/BR112023010717A2/pt unknown
- 2021-12-02 IL IL303359A patent/IL303359A/en unknown
- 2021-12-02 PE PE2023001811A patent/PE20231177A1/es unknown
- 2021-12-02 GB GB2309871.8A patent/GB2616795B/en active Active
- 2021-12-02 CN CN202180092086.3A patent/CN116801913A/zh active Pending
-
2023
- 2023-06-02 CL CL2023001593A patent/CL2023001593A1/es unknown
- 2023-06-16 CO CONC2023/0007900A patent/CO2023007900A2/es unknown
-
2024
- 2024-05-27 CL CL2024001577A patent/CL2024001577A1/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US11965174B2 (en) | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof | |
| GB2616795A (en) | Compositions and methods for the targeting of BCL11A | |
| US12529051B2 (en) | Methods of treating amyotrophic lateral sclerosis (ALS) | |
| CA3116452A1 (en) | Genome editing methods and constructs | |
| CN111718420B (zh) | 一种用于基因治疗的融合蛋白及其应用 | |
| CN116949012B (zh) | 一种融合蛋白及其应用 | |
| EP3943512A1 (en) | Fusion protein for enhancing gene editing and use thereof | |
| AU2020262281B2 (en) | Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof | |
| US20190256867A1 (en) | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof | |
| WO2025119295A1 (zh) | 脱氨酶、融合蛋白、核酸、药物组合物及其应用 | |
| JP2025515030A5 (enExample) | ||
| CN114072518B (zh) | 用于治疗地中海贫血或镰状细胞病的方法和组合物 | |
| CN117561331A (zh) | 用于产生经基因修饰的原代细胞的方法和组合物 | |
| WO2025056035A1 (en) | Compositions and methods for treating duchenne muscular dystrophy | |
| EP3952921A1 (en) | Correction of beta-thalassemia phenotype by genetically engineered hematopoietic stem cell | |
| CN120665952A (zh) | 一种修复肌萎缩侧索硬化致病突变基因的编辑系统 | |
| EP4532720A2 (en) | Crispr interference therapeutics for c9orf72 repeat expansion disease | |
| WO2026025441A1 (zh) | 一种用于显性遗传的视网膜色素变性基因编辑药物载体 | |
| HK40049414A (en) | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof | |
| WO2020187272A1 (zh) | 一种用于基因治疗的融合蛋白及其应用 | |
| HK40069063A (en) | Methods and compositions for treating thalassemia or sickle cell disease | |
| HK1240977B (en) | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof | |
| HK1240977A1 (en) | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |