JP2019508051A5 - - Google Patents
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- JP2019508051A5 JP2019508051A5 JP2018548318A JP2018548318A JP2019508051A5 JP 2019508051 A5 JP2019508051 A5 JP 2019508051A5 JP 2018548318 A JP2018548318 A JP 2018548318A JP 2018548318 A JP2018548318 A JP 2018548318A JP 2019508051 A5 JP2019508051 A5 JP 2019508051A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide sequence
- cells
- cell
- grna
- targeting domain
- 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
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- 210000004027 cell Anatomy 0.000 claims 13
- 239000002773 nucleotide Substances 0.000 claims 12
- 125000003729 nucleotide group Chemical group 0.000 claims 12
- 108020005004 Guide RNA Proteins 0.000 claims 9
- 238000000034 method Methods 0.000 claims 9
- 102000004389 Ribonucleoproteins Human genes 0.000 claims 7
- 108010081734 Ribonucleoproteins Proteins 0.000 claims 7
- 210000005260 human cell Anatomy 0.000 claims 7
- 230000000295 complement effect Effects 0.000 claims 6
- 238000012986 modification Methods 0.000 claims 6
- 230000004048 modification Effects 0.000 claims 6
- 230000008685 targeting Effects 0.000 claims 6
- 238000010362 genome editing Methods 0.000 claims 5
- 210000003743 erythrocyte Anatomy 0.000 claims 4
- 210000003924 normoblast Anatomy 0.000 claims 4
- 239000002243 precursor Substances 0.000 claims 4
- 108091033409 CRISPR Proteins 0.000 claims 3
- 102100038614 Hemoglobin subunit gamma-1 Human genes 0.000 claims 3
- 102100038617 Hemoglobin subunit gamma-2 Human genes 0.000 claims 3
- 101001031977 Homo sapiens Hemoglobin subunit gamma-1 Proteins 0.000 claims 3
- 101001031961 Homo sapiens Hemoglobin subunit gamma-2 Proteins 0.000 claims 3
- 238000006640 acetylation reaction Methods 0.000 claims 3
- 238000007069 methylation reaction Methods 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 3
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 2
- 230000007774 longterm Effects 0.000 claims 2
- 108010044495 Fetal Hemoglobin Proteins 0.000 claims 1
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 1
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 1
- 238000004581 coalescence Methods 0.000 claims 1
- 238000004520 electroporation Methods 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 229940046166 oligodeoxynucleotide Drugs 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023026918A JP2023075166A (ja) | 2016-03-14 | 2023-02-24 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662308190P | 2016-03-14 | 2016-03-14 | |
| US62/308,190 | 2016-03-14 | ||
| US201762456615P | 2017-02-08 | 2017-02-08 | |
| US62/456,615 | 2017-02-08 | ||
| PCT/US2017/022377 WO2017160890A1 (en) | 2016-03-14 | 2017-03-14 | Crispr/cas-related methods and compositions for treating beta hemoglobinopathies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023026918A Division JP2023075166A (ja) | 2016-03-14 | 2023-02-24 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019508051A JP2019508051A (ja) | 2019-03-28 |
| JP2019508051A5 true JP2019508051A5 (enExample) | 2020-04-23 |
Family
ID=58413206
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018548318A Pending JP2019508051A (ja) | 2016-03-14 | 2017-03-14 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
| JP2023026918A Pending JP2023075166A (ja) | 2016-03-14 | 2023-02-24 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023026918A Pending JP2023075166A (ja) | 2016-03-14 | 2023-02-24 | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US20200255857A1 (enExample) |
| EP (1) | EP3430142A1 (enExample) |
| JP (2) | JP2019508051A (enExample) |
| KR (2) | KR102723347B1 (enExample) |
| CN (4) | CN117821458A (enExample) |
| AU (2) | AU2017235333B2 (enExample) |
| CA (1) | CA3017956A1 (enExample) |
| IL (2) | IL313038A (enExample) |
| MX (2) | MX2018011114A (enExample) |
| SG (1) | SG11201807859WA (enExample) |
| WO (1) | WO2017160890A1 (enExample) |
Families Citing this family (66)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ヌクレアーゼ切断特異性の評価および改善 |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| WO2016135559A2 (en) | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of human genetic diseases including hemoglobinopathies |
| WO2016135558A2 (en) | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| CA2986310A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| EP3307887A1 (en) | 2015-06-09 | 2018-04-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for improving transplantation |
| EP3365356B1 (en) | 2015-10-23 | 2023-06-28 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
| EP3371306B8 (en) | 2015-11-04 | 2023-02-22 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| CN110191948A (zh) * | 2016-11-02 | 2019-08-30 | 巴塞尔大学 | 用于细胞疗法的免疫可识别的细胞表面变体 |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| TW201839136A (zh) * | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| EP3596217A1 (en) | 2017-03-14 | 2020-01-22 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| WO2018200597A1 (en) | 2017-04-24 | 2018-11-01 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Homology directed repair compositions for the treatment of hemoglobinopathies |
| WO2018209158A2 (en) | 2017-05-10 | 2018-11-15 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| WO2019003193A1 (en) * | 2017-06-30 | 2019-01-03 | Novartis Ag | METHODS FOR TREATING DISEASES USING GENE EDITING SYSTEMS |
| US11866726B2 (en) | 2017-07-14 | 2024-01-09 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| CN111757937A (zh) * | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| MA50849A (fr) * | 2017-10-26 | 2020-09-02 | Vertex Pharma | Substances et procédés pour le traitement d'hémoglobinopathies |
| CN111556893A (zh) * | 2017-11-06 | 2020-08-18 | 爱迪塔斯医药股份有限公司 | 免疫疗法t细胞中cblb的crispr-cas9编辑的方法、组合物和组分 |
| US12161674B2 (en) | 2017-12-05 | 2024-12-10 | Vertex Pharmaceuticals Incorporated | CRISPR-CAS9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof |
| JP2021505187A (ja) * | 2017-12-11 | 2021-02-18 | エディタス・メディシン、インコーポレイテッド | 遺伝子編集のためのｃｐｆ１関連方法及び組成物 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3749768A1 (en) | 2018-02-05 | 2020-12-16 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| EP3762496A2 (en) * | 2018-03-07 | 2021-01-13 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| JP7706888B2 (ja) * | 2018-03-14 | 2025-07-14 | エディタス・メディシン、インコーポレイテッド | 異常ヘモグロビン症の治療のためのシステム及び方法 |
| CA3093701A1 (en) * | 2018-03-14 | 2019-09-19 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| WO2019210005A1 (en) * | 2018-04-24 | 2019-10-31 | Ligandal, Inc. | Methods and compositions for genome editing |
| US12133884B2 (en) | 2018-05-11 | 2024-11-05 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| WO2020053224A1 (en) * | 2018-09-11 | 2020-03-19 | 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 |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US20220047637A1 (en) * | 2018-11-29 | 2022-02-17 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| CN113302292B (zh) * | 2018-12-05 | 2024-10-01 | 弗莱德哈钦森癌症中心 | 遗传修饰细胞的减少和最少的操作制造 |
| CN111321171A (zh) * | 2018-12-14 | 2020-06-23 | 江苏集萃药康生物科技有限公司 | 一种应用CRISPR/Cas9介导ES打靶技术制备基因打靶动物模型的方法 |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| CN114096666B (zh) | 2019-02-13 | 2025-03-18 | 比姆医疗股份有限公司 | 治疗血红素病变的组合物和方法 |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN112011576A (zh) * | 2019-05-31 | 2020-12-01 | 华东师范大学 | Crispr基因编辑技术在治疗地中海贫血中的应用 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CN112979823B (zh) * | 2019-12-18 | 2022-04-08 | 华东师范大学 | 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白 |
| MX2022014008A (es) | 2020-05-08 | 2023-02-09 | Broad Inst Inc | Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo. |
| CN111876416B (zh) * | 2020-07-01 | 2021-09-03 | 广州瑞风生物科技有限公司 | 激活γ-珠蛋白基因表达的方法和组合物 |
| WO2023079465A1 (en) * | 2021-11-02 | 2023-05-11 | The University Of British Columbia | Compositions and methods for preventing, ameliorating, or treating sickle cell disease |
| CN114848851A (zh) * | 2022-04-29 | 2022-08-05 | 广州医科大学附属第三医院（广州重症孕产妇救治中心、广州柔济医院） | 治疗β-地中海贫血的药物 |
| WO2024073751A1 (en) | 2022-09-29 | 2024-04-04 | Vor Biopharma Inc. | Methods and compositions for gene modification and enrichment |
| WO2025038164A1 (en) * | 2023-08-15 | 2025-02-20 | University Of Massachusetts | Nick resection in cancer |
| 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 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009013559A1 (en) * | 2007-07-23 | 2009-01-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof |
| SG11201405103SA (en) * | 2012-02-24 | 2014-09-26 | Hutchinson Fred Cancer Res | Compositions and methods for the treatment of hemoglobinopathies |
| KR20220164090A (ko) * | 2012-08-29 | 2022-12-12 | 상가모 테라퓨틱스, 인코포레이티드 | 유전적 병태를 치료하기 위한 방법 및 조성물 |
| PT3502240T (pt) * | 2012-11-27 | 2021-08-11 | Childrens Medical Ct Corp | Elementos reguladores distais de bcl11a como alvo para a reindução de hemoglobina fetal |
| AU2014265331B2 (en) * | 2013-05-15 | 2019-12-05 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| KR102733409B1 (ko) * | 2013-06-05 | 2024-11-21 | 듀크 유니버시티 | Rna-가이드 유전자 편집 및 유전자 조절 |
| PT3066201T (pt) * | 2013-11-07 | 2018-06-04 | Massachusetts Inst Technology | Métodos e composições relacionadas com crispr com arng reguladores |
| EP3116997B1 (en) | 2014-03-10 | 2019-05-15 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| US11242525B2 (en) * | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
| AU2015342749B2 (en) | 2014-11-07 | 2022-01-27 | Editas Medicine, Inc. | Methods for improving CRISPR/Cas-mediated genome-editing |
| WO2016135558A2 (en) * | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| CA2986310A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
-
2017
- 2017-03-14 SG SG11201807859WA patent/SG11201807859WA/en unknown
- 2017-03-14 AU AU2017235333A patent/AU2017235333B2/en active Active
- 2017-03-14 MX MX2018011114A patent/MX2018011114A/es unknown
- 2017-03-14 KR KR1020237015832A patent/KR102723347B1/ko active Active
- 2017-03-14 JP JP2018548318A patent/JP2019508051A/ja active Pending
- 2017-03-14 IL IL313038A patent/IL313038A/en unknown
- 2017-03-14 CN CN202311860322.9A patent/CN117821458A/zh active Pending
- 2017-03-14 CA CA3017956A patent/CA3017956A1/en active Pending
- 2017-03-14 WO PCT/US2017/022377 patent/WO2017160890A1/en not_active Ceased
- 2017-03-14 CN CN202311860310.6A patent/CN117802102A/zh active Pending
- 2017-03-14 EP EP17713843.5A patent/EP3430142A1/en active Pending
- 2017-03-14 KR KR1020187029140A patent/KR102532663B1/ko active Active
- 2017-03-14 US US16/085,480 patent/US20200255857A1/en not_active Abandoned
- 2017-03-14 CN CN201780029929.9A patent/CN109153994A/zh active Pending
- 2017-03-14 CN CN202311860300.2A patent/CN118127006A/zh active Pending
- 2017-03-14 IL IL261714A patent/IL261714B2/en unknown
-
2018
- 2018-09-13 MX MX2024012899A patent/MX2024012899A/es unknown
-
2023
- 2023-02-24 JP JP2023026918A patent/JP2023075166A/ja active Pending
- 2023-08-08 AU AU2023214243A patent/AU2023214243A1/en active Pending
- 2023-08-28 US US18/457,258 patent/US20240360471A1/en active Pending
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