JPWO2019210078A5 - - Google Patents
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- Publication number
- JPWO2019210078A5 JPWO2019210078A5 JP2020560338A JP2020560338A JPWO2019210078A5 JP WO2019210078 A5 JPWO2019210078 A5 JP WO2019210078A5 JP 2020560338 A JP2020560338 A JP 2020560338A JP 2020560338 A JP2020560338 A JP 2020560338A JP WO2019210078 A5 JPWO2019210078 A5 JP WO2019210078A5
- Authority
- JP
- Japan
- Prior art keywords
- foxp3
- sequence
- locus
- homology
- promoter
- 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
- 101000861452 Homo sapiens Forkhead box protein P3 Proteins 0.000 claims 30
- 102100027581 Forkhead box protein P3 Human genes 0.000 claims 28
- 210000004698 lymphocyte Anatomy 0.000 claims 13
- 238000000034 method Methods 0.000 claims 13
- 210000004027 cell Anatomy 0.000 claims 10
- 150000007523 nucleic acids Chemical class 0.000 claims 10
- 239000002299 complementary DNA Substances 0.000 claims 7
- 108020004707 nucleic acids Proteins 0.000 claims 7
- 102000039446 nucleic acids Human genes 0.000 claims 7
- 125000006850 spacer group Chemical group 0.000 claims 7
- 208000009329 Graft vs Host Disease Diseases 0.000 claims 4
- 108020005004 Guide RNA Proteins 0.000 claims 4
- 102100022219 NF-kappa-B essential modulator Human genes 0.000 claims 4
- 101710090077 NF-kappa-B essential modulator Proteins 0.000 claims 4
- 208000024908 graft versus host disease Diseases 0.000 claims 4
- 101001000998 Homo sapiens Protein phosphatase 1 regulatory subunit 12C Proteins 0.000 claims 3
- 101710163270 Nuclease Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 102100035620 Protein phosphatase 1 regulatory subunit 12C Human genes 0.000 claims 3
- 210000001744 T-lymphocyte Anatomy 0.000 claims 3
- 150000002632 lipids Chemical class 0.000 claims 3
- 230000000527 lymphocytic effect Effects 0.000 claims 3
- 239000003550 marker Substances 0.000 claims 3
- 239000002105 nanoparticle Substances 0.000 claims 3
- 239000013607 AAV vector Substances 0.000 claims 2
- 206010003594 Ataxia telangiectasia Diseases 0.000 claims 2
- 208000023275 Autoimmune disease Diseases 0.000 claims 2
- 208000000659 Autoimmune lymphoproliferative syndrome Diseases 0.000 claims 2
- 208000023328 Basedow disease Diseases 0.000 claims 2
- 201000010717 Bruton-type agammaglobulinemia Diseases 0.000 claims 2
- 108010019670 Chimeric Antigen Receptors Proteins 0.000 claims 2
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims 2
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 208000000398 DiGeorge Syndrome Diseases 0.000 claims 2
- 108010042407 Endonucleases Proteins 0.000 claims 2
- 102000004533 Endonucleases Human genes 0.000 claims 2
- 208000024869 Goodpasture syndrome Diseases 0.000 claims 2
- 208000015023 Graves' disease Diseases 0.000 claims 2
- 208000003352 Hyper-IgM Immunodeficiency Syndrome Diseases 0.000 claims 2
- 206010061598 Immunodeficiency Diseases 0.000 claims 2
- 208000029462 Immunodeficiency disease Diseases 0.000 claims 2
- 208000022559 Inflammatory bowel disease Diseases 0.000 claims 2
- 208000009388 Job Syndrome Diseases 0.000 claims 2
- 201000001779 Leukocyte adhesion deficiency Diseases 0.000 claims 2
- 208000029578 Muscle disease Diseases 0.000 claims 2
- 206010039710 Scleroderma Diseases 0.000 claims 2
- 238000010459 TALEN Methods 0.000 claims 2
- 206010067584 Type 1 diabetes mellitus Diseases 0.000 claims 2
- 206010047115 Vasculitis Diseases 0.000 claims 2
- 208000006110 Wiskott-Aldrich syndrome Diseases 0.000 claims 2
- 208000016349 X-linked agammaglobulinemia Diseases 0.000 claims 2
- 208000007502 anemia Diseases 0.000 claims 2
- 230000001684 chronic effect Effects 0.000 claims 2
- 102000053917 human FOXP3 Human genes 0.000 claims 2
- 206010051040 hyper-IgE syndrome Diseases 0.000 claims 2
- 206010066130 hyper-IgM syndrome Diseases 0.000 claims 2
- 230000007813 immunodeficiency Effects 0.000 claims 2
- 208000027866 inflammatory disease Diseases 0.000 claims 2
- 201000006417 multiple sclerosis Diseases 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 210000000056 organ Anatomy 0.000 claims 2
- 230000002265 prevention Effects 0.000 claims 2
- 206010039073 rheumatoid arthritis Diseases 0.000 claims 2
- 208000029138 selective IgA deficiency disease Diseases 0.000 claims 2
- 208000002491 severe combined immunodeficiency Diseases 0.000 claims 2
- 239000007787 solid Substances 0.000 claims 2
- 208000011580 syndromic disease Diseases 0.000 claims 2
- 201000000596 systemic lupus erythematosus Diseases 0.000 claims 2
- 238000002054 transplantation Methods 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 108091033409 CRISPR Proteins 0.000 claims 1
- 108091008874 T cell receptors Proteins 0.000 claims 1
- 102000016266 T-Cell Antigen Receptors Human genes 0.000 claims 1
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 1
- 239000000654 additive Substances 0.000 claims 1
- 230000000996 additive effect Effects 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 210000003289 regulatory T cell Anatomy 0.000 claims 1
- 230000009261 transgenic effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024182444A JP7844581B2 (ja) | 2018-04-27 | 2024-10-18 | 遺伝子編集ｔ細胞におけるヒトｆｏｘｐ３の発現 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862663561P | 2018-04-27 | 2018-04-27 | |
| US62/663,561 | 2018-04-27 | ||
| US201862773414P | 2018-11-30 | 2018-11-30 | |
| US62/773,414 | 2018-11-30 | ||
| PCT/US2019/029159 WO2019210078A1 (en) | 2018-04-27 | 2019-04-25 | Expression of human foxp3 in gene edited t cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024182444A Division JP7844581B2 (ja) | 2018-04-27 | 2024-10-18 | 遺伝子編集ｔ細胞におけるヒトｆｏｘｐ３の発現 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021521849A JP2021521849A (ja) | 2021-08-30 |
| JPWO2019210078A5 true JPWO2019210078A5 (https=) | 2022-11-25 |
| JP2021521849A5 JP2021521849A5 (https=) | 2022-11-25 |
| JP7575949B2 JP7575949B2 (ja) | 2024-10-31 |
Family
ID=68295504
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020560338A Active JP7575949B2 (ja) | 2018-04-27 | 2019-04-25 | 遺伝子編集ｔ細胞におけるヒトｆｏｘｐ３の発現 |
| JP2024182444A Active JP7844581B2 (ja) | 2018-04-27 | 2024-10-18 | 遺伝子編集ｔ細胞におけるヒトｆｏｘｐ３の発現 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024182444A Active JP7844581B2 (ja) | 2018-04-27 | 2024-10-18 | 遺伝子編集ｔ細胞におけるヒトｆｏｘｐ３の発現 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20210253652A1 (https=) |
| EP (1) | EP3784689A4 (https=) |
| JP (2) | JP7575949B2 (https=) |
| KR (1) | KR102912481B1 (https=) |
| CN (1) | CN112041334A (https=) |
| AU (2) | AU2019257708B2 (https=) |
| CA (1) | CA3091491A1 (https=) |
| IL (1) | IL277036A (https=) |
| SG (1) | SG11202007877SA (https=) |
| WO (1) | WO2019210078A1 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110139675B (zh) | 2016-10-31 | 2023-09-29 | 西雅图儿童医院（Dba西雅图儿童研究所） | 用具有工程化稳定的内源性foxp3基因表达的cd4t细胞治疗自身免疫疾病的方法 |
| US11713459B2 (en) | 2018-04-27 | 2023-08-01 | Seattle Children's Hospital | Expression of FOXP3 in edited CD34+ cells |
| EP3921427A1 (en) * | 2019-02-05 | 2021-12-15 | Institut National de la Santé et de la Recherche Médicale (INSERM) | Recombinant vectors suitable for the treatment of ipex syndrome |
| EP3973067A1 (en) * | 2019-05-21 | 2022-03-30 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory t cells |
| US20220380727A1 (en) * | 2019-11-08 | 2022-12-01 | Sangamo Therapeutics, Inc. | Generation of engineered regulatory t cells |
| EP4103591A4 (en) * | 2020-02-13 | 2024-09-04 | The Board of Trustees of the Leland Stanford Junior University | Crispr-based foxp3 gene engineered t cells and hematopoietic stem cell precursors to treat ipex syndrome patients |
| EP4240376B1 (en) * | 2020-11-09 | 2026-03-25 | Quell Therapeutics Limited | Method for cryopreserving engineered tregs |
| EP4355879A4 (en) * | 2021-06-14 | 2025-07-23 | Kamau Therapeutics Inc | METHODS FOR GENETIC MODIFICATION OF HEMATOPOIETIC STEM AND PROGENITOR CELLS FOR ERYTHROCYTE-SPECIFIC EXPRESSION OF THERAPEUTIC PROTEINS |
| WO2023122099A2 (en) * | 2021-12-21 | 2023-06-29 | The Board Of Trustees Of The Leland Stanford Junior University | Crispr-based gene editing to preserve splicing and expression of foxp3 isoforms 1 and 2 |
| CN115305237B (zh) * | 2022-07-08 | 2023-07-14 | 中南大学 | 一种优化的调节性t细胞的扩增及规模化生产的方法 |
| EP4634383A1 (en) * | 2022-12-16 | 2025-10-22 | Life Edit Therapeutics, Inc. | Guide rnas that target foxp3 gene and methods of use |
| AU2024308500A1 (en) * | 2023-06-24 | 2025-12-18 | Sonoma Biotherapeutics, Inc. | Compositions and methods for engineering cells |
| WO2025054503A1 (en) * | 2023-09-08 | 2025-03-13 | Sonoma Biotherapeutics, Inc. | Cells ectopically expressing foxp3 |
| JP7818147B1 (ja) * | 2025-01-23 | 2026-02-20 | 国立研究開発法人国立がん研究センター | エフェクターｔ細胞、およびそれを含む医薬組成物 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008101272A1 (en) * | 2007-02-21 | 2008-08-28 | Women's And Children's Health Research Institute Inc | Method for obtaining treg-cells |
| ES2883131T3 (es) * | 2013-05-29 | 2021-12-07 | Cellectis | Métodos para la modificación de células T para inmunoterapia utilizando el sistema de nucleasa CAS guiado por ARN |
| WO2016123578A1 (en) * | 2015-01-30 | 2016-08-04 | The Regents Of The University Of California | Protein delivery in primary hematopoietic cells |
| EP3294342A4 (en) * | 2015-05-08 | 2018-11-07 | President and Fellows of Harvard College | Universal donor stem cells and related methods |
| WO2018035141A1 (en) * | 2016-08-16 | 2018-02-22 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
| WO2018073391A1 (en) * | 2016-10-19 | 2018-04-26 | Cellectis | Targeted gene insertion for improved immune cells therapy |
| CA3041831A1 (en) * | 2016-10-27 | 2018-05-03 | Intima Bioscience, Inc. | Viral methods of making genetically modified cells |
| CN110139675B (zh) | 2016-10-31 | 2023-09-29 | 西雅图儿童医院（Dba西雅图儿童研究所） | 用具有工程化稳定的内源性foxp3基因表达的cd4t细胞治疗自身免疫疾病的方法 |
| CN109797171A (zh) * | 2017-05-08 | 2019-05-24 | 北京东方略细胞技术有限公司 | 经修饰的t细胞、其制备方法及用途 |
| EP3672617A4 (en) * | 2017-08-22 | 2021-06-23 | The Regents of the University of California | LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES |
| US11713459B2 (en) * | 2018-04-27 | 2023-08-01 | Seattle Children's Hospital | Expression of FOXP3 in edited CD34+ cells |
-
2019
- 2019-04-25 CA CA3091491A patent/CA3091491A1/en active Pending
- 2019-04-25 KR KR1020207033790A patent/KR102912481B1/ko active Active
- 2019-04-25 JP JP2020560338A patent/JP7575949B2/ja active Active
- 2019-04-25 SG SG11202007877SA patent/SG11202007877SA/en unknown
- 2019-04-25 CN CN201980028523.8A patent/CN112041334A/zh active Pending
- 2019-04-25 EP EP19792759.3A patent/EP3784689A4/en active Pending
- 2019-04-25 AU AU2019257708A patent/AU2019257708B2/en active Active
- 2019-04-25 WO PCT/US2019/029159 patent/WO2019210078A1/en not_active Ceased
- 2019-04-25 US US16/981,213 patent/US20210253652A1/en active Pending
-
2020
- 2020-08-31 IL IL277036A patent/IL277036A/en unknown
-
2024
- 2024-10-18 JP JP2024182444A patent/JP7844581B2/ja active Active
- 2024-11-22 AU AU2024266825A patent/AU2024266825A1/en active Pending
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