JPWO2019210042A5 - - Google Patents
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- Publication number
- JPWO2019210042A5 JPWO2019210042A5 JP2020560400A JP2020560400A JPWO2019210042A5 JP WO2019210042 A5 JPWO2019210042 A5 JP WO2019210042A5 JP 2020560400 A JP2020560400 A JP 2020560400A JP 2020560400 A JP2020560400 A JP 2020560400A JP WO2019210042 A5 JPWO2019210042 A5 JP WO2019210042A5
- Authority
- JP
- Japan
- Prior art keywords
- foxp3
- sequence
- promoter
- cell
- homology
- 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 26
- 102100027581 Forkhead box protein P3 Human genes 0.000 claims 24
- 210000004027 cell Anatomy 0.000 claims 21
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims 13
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims 13
- 238000000034 method Methods 0.000 claims 12
- 150000007523 nucleic acids Chemical class 0.000 claims 12
- 108020004707 nucleic acids Proteins 0.000 claims 9
- 102000039446 nucleic acids Human genes 0.000 claims 9
- 239000002299 complementary DNA Substances 0.000 claims 7
- 125000006850 spacer group Chemical group 0.000 claims 5
- 101150027879 FOXP3 gene Proteins 0.000 claims 4
- 208000009329 Graft vs Host Disease Diseases 0.000 claims 4
- 101001000998 Homo sapiens Protein phosphatase 1 regulatory subunit 12C 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
- 102100035620 Protein phosphatase 1 regulatory subunit 12C Human genes 0.000 claims 4
- 208000024908 graft versus host disease Diseases 0.000 claims 4
- 108020005004 Guide RNA Proteins 0.000 claims 3
- 101710163270 Nuclease Proteins 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 150000002632 lipids Chemical class 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
- 101710153660 Nuclear receptor corepressor 2 Proteins 0.000 claims 2
- 206010039710 Scleroderma Diseases 0.000 claims 2
- 108091008874 T cell receptors Proteins 0.000 claims 2
- 102000016266 T-Cell Antigen Receptors Human genes 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
- 108090000623 proteins and genes Proteins 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
- 101150069263 tra gene Proteins 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
- 210000001744 T-lymphocyte Anatomy 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
- 210000005260 human cell Anatomy 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 210000003289 regulatory T cell Anatomy 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024182581A JP7844582B2 (ja) | 2018-04-27 | 2024-10-18 | 遺伝子編集されたｃｄ３４＋細胞におけるｆｏｘｐ３の発現 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862663545P | 2018-04-27 | 2018-04-27 | |
| US62/663,545 | 2018-04-27 | ||
| PCT/US2019/029082 WO2019210042A1 (en) | 2018-04-27 | 2019-04-25 | Expression of foxp3 in edited cd34+ cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024182581A Division JP7844582B2 (ja) | 2018-04-27 | 2024-10-18 | 遺伝子編集されたｃｄ３４＋細胞におけるｆｏｘｐ３の発現 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021521856A JP2021521856A (ja) | 2021-08-30 |
| JPWO2019210042A5 true JPWO2019210042A5 (https=) | 2022-11-25 |
| JP2021521856A5 JP2021521856A5 (https=) | 2022-11-25 |
| JP7575950B2 JP7575950B2 (ja) | 2024-10-31 |
Family
ID=68294731
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020560400A Active JP7575950B2 (ja) | 2018-04-27 | 2019-04-25 | 遺伝子編集されたｃｄ３４＋細胞におけるｆｏｘｐ３の発現 |
| JP2024182581A Active JP7844582B2 (ja) | 2018-04-27 | 2024-10-18 | 遺伝子編集されたｃｄ３４＋細胞におけるｆｏｘｐ３の発現 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024182581A Active JP7844582B2 (ja) | 2018-04-27 | 2024-10-18 | 遺伝子編集されたｃｄ３４＋細胞におけるｆｏｘｐ３の発現 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11713459B2 (https=) |
| EP (1) | EP3784690A4 (https=) |
| JP (2) | JP7575950B2 (https=) |
| CN (1) | CN112218882A (https=) |
| AU (2) | AU2019261438B2 (https=) |
| CA (1) | CA3091688A1 (https=) |
| IL (1) | IL277039A (https=) |
| SG (1) | SG11202007878UA (https=) |
| WO (1) | WO2019210042A1 (https=) |
Families Citing this family (13)
| 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 |
| CA3091491A1 (en) | 2018-04-27 | 2019-10-31 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Expression of human foxp3 in gene edited t cells |
| US11713459B2 (en) * | 2018-04-27 | 2023-08-01 | Seattle Children's Hospital | Expression of FOXP3 in edited CD34+ cells |
| KR20210116531A (ko) * | 2019-01-18 | 2021-09-27 | 고꾸리쯔 다이가꾸 호우징 오사까 다이가꾸 | 영양 장애형 표피 수포증 치료약 |
| EP3973067A1 (en) * | 2019-05-21 | 2022-03-30 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory t cells |
| AU2020304057A1 (en) * | 2019-06-27 | 2022-01-27 | Benaroya Research Institute At Virginia Mason | Artificial antigen-specific immunoregulatory t (airt) cells |
| US20220380727A1 (en) | 2019-11-08 | 2022-12-01 | Sangamo Therapeutics, Inc. | Generation of engineered regulatory t cells |
| WO2021144692A1 (en) * | 2020-01-14 | 2021-07-22 | Crispr Therapeutics Ag | Methods for increased efficiency of homology-directed repair |
| 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 |
| WO2022093884A1 (en) * | 2020-10-27 | 2022-05-05 | Pact Pharma, Inc. | Compositions and methods for the treatment of cancer using next generation engineered t cell therapy |
| CN112851794B (zh) * | 2021-02-04 | 2023-05-23 | 苏州铂维生物科技有限公司 | 一种基于cd271的抗原表位及其应用 |
| GB202113674D0 (en) | 2021-09-24 | 2021-11-10 | Reflection Therapeutics Ltd | Targeted cell therapies |
| GB202113673D0 (en) | 2021-09-24 | 2021-11-10 | Reflection Therapeutics Ltd | Targeted cell therapies |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1430124B1 (en) | 2000-09-20 | 2013-11-06 | EMD Millipore Corporation | Artificial ubiquitous chromatin opening elements (ucoe) |
| US7507542B2 (en) | 2001-05-08 | 2009-03-24 | Ucb Sa | Method for regulating immune function using the FOXP3 protein |
| GB0614536D0 (en) | 2006-07-21 | 2006-08-30 | Metcalfe Susan M | Methods of controlling cellular response to LIF |
| EP2064350B1 (en) * | 2006-11-27 | 2013-01-02 | Ludwig Institute for Cancer Research Ltd. | Expression of foxp3 by cancer cells |
| EP2126054B1 (en) | 2007-01-31 | 2016-07-06 | Yeda Research And Development Company Limited | Redirected, genetically-engineered t regulatory cells and their use in suppression of autoimmune and inflammatory disease |
| US9249423B2 (en) | 2007-02-02 | 2016-02-02 | Yale University | Method of de-differentiating and re-differentiating somatic cells using RNA |
| WO2008101272A1 (en) | 2007-02-21 | 2008-08-28 | Women's And Children's Health Research Institute Inc | Method for obtaining treg-cells |
| US8158596B2 (en) | 2007-05-11 | 2012-04-17 | The Regents Of The University Of Michigan | Materials and methods for FOXP3 tumor suppression |
| WO2008154399A1 (en) | 2007-06-08 | 2008-12-18 | The Trustees Of The University Of Pennsylvania | Foxp3 oligomerization and intermolecular interactions |
| JP2011503232A (ja) | 2007-11-20 | 2011-01-27 | ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド | 免疫応答の調節 |
| CA2717166A1 (en) | 2008-03-03 | 2009-09-19 | Converge Biotech Inc. | Methods of modulating t cell-dependent immune responses |
| WO2012018930A1 (en) | 2010-08-03 | 2012-02-09 | University Of Miami | Methods of isolating and expanding human t regulatory cells and uses thereof for cellular therapy |
| WO2014180943A1 (en) | 2013-05-08 | 2014-11-13 | Vib Vzw | Mcl-1 as critical regulator of foxp3+ regulatory t cell survival, and use thereof to treat severe immune disorders |
| MX2015015638A (es) | 2013-05-13 | 2016-10-28 | Cellectis | Metodos para diseñar celulas t altamente activas para inmunoterapia. |
| 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 |
| WO2014191128A1 (en) | 2013-05-29 | 2014-12-04 | Cellectis | Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system |
| ES3063961T3 (en) | 2014-02-14 | 2026-04-21 | Cellectis | Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells |
| EP3119897A1 (en) | 2014-03-21 | 2017-01-25 | Cellectis | Engineering mammalian genome using dna-guided argonaute interference systems (dais) |
| KR101835554B1 (ko) | 2014-06-24 | 2018-04-19 | 서울대학교 산학협력단 | C/ebp를 포함하는 유도성 t 조절세포 분화촉진 또는 안정화 조성물 및 방법 |
| CN107250373A (zh) | 2015-01-12 | 2017-10-13 | 麻省理工学院 | 通过微流体递送实现的基因编辑 |
| 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 |
| US11499168B2 (en) | 2016-04-25 | 2022-11-15 | Universitat Basel | Allele editing and applications thereof |
| WO2018031762A1 (en) | 2016-08-10 | 2018-02-15 | Duke University | Compositions, systems and methods for programming immune cell function through targeted gene regulation |
| 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 |
| JP7206214B2 (ja) | 2016-12-13 | 2023-01-17 | シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート） | インビトロ及びインビボで操作された細胞において発現された化学誘導シグナル伝達複合体の外因性薬物活性化の方法 |
| 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 |
| CA3091491A1 (en) | 2018-04-27 | 2019-10-31 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Expression of human foxp3 in gene edited t cells |
| WO2019241549A1 (en) | 2018-06-15 | 2019-12-19 | A2 Biotherapeutics, Inc. | Foxp3-expressing car-t regulatory cells |
-
2019
- 2019-04-25 US US16/981,223 patent/US11713459B2/en active Active
- 2019-04-25 CN CN201980028370.7A patent/CN112218882A/zh active Pending
- 2019-04-25 JP JP2020560400A patent/JP7575950B2/ja active Active
- 2019-04-25 CA CA3091688A patent/CA3091688A1/en active Pending
- 2019-04-25 AU AU2019261438A patent/AU2019261438B2/en active Active
- 2019-04-25 WO PCT/US2019/029082 patent/WO2019210042A1/en not_active Ceased
- 2019-04-25 EP EP19793428.4A patent/EP3784690A4/en active Pending
- 2019-04-25 SG SG11202007878UA patent/SG11202007878UA/en unknown
-
2020
- 2020-08-31 IL IL277039A patent/IL277039A/en unknown
-
2023
- 2023-06-16 US US18/336,276 patent/US20240117352A1/en active Pending
-
2024
- 2024-10-18 JP JP2024182581A patent/JP7844582B2/ja active Active
- 2024-11-22 AU AU2024266824A patent/AU2024266824A1/en active Pending
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