IL301012A - Genetically engineered T cells with a REGNASE-1 and/or TGFBRII disorder with improved functionality and persistence - Google Patents

Genetically engineered T cells with a REGNASE-1 and/or TGFBRII disorder with improved functionality and persistence

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Publication number
IL301012A
IL301012A IL301012A IL30101223A IL301012A IL 301012 A IL301012 A IL 301012A IL 301012 A IL301012 A IL 301012A IL 30101223 A IL30101223 A IL 30101223A IL 301012 A IL301012 A IL 301012A
Authority
IL
Israel
Prior art keywords
cells
gene
car
tgfbrii
seq
Prior art date
Application number
IL301012A
Other languages
English (en)
Hebrew (he)
Original Assignee
Crispr Therapeutics Ag
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Crispr Therapeutics Ag filed Critical Crispr Therapeutics Ag
Publication of IL301012A publication Critical patent/IL301012A/en

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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
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    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
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IL301012A 2020-09-23 2021-09-23 Genetically engineered T cells with a REGNASE-1 and/or TGFBRII disorder with improved functionality and persistence IL301012A (en)

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Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113396216A (zh) * 2019-02-04 2021-09-14 Ksq治疗公司 用于改进的免疫疗法的组合基因靶标
EP3773918A4 (en) 2019-03-05 2022-01-05 Nkarta, Inc. CD19 DIRECTED CHIMERIC ANTIGEN RECEPTORS AND THEIR USES IN IMMUNOTHERAPY
AU2021286655A1 (en) 2020-06-12 2023-01-05 Nkarta, Inc. Genetically modified natural killer cells for CD70-directed cancer immunotherapy
TW202328444A (zh) * 2021-07-26 2023-07-16 瑞士商Ｃｒｉｓｐｒ治療公司 用於製造基因工程化ｃａｒ－ｔ細胞之方法
WO2023084399A1 (en) 2021-11-09 2023-05-19 Crispr Therapeutics Ag Genetically engineered immune cells expressing masked chimeric antigen receptors specific to protein tyrosine kinase 7
WO2023111913A1 (en) 2021-12-15 2023-06-22 Crispr Therapeutics Ag Engineered anti-liv1 cell with regnase-1 and/or tgfbrii disruption
US20230346836A1 (en) 2021-12-22 2023-11-02 Crispr Therapeutics Ag Genetically engineered t cells with disrupted casitas b-lineage lymphoma proto-oncogene-b (cblb) and uses thereof
US20230303713A1 (en) 2022-03-23 2023-09-28 Crispr Therapeutics Ag Anti-cd19 car-t cells with multiple gene edits and therapeutic uses thereof
US20230331841A1 (en) 2022-03-23 2023-10-19 Crispr Therapeutics Ag Anti-cd83 car-t cells with regnase-1 and/or tgfbrii disruption
WO2024003786A1 (en) 2022-06-29 2024-01-04 Crispr Therapeutics Ag Chimeric antigen receptor targeting gpc-3 and immune cells expressing such for therapeutic uses
WO2024023802A2 (en) 2022-07-29 2024-02-01 Crispr Therapeutics Ag Genetically engineered immune cells having disrupted transporter associated with antigen processing-2 (tap-2) gene
WO2024023801A2 (en) 2022-07-29 2024-02-01 Crispr Therapeutics Ag Genetically engineered immune cells having disrupted transporter associated with antigen processing-1 (tap-1) gene
WO2024023804A2 (en) 2022-07-29 2024-02-01 Crispr Therapeutics Ag Genetically engineered immune cells having disrupted transporter associated with antigen processing binding protein (tapbp) gene
EP4587477A2 (en) * 2022-09-14 2025-07-23 Ohio State Innovation Foundation Methods for reprograming exhausted t cells and boosting immune checkpoint blockade therapy for cancer
WO2024062388A2 (en) 2022-09-20 2024-03-28 Crispr Therapeutics Ag Genetically engineered immune cells expressing chimeric antigen receptor targeting cd20
US20240115703A1 (en) * 2022-10-10 2024-04-11 Crispr Therapeutics Ag Genetically engineered anti-cd19 car-t cells for use in treating b-cell malignancies
WO2024229098A2 (en) * 2023-05-01 2024-11-07 Wisconsin Alumni Research Foundation Methods and systems for predicting potent t cell products
US12500832B2 (en) 2023-08-28 2025-12-16 Ciena Corporation Establishing and advertising co-routed bidirectional paths across multiple domains
WO2025126049A2 (en) 2023-12-11 2025-06-19 Crispr Therapeutics Ag Multiplex gene editing
WO2025137439A2 (en) * 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Engineered t cells
CN117844811A (zh) * 2024-03-08 2024-04-09 上海恒润达生生物科技股份有限公司 靶向敲除CD70基因的sgRNA组合物及其应用

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6534055B1 (en) 1988-11-23 2003-03-18 Genetics Institute, Inc. Methods for selectively stimulating proliferation of T cells
US6352694B1 (en) 1994-06-03 2002-03-05 Genetics Institute, Inc. Methods for inducing a population of T cells to proliferate using agents which recognize TCR/CD3 and ligands which stimulate an accessory molecule on the surface of the T cells
US6905680B2 (en) 1988-11-23 2005-06-14 Genetics Institute, Inc. Methods of treating HIV infected subjects
US5858358A (en) 1992-04-07 1999-01-12 The United States Of America As Represented By The Secretary Of The Navy Methods for selectively stimulating proliferation of T cells
US7175843B2 (en) 1994-06-03 2007-02-13 Genetics Institute, Llc Methods for selectively stimulating proliferation of T cells
US6692964B1 (en) 1995-05-04 2004-02-17 The United States Of America As Represented By The Secretary Of The Navy Methods for transfecting T cells
US7067318B2 (en) 1995-06-07 2006-06-27 The Regents Of The University Of Michigan Methods for transfecting T cells
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
KR20030032922A (ko) 2000-02-24 2003-04-26 싸이트 테라피스 인코포레이티드 세포의 동시 자극 및 농축
US6867041B2 (en) 2000-02-24 2005-03-15 Xcyte Therapies, Inc. Simultaneous stimulation and concentration of cells
US6797514B2 (en) 2000-02-24 2004-09-28 Xcyte Therapies, Inc. Simultaneous stimulation and concentration of cells
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Ｃｎｎについての亜鉛フィンガー結合ドメイン
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
JPWO2010098429A1 (ja) 2009-02-27 2012-09-06 国立大学法人大阪大学 免疫アジュバント組成物、及びその利用
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
CN104411338A (zh) 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
EP2970985A1 (en) 2013-03-14 2016-01-20 Fred Hutchinson Cancer Research Center Compositions and methods to modify cells for therapeutic objectives
WO2016167957A1 (en) 2015-04-15 2016-10-20 Saint Louis University Tumor suppression by mcpip1
JP2017002928A (ja) 2015-06-05 2017-01-05 株式会社デンソー 複数軸駆動用アクチュエータ
WO2017002928A1 (ja) 2015-06-30 2017-01-05 岸本　忠三 新規な肺疾患治療剤および／またはそのスクリーニング方法
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
EP3389677B1 (en) * 2015-12-18 2024-06-26 Sangamo Therapeutics, Inc. Targeted disruption of the t cell receptor
MX2019013514A (es) * 2017-05-12 2020-01-20 Crispr Therapeutics Ag Materiales y metodos para modificar celulas por ingenieria genetica y usos de los mismos en inmunooncologia.
US11166985B2 (en) * 2017-05-12 2021-11-09 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
WO2019089884A2 (en) * 2017-11-01 2019-05-09 Editas Medicine, Inc. Methods, compositions and components for crispr-cas9 editing of tgfbr2 in t cells for immunotherapy
US20190284553A1 (en) * 2018-03-15 2019-09-19 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
CN112040987A (zh) 2018-03-15 2020-12-04 Ksq治疗公司 用于改进的免疫疗法的基因调控组合物和方法
MX2020012028A (es) 2018-05-11 2021-03-29 Crispr Therapeutics Ag Metodos y composiciones para tratar el cancer.
US20220125891A1 (en) 2018-06-06 2022-04-28 Osaka University Method for treating and/or preventing regnase-1-related disease
WO2020032160A1 (ja) 2018-08-09 2020-02-13 国立大学法人大阪大学 炎症性腸疾患治療薬およびそのスクリーニング方法
CN110904045A (zh) * 2018-09-17 2020-03-24 中国科学院动物研究所 经修饰的t细胞、其制备方法及用途
BR112021008041A2 (pt) 2018-11-07 2021-08-10 Crispr Therapeutics Ag terapia contra o câncer com células imunitárias anti-cd33
EP3911735A4 (en) * 2019-01-16 2023-07-12 Beam Therapeutics, Inc. MODIFIED IMMUNE CELLS WITH INCREASED ANTINEOPLASTIC ACTIVITY AND RESISTANCE TO IMMUNOSUPPRESSION
MX2021013359A (es) * 2019-04-30 2022-01-31 Crispr Therapeutics Ag Terapia de celulas alogénicas de neoplasias malignas de células b usando células t modificadas genéticamente dirigidas a cd19.
JP6957572B2 (ja) 2019-09-19 2021-11-02 フランスベッド株式会社 ベッド装置
CN114929862A (zh) 2019-11-13 2022-08-19 克里斯珀医疗股份公司 用于制备表达嵌合抗原受体的t细胞的生产方法

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