CA3157027A1 - Methods and compositions for modification of a t-cell receptor gene - Google Patents
Methods and compositions for modification of a t-cell receptor gene Download PDFInfo
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| CA2993567A CA2993567C (en) | 2010-07-21 | 2011-07-21 | Methods and compositions for modification of a t-cell receptor gene |
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Families Citing this family (184)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7888121B2 (en) * | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US11311574B2 (en) | 2003-08-08 | 2022-04-26 | Sangamo Therapeutics, Inc. | Methods and compositions for targeted cleavage and recombination |
| RU2568056C2 (ru) | 2009-10-22 | 2015-11-10 | ДАУ АГРОСАЙЕНСИЗ ЭлЭлСи | Конструируемые белки с цинковыми пальцами, направленные на гены растений, вовлеченные в биосинтез жирных кислот |
| US12492376B2 (en) | 2009-10-29 | 2025-12-09 | The Trustees Of Dartmouth College | T-cell receptor-deficient T cell compositions |
| WO2011059836A2 (en) | 2009-10-29 | 2011-05-19 | Trustees Of Dartmouth College | T cell receptor-deficient t cell compositions |
| US9273283B2 (en) * | 2009-10-29 | 2016-03-01 | The Trustees Of Dartmouth College | Method of producing T cell receptor-deficient T cells expressing a chimeric receptor |
| US20140134195A1 (en) | 2011-04-20 | 2014-05-15 | University Of Washington Through Its Center For Commercialization | Beta-2 microglobulin-deficient cells |
| JP6072788B2 (ja) * | 2011-07-25 | 2017-02-01 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 嚢胞性線維症膜コンダクタンス制御因子（ｃｆｔｒ）遺伝子を改変するための方法および組成物 |
| WO2013033626A2 (en) | 2011-08-31 | 2013-03-07 | Trustees Of Dartmouth College | Nkp30 receptor targeted therapeutics |
| WO2013074916A1 (en) | 2011-11-18 | 2013-05-23 | Board Of Regents, The University Of Texas System | Car+ t cells genetically modified to eliminate expression of t- cell receptor and/or hla |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
| AU2013249820C1 (en) * | 2012-04-17 | 2018-10-25 | University Of Washington Through Its Center For Commercialization | HLA Class II Deficient Cells, HLA Class I Deficient Cells Capable of Expressing HLA Class II Proteins, and Uses Thereof |
| KR101904508B1 (ko) * | 2012-04-25 | 2018-10-04 | 리제너론 파마슈티칼스 인코포레이티드 | 큰 표적화 벡터를 사용한 뉴클레아제-중재된 표적화 |
| EP3505537A1 (en) | 2012-05-07 | 2019-07-03 | Trustees of Dartmouth College | Anti-b7-h6 antibody, fusion proteins, and methods of using the same |
| CN111676196A (zh) | 2012-05-25 | 2020-09-18 | 塞勒克提斯公司 | 工程化异体和免疫抑制耐受性t细胞的方法 |
| US20150017136A1 (en) * | 2013-07-15 | 2015-01-15 | Cellectis | Methods for engineering allogeneic and highly active t cell for immunotherapy |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| ES2824024T3 (es) | 2012-10-10 | 2021-05-11 | Sangamo Therapeutics Inc | Compuestos modificadores de células T y usos de los mismos |
| US20140115728A1 (en) | 2012-10-24 | 2014-04-24 | A. Joseph Tector | Double knockout (gt/cmah-ko) pigs, organs and tissues |
| EP4481048A3 (en) | 2012-12-17 | 2025-02-26 | President and Fellows of Harvard College | Rna-guided human genome engineering |
| EP2970886B1 (en) * | 2013-03-12 | 2018-05-23 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of hla |
| US9587237B2 (en) | 2013-03-14 | 2017-03-07 | Elwha Llc | Compositions, methods, and computer systems related to making and administering modified T cells |
| US9499855B2 (en) | 2013-03-14 | 2016-11-22 | Elwha Llc | Compositions, methods, and computer systems related to making and administering modified T cells |
| JP6346266B2 (ja) * | 2013-03-21 | 2018-06-20 | サンガモ セラピューティクス， インコーポレイテッド | 操作されたジンクフィンガータンパク質ヌクレアーゼを使用するｔ細胞受容体遺伝子の標的化された破壊 |
| CA3185368A1 (en) * | 2013-04-03 | 2014-10-09 | Memorial Sloan-Kettering Cancer Center | Effective generation of tumor-targeted t cells derived from pluripotent stem cells |
| RS62263B1 (sr) | 2013-04-16 | 2021-09-30 | Regeneron Pharma | Ciljana modifikacija genoma pacova |
| US11077144B2 (en) | 2013-05-13 | 2021-08-03 | Cellectis | CD19 specific chimeric antigen receptor and uses thereof |
| EP2997134B1 (en) | 2013-05-14 | 2020-06-24 | Board of Regents, The University of Texas System | Human application of engineered chimeric antigen receptor (car) t-cells |
| EP3004168A4 (en) | 2013-05-24 | 2017-03-01 | Board of Regents, The University of Texas System | Chimeric antigen receptor-targeting monoclonal antibodies |
| WO2015057976A1 (en) | 2013-10-17 | 2015-04-23 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
| RU2725520C2 (ru) | 2013-12-11 | 2020-07-02 | Регенерон Фармасьютикалс, Инк. | Способы и композиции для направленной модификации генома |
| EP4026909A1 (en) | 2013-12-19 | 2022-07-13 | Novartis AG | Human mesothelin chimeric antigen receptors and uses thereof |
| WO2015105955A1 (en) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| CA2937711C (en) | 2014-02-14 | 2020-10-20 | Cellectis | Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells |
| MX369513B (es) | 2014-02-14 | 2019-11-11 | Univ Texas | Receptores de antígenos quiméricos y métodos de realización. |
| KR102228828B1 (ko) | 2014-03-11 | 2021-03-16 | 셀렉티스 | 동종이형 이식에 양립성인 t-세포들을 만들어내는 방법 |
| EP3593812A3 (en) | 2014-03-15 | 2020-05-27 | Novartis AG | Treatment of cancer using chimeric antigen receptor |
| MX370272B (es) * | 2014-03-19 | 2019-12-09 | Cellectis | Receptores de antigeno quimerico especifico de cd123 para inmunoterapia de cancer. |
| SI3888674T1 (sl) | 2014-04-07 | 2024-08-30 | Novartis Ag | Zdravljenje raka z uporabo antigenskega himernega receptorja proti-CD19 |
| WO2015155341A1 (en) | 2014-04-11 | 2015-10-15 | Cellectis | Method for generating immune cells resistant to arginine and/or tryptophan depleted microenvironment |
| JP6698546B2 (ja) * | 2014-04-14 | 2020-05-27 | セレクティスＣｅｌｌｅｃｔｉｓ | 癌免疫療法のためのｂｃｍａ（ｃｄ２６９）特異的キメラ抗原受容体 |
| CN106459918A (zh) * | 2014-04-24 | 2017-02-22 | 得克萨斯州大学系统董事会 | 施加诱导多能干细胞产生过继细胞疗法产品 |
| JP6688231B2 (ja) | 2014-06-06 | 2020-04-28 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | 標的遺伝子座を修飾するための方法及び組成物 |
| EP3161128B1 (en) | 2014-06-26 | 2018-09-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modifications and methods of use |
| GB201412494D0 (en) | 2014-07-14 | 2014-08-27 | Ospedale San Raffaele And Fond Telethon | Vector production |
| JP6831777B2 (ja) | 2014-07-21 | 2021-02-17 | ノバルティス アーゲー | Ｃｄ３３キメラ抗原受容体を使用する癌の処置 |
| JP7054622B2 (ja) | 2014-07-21 | 2022-04-14 | ノバルティス アーゲー | ヒト化抗ｂｃｍａキメラ抗原受容体を使用した癌の処置 |
| US11542488B2 (en) | 2014-07-21 | 2023-01-03 | Novartis Ag | Sortase synthesized chimeric antigen receptors |
| EP3193915A1 (en) | 2014-07-21 | 2017-07-26 | Novartis AG | Combinations of low, immune enhancing. doses of mtor inhibitors and cars |
| US9757420B2 (en) | 2014-07-25 | 2017-09-12 | Sangamo Therapeutics, Inc. | Gene editing for HIV gene therapy |
| US9616090B2 (en) | 2014-07-30 | 2017-04-11 | Sangamo Biosciences, Inc. | Gene correction of SCID-related genes in hematopoietic stem and progenitor cells |
| WO2016019300A1 (en) | 2014-07-31 | 2016-02-04 | Novartis Ag | Subset-optimized chimeric antigen receptor-containing t-cells |
| AU2015301460B2 (en) | 2014-08-14 | 2021-04-08 | Novartis Ag | Treatment of cancer using GFR alpha-4 chimeric antigen receptor |
| WO2016028896A1 (en) | 2014-08-19 | 2016-02-25 | Novartis Ag | Anti-cd123 chimeric antigen receptor (car) for use in cancer treatment |
| WO2016044416A1 (en) | 2014-09-16 | 2016-03-24 | Sangamo Biosciences, Inc. | Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells |
| US10577417B2 (en) | 2014-09-17 | 2020-03-03 | Novartis Ag | Targeting cytotoxic cells with chimeric receptors for adoptive immunotherapy |
| WO2016081518A2 (en) | 2014-11-17 | 2016-05-26 | Adicet Bio, Inc. | Engineered gamma delta t-cells |
| IL301900A (en) | 2014-11-21 | 2023-06-01 | Regeneron Pharma | Methods and compositions for targeted genetic modification using paired guide rnas |
| EP3025719B1 (en) | 2014-11-26 | 2018-09-26 | Miltenyi Biotec GmbH | Combination immunotherapy of antigen-recognizing receptors and hematopoietic cells for the treatment of diseases |
| US10889834B2 (en) | 2014-12-15 | 2021-01-12 | Sangamo Therapeutics, Inc. | Methods and compositions for enhancing targeted transgene integration |
| EP3653048B9 (en) | 2014-12-19 | 2023-10-04 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification through single-step multiple targeting |
| WO2016112779A1 (en) * | 2015-01-17 | 2016-07-21 | Zhejiang University | Modified cells evoking reduced immunogenic responses |
| WO2016118726A2 (en) | 2015-01-21 | 2016-07-28 | Sangamo Biosciences, Inc. | Methods and compositions for identification of highly specific nucleases |
| SG10202101358XA (en) | 2015-01-26 | 2021-03-30 | Fate Therapeutics Inc | Methods and compositions for inducing hematopoietic cell differentiation |
| EP3286211A1 (en) | 2015-04-23 | 2018-02-28 | Novartis AG | Treatment of cancer using chimeric antigen receptor and protein kinase a blocker |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US10179918B2 (en) | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
| US10808020B2 (en) * | 2015-05-12 | 2020-10-20 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| EP3313176A4 (en) * | 2015-06-26 | 2019-06-26 | Indiana University Research&technology Corporation | TRANSGENIC PIGS WITH GENETIC MODIFICATIONS OF SLA |
| AU2016291778B2 (en) | 2015-07-13 | 2021-05-06 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| BR112018002600A2 (pt) | 2015-08-11 | 2018-10-23 | Cellectis | células para imunoterapia modificadas para direcionamento ao antígeno cd38 e para inativação do gene cd38 |
| PT3352776T (pt) | 2015-09-23 | 2025-06-27 | Sangamo Therapeutics Inc | Repressores de htt e utilizações dos mesmos |
| JP6816133B2 (ja) | 2015-10-05 | 2021-01-20 | プレシジョン バイオサイエンシズ，インク． | 改変ヒトｔ細胞受容体アルファ定常領域遺伝子を含む遺伝子改変細胞 |
| US10799535B2 (en) * | 2015-10-05 | 2020-10-13 | Precision Biosciences, Inc. | Engineered meganucleases with recognition sequences found in the human T cell receptor alpha constant region gene |
| JP7017506B2 (ja) | 2015-10-16 | 2022-02-08 | ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク | 系統特異的抗原の阻害のための組成物および方法 |
| EP3368664A4 (en) | 2015-10-28 | 2019-08-28 | Sangamo Therapeutics, Inc. | LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF |
| US10858628B2 (en) | 2015-11-04 | 2020-12-08 | Fate Therapeutics, Inc. | Methods and compositions for inducing hematopoietic cell differentiation |
| ES2953925T3 (es) | 2015-11-04 | 2023-11-17 | Fate Therapeutics Inc | Ingeniería genómica de células pluripotentes |
| AU2016361350B2 (en) | 2015-11-23 | 2023-04-06 | Sangamo Therapeutics, Inc. | Methods and compositions for engineering immunity |
| SG10202109655VA (en) | 2015-12-04 | 2021-10-28 | Novartis Ag | Compositions and methods for immunooncology |
| WO2017106528A2 (en) | 2015-12-18 | 2017-06-22 | Sangamo Biosciences, Inc. | Targeted disruption of the t cell receptor |
| KR20180088911A (ko) | 2015-12-18 | 2018-08-07 | 상가모 테라퓨틱스, 인코포레이티드 | Mhc 세포 수용체의 표적화된 붕괴 |
| CN109072195A (zh) | 2015-12-30 | 2018-12-21 | 诺华股份有限公司 | 具有增强功效的免疫效应细胞疗法 |
| ES2895430T3 (es) | 2016-01-15 | 2022-02-21 | Univ Minnesota | Métodos y composiciones para el tratamiento de enfermedad neurológica |
| JP7012650B2 (ja) | 2016-02-02 | 2022-01-28 | サンガモ セラピューティクス， インコーポレイテッド | Ｄｎａ結合ドメインと切断ドメインとを連結するための組成物 |
| ES2933961T3 (es) | 2016-04-15 | 2023-02-15 | Memorial Sloan Kettering Cancer Center | Células T transgénicas y composiciones de células T de receptor de antígeno quimérico y métodos relacionados |
| PL3443096T3 (pl) | 2016-04-15 | 2023-06-19 | Novartis Ag | Kompozycje i sposoby do selektywnej ekspresji chimerycznych receptorów antygenowych |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| EP3454870A4 (en) | 2016-05-12 | 2020-03-04 | Adicet Bio Inc. | METHOD FOR SELECTIVE EXPANSION OF GAMMA DELTA T CELL POPULATIONS AND COMPOSITIONS THEREOF |
| JP7324583B2 (ja) | 2016-05-20 | 2023-08-10 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 複数のガイドｒｎａを使用して免疫寛容を破綻させるための方法 |
| AU2017295886C1 (en) | 2016-07-15 | 2024-05-16 | Novartis Ag | Treatment and prevention of cytokine release syndrome using a chimeric antigen receptor in combination with a kinase inhibitor |
| HUE056707T2 (hu) | 2016-08-24 | 2022-03-28 | Sangamo Therapeutics Inc | Génexpresszió szabályozása szerkezeti nukleázok alkalmazásával |
| KR20220145913A (ko) | 2016-08-24 | 2022-10-31 | 상가모 테라퓨틱스, 인코포레이티드 | 가공된 표적 특이적 뉴클레아제 |
| CA3035534A1 (en) | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| US20190203230A1 (en) | 2016-09-28 | 2019-07-04 | Novartis Ag | Porous membrane-based macromolecule delivery system |
| EP4667565A2 (en) | 2016-10-20 | 2025-12-24 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of fabry disease |
| WO2018081531A2 (en) | 2016-10-28 | 2018-05-03 | Ariad Pharmaceuticals, Inc. | Methods for human t-cell activation |
| WO2018081775A1 (en) | 2016-10-31 | 2018-05-03 | Sangamo Therapeutics, Inc. | Gene correction of scid-related genes in hematopoietic stem and progenitor cells |
| EP3535396A1 (en) | 2016-11-01 | 2019-09-11 | Novartis AG | Methods and compositions for enhancing gene editing |
| EP3346001A1 (en) | 2017-01-06 | 2018-07-11 | TXCell | Monospecific regulatory t cell population with cytotoxicity for b cells |
| WO2018127585A1 (en) | 2017-01-06 | 2018-07-12 | Txcell | Monospecific regulatory t cell population with cytotoxicity for b cells |
| US11535662B2 (en) | 2017-01-26 | 2022-12-27 | Novartis Ag | CD28 compositions and methods for chimeric antigen receptor therapy |
| BR112019017751A2 (pt) | 2017-02-28 | 2020-04-07 | Vor Biopharma, Inc. | composições e métodos de inibição de proteínas de linhagem específica |
| EP3589647A1 (en) | 2017-02-28 | 2020-01-08 | Novartis AG | Shp inhibitor compositions and uses for chimeric antigen receptor therapy |
| JP2020513832A (ja) | 2017-03-22 | 2020-05-21 | ノバルティス アーゲー | 免疫腫瘍学のための組成物および方法 |
| CN116555353A (zh) | 2017-04-20 | 2023-08-08 | E开创生物技术股份有限公司 | 产生基因修改的动物的方法 |
| EP3615055A1 (en) | 2017-04-28 | 2020-03-04 | Novartis AG | Cells expressing a bcma-targeting chimeric antigen receptor, and combination therapy with a gamma secretase inhibitor |
| CN116693411A (zh) | 2017-04-28 | 2023-09-05 | 爱康泰生治疗公司 | 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂 |
| US20200179511A1 (en) | 2017-04-28 | 2020-06-11 | Novartis Ag | Bcma-targeting agent, and combination therapy with a gamma secretase inhibitor |
| CA3059793A1 (en) | 2017-05-03 | 2018-11-08 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (cftr) gene |
| US11512287B2 (en) | 2017-06-16 | 2022-11-29 | Sangamo Therapeutics, Inc. | Targeted disruption of T cell and/or HLA receptors |
| US11053484B2 (en) | 2017-06-30 | 2021-07-06 | Precision Biosciences, Inc. | Genetically-modified T cells comprising a modified intron in the T cell receptor alpha gene |
| WO2019003193A1 (en) | 2017-06-30 | 2019-01-03 | Novartis Ag | METHODS FOR TREATING DISEASES USING GENE EDITING SYSTEMS |
| JP7281774B2 (ja) | 2017-09-19 | 2023-05-26 | ザ・ユニバーシティ・オブ・ブリティッシュ・コロンビア | 抗ｈｌａ－ａ２抗体及びその使用方法 |
| JP7654240B2 (ja) | 2017-09-19 | 2025-04-01 | マサチューセッツ インスティテュート オブ テクノロジー | キメラ抗原受容体ｔ細胞治療のための組成物およびその使用 |
| IL273401B2 (en) | 2017-09-20 | 2025-05-01 | Univ British Columbia | Novel antibodies against human leukocyte antigens type A2 and their uses |
| WO2019089798A1 (en) | 2017-10-31 | 2019-05-09 | Novartis Ag | Anti-car compositions and methods |
| JP2021502085A (ja) | 2017-11-09 | 2021-01-28 | サンガモ セラピューティクス， インコーポレイテッド | サイトカイン誘導性ｓｈ２含有タンパク質（ｃｉｓｈ）遺伝子の遺伝子改変 |
| IL274640B1 (en) | 2017-11-15 | 2025-10-01 | Adicet Bio Inc | Methods for selective expansion of delta 3 gamma-delta T-cell populations and preparations thereof |
| CN111601882A (zh) | 2017-11-22 | 2020-08-28 | 拉霍拉敏感及免疫学研究所 | 工程化的免疫细胞的应用和生产 |
| BR112020009889A2 (pt) | 2017-12-14 | 2020-11-03 | Flodesign Sonics, Inc. | acionador e controlador de transdutor acústico |
| US20190201548A1 (en) | 2017-12-29 | 2019-07-04 | Rubius Therapeutics, Inc. | Gene editing and targeted transcriptional modulation for engineering erythroid cells |
| AU2019216269B2 (en) | 2018-01-30 | 2025-05-01 | Cellectis | Combination comprising allogeneic immune cells deficient for an antigen present on both T-cells and pathological cells and therapeutic antibody against said antigen |
| RU2020129448A (ru) | 2018-02-08 | 2022-03-09 | Сангамо Терапьютикс, Инк. | Сконструированные специфические для мишени нуклеазы |
| CA3095084A1 (en) | 2018-04-05 | 2019-10-10 | Juno Therapeutics, Inc. | T cells expressing a recombinant receptor, related polynucleotides and methods |
| KR20210029707A (ko) | 2018-04-05 | 2021-03-16 | 주노 쎄러퓨티크스 인코퍼레이티드 | 재조합 수용체를 발현하는 세포의 생산 방법 및 관련 조성물 |
| IL304025A (en) | 2018-04-12 | 2023-08-01 | Prec Biosciences Inc | Optimized engineered nucleases with specificity for the human T cell receptor alpha constant region gene |
| US11981739B2 (en) | 2018-04-13 | 2024-05-14 | Sangamo Therapeutics France | Chimeric antigen receptor specific for interleukin-23 receptor |
| CN112313246B (zh) | 2018-04-18 | 2025-05-13 | 桑格摩生物治疗股份有限公司 | 用于调节亨廷顿蛋白(htt)的锌指蛋白组合物 |
| WO2019210153A1 (en) | 2018-04-27 | 2019-10-31 | Novartis Ag | Car t cell therapies with enhanced efficacy |
| US11690921B2 (en) | 2018-05-18 | 2023-07-04 | Sangamo Therapeutics, Inc. | Delivery of target specific nucleases |
| EP3802825A1 (en) | 2018-06-08 | 2021-04-14 | Intellia Therapeutics, Inc. | Compositions and methods for immunooncology |
| CN112203725A (zh) | 2018-06-13 | 2021-01-08 | 诺华股份有限公司 | Bcma嵌合抗原受体及其用途 |
| US20210292389A1 (en) | 2018-08-10 | 2021-09-23 | Sangamo Therapeutics France | New car constructs comprising tnfr2 domains |
| KR102712986B1 (ko) | 2018-08-23 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 조작된 표적 특이적 염기 편집기 |
| CN113423725A (zh) | 2018-08-28 | 2021-09-21 | Vor生物制药股份有限公司 | 遗传工程化造血干细胞及其用途 |
| JP2022500052A (ja) | 2018-09-18 | 2022-01-04 | サンガモ セラピューティクス， インコーポレイテッド | プログラム細胞死１（ｐｄ１）特異的ヌクレアーゼ |
| US12329857B2 (en) | 2018-09-21 | 2025-06-17 | Acuitas Therapeutics, Inc. | Systems and methods for manufacturing lipid nanoparticles and liposomes |
| SG11202100935TA (en) | 2018-09-28 | 2021-02-25 | Massachusetts Inst Technology | Collagen-localized immunomodulatory molecules and methods thereof |
| AU2020205717B2 (en) | 2019-01-11 | 2025-09-11 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| BR112021019448A2 (pt) | 2019-04-02 | 2021-11-30 | Sangamo Therapeutics Inc | Métodos para o tratamento de beta-talassemia |
| US20220251152A1 (en) | 2019-04-24 | 2022-08-11 | Novartis Ag | Compositions and methods for selective protein degradation |
| JP7754722B2 (ja) | 2019-05-01 | 2025-10-15 | ジュノー セラピューティクス インコーポレイテッド | 改変されたｃｄ２４７遺伝子座からキメラ受容体を発現する細胞、関連ポリヌクレオチド、および方法 |
| MA55811A (fr) | 2019-05-01 | 2022-03-09 | Editas Medicine Inc | Cellules exprimant un récepteur recombinant à base d'un locus modifié du tgfbr2, et polynucléotides et méthodes associés |
| US11642409B2 (en) | 2019-06-26 | 2023-05-09 | Massachusetts Insttute of Technology | Immunomodulatory fusion protein-metal hydroxide complexes and methods thereof |
| WO2021028359A1 (en) | 2019-08-09 | 2021-02-18 | Sangamo Therapeutics France | Controlled expression of chimeric antigen receptors in t cells |
| EP3808766A1 (en) | 2019-10-15 | 2021-04-21 | Sangamo Therapeutics France | Chimeric antigen receptor specific for interleukin-23 receptor |
| CN114761560A (zh) * | 2019-11-25 | 2022-07-15 | 国立大学法人京都大学 | T细胞主细胞库 |
| CN115485300A (zh) | 2020-02-26 | 2022-12-16 | 索伦托药业有限公司 | 具有通用掩蔽部分的可活化的抗原结合蛋白 |
| CA3173527A1 (en) | 2020-03-10 | 2021-09-16 | Massachusetts Institute Of Technology | Methods for generating engineered memory-like nk cells and compositions thereof |
| US11896619B2 (en) | 2020-03-10 | 2024-02-13 | Massachusetts Institute Of Technology | Compositions and methods for immunotherapy of NPM1c-positive cancer |
| JP2023518538A (ja) | 2020-03-20 | 2023-05-02 | インセルム（インスティチュート ナショナル デ ラ サンテ エ デ ラ リシェルシェ メディカル） | ヒトｃｄ４５ｒｃに特異的なキメラ抗原受容体およびその使用 |
| EP4127143A1 (en) * | 2020-04-03 | 2023-02-08 | Progenitor Life Sciences | Targeting tapasin and tap complex to improve cellular immune-compatibility |
| US20230167191A1 (en) | 2020-04-24 | 2023-06-01 | Sorrento Therapeutics, Inc. | Memory Dimeric Antigen Receptors (mDARs) |
| US20210340524A1 (en) | 2020-05-01 | 2021-11-04 | Massachusetts Institute Of Technology | Methods for identifying chimeric antigen receptor-targeting ligands and uses thereof |
| US12433954B2 (en) | 2020-05-01 | 2025-10-07 | Massachusetts Institute Of Technology | Methods of activating anti-CD19 chimeric antigen receptor (CAR) T cells using amphiphilic ligand conjugates comprising CAR-targeting protein sequence motifs |
| US20230181641A1 (en) | 2020-05-13 | 2023-06-15 | Juno Therapeutics, Inc. | Process for producing donor-batched cells expressing a recombinant receptor |
| CA3185455A1 (en) | 2020-06-11 | 2021-12-16 | Novartis Ag | Zbtb32 inhibitors and uses thereof |
| US20230227901A1 (en) | 2020-06-26 | 2023-07-20 | The Regents Of The University Of California | Selective Addition of Reagents to Droplets |
| EP4171616A1 (en) | 2020-06-26 | 2023-05-03 | Juno Therapeutics GmbH | Engineered t cells conditionally expressing a recombinant receptor, related polynucleotides and methods |
| AU2021308681A1 (en) | 2020-07-16 | 2023-03-09 | Acuitas Therapeutics, Inc. | Cationic lipids for use in lipid nanoparticles |
| JP2023549780A (ja) | 2020-11-04 | 2023-11-29 | ジュノー セラピューティクス インコーポレイテッド | 改変されたインバリアントｃｄ３免疫グロブリンスーパーファミリー鎖遺伝子座からキメラ受容体を発現する細胞ならびに関連するポリヌクレオチドおよび方法 |
| US20240033358A1 (en) | 2020-11-13 | 2024-02-01 | Novartis Ag | Combination therapies with chimeric antigen receptor (car)-expressing cells |
| AR125199A1 (es) | 2021-03-23 | 2023-06-21 | Iovance Biotherapeutics Inc | Edición génica cish de linfocitos infiltrantes de tumores y usos de los mismos en inmunoterapia |
| AU2022277649A1 (en) | 2021-05-21 | 2023-11-30 | Cellectis S.A. | Enhancing efficacy of t-cell-mediated immunotherapy by modulating cancer-associated fibroblasts in solid tumors |
| CN117597439A (zh) * | 2021-05-24 | 2024-02-23 | 桑格摩生物治疗股份有限公司 | Ciita靶向锌指核酸酶 |
| WO2023035011A1 (en) | 2021-09-03 | 2023-03-09 | North Carolina State University | Compositions and methods for conferring resistance to geminivirus |
| WO2023081715A1 (en) | 2021-11-03 | 2023-05-11 | Viracta Therapeutics, Inc. | Combination of car t-cell therapy with btk inhibitors and methods of use thereof |
| WO2023081900A1 (en) | 2021-11-08 | 2023-05-11 | Juno Therapeutics, Inc. | Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods |
| CN118632860A (zh) | 2021-11-24 | 2024-09-10 | 诺华股份有限公司 | 腺相关病毒转导调节剂及其用途 |
| KR20240123832A (ko) | 2021-12-16 | 2024-08-14 | 아퀴타스 테라퓨틱스 인크. | 지질 나노입자 제형에 사용하기 위한 지질 |
| EP4544058A1 (en) * | 2022-06-23 | 2025-04-30 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of ciita expression |
| WO2024003334A1 (en) | 2022-06-30 | 2024-01-04 | Cellectis S.A. | Enhancing safety of t-cell-mediated immunotherapy |
| WO2024086591A2 (en) * | 2022-10-17 | 2024-04-25 | The Regents Of The University Of California | Stem cell based delivery of tumor-specific retroviral vectors |
| WO2024094775A1 (en) | 2022-11-03 | 2024-05-10 | Cellectis S.A. | Enhancing efficacy and safety of t-cell-mediated immunotherapy |
| WO2024100604A1 (en) | 2022-11-09 | 2024-05-16 | Juno Therapeutics Gmbh | Methods for manufacturing engineered immune cells |
| WO2024102954A1 (en) | 2022-11-10 | 2024-05-16 | Massachusetts Institute Of Technology | Activation induced clipping system (aics) |
| CN121002052A (zh) | 2023-02-03 | 2025-11-21 | C3S2 有限公司 | 用于非病毒生产工程化免疫细胞的方法 |
| WO2025101877A1 (en) * | 2023-11-08 | 2025-05-15 | The Children's Hospital Of Philadelphia | Platform for increasing hla class i antigen presentation utilizing engineered chaperone fusion proteins |
| US20250345431A1 (en) | 2024-05-10 | 2025-11-13 | Juno Therapeutics, Inc. | Genetically engineered t cells expressing a cd19 chimeric antigen receptor (car) and uses thereof for allogeneic cell therapy |
| WO2025235862A1 (en) | 2024-05-10 | 2025-11-13 | Inndura Therapeutics Inc. | A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a |
Family Cites Families (69)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US789538A (en) | 1904-11-11 | 1905-05-09 | Colin E Ham | Dumb-bell. |
| US5420032A (en) | 1991-12-23 | 1995-05-30 | Universitge Laval | Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence |
| US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
| US5792632A (en) | 1992-05-05 | 1998-08-11 | Institut Pasteur | Nucleotide sequence encoding the enzyme I-SceI and the uses thereof |
| DE69534629D1 (de) | 1994-01-18 | 2005-12-29 | Scripps Research Inst | Derivate von zinkfingerproteinen und methoden |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| GB9824544D0 (en) | 1998-11-09 | 1999-01-06 | Medical Res Council | Screening system |
| DE69535829D1 (de) | 1994-08-20 | 2008-10-16 | Gendaq Ltd | Verbesserung in bezug auf bindungsproteine bei der erkennung von dna |
| US5789538A (en) | 1995-02-03 | 1998-08-04 | Massachusetts Institute Of Technology | Zinc finger proteins with high affinity new DNA binding specificities |
| US5925523A (en) | 1996-08-23 | 1999-07-20 | President & Fellows Of Harvard College | Intraction trap assay, reagents and uses thereof |
| GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
| GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
| 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 |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| AU746454B2 (en) | 1998-03-02 | 2002-05-02 | Massachusetts Institute Of Technology | Poly zinc finger proteins with improved linkers |
| 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 |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | 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 |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| AU776576B2 (en) | 1999-12-06 | 2004-09-16 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| KR20020086508A (ko) | 2000-02-08 | 2002-11-18 | 상가모 바이오사이언스 인코포레이티드 | 약물 발견용 세포 |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| WO2001088197A2 (en) | 2000-05-16 | 2001-11-22 | Massachusetts Institute Of Technology | Methods and compositions for interaction trap assays |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| EP1421177A4 (en) | 2001-08-20 | 2006-06-07 | Scripps Research Inst | ZINC FINGER FASTENING DOMAINS FOR CNN |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| WO2003087341A2 (en) | 2002-01-23 | 2003-10-23 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
| EP1504092B2 (en) | 2002-03-21 | 2014-06-25 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| US20030194704A1 (en) * | 2002-04-03 | 2003-10-16 | Penn Sharron Gaynor | Human genome-derived single exon nucleic acid probes useful for gene expression analysis two |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| US9447434B2 (en) | 2002-09-05 | 2016-09-20 | California Institute Of Technology | Use of chimeric nucleases to stimulate gene targeting |
| US7833706B2 (en) * | 2003-01-30 | 2010-11-16 | Celera Corporation | Genetic polymorphisms associated with rheumatoid arthritis, methods of detection and uses thereof |
| US20070134796A1 (en) | 2005-07-26 | 2007-06-14 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| 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 |
| WO2005099771A2 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Methods and compositions for treating neuropathic and neurodegenerative conditions |
| CA2562193A1 (en) | 2004-04-08 | 2005-10-27 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| CA2579677A1 (en) | 2004-09-16 | 2006-03-30 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| EP1877583A2 (en) | 2005-05-05 | 2008-01-16 | Arizona Board of Regents on behalf of the Unversity of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
| DK2628794T3 (en) | 2005-10-18 | 2016-08-15 | Prec Biosciences | RATIONALE CONSTRUCTED MECHANUCLEAS WITH CHANGED SEQUENCE SPECIFICITY AND DNA BINDING EFFICIENCY |
| GB0609121D0 (en) | 2006-05-09 | 2006-06-21 | Univ Birmingham | Peptide Therapy |
| JP2009537140A (ja) | 2006-05-19 | 2009-10-29 | サンガモ バイオサイエンシーズ， インコーポレイテッド | ジヒドロ葉酸還元酵素の不活性化のための方法及び組成物 |
| JP5551432B2 (ja) | 2006-05-25 | 2014-07-16 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 遺伝子不活性化のための方法と組成物 |
| EP2027262B1 (en) | 2006-05-25 | 2010-03-31 | Sangamo Biosciences Inc. | Variant foki cleavage half-domains |
| US20070283453A1 (en) * | 2006-05-31 | 2007-12-06 | Juan Carlos Rodriguez Cimadevilla | Animal models of tumor metastasis and toxicity |
| RU2355763C2 (ru) * | 2006-09-13 | 2009-05-20 | Закрытое акционерное общество "Научно-исследовательский институт Аджиномото-Генетика" (ЗАО АГРИ) | Мутантная ацетолактатсинтаза и способ продукции разветвленных l-аминокислот |
| DE602008003684D1 (de) * | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| EP2188384B1 (en) | 2007-09-27 | 2015-07-15 | Sangamo BioSciences, Inc. | Rapid in vivo identification of biologically active nucleases |
| JP2010540534A (ja) | 2007-09-28 | 2010-12-24 | イントレキソン コーポレーション | 生体治療分子の発現のための治療遺伝子スイッチ構築物およびバイオリアクター、ならびにその使用 |
| DE102007056956B4 (de) | 2007-11-27 | 2009-10-29 | Moosbauer, Peter, Dipl.-Ing.(FH) | Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung |
| AU2009238629C1 (en) * | 2008-04-14 | 2015-04-30 | Sangamo Therapeutics, Inc. | Linear donor constructs for targeted integration |
| US8772008B2 (en) | 2009-05-18 | 2014-07-08 | Sangamo Biosciences, Inc. | Methods and compositions for increasing nuclease activity |
| US8956828B2 (en) | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| ES2696825T3 (es) | 2009-12-10 | 2019-01-18 | Univ Minnesota | Modificación del ADN inducida por el efector TAL |
| EP2534173B1 (en) | 2010-02-08 | 2019-09-11 | Sangamo Therapeutics, Inc. | Engineered cleavage half-domains |
| WO2011139349A1 (en) | 2010-05-03 | 2011-11-10 | Sangamo Biosciences, Inc. | Compositions for linking zinc finger modules |
| EP2571512B1 (en) | 2010-05-17 | 2017-08-23 | Sangamo BioSciences, Inc. | Novel dna-binding proteins and uses thereof |
| US9591835B2 (en) * | 2011-10-28 | 2017-03-14 | Regeneron Pharmaceuticals, Inc. | Genetically modified major histocompatibility complex animals |
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- 2011-07-21 EP EP11810427.2A patent/EP2596011B1/en not_active Not-in-force
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- 2011-07-21 JP JP2013520872A patent/JP6050230B2/ja not_active Expired - Fee Related
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- 2011-07-21 US US13/188,417 patent/US8945868B2/en active Active
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| CA2993567A1 (en) | 2012-01-26 |
| US20120060230A1 (en) | 2012-03-08 |
| EP2596011A2 (en) | 2013-05-29 |
| CA2805442C (en) | 2020-05-12 |
| US8945868B2 (en) | 2015-02-03 |
| EP2596011A4 (en) | 2014-03-19 |
| CA2993567C (en) | 2022-06-28 |
| US20210054046A1 (en) | 2021-02-25 |
| JP6050230B2 (ja) | 2016-12-21 |
| JP2013538562A (ja) | 2013-10-17 |
| US10858416B2 (en) | 2020-12-08 |
| US20150252094A1 (en) | 2015-09-10 |
| US10072062B2 (en) | 2018-09-11 |
| EP2596011B1 (en) | 2018-10-03 |
| CA2805442A1 (en) | 2012-01-26 |
| WO2012012667A3 (en) | 2012-05-10 |
| AU2011281062A1 (en) | 2013-01-31 |
| WO2012012667A2 (en) | 2012-01-26 |
| AU2011281062B2 (en) | 2015-01-22 |
| US20180319864A1 (en) | 2018-11-08 |
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