公开(公告)号：US20210147868A1

公开(公告)日：2021-05-20

申请号：US16953473

申请日：2020-11-20

Applicant: Cellectis

Inventor： Philippe DUCHATEAU ,  André CHOULIKA ,  Laurent POIROT

IPC: C12N15/85 ,  A61K35/17 ,  C12N5/0783 ,  C12N9/22 ,  C12N15/90

Abstract: The present invention relates to methods of developing genetically engineered, preferably non-alloreactive T-cells for immunotherapy. This method involves the use of RNA-guided endonucleases, in particular Cas9/CRISPR system, to specifically target a selection of key genes in T-cells. The engineered T-cells are also intended to express chimeric antigen receptors (CAR) to redirect their immune activity towards malignant or infected cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies using T-Cells for treating cancer and viral infections.

公开(公告)号：US20200237823A1

公开(公告)日：2020-07-30

申请号：US16755082

申请日：2018-03-09

Applicant: CELLECTIS

Inventor： Brian BUSSER ,  Philippe DUCHATEAU ,  Alexandre JUILLERAT ,  Laurent POIROT ,  Julien VALTON

IPC: A61K35/17 ,  C07K16/28 ,  C07K16/30

Abstract: The invention pertains to the field of adaptive cell immunotherapy. It provides with the genetic insertion of exogenous coding sequence(s) that help the immune cells to direct their immune response against infected or malignant cells. These exogenous coding sequences are more particularly inserted under the transcriptional control of endogenous gene promoters that are sensitive to immune cells activation. Such method allows the production of safer immune primary cells of higher therapeutic potential.

公开(公告)号：US20200181643A1

公开(公告)日：2020-06-11

申请号：US16793918

申请日：2020-02-18

Applicant: Cellectis

Inventor： Philippe DUCHATEAU ,  André CHOULIKA ,  Laurent POIROT

IPC: C12N15/85 ,  C12N5/0783 ,  C12N15/90 ,  C12N9/22 ,  A61K35/17

Abstract: The present invention relates to methods of developing genetically engineered, preferably non-alloreactive T-cells for immunotherapy. This method involves the use of RNA-guided endonucleases, in particular Cas9/CRISPR system, to specifically target a selection of key genes in T-cells. The engineered T-cells are also intended to express chimeric antigen receptors (CAR) to redirect their immune activity towards malignant or infected cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies using T-Cells for treating cancer and viral infections.

公开(公告)号：US20190338015A1

公开(公告)日：2019-11-07

申请号：US16342139

申请日：2017-10-19

Applicant: CELLECTIS

Inventor： Alexandre JUILLERAT ,  Philippe DUCHATEAU ,  Laurent POIROT ,  Murielle DERRIEN ,  Donna Marie STONE ,  Javier Fernando CHAPARRO RIGGERS

IPC: C07K14/705 ,  C12N5/0783 ,  A61K35/17 ,  C07K14/725 ,  C07K16/28

Abstract: The invention relates to cell death inducing chimeric antigen receptors (D-CAR). In particular, the present invention relates to cell death inducing chimeric antigen receptors which comprise at least one death domain in their endodomain, including cell death inducing chimeric antigen receptors comprising within their death domains modifications which attenuate the self-association and/or binding to pro-apoptotic or pro-necrotic adaptor proteins, such as FADD or TRADD. Moreover, the present invention relates to an engineered immune cell expressing at its surface a cell death inducing CAR of the present invention and, optionally, an activating chimeric antigen receptor, wherein the extracellular ligand-binding domains of the cell death inducing CAR and the activating CAR bind to different antigens. The engineered immune cell may furthermore comprise at least one edited (e.g., inactivated) gene selected from TCR genes, immune check point genes, genes involved in drug resistance, and combinations thereof.

公开(公告)号：US20190328783A1

公开(公告)日：2019-10-31

申请号：US16092414

申请日：2017-04-13

Applicant: CELLECTIS

Inventor： Julien VALTON ,  Philippe DUCHATEAU ,  Laurent POIROT ,  Barbra Johnsson SASU ,  Arvind RAJPAL

IPC: A61K35/17 ,  C07K14/725 ,  C12N5/0783 ,  C12N15/85

Abstract: The present invention relates to therapeutic cells for immunotherapy to treat patients with cancer. In particular, the inventors develop a method of engineering prodrug-specific hypersensitive T-cell, which can be depleted in vivo by the administration of said specific prodrug in case of occurrence of a serious adverse event. The invention opens the way to safer and tunable adoptive immunotherapy strategies for treating cancer.

公开(公告)号：US20190151478A1

公开(公告)日：2019-05-23

申请号：US16092417

申请日：2017-04-13

Applicant: CELLECTIS

Inventor： Julien VALTON ,  Veronique ZENNOU ,  Philippe DUCHATEAU ,  Laurent POIROT

IPC: A61K48/00 ,  A61P35/00 ,  A61P31/00

Abstract: The present invention relates to therapeutic cells for immunotherapy to treat patients with cancer. In particular, the inventors develop a method of engineering drug-specific hypersensitive T-cell, which can be depleted in vivo by the administration of said specific drug in case of occurrence of a serious adverse even. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer.

公开(公告)号：US20180320138A1

公开(公告)日：2018-11-08

申请号：US15773960

申请日：2016-11-07

Applicant: CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED (CIBER) ,  CENTRO DE INVESTIGACIONES ENERGÉTICAS, MEDIOAMBIENTALES Y TECNOLÓGICAS ,  FUNDACIÓN INSTITUTO DE INVESTIGACIÓ SANITARIA FUNDACIÓN JIMÉNEZ DÍAZ ,  CELLECTIS

Inventor： José Carlos SEGOVIA SANZ ,  Oscar QUINTANA BUSTAMANTE ,  Zita Maite GÁRATE MUTILOA ,  Juan Antonio BUEREN RONCERO ,  Brian R. DAVIS ,  Roman GALETTO ,  Agnes GOUBLE ,  Laurent POIROT

IPC: C12N5/0789 ,  C12N5/074

CPC classification number: C12N5/0647 ,  C12N5/0696 ,  C12N2500/25 ,  C12N2501/105 ,  C12N2501/113 ,  C12N2501/115 ,  C12N2501/125 ,  C12N2501/14 ,  C12N2501/145 ,  C12N2501/155 ,  C12N2501/16 ,  C12N2501/165 ,  C12N2501/22 ,  C12N2501/2303 ,  C12N2501/2311 ,  C12N2501/26 ,  C12N2501/415 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2501/727 ,  C12N2501/91 ,  C12N2506/11 ,  C12N2506/45 ,  C12N2510/00

Abstract: The present invention relates to the medical field, in particular to gene editing as a therapeutic approach for the treatment of metabolic diseases affecting the erythroid lineage in a mammalian subject. In invention particular embodiment it refers to the combination of cell reprograming and gene editing for PKD correction as a first example of the potential application of these advanced technologies to metabolic diseases affecting the erythroid lineage. In this sense, PKD patient-specific iPSCs were efficiently generated from PB-MNCs (peripheral blood mononuclear cells) by a SeV non-integrative system and efficiently use to treat pyruvate kinase deficiency. The gene editing strategy for PKLR gene correction was also successfully applied directly to hematopoietic progenitors.

公开(公告)号：US20180291343A1

公开(公告)日：2018-10-11

申请号：US15556558

申请日：2016-03-11

Applicant: Cellectis

Inventor： Philippe DUCHATEAU ,  Jean-Pierre CABANIOLS ,  Julien VALTON ,  Laurent POIROT

IPC: C12N5/0783 ,  C07K16/28 ,  C07K14/705 ,  C12N15/11 ,  C12N9/22 ,  C07K14/74 ,  C07K14/005 ,  A61K35/17 ,  C12N5/00

Abstract: The present invention relates to methods for developing engineered immune cells such as T-cells for immunotherapy that have a higher potential of persistence and/or engraftment in host organism. IN particular, this method involves an inactivation of at least one gene involved in self/non self recognition, combined with a step of contact with at least one non-endogenous immunosuppressive polypeptide. The invention allows the possibility for a standard and affordable adoptive immunotherapy, whereby the risk of GvH is reduced.

公开(公告)号：US20160120905A1

公开(公告)日：2016-05-05

申请号：US14889686

申请日：2014-05-13

Applicant: CELLECTIS

Inventor： Roman GALETTO ,  Agnes GOUBLE ,  Stephanie GROSSE ,  Cécile SCHIFFER-MANNIOUI ,  Laurent POIROT ,  Andrew SCHARENBERG ,  Julianne SMITH

IPC: A61K35/17 ,  C12N5/0783

CPC classification number: A61K35/17 ,  A61K38/00 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70521 ,  C07K14/70578 ,  C07K16/28 ,  C07K16/2803 ,  C07K2317/14 ,  C07K2317/24 ,  C07K2317/569 ,  C07K2317/622 ,  C07K2319/00 ,  C07K2319/03 ,  C07K2319/74 ,  C12N5/0636 ,  C12N2501/39 ,  C12N2501/51 ,  C12N2501/515 ,  C12N2501/599 ,  C12N2502/99 ,  C12N2510/00

Abstract: The present invention relates to methods for developing engineered T-cells for immunotherapy that are non-alloreactive. The present invention relates to methods for modifying T-cells by inactivating both genes encoding T-cell receptor and an immune checkpoint gene to unleash the potential of the immune response. This method involves the use of specific rare cutting endonucleases, in particular TALE-nucleases (TAL effector endonuclease) and polynucleotides encoding such polypeptides, to precisely target a selection of key genes in T-cells, which are available from donors or from culture of primary cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer and viral infections.

Abstract translation: 本发明涉及用于开发非同种异体反应的用于免疫治疗的工程化T细胞的方法。 本发明涉及通过使编码T细胞受体的两个基因和免疫检查点基因失活来释放免疫应答的潜力来修饰T细胞的方法。 该方法包括使用特定的稀有切割内切核酸酶，特别是TALE-核酸酶（TAL效应子内切核酸酶）和编码这种多肽的多核苷酸，以精确地靶向T细胞中的关键基因的选择，其可从供体或从原代培养获得 细胞。 本发明开启了治疗癌症和病毒感染的标准和负担得起的过继免疫治疗策略的方法。

公开(公告)号：US20230279440A1

公开(公告)日：2023-09-07

申请号：US17996701

申请日：2021-05-06

Applicant: CELLECTIS S.A.

Inventor： Alexandre JUILLERAT ,  Philippe DUCHATEAU ,  Patrick HONG ,  Laurent POIROT ,  Brian BUSSER ,  Alex BOYNE

IPC: C12N15/90 ,  C12N15/86

CPC classification number: C12N15/907 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/42

Abstract: The present disclosure provides methods to genetically modify cells by insertion of an artificial exon (ArtEx) for delivery of therapeutic proteins in specific cell types and more particularly engineered cells for expression of a transgene into the brain of a patient.