公开(公告)号：US20160333094A1

公开(公告)日：2016-11-17

申请号：US15111441

申请日：2015-01-14

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Julien VALTON

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

CPC classification number: C07K16/2803 ,  A61K35/17 ,  A61K38/00 ,  A61K39/001112 ,  A61K2039/5156 ,  A61K2039/804 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70578 ,  C07K2317/20 ,  C07K2317/24 ,  C07K2317/53 ,  C07K2317/565 ,  C07K2317/569 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/33 ,  C07K2319/74 ,  C12N5/0636 ,  C12N2510/00

Abstract: The present invention relates to a new generation of chimeric antigen receptors (CAR), under single-chain or multi-chain forms, the specificity of which, to a desired antigen, is conferred by a VNAR polypeptide derived from monomeric antibodies from cartilaginous fish. Such CARs, which aim to redirect immune cell specificity toward selected undesired malignant cells, are compact and thus particularly adapted to target hollow antigens such as ions channels of efflux pumps present at the surface of drug-resistant cells. The invention encompasses the polynucleotides, vectors encoding said multi-chain CAR and the isolated cells expressing them at their surface, in particularly for their use in immunotherapy.

Abstract translation: 本发明涉及单链或多链形式的新一代嵌合抗原受体（CAR），其特异性为所需抗原，由来自软骨鱼的单体抗体衍生的VNAR多肽赋予其特异性。 旨在将免疫细胞特异性转向所选择的不希望的恶性细胞的这种CAR是紧凑的，因此特别适用于靶向中空抗原，例如存在于耐药细胞表面的外排泵的离子通道。 本发明包括多核苷酸，编码所述多链CAR的载体和在其表面表达它们的分离细胞，特别是其用于免疫治疗。

公开(公告)号：US20160272999A1

公开(公告)日：2016-09-22

申请号：US14892934

申请日：2014-04-01

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  André CHOULIKA ,  Laurent POIROT

IPC: C12N15/85

CPC classification number: C12N15/85 ,  C12N5/0636 ,  C12N5/0637 ,  C12N5/0638 ,  C12N9/22 ,  C12N2510/00

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.

Abstract translation: 本发明涉及开发用于免疫治疗的遗传工程化，优选非同种异体反应性T细胞的方法。 该方法涉及使用RNA引导的核酸内切酶，特别是Cas9 / CRISPR系统，以特异性靶向T细胞中关键基因的选择。 工程化T细胞还旨在表达嵌合抗原受体（CAR）以将其免疫活性重定向于恶性或感染细胞。 本发明开启了使用T细胞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：US20160130599A1

公开(公告)日：2016-05-12

申请号：US14899107

申请日：2014-06-25

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Fayza DABOUSSI ,  David SOURDIVE ,  Jean-Charles EPINAT

IPC: C12N15/82 ,  C12P7/64

CPC classification number: C12N15/8247 ,  C12N9/0006 ,  C12N9/001 ,  C12N9/0071 ,  C12N9/1029 ,  C12N9/1241 ,  C12N9/16 ,  C12N15/79 ,  C12P7/64 ,  C12P7/6427 ,  C12Y101/01008 ,  C12Y103/01009 ,  C12Y114/19006 ,  C12Y203/01 ,  C12Y207/07009 ,  C12Y301/02022

Abstract: The invention provides engineered diatoms and methods of producing oil using diatoms. The invention also provides methods of modifying the lipids quantity and/or quality produced by diatom organisms through genome engineering. Also provided are oils, fuels, oleochemicals, chemical precursors, and other compounds manufactured from such modified diatoms.

Abstract translation: 本发明提供工程硅藻和使用硅藻生产油的方法。 本发明还提供了通过基因组工程改变由硅藻生物产生的脂质量和/或质量的方法。 还提供油​​，燃料，油化学品，化学前体和由这种改性硅藻制造的其它化合物。

公开(公告)号：US20250057952A1

公开(公告)日：2025-02-20

申请号：US18562603

申请日：2022-05-23

Applicant: CELLECTIS S.A.

Inventor： Shipra DAS ,  Julien VALTON ,  Laurent POIROT ,  Philippe DUCHATEAU

IPC: A61K39/00 ,  A61K39/395 ,  A61P35/00 ,  C12N15/90

Abstract: The invention relates to methods of treatment of a solid tumor in a patient in need thereof, comprising administering to the patient: (i) an effective amount of engineered immune cells originating from a donor expressing at their cell surface a Chimeric Antigen Receptor (CAR) directed against Fibroblast Activation Protein (FAP), and (ii) an effective amount of an immunotherapy treatment that elicits an immune response in the patient.

公开(公告)号：US20250034641A1

公开(公告)日：2025-01-30

申请号：US18914680

申请日：2024-10-14

Applicant: CELLECTIS

Inventor： David SOURDIVE ,  Aymeric DUCLERT ,  Mathieu SIMON ,  Philippe DUCHATEAU ,  Alan Marc WILLIAMS ,  Laurent POIROT

IPC: C12Q1/6881 ,  A61K39/00

Abstract: The present invention provides composition kits and methods for treating cancer in a human by immunotherapy using successive doses of CAR-T cells with no or reduced anamnestic immune reaction in one individual (P).

公开(公告)号：US20240318154A1

公开(公告)日：2024-09-26

申请号：US18561938

申请日：2022-05-20

Applicant: CELLECTIS ,  Albert-Ludwigs-Universitat Freiburg

Inventor： Toni CATHOMEN ,  Tatjana CORNU ,  Julia KLERMUND ,  Manuel RHIEL ,  Julia ROSITZKA ,  Philippe DUCHATEAU ,  Alexandre JUILLERAT

IPC: C12N9/22 ,  A61K35/28 ,  C07K14/47 ,  C12N5/0789 ,  C12N15/90

CPC classification number: C12N9/22 ,  A61K35/28 ,  C07K14/4705 ,  C12N5/0647 ,  C12N15/907 ,  C12N2510/00

Abstract: The present invention generally relates to the field of genome engineering (gene editing), and more specifically to gene therapy for the treatment of Severe Combined Immunodeficiency (SCID) related to RAG1. Particularly, the present invention pertains to the treatment of RAG1 deficiency in long-term repopulating hematopoietic stem cells (HSCs). The present invention provides means and methods for genetically modifying HSCs involving gene editing reagents, such as TALE-nucleases, that specifically target a non-functional endogenous RAG1 gene, comprising at least one mutation causing Severe Combined Immunodeficiency (SCID), thereby allowing the restoration of the normal cellular phenotype. The present invention also provides engineered RAG1-edited HSCs comprising an exogenous sequence comprising a nucleic acid sequence encoding a functional RAG1 protein which is integrated in said HSCs' genome into a non-functional RAG1 endogenous locus, resulting in the expression of a functional RAG1 polypeptide. The present invention further provides populations of cells comprising said engineered HSCs, pharmaceutical compositions comprising said engineered HSCs or populations of cells, as well as their use in gene therapy for the treatment of Severe Combined Immunodeficiency (SCID) related to RAG1.

公开(公告)号：US20230357719A1

公开(公告)日：2023-11-09

申请号：US18321481

申请日：2023-05-22

Applicant: Cellectis

Inventor： Philippe DUCHATEAU ,  Laurent POIROT

IPC: C12N5/0783 ,  A61K35/17 ,  A61P35/02 ,  A61P35/00

CPC classification number: C12N5/0636 ,  A61K35/17 ,  A61P35/02 ,  A61P35/00 ,  C12N2510/00 ,  C12N2501/599 ,  Y02A50/30

Abstract: Methods of developing genetically engineered immune cells for immunotherapy, which can be endowed with Chimeric Antigen Receptors targeting an antigen marker that is common to both the pathological cells and said immune cells (ex: CD38, CSI or CD70) by the fact that the genes encoding said markers are inactivated in said immune cells by a rare cutting endonuclease such as TALEN, Cas9 or argonaute.

公开(公告)号：US20230158070A1

公开(公告)日：2023-05-25

申请号：US16965834

申请日：2019-01-30

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU

IPC: A61K35/17 ,  C07K14/725 ,  C07K16/28

CPC classification number: A61K35/17 ,  C07K14/7051 ,  C07K16/2803 ,  A61K2039/505

Abstract: The present invention relates to a therapeutic combination of immune cells, preferably allogeneic non-alloreactive TCR-KO immune T cells, wherein a gene coding an antigen marker X present on both T-cells and pathological cells is inactivated and a corresponding therapeutic antibody specific for said antigen marker X, method for preparing the same and use in immunotherapy.

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.