公开(公告)号：US20170183418A1

公开(公告)日：2017-06-29

申请号：US15302450

申请日：2015-04-13

Applicant: CELLECTIS

Inventor： Roman GALLETTO

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

CPC classification number: C07K16/2878 ,  A61K35/17 ,  A61K2035/124 ,  A61K2039/5156 ,  A61K2039/585 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70575 ,  C07K14/70578 ,  C07K2317/24 ,  C07K2317/53 ,  C07K2317/565 ,  C07K2317/622 ,  C07K2317/70 ,  C07K2317/73 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/33 ,  C07K2319/74 ,  C12N5/0636 ,  C12N2501/599 ,  C12N2510/00

Abstract: The present invention relates to Chimeric Antigen Receptors (CAR) that are recombinant chimeric proteins able to redirect immune cell specificity and reactivity toward selected membrane antigens, and more particularly in which extracellular ligand binding is a scFV derived from a BCMA monoclonal antibody, conferring specific immunity against BCMA positive cells. The engineered immune cells endowed with such CARs are particularly suited for treating lymphomas, multiple myeloma and leukemia.

公开(公告)号：US20170145094A1

公开(公告)日：2017-05-25

申请号：US15301686

申请日：2015-04-02

Applicant: CELLECTIS

Inventor： Roman GALETTO

IPC: C07K16/28 ,  C07K14/725 ,  C07K14/705

CPC classification number: C07K16/2803 ,  A61K2039/505 ,  C07K14/7051 ,  C07K14/70517 ,  C07K2317/24 ,  C07K2317/53 ,  C07K2317/62 ,  C07K2317/622 ,  C07K2317/73 ,  C07K2319/03

Abstract: The present invention relates to Chimeric Antigen Receptors (CAR) that are recombinant chimeric proteins able to redirect immune cell specificity and reactivity toward selected membrane antigens, and more particularly in which extracellular ligand binding is a scFV derived from a CD33 monoclonal antibody, conferring specific immunity against CD33 positive cells. The engineered immune cells endowed with such CARs are particularly suited for treating lymphomas and leukemia.

公开(公告)号：US20170035866A1

公开(公告)日：2017-02-09

申请号：US15302913

申请日：2015-04-10

Applicant: CELLECTIS

Inventor： Laurent POIROT ,  Mathieu SIMON

IPC: A61K39/00 ,  C07K14/725 ,  C12N9/12

Abstract: The present invention pertains to engineered immune cells, method for their preparation and their use as medicament, particularly for immunotherapy. The engineered immune cells of the present invention are characterized in that at least one gene selected from a gene encoding GCN2 and a gene encoding PRDM1 is inactivated or repressed. Such modified Immune cells are resistant to an arginine and/or tryptophan depleted microenvironment caused by, e.g., tumor cells, which makes the immune cells of the invention particularly suitable for immunotherapy. The invention opens the way to standard and affordable adoptive immunotherapy strategies using immune cells for treating different types of malignancies.

Abstract translation: 本发明涉及工程改造的免疫细胞，其制备方法及其用作药物，特别是用于免疫治疗。 本发明的工程化免疫细胞的特征在于，选自编码GCN2的基因和编码PRDM1的基因的至少一种基因被灭活或抑制。 这种修饰的免疫细胞对由例如肿瘤细胞引起的精氨酸和/或色氨酸消耗的微环境具有抗性，这使得本发明的免疫细胞特别适用于免疫治疗。 本发明开启了使用免疫细胞治疗不同类型恶性肿瘤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：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：US09365864B2

公开(公告)日：2016-06-14

申请号：US13900099

申请日：2013-05-22

Applicant: CELLECTIS

Inventor： Jean-Pierre Cabaniols ,  Andre Choulika ,  Christophe Delenda

IPC: C12N15/85 ,  C12N15/90

CPC classification number: C12N15/85 ,  C12N15/902

Abstract: The invention relates to a set of genetic constructs which allow the efficient and reproducible introduction of a specific nucleotide sequence at a fixed position in the genome by generating a double strand break at a specific position in the genome using a meganuclease and so stimulating a homologous recombination event at this locus between the genomic site and a transfected donor sequence. The present invention also relates to methods using these constructs and to these materials in the form of a kit.

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：US20160037742A1

公开(公告)日：2016-02-11

申请号：US14653604

申请日：2013-12-20

Applicant: CELLECTIS

Inventor： Luc Mathis ,  Daniel F. Voytas ,  Feng Zhang ,  Benjamin Clasen ,  William Haun ,  Thomas Stoddard

IPC: A01H5/04 ,  A23L1/217

CPC classification number: C12N15/01 ,  A23V2002/00 ,  C12N15/8245

Abstract: Materials and methods are provided for making Solanum varieties with decreased accumulation of reducing sugars and acrylamide in cold-stored potatoes, specifically, by making TALE-nuclease-induced mutations in genes encoding vacuolar invertase.

Abstract translation: 提供了材料和方法，用于制备在冷藏土豆中还原糖和丙烯酰胺积累减少的茄属品种，特别是通过在编码液泡转化酶的基因中产生TALE-核酸酶诱导的突变。

公开(公告)号：US20150291967A1

公开(公告)日：2015-10-15

申请号：US14439413

申请日：2013-10-31

Applicant: Luc MATHIS ,  Daniel VOYTAS ,  Jin LI ,  Feng ZHANG ,  Song LUO ,  CELLECTIS

Inventor： Luc Mathis ,  Daniel F. Voytas ,  Jin Li ,  Feng Zhang ,  Song Luo

IPC: C12N15/82

CPC classification number: C12N15/8213 ,  C12N15/8209 ,  C12N15/8274 ,  C12N15/8278

Abstract: The present invention relates to methods allowing the targeted insertion of transgenes into a plant genome at desired loci by using homologous recombination combined with rare-cutting endonucleases without the need of inserting an exogenous selectable marker.

Abstract translation: 本发明涉及通过使用与稀有切割内切核酸酶组合的同源重组而不需要插入外源选择性标记的方法，允许将转基因靶向插入到所需基因座的植物基因组中。

公开(公告)号：US20150225465A1

公开(公告)日：2015-08-13

申请号：US14696920

申请日：2015-04-27

Applicant: Cellectis

Inventor： Claudia BERTONATI ,  Philippe Duchateau ,  Alexandre Juillerat ,  George Silva ,  Julien Valton

IPC: C07K14/195 ,  C12N15/90 ,  C12N9/22

CPC classification number: C07K14/195 ,  A01K67/0275 ,  A01K2217/07 ,  C07K2319/80 ,  C12N9/22 ,  C12N15/62 ,  C12N15/87 ,  C12N15/902 ,  C12N15/907 ,  C12Y301/21

Abstract: The present invention concerns new modular base-per-base specific nucleic acid binding domains (MBBBD) derived from newly identified proteins from the bacterial endosymbiont Burkholderia Rhizoxinica and their use for engineering nucleic acid processing enzymes, such as specific endonucleases or transcription activators.

Abstract translation: 本发明涉及从新鉴定的来自细菌内分泌杆菌伯克霍尔德杆菌（Burkholderia Rhizoxinica）的新鉴定的蛋白质中获得的新的基于碱基的碱基特异性核酸结合结构域（MBBBD）及其用于工程化核酸加工酶如特异性内切核酸酶或转录激活子的用途。