公开(公告)号：US20200208174A1

公开(公告)日：2020-07-02

申请号：US16314697

申请日：2017-06-30

申请人： CELLECTIS

发明人： Jean-Pierre CABANIOLS ,  Jean-Charles EPINAT ,  Philippe DUCHATEAU

IPC分类号： C12N15/86 ,  C12N13/00 ,  C12N5/0783 ,  C12N9/22 ,  C12N15/113 ,  C12N15/90

摘要： The invention pertains to the field of adaptive cell immunotherapy. It aims at reducing the occurrence of translocations and cell deaths when several specific endonuclease reagents are used altogether to genetically modify primary immune cells at different genetic loci. The method of the invention allows to yield safer immune primary cells harboring several genetic modifications, such as triple or quadruple gene inactivated cells, from populations or sub-populations of cells originating from a single donor or patient,for their subsequent use in therapeutic treatments.

公开(公告)号：US20180171298A1

公开(公告)日：2018-06-21

申请号：US15578946

申请日：2016-06-30

申请人： Cellectis

发明人： Philippe DUCHATEAU ,  Jean-Pierre CABANIOLS ,  Julien VALTON

IPC分类号： C12N5/0783 ,  C12N15/63 ,  A61K35/17 ,  A61K45/06 ,  C12N9/22 ,  C07K14/47 ,  C07K14/725 ,  C12N9/12

CPC分类号： C12N5/0646 ,  A61K35/17 ,  A61K45/06 ,  A61K2039/515 ,  A61K2039/572 ,  C07K14/4703 ,  C07K14/7051 ,  C12N9/12 ,  C12N9/22 ,  C12N15/63 ,  C12N2510/00

摘要： The present invention relates to methods for improving therapeutic activity of NK cell, such as their cytotoxic/cytolytic activity, to be used in immunotherapy, by gene editing. In particular, these methods comprise a step of reduction or inactivation of gene expression using specific endonuclease such as TAL-nuclease, CRISPR or Argonaute. An additional genetic modification can be performed by (over)expressing at least one gene involved in N K function. The present invention encompasses also engineered NK cell, pharmaceutical composition containing the same.

公开(公告)号：US20180051089A1

公开(公告)日：2018-02-22

申请号：US15546623

申请日：2016-01-25

申请人： Cellectis

发明人： Roman GALETTO ,  Barbara Johnson SASU ,  Arvind RAJPAL ,  Philippe DUCHATEAU ,  Alexandre JUILLERAT ,  Julien VALTON ,  Mathieu SIMON

IPC分类号： C07K16/28 ,  A61K35/17 ,  A61K39/00 ,  C07K14/725 ,  C07K16/30 ,  C12N5/00 ,  C12N5/0783

CPC分类号： C07K16/2866 ,  A61K35/17 ,  A61K39/0011 ,  A61K39/39558 ,  A61K2039/505 ,  A61K2039/5156 ,  A61K2039/5158 ,  A61K2039/6056 ,  A61K2039/64 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70535 ,  C07K14/70578 ,  C07K16/2803 ,  C07K16/2887 ,  C07K16/3061 ,  C07K2317/24 ,  C07K2317/53 ,  C07K2317/56 ,  C07K2317/622 ,  C07K2319/00 ,  C07K2319/02 ,  C07K2319/03 ,  C12N5/0093 ,  C12N5/0636 ,  C12N5/0638 ,  C12N2510/00 ,  G01N15/1031 ,  G01N15/14 ,  G01N2015/008 ,  G01N2015/1006 ,  G01N2015/1081 ,  G01N2015/149

摘要： The present invention relates to a TCR KO—or TCR KO and dCK KO—engineered immune cells expressing a Chimeric Antigen Receptors (CAR) specific for CD123 that is a recombinant chimeric protein able to redirect immune cell specificity and reactivity toward CD123-expressing cells, and more particularly in which extracellular ligand binding is a scFV derived from a CD123 monoclonal antibody, conferring specific immunity against CD123 positive cells. The engineered immune cells endowed with such CD123 CARs are particularly suited for treating relapse refractory AML and blastic plasmacytoid dendritic cell neoplasm and for use as a treatment before bone marrow transplantation.

公开(公告)号：US20160333094A1

公开(公告)日：2016-11-17

申请号：US15111441

申请日：2015-01-14

申请人： CELLECTIS

发明人： Philippe DUCHATEAU ,  Julien VALTON

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

CPC分类号： 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

摘要： 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.

摘要翻译： 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

申请人： CELLECTIS

发明人： Philippe DUCHATEAU ,  André CHOULIKA ,  Laurent POIROT

IPC分类号： C12N15/85

CPC分类号： C12N15/85 ,  C12N5/0636 ,  C12N5/0637 ,  C12N5/0638 ,  C12N9/22 ,  C12N2510/00

摘要： 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.

摘要翻译： 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

申请人： CELLECTIS

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

IPC分类号： C12N15/82 ,  C12P7/64

CPC分类号： 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

摘要： 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.

摘要翻译： 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：US20230279440A1

公开(公告)日：2023-09-07

申请号：US17996701

申请日：2021-05-06

申请人： CELLECTIS S.A.

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

IPC分类号： C12N15/90 ,  C12N15/86

CPC分类号： C12N15/907 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/42

摘要： 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.

公开(公告)号：US20230279350A1

公开(公告)日：2023-09-07

申请号：US18016972

申请日：2021-07-30

申请人： CELLECTIS S.A.

发明人： André CHOULIKA ,  Laurent POIROT ,  Beatriz ARANDA ORGILLES ,  Philippe DUCHATEAU

IPC分类号： C12N5/0783 ,  C07K16/28 ,  A61P35/00

CPC分类号： C12N5/0636 ,  C07K16/2887 ,  C07K16/2896 ,  A61P35/00 ,  C07K2317/53 ,  C07K2317/565 ,  C07K2319/03

摘要： The present invention concerns new engineered immune cells expressing two CARs directed against two different targets, polynucleotides for preparing said immune cells, pharmaceutical compositions comprising said immune cells, and the use of said immune cells in the treatment of cancers.

公开(公告)号：US20230068949A1

公开(公告)日：2023-03-02

申请号：US17788133

申请日：2020-12-22

申请人： CELLECTIS

发明人： Cecile SCHIFFER-MANNIOUI ,  Philippe DUCHATEAU

IPC分类号： A61K35/17 ,  C07K16/30 ,  C07K14/725 ,  C07K16/28 ,  C07K14/705 ,  C12N15/90 ,  A61P35/00

摘要： The present invention relates to engineered immune cells expressing new mesothelin (MLSN) specific chimeric antigen receptors (anti-mesothelin CAR) and their use in the treatment of solid tumors, particularly suited for allogeneic cell immunotherapy.

公开(公告)号：US20190338273A1

公开(公告)日：2019-11-07

申请号：US16379073

申请日：2019-04-09

申请人： CELLECTIS

发明人： Philippe DUCHATEAU ,  Julien VALTON

IPC分类号： C12N15/10 ,  G01N33/53 ,  C12N9/22 ,  C07K14/47

摘要： The present invention relates to polypeptides and more particularly to Transcription Activator-Like Effector derived proteins that allow to efficiently target and/or process nucleic acids. Particularly, the present invention reports the characterization of TALE derived proteins that can efficiently target methylated DNA. The present invention more specifically relates to TALE derived proteins that allow activation of methylated promoters responsible for gene silencing.