公开(公告)号：US20230212613A1

公开(公告)日：2023-07-06

申请号：US17996733

申请日：2021-05-06

Applicant: CELLECTIS S.A.

Inventor： Ming YANG ,  Alexandre JUILLERAT ,  Philippe DUCHATEAU ,  Patrick HONG

IPC: C12N15/90 ,  C07K14/725 ,  C12N9/22 ,  C12N5/0783

CPC classification number: C12N15/907 ,  C07K14/7051 ,  C12N9/22 ,  C12N5/0636 ,  C12N2310/20

Abstract: The present disclosure provides methods for targeted insertion of an exogenous sequence at a genomic locus in a cell, wherein said insertion is induced by a sequence-specific endonuclease that has cleavage activity at said locus, at least 5 hours before the introduction into said cell of a DNA template comprising said exogenous sequence.

公开(公告)号：US20200216515A1

公开(公告)日：2020-07-09

申请号：US16633604

申请日：2018-07-26

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Anne-Sophie GAUTRON ,  Laurent POIROT

IPC: C07K14/725 ,  C12N15/10

Abstract: The present disclosure provides in vitro and in vivo methods for selecting a candidate CAR polynucleotide to be expressed in immune cells for its preferential capability to make immune cells proliferate in an antigen-dependent manner.

公开(公告)号：US20190032088A1

公开(公告)日：2019-01-31

申请号：US16073778

申请日：2017-02-24

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Veronique ZENNOU

IPC: C12N15/88 ,  A61K9/00 ,  C12N9/22 ,  C12N15/11 ,  A61K48/00 ,  A61K9/127

Abstract: The invention pertains to therapies that require gene editing, and more specifically to non-viral methods for in vivo delivery of endonuclease reagents to specific tissues or cells. According to the invention, the endonuclease reagents are encapsulated into micelle structures of 50 to 150 nm diameter for intravenous injection. The invention thus provides therapeutic composition including such micelles structures, by which endonuclease reagents can be released into cell under RNA form for their use in the treatment of gene related diseases.

公开(公告)号：US20180201901A1

公开(公告)日：2018-07-19

申请号：US15118801

申请日：2015-02-13

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Laurent POIROT

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

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, CS1 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：US20180002435A1

公开(公告)日：2018-01-04

申请号：US15546630

申请日：2016-01-25

Applicant: Cellectis ,  RINAT NEUROSCIENCE CORPORATION

Inventor： Barbara Johnson SASU ,  Arvind RAJPAL ,  Philippe DUCHATEAU ,  Alexandre JUILLERAT ,  Julien VALTON

IPC: C07K16/28 ,  C07K14/705 ,  G01N15/10 ,  G01N15/14 ,  C12N5/0783 ,  C07K14/725 ,  G01N15/00

CPC classification number: 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

Abstract: A polypeptide encoding a chimeric antigen receptor (CAR) comprising at least one extracellular binding domain that comprises a scFv formed by at least a VH chain and a VL chain specific to an antigen, wherein said extracellular binding domain comprises at least one mAb-specific epitope.

公开(公告)号：US20180002427A1

公开(公告)日：2018-01-04

申请号：US15546619

申请日：2016-01-25

Applicant: CELLECTIS

Inventor： Julianne SMITH ,  Julien VALTON ,  Philippe DUCHATEAU ,  Alexandre JUILLERAT ,  Arvind RAJPAL ,  Barbra Johnson SASU

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

CPC classification number: 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

Abstract: The present invention relates to a new generation of chimeric antigen receptors (CAR) referred to as multi-chain CARs, which are made specific to the antigen CLL1. Such CARs aim to redirect immune cell specificity and reactivity toward malignant cells expressing the tumor antigen CLL1. The alpha, beta and gamma polypeptides composing these CARs are designed to assemble in juxtamembrane position, which forms flexible architecture closer to natural receptors, that confers optimal signal transduction. 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. The invention opens the way to efficient adoptive immunotherapy strategies for treating cancer, especially leukemia.

公开(公告)号：US20160312233A1

公开(公告)日：2016-10-27

申请号：US15103751

申请日：2014-12-12

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Fayza DABOUSSI

IPC: C12N15/79 ,  C12N15/90 ,  C12N9/22 ,  C12N15/10

CPC classification number: C12N15/79 ,  C12N9/22 ,  C12N15/102 ,  C12N15/8209 ,  C12N15/821 ,  C12N15/8213 ,  C12N15/902 ,  C12N2800/80 ,  C12Y301/00

Abstract: The invention relates to a method to select transformed cells. In particular, the present invention relates to the use of a nuclease engineered to inactivate selectable marker which confers cell resistance to a toxic compound. The present invention relates to methods of modifying genome of a cell, preferably an algal cell comprising the present selection step. The present invention also relates to specific engineered nucleases, polynucleotides, vectors encoding thereof, kits and isolated cells comprising said nuclease.

Abstract translation: 本发明涉及一种选择转化细胞的方法。 特别地，本发明涉及工程化的核酸酶在使有毒化合物赋予细胞耐药性的选择性标记失活的用途。 本发明涉及修饰细胞基因组的方法，所述方法优选包含本选择步骤的藻细胞。 本发明还涉及特异性工程化核酸酶，多核苷酸，编码它们的载体，试剂盒和包含所述核酸酶的分离的细胞。

公开(公告)号：US20160273002A1

公开(公告)日：2016-09-22

申请号：US15031996

申请日：2014-10-24

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Alexandre JUILLERAT

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

CPC classification number: C12N15/907 ,  A61K38/00 ,  A61K48/00 ,  A61K48/005 ,  C07K14/195 ,  C07K2319/80 ,  C12N9/22 ,  C12Y301/00 ,  C12Y301/21004

Abstract: The present invention is in the field of genetic editing tools and methods of genetic engineering. It relates to the engineering of rare-cutting endonucleases designed to contract highly repetitive motives in chromosomes, which are at the origin of certain genetic diseases, in particular the so-called “triplet repeat diseases”, such as the Huntington disease. The invention encompasses the method for contracting the repetitive motives, the rare-cutting endonucleases for use to contract repetitive motives in a gene subjected to repeat disorder, the polynucleotides and vectors encoding thereof as well as the resulting pharmaceutical compositions.

Abstract translation: 本发明在遗传编辑工具和基因工程方法领域。 它涉及设计用于在染色体中收缩高度重复动机的稀有内切核酸酶的工程，这些动机是某些遗传疾病的起源，特别是所谓的“三重重复疾病”，如亨廷顿疾病。 本发明包括用于收缩重复动机的方法，用于在重复病症的基因中重复动作的稀有内切核酸酶，编码它们的多核苷酸和载体以及所得药物组合物。

公开(公告)号：US20160184362A1

公开(公告)日：2016-06-30

申请号：US15045368

申请日：2016-02-17

Applicant: Cellectis

Inventor： Philippe DUCHATEAU ,  André CHOULIKA ,  Laurent POIROT

IPC: A61K35/17 ,  C12N9/22 ,  C12N15/90 ,  A61K47/48 ,  C12N5/0783

CPC classification number: C12N15/85 ,  A61K35/17 ,  C12N5/0636 ,  C12N5/0637 ,  C12N5/0638 ,  C12N9/22 ,  C12N15/1138 ,  C12N15/90 ,  C12N2310/20 ,  C12N2510/00 ,  C12Y301/00

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.

公开(公告)号：US20160102324A1

公开(公告)日：2016-04-14

申请号：US14892707

申请日：2014-05-28

Applicant: CELLECTIS

Inventor： Philippe DUCHATEAU ,  Claudia BERTONATI

IPC: C12N15/90 ,  C12N9/22

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N15/1082 ,  C12N15/63 ,  C12N15/902 ,  C12Y301/00 ,  C12Y301/26004

Abstract: The present invention is in the field of CRISPR-Cas system for genome targeting. The present invention relates to new engineered Cas9 scaffolds and uses thereof. More particularly, the present invention relates to methods for genome targeting, cell engineering and therapeutic application. The present invention also relates to vectors, compositions and kits in which the new Cas9 scaffolds of the present invention are used.

Abstract translation: 本发明涉及用于基因组靶向的CRISPR-Cas系统。 本发明涉及新的工程化Cas9支架及其用途。 更具体地，本发明涉及用于基因组靶向，细胞工程和治疗应用的方法。 本发明还涉及其中使用本发明的新的Cas9支架的载体，组合物和试剂盒。