公开(公告)号：US20170198306A1

公开(公告)日：2017-07-13

申请号：US15127128

申请日：2015-03-23

Applicant: CELLECTIS

Inventor： Julien Valton ,  Philippe Duchateau

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/11 ,  C12N5/0783 ,  C12N15/85 ,  C12N15/86 ,  C12N5/074

CPC classification number: C12N15/907 ,  C12N5/0607 ,  C12N5/0636 ,  C12N9/22 ,  C12N15/102 ,  C12N15/111 ,  C12N15/8509 ,  C12N15/86 ,  C12N15/90 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2320/30 ,  C12N2510/00 ,  C12N2517/02 ,  C12N2740/15043 ,  C12Y301/26

Abstract: This invention relates to materials and methods for gene editing in mammalian cells, and more particularly to methods for gene editing using DNA-guided Argonaute (Ago) interference systems (DAIS) in T-cells.

公开(公告)号：US20160298098A1

公开(公告)日：2016-10-13

申请号：US15069672

申请日：2016-03-14

Applicant: CELLECTIS, S.A.

Inventor： Philippe Duchateau ,  Julien Valton ,  Claudia Bertonati ,  Jean-Charles Epinat ,  George H. Silva ,  Alexandre Juillerat ,  Marine Beurdeley

IPC: C12N9/22 ,  C07K14/47 ,  C12P19/34

CPC classification number: C12N9/16 ,  A61K38/00 ,  C07K14/4703 ,  C07K2319/00 ,  C07K2319/80 ,  C12N9/22 ,  C12P19/34

Abstract: The present invention relates to a method for the generation of compact Transcription Activator-Like Effector Nucleases (TALENS) that can efficiently target and process double-stranded DNA. More specifically, the present invention concerns a method for the creation of TALENs that consist of a single TALE DNA binding domain fused to at least one catalytic domain such that the active entity is composed of a singe polypeptide chain for simple and efficient vectorization and does not require dimerization to target a specific single double-stranded DNA tar et sequence of interest and process DNA nearby said DNA target sequence. The present invention also relates to compact TALENs, vectors, compositions and kits used to implement the method.

Abstract translation: 本发明涉及能够有效靶向和加工双链DNA的致密转录激活子样效应核酸酶（TALENS）的生成方法。 更具体地，本发明涉及一种用于产生TALEN的方法，其由与至少一个催化结构域融合的单个TALE DNA结合结构域组成，使得活性实体由简单且有效的载体化的单个多肽链组成，并且不 需要二聚化来靶向目标特定的单链双链DNA焦油和序列，并在DNA附近处理DNA靶序列。 本发明还涉及用于实施该方法的紧凑型TALEN，载体，组合物和试剂盒。

公开(公告)号：US20140230083A1

公开(公告)日：2014-08-14

申请号：US13949880

申请日：2013-07-24

Applicant: CELLECTIS

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

IPC: C12N15/87 ,  A01K67/027

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。

公开(公告)号：US12252699B2

公开(公告)日：2025-03-18

申请号：US18480890

申请日：2023-10-04

Applicant: CELLECTIS

Inventor： Laurent Poirot ,  David Sourdive ,  Philippe Duchateau ,  Jean-Pierre Cabaniols

IPC: C12N15/85 ,  A61K35/17 ,  A61K39/00 ,  C07K14/705 ,  C07K14/74 ,  C12N5/0783 ,  C12N15/113 ,  C12N15/90

Abstract: The present invention pertains to engineered T-cells, method for their preparation and their use as medicament, particularly for immunotherapy. The engineered T-cells of the invention are characterized in that the expression of beta 2-microglobulin (B2M) and/or class II major histocompatibility complex transactivator (CIITA) is inhibited, e.g., by using rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding B2M and/or CIITA, or by using nucleic acid molecules which inhibit the expression of B2M and/or CIITA. In order to further render the T-cell non-alloreactive, at least one gene encoding a component of the T-cell receptor is inactivated, e.g., by using a rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding said TCR component.

公开(公告)号：US12221478B2

公开(公告)日：2025-02-11

申请号：US16755093

申请日：2018-04-16

Applicant: CELLECTIS

Inventor： Brian Busser ,  Philippe Duchateau ,  Alexandre Juillerat ,  Laurent Poirot ,  Julien Valton ,  Mohit Sachdeva

IPC: C07K16/28 ,  A61K39/00 ,  C07K16/30 ,  C12N5/0783 ,  C12N5/10

Abstract: The invention pertains to the field of adaptive cell immunotherapy. It provides with the genetic insertion of exogenous coding sequence(s) into genetically engineered immune cells to prevent cytokine release syndrome to arise during the course of cell therapy. These exogenous coding sequences are more particularly soluble human polypeptides placed 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：US20240299453A1

公开(公告)日：2024-09-12

申请号：US18562536

申请日：2022-05-20

Applicant: CELLECTIS S.A. ,  ALBERT-LUDWIGS-UNIVERSITÄT FREIBURG

Inventor： Toni Cathomen ,  Tatjana Cornu ,  Viviane Dettmer-Monaco ,  Simone Haas ,  Julia Rositzka ,  Philippe Duchateau ,  Alexandre Juillerat

IPC: A61K35/17 ,  A61K48/00 ,  C12N5/0783 ,  C12N9/22 ,  C12N15/86 ,  C12N15/90

CPC classification number: A61K35/17 ,  A61K48/005 ,  C12N5/0636 ,  C12N9/22 ,  C12N15/86 ,  C12N15/907 ,  C12N2501/2302 ,  C12N2501/51 ,  C12N2501/515 ,  C12N2501/53 ,  C12N2510/00 ,  C12N2750/14143 ,  C12N2800/80 ,  C12N2830/42

Abstract: The present invention generally relates to the field of genome engineering (gene editing), and more specifically to gene therapy for the treatment of Hyper-lgE syndrome (HIES). In particular, the present invention provides means and methods for genetically modifying HSCs or T-cells involving gene editing reagents, such as TALE-nucleases, that specifically target an endogenous STATS gene comprising at least one mutation causing Hyper-lgE syndrome (HIES), thereby allowing the restoration of the normal cellular phenotype. The present invention also provides populations of engineered HSCs or T-cells which comprise cells comprising an exogenous polynucleotide sequence comprising at least a partial or complete sequence of a functional STATS gene, said exogenous polynucleotide sequence being integrated in an endogenous STATS gene comprising at least one mutation causing Hyper-lgE syndrome (HIES), resulting in the expression of a functional STATS polypeptide. The present invention further provides pharmaceutical compositions comprising the cell populations of the invention, and their use in gene therapy for the treatment of Hyper-lgE syndrome (HIES).

公开(公告)号：US11959091B2

公开(公告)日：2024-04-16

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 ,  C12N15/113

CPC classification number: C12N15/85 ,  A61K35/17 ,  C12N5/0636 ,  C12N5/0637 ,  C12N5/0638 ,  C12N9/22 ,  C12N15/90 ,  C12N15/1138 ,  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：US11944643B2

公开(公告)日：2024-04-02

申请号：US16498899

申请日：2018-03-30

Applicant: CELLECTIS SA

Inventor： Julianne Smith ,  Philippe Duchateau ,  Murielle Derrien

IPC: A61K35/17 ,  A61K31/365 ,  A61K39/395 ,  A61P35/02 ,  C07K14/705 ,  C07K14/725 ,  C07K16/28

CPC classification number: A61K35/17 ,  A61K31/365 ,  A61K39/39558 ,  A61P35/02 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70578 ,  C07K16/2803 ,  C07K2317/34 ,  C07K2317/53 ,  C07K2317/622

Abstract: The present invention relates to an engineered immune cell endowed with CD22 Chimeric Antigen Receptors (CD22 CAR) with a deletion in the TRAC gene that is able to redirect immune cell specificity and reactivity toward selected tumor cells. The engineered immune cells endowed with such CARs are particularly suited for treating relapsed refractory CD22 expressing cancers.

公开(公告)号：US11820996B2

公开(公告)日：2023-11-21

申请号：US16138908

申请日：2018-09-21

Applicant: CELLECTIS

Inventor： Laurent Poirot ,  David Sourdive ,  Philippe Duchateau ,  Jean-Pierre Cabaniols

IPC: A61K35/17 ,  A61K48/00 ,  C12N5/0783 ,  C12N5/00 ,  C07K14/005 ,  C07K14/705 ,  C07K14/74 ,  C07K16/28 ,  C12N9/22 ,  C12N15/11 ,  A61K39/00 ,  C12N15/85 ,  C12N15/113 ,  C12N15/90

CPC classification number: C12N15/85 ,  C07K14/70503 ,  C07K14/70539 ,  C12N5/0636 ,  C12N15/1138 ,  C12N15/907 ,  C07K2317/24 ,  C07K2317/622

Abstract: The present invention pertains to engineered T-cells, method for their preparation and their use as medicament, particularly for immunotherapy. The engineered T-cells of the invention are characterized in that the expression of beta 2-microglobulin (B2M) and/or class II major histocompatibility complex transactivator (CIITA) is inhibited, e.g., by using rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding B2M and/or CIITA or by using nucleic acid molecules which inhibit the expression of B2M and/or CIITA. In order to further render the T-cell non-alloreactive, at least one gene encoding a component of the T-cell receptor is inactivated, e.g., by using a rare-cutting endonucleases able to selectively inactivating by DNA cleavage the gene encoding said TCR component. In addition, expression of immunosuppressive polypeptide can be performed on those modified T-cells in order to prolong the survival of these modified T cells in host organism. Such modified T-cell is particularly suitable for allogeneic transplantations, especially because it reduces both the risk of rejection by the host's immune system and the risk of developing graft versus host disease. The invention opens the way to standard and affordable adoptive immunotherapy strategies using T-Cells for treating cancer, infections and auto-immune diseases.

公开(公告)号：US11685935B2

公开(公告)日：2023-06-27

申请号：US14892707

申请日：2014-05-28

Applicant: CELLECTIS

Inventor： Philippe Duchateau ,  Claudia Bertonati

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/10 ,  C12N15/63

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N15/902 ,  C12N15/1082 ,  C12N15/63 ,  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.