公开(公告)号：US20210017545A1

公开(公告)日：2021-01-21

申请号：US17041359

申请日：2019-03-29

Applicant: CELLECTIS

Inventor： Alex BOYNE ,  Brian BUSSER ,  Philippe DUCHATEAU ,  Aymeric DUCLERT

IPC: C12N15/90 ,  C12N15/86 ,  C07K14/805 ,  C12N15/10 ,  C12N9/22

Abstract: The present invention relates to the field of genome engineering (gene editing). More specifically the invention provides with allele specific TALE-nucleases and methods to operate allele specific gene repair by homologous recombination in primary cells, such as hematopoietic stem cells, blood cells and hepatocytes. These reagents and methods can be used for the genetic treatment of inherited disease, such as sickle cell disease betathalassemia.

公开(公告)号：US20200332004A1

公开(公告)日：2020-10-22

申请号：US16958250

申请日：2018-12-28

Applicant: CELLECTIS

Inventor： Alex BOYNE ,  Laurent POIROT ,  Philippe DUCHATEAU ,  Alexandre JUILLERAT

IPC: C07K16/28 ,  C12N5/0783 ,  C12N15/87 ,  C12N15/86 ,  A61K35/17 ,  A61P35/00

Abstract: A method for engineering less alloreactive immune cells, including T-cells that express chimeric antigen receptors (CARs), using a nucleotide sequence in form of an RNA encoding a anti-TCR CAR to achieve the transient expression of anti-TCR CAR at the cell surface. The transient expression of the anti-TCR CAR recognized by the alpha beta TCR on the cell surface unexpectedly enabled the a purification of the TCR-negative CAR expressing cells. The TCR-negative CAR expressing immune cells can be used in adoptive therapy to treat diseases associated with cell surface antigens, such as cancer with less side effects, in particular less GVHD.

公开(公告)号：US20250002945A1

公开(公告)日：2025-01-02

申请号：US18712640

申请日：2022-11-23

Applicant: CELLECTIS SA

Inventor： Philippe DUCHATEAU ,  Alexandre JUILLERAT ,  Alex BOYNE ,  Selena KAZANCIOGLU

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

Abstract: The present invention relates to the design of improved TALE protein fusions useful as sequence-specific genomic reagents, such as TALE-nucleases and TALE base editors, displaying higher on-target/off-target activity ratios. Its goal is to produce safer reagents to genetically modify the genomes of different types of cells, especially mammalian cells, in particular for their use in gene therapy.

公开(公告)号：US20230279440A1

公开(公告)日：2023-09-07

申请号：US17996701

申请日：2021-05-06

Applicant: CELLECTIS S.A.

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

IPC: C12N15/90 ,  C12N15/86

CPC classification number: C12N15/907 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/42

Abstract: 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.