公开(公告)号：US20240360471A1

公开(公告)日：2024-10-31

申请号：US18457258

申请日：2023-08-28

申请人： EDITAS MEDICINE, INC.

发明人： Jennifer Leah GORI ,  Luis A. BARRERA

IPC分类号： C12N15/85 ,  C12N5/071 ,  C12N9/22 ,  C12N15/113

CPC分类号： C12N15/85 ,  C12N5/0602 ,  C12N9/22 ,  C12N15/113 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2510/00 ,  C12N2800/80

摘要： Provided herein are CRISPR/Cas-related methods and components for editing a target nucleic acid sequence in a HBG1 and/or HBG2 gene regulatory region, and applications thereof in connection with methods of increasing expression of fetal hemoglobin and treating β-hemoglobinopathies including sickle cell disease and β-thalassemia.

公开(公告)号：US20240293543A1

公开(公告)日：2024-09-05

申请号：US18573231

申请日：2022-06-23

申请人： Editas Medicine, Inc.

发明人： John Anthony Zuris

IPC分类号： A61K39/00 ,  C07K14/74 ,  C12N5/0783 ,  C12N9/02 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90

CPC分类号： A61K39/4613 ,  A61K39/4634 ,  C07K14/70539 ,  C12N5/0646 ,  C12N9/0008 ,  C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  C12Y102/01012 ,  A61K2239/26 ,  C12N2310/20 ,  C12N2501/16 ,  C12N2506/45 ,  C12N2800/80

摘要： Edited cells, e.g., genomically edited cells, with reduced levels of immune rejection and/or improved persistence are described.

公开(公告)号：US12049651B2

公开(公告)日：2024-07-30

申请号：US17555974

申请日：2021-12-20

申请人： Editas Medicine, Inc.

发明人： Cecilia Cotta-Ramusino ,  Hariharan Jayaram ,  John Anthony Zuris

IPC分类号： C12N9/22 ,  C07K19/00 ,  C12N15/10 ,  C12N15/11 ,  C12N15/63 ,  C12N15/90

CPC分类号： C12N9/22 ,  C07K19/00 ,  C12N15/10 ,  C12N15/102 ,  C12N15/11 ,  C12N15/63 ,  C12N15/907 ,  C12Y301/00 ,  C07K2319/80 ,  C12N2310/20 ,  C12N2800/80

摘要： Disclosed herein are enzymatically active Cas9 (eaCas9) fusion molecules, comprising an eaCas9 molecule linked, e.g., covalently or non-covalently, to a template nucleic acid; gene editing systems comprising the eaCas9 fusion molecules, and methods of use thereof.

公开(公告)号：US20240132877A1

公开(公告)日：2024-04-25

申请号：US18104461

申请日：2023-02-01

申请人： Editas Medicine, Inc.

发明人： Cecilia Cotta-Ramusino ,  Anne Helen Bothmer

IPC分类号： C12N15/11 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/46 ,  A61K48/00 ,  C12N9/12 ,  C12N9/22

CPC分类号： C12N15/11 ,  A61K31/7088 ,  A61K38/45 ,  A61K38/465 ,  A61K48/005 ,  C12N9/1264 ,  C12N9/22 ,  C12Y207/07031 ,  C12Y301/16001 ,  C12N2310/20 ,  C12N2800/80

摘要： This application provides improved methods of genome editing. The genome editing systems described herein comprise a RNA-guided nuclease molecule and a Repair-Modulating Enzyme Molecule (RMEM).

公开(公告)号：US20230365980A1

公开(公告)日：2023-11-16

申请号：US18138988

申请日：2023-04-25

申请人： Editas Medicine, Inc.

发明人： Cecilia Cotta-Ramusino

IPC分类号： C12N15/64 ,  C12N15/10 ,  C12N9/12

CPC分类号： C12N15/64 ,  C12N15/102 ,  C12N9/1241 ,  C12N2310/20 ,  C12N2310/122

摘要： This application provides improved methods of editing the genome of a target cell. Cas9 molecules can be used to create a break in a genomic region of interest. To increase the likelihood that the break is repaired by homology-directed repair (HDR), the cell can be contacted with an HDR-enhancer. The cell may be, e.g., a human cell, a non-human animal cell, a bacterial cell, or a plant cell.

公开(公告)号：US20230053028A1

公开(公告)日：2023-02-16

申请号：US17786753

申请日：2020-12-18

申请人： Editas Medicine, Inc.

发明人： G. Grant Welstead ,  Jung Il Moon

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

摘要： Methods of culturing embryonic stem cells, induced pluripotent stem cells and/or differentiated cells in culture medium comprising activin are described. In one aspect, the disclosure features a pluripotent human stem cell, wherein the stem cell comprises: (i) a genomic edit that results in loss of function of Cytokine Inducible SH2 Containing Protein (CISH) and (ii) a genomic edit that results in a loss of function of an agonist of the TGF beta signaling pathway, or a genomic edit that results in a loss of function of adenosine Ata receptor.

公开(公告)号：US20220025363A1

公开(公告)日：2022-01-27

申请号：US17115791

申请日：2020-12-08

申请人： EDITAS MEDICINE, INC.

发明人： Jennifier Leah GORI ,  Edouard AUPEPIN DE LAMOTHE-DREUZY ,  Jack HEATH ,  John Anthony ZURIS ,  KaiHsin CHANG

IPC分类号： C12N15/11 ,  C12N15/10 ,  C12N9/22

摘要： Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.

公开(公告)号：US20220010313A1

公开(公告)日：2022-01-13

申请号：US17234145

申请日：2021-04-19

申请人： EDITAS MEDICINE, INC.

发明人： Ari E. Friedland ,  Penrose O'Donnell ,  David A. Bumcrot

IPC分类号： C12N15/113 ,  C12N15/10 ,  C12N9/22

摘要： CRISPR/CAS-related systems, compositions and methods for editing RS1, RL2, and/or LAT genes in human cells are described, as are cells and compositions including cells edited according to the same.

公开(公告)号：US11098297B2

公开(公告)日：2021-08-24

申请号：US16544452

申请日：2019-08-19

申请人： Editas Medicine, Inc.

发明人： Barrett Ethan Steinberg ,  Derek Cerchione

IPC分类号： C12N9/22 ,  C12N15/55 ,  C12N15/11 ,  C12N15/115 ,  C12N15/10

摘要： The present disclosure relates to Cas9 nuclease variants and methods of producing and using such variants.

公开(公告)号：US20210254061A1

公开(公告)日：2021-08-19

申请号：US17019154

申请日：2020-09-11

申请人： EDITAS MEDICINE, INC.

发明人： Jennifer Leah GORI ,  Edouard AUPEPIN DE LAMOTHE-DREUZY ,  Jack HEATH ,  John Anthony ZURIS ,  KaiHsin CHANG

IPC分类号： C12N15/113 ,  A61K9/00

摘要： Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.