Abstract: Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Application
Filed:
April 12, 2021
Publication date:
August 19, 2021
Applicant:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: Provided include compositions, methods, and systems for modulating the expression, function, and/or activity of a target gene, for example a blood-clotting protein such as Factor VIII (FVIII), in a cell by genome editing. Also provided include compositions, methods, and systems for treating a subject having or suspected of having a disorder or health condition, e.g., Hemophilia A, employing ex vivo and/or in vivo genome editing.
Abstract: Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) gene in a cell by genome editing.
Type:
Grant
Filed:
March 16, 2017
Date of Patent:
August 10, 2021
Assignee:
CRISPR THERAPEUTICS AG
Inventors:
Roman Lvovitch Bogorad, Chad Albert Cowan, Ante Sven Lundberg
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Application
Filed:
April 12, 2021
Publication date:
August 5, 2021
Applicant:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander TERRETT, Demetrios KALAITZIDIS, Lawrence KLEIN
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Application
Filed:
April 12, 2021
Publication date:
August 5, 2021
Applicant:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Grant
Filed:
June 15, 2020
Date of Patent:
July 27, 2021
Assignee:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: The present application provides materials and methods for treating a patient with Usher Syndrome Type 2A, both ex vivo and in vivo; materials and methods for editing a USH2A gene in a human cell; materials and methods for editing an USH2A gene containing an IVS40 mutation; materials and methods for treating a patient with an USH2A gene containing an IVS40 mutation; and a method for deleting a sequence comprising an IVS40 mutation within a USH2A gene of a cell. The present application also provides one or more gRNAs or sgRNAs for editing an USH2A gene containing an IVS40 mutation. The present application provides a therapeutic for treating a patient with Usher Syndrome Type 2A. The present application also provides a kit for treating a patient with Usher Syndrome Type 2A.
Type:
Grant
Filed:
June 18, 2020
Date of Patent:
July 27, 2021
Assignees:
BAYER HEALTHCARE LLC, CRISPR THERAPEUTICS AG
Inventors:
Albena Kantardzhieva, Akiko Noma, Abraham Scaria
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Grant
Filed:
August 3, 2020
Date of Patent:
May 25, 2021
Assignee:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.
Abstract: Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating the genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or component or transcriptional regulator of the MHC-I or MHC-II complex, at least one genetic modification that increases the expression of at least one polynucleotide that encodes a tolerogenic factor, and optionally at least one genetic modification that increases or decreases the expression of at least one gene that encodes a survival factor.
Abstract: Provided include compositions, methods, and systems for modulating the expression, function, and/or activity of a target gene, for example a blood-clotting protein such as Factor VIII (FVIII), in a cell by genome editing. Also provided include compositions, methods, and systems for treating a subject having or suspected of having a disorder or health condition, e.g., hemophilia A, employing ex vivo and/or in vivo genome editing.
Abstract: The present application provides materials and methods for treating a patient with autosomal dominant CORD, both ex vivo and in vivo; materials and methods for editing a GUCY2D gene in a human cell; and materials and methods for editing a R838H, R838C, or R838S mutation in a GUCY2D gene in a human cell. The present application also provides one or more gRNAs or sgRNAs for editing a GUCY2D gene; one or more gRNAs or sgRNAs for editing a R838H, R838C, or R838S mutation in a GUCY2D gene; and a therapeutic comprising at least one or more gRNAs or sgRNAs for editing a R838H, R838C, or R838S mutation in a GUCY2D gene. The present application provides a therapeutic for treating a patient with autosomal dominant CORD. The present application also provides a kit for treating a patient with autosomal dominant CORD. In addition, the present application provides a self-inactivating CRISPR-Cas system.
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Application
Filed:
May 11, 2018
Publication date:
April 15, 2021
Applicant:
CRISPR THERAPEUTICS AG
Inventors:
Jonathan Alexander TERRETT, Demetrios KALAITZIDIS, Lawrence KLEIN
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Application
Filed:
September 29, 2020
Publication date:
March 25, 2021
Applicant:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Grant
Filed:
June 7, 2019
Date of Patent:
January 5, 2021
Assignee:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Application
Filed:
August 3, 2020
Publication date:
December 31, 2020
Applicant:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
Type:
Application
Filed:
July 29, 2020
Publication date:
December 31, 2020
Applicant:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein