Abstract: A population of genetically engineered immune cells (e.g., T cells), which express a chimeric antigen receptor (CAR) specific to CD19 and contain a disrupted TRAC gene, a disrupted B2M gene, or both, for use in treating a B cell malignancy.
Type:
Grant
Filed:
April 22, 2021
Date of Patent:
July 19, 2022
Assignee:
CRISPR Therapeutics AG
Inventors:
Mark Benton, Tony Ho, Demetrios Kalaitzidis, Ewelina Morawa, Jonathan Alexander Terrett
Abstract: The present disclosure provides donor polynucleotides, genome editing systems, methods, pharmaceutical compositions, and kits which correct or induce a mutation that causes Glycogen Storage Disease 1a in a genomic DNA (gDNA) molecule in a cell. In some embodiments the present disclosure provides donor polynucleotides comprising two strands capable of correcting a mutation that causes Glycogen Storage Disease 1a.
Type:
Grant
Filed:
February 18, 2021
Date of Patent:
May 17, 2022
Assignee:
CRISPR THERAPEUTICS AG
Inventors:
Troy Dean Carlo, Roman Lvovitch Bogorad
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:
April 12, 2021
Date of Patent:
April 12, 2022
Assignee:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: Provided herein, in some embodiments, are methods and compositions (e.g., cell compositions) for the treatment of cancer. The methods and compositions involve genetically engineered immune cells (e.g., T cells), in which the endogenous CD70 gene is disrupted by genetic editing, for example, the CRISPR/Cas9 gene editing technology.
Abstract: The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with ATXN1 whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Spinocerebellar ataxia type 1 (SCA1). Also provided are materials and methods for editing a ATXN1 gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a ATXN1 gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a ATXN1 gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a ATXN1 gene. In addition, the present disclosure provides therapeutics for treating patients with a ATXN1 related condition or disorder.
Type:
Application
Filed:
February 20, 2018
Publication date:
February 17, 2022
Applicant:
CRISPR THERAPEUTICS AG
Inventors:
Ante Sven LUNDBERG, Samarth KULKARNI, Lawrence KLEIN, Hari Kumar PADMANABHAN
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:
April 12, 2021
Date of Patent:
December 28, 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:
Grant
Filed:
April 12, 2021
Date of Patent:
December 21, 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:
Grant
Filed:
September 29, 2020
Date of Patent:
December 7, 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 the genetically 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: The present disclosure provides materials and methods for treating a patient with one or more conditions associated with SOD1 whether ex vivo or in vivo. In addition, the present disclosure provides materials and methods for editing and/or modulating the expression of SOD1 gene in a cell by genome editing.
Type:
Grant
Filed:
June 15, 2017
Date of Patent:
November 16, 2021
Assignee:
CRISPR THERAPEUTICS AG
Inventors:
Ante Sven Lundberg, Samarth Kulkarni, Lawrence Klein, Hari Kumar Padmanabhan
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:
July 29, 2020
Date of Patent:
November 9, 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:
Grant
Filed:
July 8, 2020
Date of Patent:
October 5, 2021
Assignee:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Demetrios Kalaitzidis, Lawrence Klein
Abstract: Provided herein, in some embodiments, are methods and compositions (e.g., cell compositions) for the treatment of cancer, such as PTK7+ malignancies.
Type:
Application
Filed:
November 7, 2019
Publication date:
September 23, 2021
Applicant:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Jason Sagert
Abstract: Provided herein, in some embodiments, are methods and compositions (e.g., cell compositions) for the treatment of cancer, such as LIV1+ malignancies.
Type:
Application
Filed:
November 7, 2019
Publication date:
September 23, 2021
Applicant:
CRISPR Therapeutics AG
Inventors:
Jonathan Alexander Terrett, Jason Sagert
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: 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: The present application provides materials and methods for treating a patient with one or more of Usher Syndrome Type 2A and ARRP, both ex vivo and in vivo; materials and methods for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299. In addition, the present application provides one or more gRNAs or sgRNAs for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299; a therapeutic comprising at least one or more gRNAs or sgRNAs for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299; and a therapeutic for treating a patient with one or more of Usher Syndrome Type 2A and ARRP. The present application also provides a kit for treating a patient with one or more of Usher Syndrome Type 2A and ARRP.
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: 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: A population of genetically engineered immune cells (e.g., T cells), which express a chimeric antigen receptor (CAR) specific to CD19 and contain a disrupted TRAC gene, a disrupted B2M gene, or both, for use in treating a B cell malignancy.
Type:
Application
Filed:
April 22, 2021
Publication date:
September 2, 2021
Applicant:
CRISPR Therapeutics AG
Inventors:
Mark Benton, Tony Ho, Demetrios Kalaitzidis, Ewelina Morawa, Jonathan Alexander Terrett