Patents by Inventor Daisuke Kajimura
Daisuke Kajimura has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).
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Publication number: 20240093316Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: ApplicationFiled: November 27, 2023Publication date: March 21, 2024Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Patent number: 11866794Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: GrantFiled: August 24, 2021Date of Patent: January 9, 2024Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Publication number: 20230337645Abstract: Nuclease-mediated methods for expanding repeats already present at a genomic locus are provided. Non-human animal genomes, non-human animal cells, and non-human animals comprising a heterologous hexanucleotide repeat expansion sequence inserted at an endogenous C9orf72 locus and methods of making such non-human animal cells and non-human animals through nuclease-mediated repeat expansion are also provided. Methods of using the non-human animal cells or non-human animals to identify therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative disorders associated with repeat expansion at the C9orf72 locus are also provided.Type: ApplicationFiled: May 12, 2023Publication date: October 26, 2023Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Daisuke Kajimura, Aarti Sharma-Kanning, Brittany Dubose, Gustavo Droguett, Chia-Jen Siao, Junko Kuno, David Frendewey, Brian Zambrowicz
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Patent number: 11690362Abstract: Nuclease-mediated methods for expanding repeats already present at a genomic locus are provided. Non-human animal genomes, non-human animal cells, and non-human animals comprising a heterologous hexanucleotide repeat expansion sequence inserted at an endogenous C9orf72 locus and methods of making such non-human animal cells and non-human animals through nuclease-mediated repeat expansion are also provided. Methods of using the non-human animal cells or non-human animals to identify therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative disorders associated with repeat expansion at the C9orf72 locus are also provided.Type: GrantFiled: December 13, 2019Date of Patent: July 4, 2023Assignee: Regeneran Pharmaceuticals, Inc.Inventors: Daisuke Kajimura, Aarti Sharma-Kanning, Brittany Dubose, Gustavo Droguett, Chia-Jen Siao, Junko Kuno, David Frendewey, Brian Zambrowicz
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Publication number: 20230081547Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized KLKB1 locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized KLKB1 locus express a human plasma kallikrein protein or a chimeric plasma kallikrein protein, fragments of which are from human plasma kallikrein. Methods are provided for using such non-human animals comprising a humanized KLKB1 locus to assess in vivo efficacy of human-KLKB 1-targeting reagents such as nuclease agents designed to target human KLKB1.Type: ApplicationFiled: February 5, 2021Publication date: March 16, 2023Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Daisuke Kajimura, Susannah Brydges, Andrew J. Murphy
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Publication number: 20210392862Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: ApplicationFiled: August 24, 2021Publication date: December 23, 2021Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Patent number: 11130999Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: GrantFiled: July 31, 2018Date of Patent: September 28, 2021Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Publication number: 20200370054Abstract: A non-human animal (e.g., a rodent) model for diseases associated with a C9ORF72 heterologous hexanucleotide repeat expansion sequence is provided, which non-human animal comprises a heterologous hexanucleotide repeat (GGGGCC) in an endogenous C9ORF72 locus. A non-human animal disclosed herein comprising a heterologous hexanucleotide repeat expansion sequence comprising at least one instance, e.g., repeat, of a hexanucleotide (GGGGCC) sequence may further exhibit a characteristic and/or phenotype associated with one or more neurodegenerative disorders (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), etc.). Methods of identifying therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative (e.g., amyotrophic lateral sclerosis (ALS, also referred to as Lou Gehrig's disease) and frontotemporal dementia (FTD)) are also provided.Type: ApplicationFiled: August 5, 2020Publication date: November 26, 2020Inventors: David Heslin, Roxanne Ally, Chia-Jen Siao, Ka-Man Venus Lai, David M. Valenzuela, Chunguang Guo, Michael LaCroix-Fralish, Lynn Macdonald, Aarti Sharma-Kanning, Daisuke Kajimura, Gustavo Droguett, David Frendewey, Alexander O. Mujica
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Patent number: 10781453Abstract: A non-human animal (e.g., a rodent) model for diseases associated with a C9ORF72 heterologous hexanucleotide repeat expansion sequence is provided, which non-human animal comprises a heterologous hexanucleotide repeat (GGGGCC) in an endogenous C9ORF72 locus. A non-human animal disclosed herein comprising a heterologous hexanucleotide repeat expansion sequence comprising at least one instance, e.g., repeat, of a hexanucleotide (GGGGCC) sequence may further exhibit a characteristic and/or phenotype associated with one or more neurodegenerative disorders (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), etc.). Methods of identifying therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative (e.g., amyotrophic lateral sclerosis (ALS, also referred to as Lou Gehrig's disease) and frontotemporal dementia (FTD)) are also provided.Type: GrantFiled: September 29, 2017Date of Patent: September 22, 2020Assignee: Regeneron Pharmaceuticals, Inc.Inventors: David Heslin, Roxanne Ally, Chia-Jen Siao, Ka-Man Venus Lai, David M. Valenzuela, Aarti Sharma-Kanning, Daisuke Kajimura, Gustavo Droguett, David Frendewey, Alexander O. Mujica
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Publication number: 20200196581Abstract: Nuclease-mediated methods for expanding repeats already present at a genomic locus are provided. Non-human animal genomes, non-human animal cells, and non-human animals comprising a heterologous hexanucleotide repeat expansion sequence inserted at an endogenous C9orf72 locus and methods of making such non-human animal cells and non-human animals through nuclease-mediated repeat expansion are also provided. Methods of using the non-human animal cells or non-human animals to identify therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative disorders associated with repeat expansion at the C9orf72 locus are also provided.Type: ApplicationFiled: December 13, 2019Publication date: June 25, 2020Inventors: Daisuke Kajimura, Aarti Sharma-Kanning, Brittany Dubose, Gustavo Droguett, Chia-Jen Siao, Junko Kuno, David Frendewey, Brian Zambrowicz
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Publication number: 20190032155Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: ApplicationFiled: July 31, 2018Publication date: January 31, 2019Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Publication number: 20180094267Abstract: A non-human animal (e.g., a rodent) model for diseases associated with a C9ORF72 heterologous hexanucleotide repeat expansion sequence is provided, which non-human animal comprises a heterologous hexanucleotide repeat (GGGGCC) in an endogenous C9ORF72 locus. A non-human animal disclosed herein comprising a heterologous hexanucleotide repeat expansion sequence comprising at least one instance, e.g., repeat, of a hexanucleotide (GGGGCC) sequence may further exhibit a characteristic and/or phenotype associated with one or more neurodegenerative disorders (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), etc.). Methods of identifying therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative (e.g., amyotrophic lateral sclerosis (ALS, also referred to as Lou Gehrig's disease) and frontotemporal dementia (FTD)) are also provided.Type: ApplicationFiled: September 29, 2017Publication date: April 5, 2018Inventors: David Heslin, Roxanne Ally, Chia-Jen Siao, Ka-Man Venus Lai, David M. Valenzuela, Chunguang Guo, Michael LaCroix-Fralish, Lynn Macdonald, Aarti Sharma, Daisuke Kajimura, Gustavo Droguett, David Frendewey