Patents by Inventor Gustavo Droguett
Gustavo Droguett 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: 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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Publication number: 20230332185Abstract: Compositions and methods are provided for creating and promoting biallelic targeted modifications to genomes within cells and for producing non-human animals comprising the modified genomes. Also provided are compositions and methods for modifying a genome within a cell that is heterozygous for an allele to become homozygous for that allele. The methods make use of Cas proteins and two or more guide RNAs that target different locations within the same genomic target locus. Also provided are methods of identifying cells with modified genomes.Type: ApplicationFiled: May 15, 2023Publication date: October 19, 2023Applicant: Regeneron Pharmaceuticals, Inc.Inventors: David Frendewey, Ka-Man Venus Lai, Wojtek Auerbach, Gustavo Droguett, Anthony Gagliardi, David M. Valenzuela, Vera Voronina, Lynn Macdonald, Andrew J. Murphy, George D. Yancopoulos
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Patent number: 11697828Abstract: Compositions and methods are provided for creating and promoting biallelic targeted modifications to genomes within cells and for producing non-human animals comprising the modified genomes. Also provided are compositions and methods for modifying a genome within a cell that is heterozygous for an allele to become homozygous for that allele. The methods make use of Cas proteins and two or more guide RNAs that target different locations within the same genomic target locus. Also provided are methods of identifying cells with modified genomes.Type: GrantFiled: September 16, 2019Date of Patent: July 11, 2023Assignee: Regeneran Pharmaceuticals, Inc.Inventors: David Frendewey, Ka-Man Venus Lai, Wojtek Auerbach, Gustavo Droguett, Anthony Gagliardi, David M. Valenzuela, Vera Voronina, Lynn Macdonald, Andrew J. Murphy, George D. Yancopoulos
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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: 20230114649Abstract: The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods of inhibiting expression of a C9orf72 gene and methods of treating subjects having a C9orf72-associated disease or disorder, e.g., C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia or Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.Type: ApplicationFiled: June 9, 2022Publication date: April 13, 2023Inventors: Elane Fishilevich, Stuart Milstein, Kirk Brown, Tracy Zimmermann, James D. McIninch, David Frendewey, Eric Chiao, Aarti Sharma-Kanning, Anthony Gagliardi, Gustavo Droguett, Brittany Dubose, Brian Zambrowicz
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Publication number: 20220167600Abstract: Methods and compositions are provided for generating antigen-binding proteins against a foreign antigen of interest.Type: ApplicationFiled: December 16, 2021Publication date: June 2, 2022Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Vera Voronina, Lynn Macdonald, Marine Prissette, Ka-Man Venus Lai, Ashok Badithe, Andrew J. Murphy, Gustavo Droguett, David Frendewey, Brian Zambrowicz
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Publication number: 20210112788Abstract: Methods and compositions are provided for generating F0 fertile XY female animals. The methods and compositions involve making XY pluripotent or totipotent animal cells, in vitro cell cultures, or embryos that are capable of producing a fertile female XY animal in an F0 generation. Such cells, embryos, and animals can be made by silencing a region of the Y chromosome. Optionally, the cells can also be cultured in feminizing medium such as a low-osmolality medium and/or can be modified to decrease the level and/or activity of an Sry protein. Methods and compositions are also provided for silencing a region of the Y chromosome in an XY pluripotent or totipotent animal cell, or in vitro cell cultures, embryos, or animals derived therefrom, by maintaining an XY pluripotent or totipotent animal cell in a feminizing medium.Type: ApplicationFiled: December 11, 2020Publication date: April 22, 2021Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Jennifer Schmahl, David Frendewey, Junko Kuno, Chia-Jen Siao, Gustavo Droguett, Yu Bai, Wojtek Auerbach
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Publication number: 20210024953Abstract: Methods and compositions are provided for generating targeted genetic modifications on the Y chromosome or a challenging target locus. Compositions include an in vitro culture comprising an XY pluripotent and/or totipotent animal cell (i.e., XY ES cells or XY iPS cells) having a modification that decreases the level and/or activity of an Sry protein; and, culturing these cells in a medium that promotes development of XY F0 fertile females. Such compositions find use in various methods for making a fertile female XY non-human mammal in an F0 generation.Type: ApplicationFiled: August 31, 2020Publication date: January 28, 2021Applicant: Regeneron Pharmaceuticals, Inc.Inventors: David Frendewey, Gustavo Droguett, Anthony Gagliardi, Junko Kuno, Wojtek Auerbach, David Valenzuela
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Patent number: 10893666Abstract: Methods and compositions are provided for generating F0 fertile XY female animals. The methods and compositions involve making XY pluripotent or totipotent animal cells, in vitro cell cultures, or embryos that are capable of producing a fertile female XY animal in an F0 generation. Such cells, embryos, and animals can be made by silencing a region of the Y chromosome. Optionally, the cells can also be cultured in feminizing medium such as a low-osmolality medium and/or can be modified to decrease the level and/or activity of an Sry protein. Methods and compositions are also provided for silencing a region of the Y chromosome in an XY pluripotent or totipotent animal cell, or in vitro cell cultures, embryos, or animals derived therefrom, by maintaining an XY pluripotent or totipotent animal cell in a feminizing medium.Type: GrantFiled: September 16, 2016Date of Patent: January 19, 2021Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Jennifer Schmahl, David Frendewey, Junko Kuno, Chia-Jen Siao, Gustavo Droguett, Yu Bai, Wojtek Auerbach
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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: 10793874Abstract: Methods and compositions are provided for generating targeted genetic modifications on the Y chromosome or a challenging target locus. Compositions include an in vitro culture comprising an XY pluripotent and/or totipotent animal cell (i.e., XY ES cells or XY iPS cells) having a modification that decreases the level and/or activity of an Sry protein; and, culturing these cells in a medium that promotes development of XY F0 fertile females. Such compositions find use in various methods for making a fertile female XY non-human mammal in an F0 generation.Type: GrantFiled: February 22, 2018Date of Patent: October 6, 2020Assignee: Regeneron Pharmaceuticals, Inc.Inventors: David Frendewey, Gustavo Droguett, Anthony Gagliardi, Junko Kuno, Wojtek Auerbach, David Valenzuela
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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: 20200002731Abstract: Compositions and methods are provided for creating and promoting biallelic targeted modifications to genomes within cells and for producing non-human animals comprising the modified genomes. Also provided are compositions and methods for modifying a genome within a cell that is heterozygous for an allele to become homozygous for that allele. The methods make use of Cas proteins and two or more guide RNAs that target different locations within the same genomic target locus. Also provided are methods of identifying cells with modified genomes.Type: ApplicationFiled: September 16, 2019Publication date: January 2, 2020Applicant: Regeneron Pharmaceuticals, Inc.Inventors: David Frendewey, Ka-Man Venus Lai, Wojtek Auerbach, Gustavo Droguett, Anthony Gagliardi, David M. Valenzuela, Vera Voronina, Lynn Macdonald, Andrew J. Murphy, George D. Yancopoulos
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Publication number: 20200002730Abstract: Compositions and methods are provided for creating and promoting biallelic targeted modifications to genomes within cells and for producing non-human animals comprising the modified genomes. Also provided are compositions and methods for modifying a genome within a cell that is heterozygous for an allele to become homozygous for that allele. The methods make use of Cas proteins and two or more guide RNAs that target different locations within the same genomic target locus. Also provided are methods of identifying cells with modified genomes.Type: ApplicationFiled: September 16, 2019Publication date: January 2, 2020Applicant: Regeneron Pharmaceuticals, Inc.Inventors: David Frendewey, Ka-Man Venus Lai, Wojtek Auerbach, Gustavo Droguett, Anthony Gagliardi, David M. Valenzuela, Vera Voronina, Lynn Macdonald, Andrew J. Murphy, George D. Yancopoulos
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Patent number: 10457960Abstract: Compositions and methods are provided for creating and promoting biallelic targeted modifications to genomes within cells and for producing non-human animals comprising the modified genomes. Also provided are compositions and methods for modifying a genome within a cell that is heterozygous for an allele to become homozygous for that allele. The methods make use of Cas proteins and two or more guide RNAs that target different locations within the same genomic target locus. Also provided are methods of identifying cells with modified genomes.Type: GrantFiled: November 20, 2015Date of Patent: October 29, 2019Assignee: Regeneron Pharmaceuticals, Inc.Inventors: David Frendewey, Ka-Man Venus Lai, Wojtek Auerbach, Gustavo Droguett, Anthony Gagliardi, David M. Valenzuela, Vera Voronina, Lynn Macdonald, Andrew J. Murphy, George D. Yancopoulos
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Publication number: 20190225992Abstract: Methods and compositions are provided for modifying one or more target loci in a cell. Such methods comprise providing a cell comprising a first polynucleotide encoding a first selection marker operably linked to a first promoter active in the cell, wherein the first polynucleotide further comprises a first recognition site for a first nuclease agent. A first nuclease agent is introduced into a cell, wherein the first nuclease agent induces a nick or double-strand break at the first recognition site. Further introduced into the cell is a first targeting vector comprising a first insert polynucleotide flanked by a first and a second homology arm that correspond to a first and a second target site located in sufficient proximity to the first recognition site. At least one cell is then identified comprising in its genome the first insert polynucleotide integrated at the target locus.Type: ApplicationFiled: March 29, 2019Publication date: July 25, 2019Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Wojtek Auerbach, David Frendewey, Gustavo Droguett, Anthony Gagliardi, Junko Kuno, David M. Valenzuela
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Patent number: 10294494Abstract: Methods and compositions are provided for modifying one or more target loci in a cell. Such methods comprise providing a cell comprising a first polynucleotide encoding a first selection marker operably linked to a first promoter active in the cell, wherein the first polynucleotide further comprises a first recognition site for a first nuclease agent. A first nuclease agent is introduced into a cell, wherein the first nuclease agent induces a nick or double-strand break at the first recognition site. Further introduced into the cell is a first targeting vector comprising a first insert polynucleotide flanked by a first and a second homology arm that correspond to a first and a second target site located in sufficient proximity to the first recognition site. At least one cell is then identified comprising in its genome the first insert polynucleotide integrated at the target locus.Type: GrantFiled: April 7, 2017Date of Patent: May 21, 2019Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Wojtek Auerbach, David Frendewey, Gustavo Droguett, Anthony Gagliardi, Junko Kuno, David M. Valenzuela
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Patent number: 10106820Abstract: Methods and compositions are provided for modifying one or more target loci in a cell. Such methods comprise providing a cell comprising a first polynucleotide encoding a first selection marker operably linked to a first promoter active in the cell, wherein the first polynucleotide further comprises a first recognition site for a first nuclease agent. A first nuclease agent is introduced into a cell, wherein the first nuclease agent induces a nick or double-strand break at the first recognition site. Further introduced into the cell is a first targeting vector comprising a first insert polynucleotide flanked by a first and a second homology arm that correspond to a first and a second target site located in sufficient proximity to the first recognition site. At least one cell is then identified comprising in its genome the first insert polynucleotide integrated at the target locus.Type: GrantFiled: June 5, 2015Date of Patent: October 23, 2018Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Wojtek Auerbach, David Frendewey, Gustavo Droguett, Anthony Gagliardi, Junko Kuno, David M. Valenzuela
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Publication number: 20180291468Abstract: Compositions and methods for determining circulating biomolecules before, during, and/or after treatment of a patient with an anti-cancer or anti-tumor drug (or putative drug) are described. Methods of treatments based on the compositions and methods described herein are also provided. Noninvasive methods and kits are provided for assessing the efficacy of an anti-cancer therapy for killing or damaging cancer cells. Embodiments are used to determine the cancer-killing efficacy of an anti-cancer drug in a patient, to optimize the selection of an anti-cancer drug for treatment of a patient, to adjust the dosage of an anti-cancer drug for treatment of a particular cancer in a patient and for identifying useful anti-cancer therapeutics for any one particular type of cancer.Type: ApplicationFiled: June 12, 2018Publication date: October 11, 2018Inventors: David Frendewey, Gavin Thurston, George D. Yancopoulos, Matthew Koss, Gustavo Droguett