Patents by Inventor Barry J. Byrne
Barry J. Byrne 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: 20210340503Abstract: The present invention includes methods and compositions for the production of high titer recombinant Adeno-Associated Virus (rAAV) in a variety of mammalian cells. The disclosed rAAV are useful in gene therapy applications. Disclosed methods based on co-infection of cells with two or more replication-defective recombinant herpes virus (rHSV) vectors are suitable for high-titer, large-scale production of infectious rAAV.Type: ApplicationFiled: March 15, 2021Publication date: November 4, 2021Inventors: Kyu-Kye Hwang, David R. Knop, James Conway, Barry J. Byrne
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Publication number: 20190017033Abstract: The present invention includes methods and compositions for the production of high titer recombinant Adeno-Associated Virus (rAAV) in a variety of mammalian cells. The disclosed rAAV are useful in gene therapy applications. Disclosed methods based on co-infection of cells with two or more replication-defective recombinant herpes virus (rHSV) vectors are suitable for high-titer, large-scale production of infectious rAAV.Type: ApplicationFiled: April 10, 2018Publication date: January 17, 2019Inventors: Kyu-Kye Hwang, David R. Knop, James Conway, Barry J. Byrne
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Publication number: 20170362577Abstract: The present invention includes methods and compositions for the production of high titer recombinant Adeno-Associated Virus (rAAV) in a variety of mammalian cells. The disclosed rAAV are useful in gene therapy applications. Disclosed methods based on co-infection of cells with two or more replication-defective recombinant herpes virus (rHSV) vectors are suitable for high-titer, large-scale production of infectious rAAV.Type: ApplicationFiled: February 3, 2017Publication date: December 21, 2017Inventors: Kyu-Kye Hwang, David Knop, James Conway, Barry J. Byrne
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Patent number: 9644216Abstract: The present disclosure describes improved adeno-associated virus (AAV) vectors for gene therapy applications in the treatment of glycogen storage disease, particularly glycogen storage disease type Ia (GSD-Ia). Described are recombinant nucleic acid molecules, vectors and recombinant AAV that include a G6PC promoter/enhancer, a synthetic intron, a G6PC coding sequence (such as a wild-type or codon-optimized G6PC coding sequence), and stuffer nucleic acid sequence situated between the G6PC promoter/enhancer and the intron, as well as between the intron and the G6PC coding sequence. The recombinant AAVs disclosed herein exhibit highly efficient liver transduction and are capable of correcting metabolic abnormalities in an animal model of GSD-Ia.Type: GrantFiled: November 25, 2014Date of Patent: May 9, 2017Assignees: The United States of America, as represented by the Secretary, Department of Health and Human Services, University of Florida Research Foundation, IncorporatedInventors: Janice J. Chou, Barry J. Byrne
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Publication number: 20160376608Abstract: The present disclosure describes improved adeno-associated virus (AAV) vectors for gene therapy applications in the treatment of glycogen storage disease, particularly glycogen storage disease type Ia (GSD-Ia). Described are recombinant nucleic acid molecules, vectors and recombinant AAV that include a G6PC promoter/enhancer, a synthetic intron, a G6PC coding sequence (such as a wild-type or codon-optimized G6PC coding sequence), and stuffer nucleic acid sequence situated between the G6PC promoter/enhancer and the intron, as well as between the intron and the G6PC coding sequence. The recombinant AAVs disclosed herein exhibit highly efficient liver transduction and are capable of correcting metabolic abnormalities in an animal model of GSD-Ia.Type: ApplicationFiled: November 25, 2014Publication date: December 29, 2016Applicants: The U.S.A., as represented by the Secretary, Department of Health and Human Services, University of Florida Research Foundation, IncorporatedInventors: Janice J. Chou, Barry J. Byrne
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Publication number: 20160024480Abstract: The present invention includes methods and compositions for the production of high titer recombinant Adeno-Associated Virus (rAAV) in a variety of mammalian cells. The disclosed rAAV are useful in gene therapy applications. Disclosed methods based on co-infection of cells with two or more replication-defective recombinant herpes virus (rHSV) vectors are suitable for high-titer, large-scale production of infectious rAAV.Type: ApplicationFiled: October 2, 2015Publication date: January 28, 2016Inventors: Kyu-Kye Hwang, David Knop, James Conway, Barry J. Byrne
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Patent number: 7829078Abstract: The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue in the treatment of anemia is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of a delivered nucleotide sequence encoding erythropoietin, and for in vivo secretion thereof from transduced muscle cells such that systemic delivery is achieved.Type: GrantFiled: July 15, 2008Date of Patent: November 9, 2010Assignees: Genzyme Corporation, Johns Hopkins UniversityInventors: Gregory M. Podsakoff, Paul D. Kessler, Barry J. Byrne, Gary J. Kurtzman
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Patent number: 7704492Abstract: The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.Type: GrantFiled: July 15, 2008Date of Patent: April 27, 2010Assignee: Genzyme CorporationInventors: Gregory M. Podsakoff, Paul D. Kessler, Barry J. Byrne, Gary J. Kurtzman
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Publication number: 20090208459Abstract: The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue in the treatment of anemia is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of a delivered nucleotide sequence encoding erythropoietin, and for in vivo secretion thereof from transduced muscle cells such that systemic delivery is achieved.Type: ApplicationFiled: July 15, 2008Publication date: August 20, 2009Inventors: Gregory M. Podsakoff, Paul D. Kessler, Barry J. Byrne, Gary J. Kurtzman
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Publication number: 20090186002Abstract: The subject invention concerns materials and methods for gene therapy. One aspect of the invention pertains to vectors which can be used to effect genetic therapy in animals or humans having genetic disorders where expression of high levels of a protein of interest are required to treat or correct the disorder. The subject invention also pertains to methods for treating animals or humans in need of gene therapy to treat or correct a genetic disorder. The materials and methods of the invention can be used to provide therapeutically effective levels of a protein that is non-functional, or that is absent or deficient in the animal or human to be treated. In one embodiment, the materials and methods can be used to treat alpha-1-antitrypsin deficiency.Type: ApplicationFiled: October 31, 2007Publication date: July 23, 2009Applicant: University of Florida Research Foundation, Inc.Inventors: Terence R. Flotte, Sihong Song, Barry J. Byrne, Michael Morgan
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Publication number: 20080305084Abstract: The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.Type: ApplicationFiled: July 15, 2008Publication date: December 11, 2008Inventors: Gregory M. Podsakoff, Paul D. Kessler, Barry J. Byrne, Gary J. Kurtzman
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Publication number: 20080279945Abstract: Disclosed are water-soluble gel-based compositions for the delivery of recombinant adeno-associated virus (rAAV) vectors that express nucleic acid segments encoding therapeutic constructs including peptides, polypeptides, ribozymes, and catalytic RNA molecules, to selected cells and tissues of vertebrate animals. Also disclosed are gel-based rAAV compositions are useful in the treatment of mammalian, and in particular, human diseases, including for example, cardiac disease or dysfunction, and musculoskeletal disorders and congenital myopathies, including, for example, muscular dystrophy, acid maltase deficiency (Pompe's disease), and the like. In illustrative embodiments, the invention provides rAAV vectors comprised within a biocompatible gel composition for enhanced viral delivery/transfection to mammalian tissues, and in particular to vertebrate muscle tissues such as a human heart or diaphragm tissue.Type: ApplicationFiled: April 10, 2008Publication date: November 13, 2008Applicant: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCInventors: Cathryn S. Mah, Thomas J. Fraites, JR., Barry J. Byrne
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Publication number: 20080199442Abstract: The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.Type: ApplicationFiled: June 22, 2007Publication date: August 21, 2008Applicants: Genzyme Corporation, Johns Hopkins UniversityInventors: Gregory M. Podsakoff, Paul D. Kessler, Barry J. Byrne, Gary J. Kurtzman
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Patent number: 7238674Abstract: The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.Type: GrantFiled: March 5, 2002Date of Patent: July 3, 2007Assignee: Genzyme CorporationInventors: Gregory M. Podsakoff, Paul D. Kessler, Barry J. Byrne, Gary J. Kurtzman
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Patent number: 7094604Abstract: Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1×1012–1×1013 vector genomes/ml.Type: GrantFiled: March 11, 2004Date of Patent: August 22, 2006Assignee: University of Florida Research Foundation, Inc.Inventors: Richard O. Snyder, Sergei Zolotukhin, Yoshihisa Sakai, Barry J. Byrne, Mark R. Potter, Irine Zolotukhin, Scott Loiler, Vince A. Chiodo, Nicholas Muzyczka, William W. Hauswirth, Terence R. Flotte, Corinna Burger, Edgardo Rodriguez, Kevin R. Nash, Thomas J. Fraites
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Publication number: 20040209245Abstract: Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1×1012-1×1013 vector genomes/ml.Type: ApplicationFiled: March 11, 2004Publication date: October 21, 2004Inventors: Richard O. Snyder, Sergei Zolotukhin, Yoshihisa Sakai, Barry J. Byrne, Mark R. Potter, Irine Zolotukhin, Scott Loiler, Vince A. Chiodo, Nicholas Muzyczka, William W. Hauswirth, Terence R. Flotte, Corinna Burger, Edgardo Rodriguez, Kevin R. Nash, Thomas J. Fraites
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Patent number: 6783972Abstract: Disclosed are HSV-1 amplicons that supply all necessary helper functions required for rAAV packaging and methods for their use. These HSV-1 amplicons have been shown to be capable of rescuing and replicating all forms of rAAV genomes including rAAV genomes introduced into cells by infection of rAAV virions, rAAV genomes transfected into cells on plasmids or proviral rAAV genomes integrated into cellular chromosomal DNA. Also provided are methods for preparing high-titer rAAV vector compositions suitable for gene therapy and the delivery of exogenous polynucleotides to selected host cells.Type: GrantFiled: September 22, 1999Date of Patent: August 31, 2004Assignee: University of Florida Research FoundationInventors: Barry J. Byrne, James E. Conway, Gary S. Hayward, Nicholas Muzyzcka, Sergei Zolotukhin
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Publication number: 20040121444Abstract: Disclosed are methods for the isolation and purification of high-titer recombinant adeno-associated virus (rAAV) compositions. Also disclosed are methods for reducing or eliminating the concentration of helper adenovirus in rAAV samples. Methods are disclosed that provide highly-purified rAAV stocks having titers up to about 1013 particles/ml at particle-to-infectivity ratios of less than 100 in processes that are accomplished about 24 hours or less.Type: ApplicationFiled: December 9, 2003Publication date: June 24, 2004Applicant: University of Florida Research FoundationInventors: Sergei Zolotukhin, Barry J. Byrne, Nicholas Muzyczka
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Patent number: 6660514Abstract: Disclosed are methods for the isolation and purification of high-titer recombinant adeno-associated virus (rAAV) compositions. Also disclosed are methods for reducing or eliminating the concentration of helper adenovirus in rAAV samples. Methods are disclosed that provide highly-purified rAAV stocks having titers up to about 1013 particles/ml at particle-to-infectivity ratios of less than 100 in processes that are accomplished about 24 hours or less.Type: GrantFiled: July 21, 2000Date of Patent: December 9, 2003Assignee: University of Florida Research FoundationInventors: Sergei Zolotukhin, Barry J. Byrne, Nicholas Muzyczka
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Publication number: 20030223966Abstract: Serotype 1 recombinant adeno-associated virus (rAAV) vectors were used to deliver functional acid alpha-glucosidase genes in vitro and in vivo to muscle cells deficient in acid alpha-glucosidase. The vector-treated cells overexpressed acid alpha-glucosidase. Vector-treated animals displayed restored enzymatic activity and muscle function. Serotype 1 rAAV vectors induced significantly greater acid alpha-glucosidase expression compared to serotype 2 rAAV vectors.Type: ApplicationFiled: April 30, 2003Publication date: December 4, 2003Inventors: Thomas J. Fraites, Mary B. Rucker, Barry J. Byrne