Patents by Inventor Sanford L. Boye
Sanford L. Boye 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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Patent number: 10648000Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian vascular system, and complications from Type I diabetes. Also disclosed are methods for systemic and tissue-localized delivery of therapeutic rAAV-based gene expression cassettes to vascular endothelial cells, tissues, and organs, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of vasculitis, and complications arising from Type I diabetes, such as macular edema, nephropathy, diabetic retinopathy, and the like.Type: GrantFiled: February 16, 2016Date of Patent: May 12, 2020Assignee: University of Florida Research Foundation, IncorporatedInventors: William W. Hauswirth, Sanford L. Boye, Daniel M. Lipinski, Michael E. Boulton, Shannon E. Boye
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Publication number: 20200002386Abstract: Adeno associated viral (AAV) particles are emerging as useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (e.g., variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.Type: ApplicationFiled: August 21, 2019Publication date: January 2, 2020Applicants: University of Florida Research Foundation, Incorporated, The UAB Research FoundationInventors: Sergei Zolotukhin, Sanford L. Boye, Shannon E. Boye, Damien Marsic, Nicholas Muzyczka, Hector Ruben Mendez-Gomez, Paul D. Gamlin
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Publication number: 20190343920Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with Leber congenital amaurosis (LCA) in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal NPHP5 protein, or fragment thereof, under the control of regulatory sequences which express the NPHP5 protein in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: ApplicationFiled: July 12, 2019Publication date: November 14, 2019Inventors: Gustavo Aguirre, William A. Beltran, Samuel G. Jacobson, Artur V. Cideciyan, William W. Hauswirth, Sanford L. Boye
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Patent number: 10383922Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to the subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: GrantFiled: September 8, 2017Date of Patent: August 20, 2019Assignees: The Trustees of the University of Pennsylvania, University of Florida Research Foundation, IncorporatedInventors: William A Beltran, Gustavo D Aguirre, Samuel G Jacobson, Artur V Cideciyan, Alfred S Lewin, Sanford L Boye, William W Hauswirth, Wen-Tao Deng
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Patent number: 10308957Abstract: Disclosed are capsid-modified rAAV particles and expression vectors, as well as compositions and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and using novel capsid-protein-mutated particle or rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian eye. Also disclosed are methods for subretinal delivery of therapeutic gene constructs to mammalian photoreceptors and retinal pigment epithelial cells, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of a variety of inherited retinal diseases.Type: GrantFiled: March 4, 2015Date of Patent: June 4, 2019Assignee: University of Florida Research Foundation, Inc.Inventors: Shannon E. Boye, Sanford L. Boye, Mavis Agbandje-McKenna, Arun Srivastava
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Publication number: 20190153050Abstract: Disclosed are materials and methods for treating diseases of the mammalian eye, and in particular, Usher syndrome 1B (USH1B). The invention provides AAV-based, dual-vector systems that facilitate the expression of full-length proteins whose coding sequences exceed that of the polynucleotide packaging capacity of an individual AAV vector. In one embodiment, vector systems are provided that include i) a first AAV vector polynucleotide that includes an inverted terminal repeat at each end of the polynucleotide and a suitable promoter followed by a partial coding sequence that encodes an N-terminal portion of a full-length polypeptide; and ii) a second AAV vector polynucleotide that includes an inverted terminal repeat at each end of the polynucleotide and a partial coding sequence that encodes a C-terminal portion of a full-length polypeptide, optionally followed by a polyadenylation (pA) signal sequence.Type: ApplicationFiled: February 1, 2019Publication date: May 23, 2019Applicant: University of Florida Research Foundation, IncorporatedInventors: Sanford L. Boye, Shannon E. Boye, Frank Dyka, William W. Hauswirth
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Publication number: 20180100165Abstract: Disclosed are viral vector compositions comprising polynucleotide sequences that express one or more biologically-active mammalian guanylate cyclase proteins. Also disclosed are methods for their use in preventing, treating, and/or ameliorating at least one or more symptoms of a disease, disorder, abnormal condition, or dysfunction resulting at least in part from a guanylate cyclase deficiency in vivo. In particular embodiments, the use of recombinant adeno-associated viral (rAAV) vectors to treat or ameliorate symptoms of Leber's congenital amaurosis, as well as other conditions caused by an absence or reduction in the expression of a functional retinal-specific guanylate cyclase 1 (retGC1).Type: ApplicationFiled: October 10, 2017Publication date: April 12, 2018Applicant: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATEDInventors: Shannon E. Boye, William W. Hauswirth, Sanford L. Boye
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Publication number: 20180057840Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian vascular system, and complications from Type I diabetes. Also disclosed are methods for systemic and tissue-localized delivery of therapeutic rAAV-based gene expression cassettes to vascular endothelial cells, tissues, and organs, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of vasculitis, and complications arising from Type I diabetes, such as macular edema, nephropathy, diabetic retinopathy, and the like.Type: ApplicationFiled: February 16, 2016Publication date: March 1, 2018Applicant: University of Florida Research Foundation, Inc.Inventors: William W. Hauswirth, Sanford L. Boye, Daniel M. Lipinski, Michael E. Boulton, Shannon E. Boye
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Publication number: 20180036385Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: ApplicationFiled: September 8, 2017Publication date: February 8, 2018Inventors: William A. BELTRAN, Gustavo D. AGUIRRE, Samuel G. JACOBSON, Artur V. CIDECIYAN, Alfred S. LEWIN, Sanford L. BOYE, William W. HAUSWIRTH, Wen-Tao DENG
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Publication number: 20170348387Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with Leber congenital amaurosis (LCA) in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal NPHP5 protein, or fragment thereof, under the control of regulatory sequences which express the NPHP5 protein in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: ApplicationFiled: February 28, 2017Publication date: December 7, 2017Inventors: Gustavo Aguirre, William A. Beltran, Samuel G. Jacobson, Artur V. Cideciyan, William W. Hauswirth, Sanford L. Boye
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Patent number: 9770491Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to a subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: GrantFiled: January 23, 2013Date of Patent: September 26, 2017Assignees: The Trustees of the University of Pennsylvania, Univeristy of Florida Research Foundation, IncorporatedInventors: William A Beltran, Gustavo D Aguirre, Samuel G Jacobson, Artur V Cideciyan, Alfred S Lewin, Sanford L Boye, William W Hauswirth, Wen-Tao Deng
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Publication number: 20170007720Abstract: Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using novel capsid-protein-mutated rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian eye. Also disclosed are methods for intravitreal delivery of therapeutic gene constructs to retinal neuron cells, and specifically to ON bipolar cells, of the mammalian eye, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of retinitis pigmentosa, melanoma-associated retinopathy, and congenital stationary night blindness.Type: ApplicationFiled: February 18, 2015Publication date: January 12, 2017Applicant: University of Florida Research Foundation, Inc.Inventors: Shannon E. Boye, Frank Dyka, Sanford L. Boye, William W. Hauswirth
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Publication number: 20160369299Abstract: Disclosed are capsid-modified rAAV particles and expression vectors, as well as compositions and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and using novel capsid-protein-mutated particle or rAAV vector constructs in a variety of diagnostic and therapeutic applications including, inter alia, as delivery agents for diagnosis, treatment, or amelioration of one or more diseases, disorders, or dysfunctions of the mammalian eye. Also disclosed are methods for subretinal delivery of therapeutic gene constructs to mammalian photoreceptors and retinal pigment epithelial cells, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications including the treatment of a variety of inherited retinal diseases.Type: ApplicationFiled: March 4, 2015Publication date: December 22, 2016Applicant: University of Florida Research Foundation, Inc.Inventors: Shannon E. Boye, Sanford L. Boye, Mavis Agbandje-McKenna, Arun Srivastava
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Publication number: 20150202269Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to said subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.Type: ApplicationFiled: January 23, 2013Publication date: July 23, 2015Inventors: William A Beltran, Gustavo D Aguirre, Samuel G Jacobson, Artur V Cideciyan, Alfred S Lewin, Sanford L Boye, William W Hauswirth, Wen-Tao Deng
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Publication number: 20100069467Abstract: The present inventors concerns vectors carrying a truncated chimeric CMV-chicken ?-actin (smCBA) promoter in which the hybrid chicken ?-actin/rabbit ?-globin intron is greatly shortened, and their use to deliver to an operatively linked polynucleotide to host cells in vitro or in vivo, resulting in expression of the polynucleotide in the host cells. In one embodiment, the vector carrying the smCBA promoter is administered to the eye. In another embodiment, the vector carrying the smCBA promoter is a self-complementary adeno-associated virus (AAV). The AAV vector may be of any serotype (e.g., type 1, type 2, type 3, type 4, type 5, type 6, type 7, type 8, type 9, type 10). In another embodiment, a self-complementary vector carrying the smCBA promoter is administered to the eye. Another aspect of the invention concerns host cells carrying a vector of the invention.Type: ApplicationFiled: April 27, 2007Publication date: March 18, 2010Applicant: University of Florida Research Foundation, Inc.Inventors: Sanford L. Boye, William W. Hauswirth, Barry Jonh Byrne