Patents by Inventor William W. Hauswirth
William W. Hauswirth 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: 20190093111Abstract: Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.Type: ApplicationFiled: March 1, 2017Publication date: March 28, 2019Applicants: University of Florida Research Foundation, Incorporated, The Trustees of the University of PennsylvaniaInventors: Alfred S. Lewin, William W. Hauswirth, MIchael T. Massengill, William Beltran, Gustavo D. Aguirre, Artur Cideciyan, Samuel Jacovson
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Patent number: 10214572Abstract: 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: GrantFiled: May 15, 2014Date of Patent: February 26, 2019Assignee: University of Florida Research Foundation, Inc.Inventors: Sanford Leon Boye, Shannon Elizabeth Boye, Frank Markus Dyka, William W. Hauswirth
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Publication number: 20180344197Abstract: The present invention provides reagents and methods for modulating cone photoreceptor activity, and devices for assessment of cone photoreceptor activity.Type: ApplicationFiled: March 29, 2018Publication date: December 6, 2018Inventors: Jay NEITZ, Maureen NEITZ, James A. KUCHENBECKER, William W. HAUSWIRTH
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Publication number: 20180112231Abstract: Provided are nucleic acid sequences, recombinant adeno-associated viral particles, compositions, and methods related to treating cone monochromacies, such as blue cone monochromacy (BCM). Specifically, the nucleic acid sequences, recombinant adeno-associated viral particles, compositions, and methods involve use of an M-opsin gene operably linked to a cone-specific promoter. Further disclosed is the sequence of a cone-specific PR2.1 promoter.Type: ApplicationFiled: March 18, 2016Publication date: April 26, 2018Applicant: University of Florida Research Foundation, IncorporatedInventors: William W. Hauswirth, Ji-Jing Pang
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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: 9816108Abstract: 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: GrantFiled: April 22, 2011Date of Patent: November 14, 2017Assignee: University of Florida Research Foundation, Inc.Inventors: Shannon Elizabeth Boye, William W. Hauswirth, Sanford Leon 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: 20160361439Abstract: Disclosed are capsid-mutated rAAV vectors and methods for their use in gene therapy, and particularly for use in delivering therapeutic transgenes to treat a variety of mammalian diseases and disorders, including dysfunctions and abnormal conditions of the human eye. VP3 capsid proteins comprising a modification of one or more of the surface-exposed tyrosine residues are disclosed, and in particular, VP3 capsid protein comprising tyrosine-to-phenylalanine mutations at positions corresponding to Y444F, Y500F, and Y730F of the wild-type AAV2 sequence. Also provided are rAAV virions and viral particles that comprise such a mutated AAV capsid protein and a nucleic acid molecule that expresses one or more selected therapeutic or reporter transgenes in one or more mammalian cells of interest. Advantageously, the capsid-mutated rAAV vectors and virions disclosed herein afford improved transduction efficiency in a variety of cells, tissues and organs of interest, when compared to their unmodified (i.e.Type: ApplicationFiled: August 24, 2016Publication date: December 15, 2016Inventors: Mavis AGBANDJE-MCKENNA, William W. HAUSWIRTH, Arun SRIVASTAVA, Li ZHONG
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Publication number: 20160263246Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: ApplicationFiled: May 4, 2016Publication date: September 15, 2016Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Patent number: 9433688Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: GrantFiled: June 20, 2014Date of Patent: September 6, 2016Assignees: The Trustees of the University of Pennsylvania, University of Florida Research Foundation, Incorporated, Cornell Research Foundation, Inc.Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Patent number: 9375491Abstract: The subject invention concerns materials and methods for providing for cone cell specific expression of a polynucleotide in a human or animal. One aspect of the invention concerns a polynucleotide promoter sequence that directs expression of an operably linked polynucleotide in cone cells. In one embodiment, a polynucleotide of the invention comprises a nucleotide sequence of an interphotoreceptor retinoid-binding protein (IRBP) gene that is positioned upstream of a promoter nucleotide sequence of a cone transducin alpha-subunit (GNAT2) gene. Another aspect of the subject invention concerns methods for expressing a selected polynucleotide in cone cells. The selected polynucleotide can be provided in a polynucleotide of the invention wherein the selected polynucleotide is operably linked to a polynucleotide promoter sequence of the invention. In one embodiment, the selected polynucleotide sequence is provided in a polynucleotide vector of the invention.Type: GrantFiled: October 29, 2012Date of Patent: June 28, 2016Assignee: University of Florida Research Foundation, Inc.Inventors: Sanford Leon Boye, Frank Markus Dyka, William W. Hauswirth
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Publication number: 20160015288Abstract: The present invention provides reagents and methods for modulating cone photoreceptor activity, and devices for assessment of cone photoreceptor activity.Type: ApplicationFiled: August 27, 2015Publication date: January 21, 2016Inventors: Jay NEITZ, Maureen NEITZ, James A. KUCHENBECKER, William W. HAUSWIRTH
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Patent number: 9198595Abstract: The present invention provides reagents and methods for modulating cone photoreceptor activity, and devices for assessment of cone photoreceptor activity.Type: GrantFiled: November 8, 2013Date of Patent: December 1, 2015Assignees: University of Washington Through its Center for Commercialization, University of Florida Research Foundation, Incorporated, Medical College of WisconsinInventors: Jay Neitz, Maureen Neitz, James A. Kuchenbecker, William W. Hauswirth
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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: 20140377224Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.Type: ApplicationFiled: June 20, 2014Publication date: December 25, 2014Applicants: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA, UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED, CORNELL RESEARCH FOUNDATION, INC.Inventors: Gregory M. Acland, Gustavo D. Aguirre, Jean Bennett, William W. Hauswirth, Samuel G. Jacobson, Albert M. Maguire
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Publication number: 20140275231Abstract: The subject invention concerns materials and methods for providing for cone cell specific expression of a polynucleotide in a human or animal. One aspect of the invention concerns a polynucleotide promoter sequence that directs expression of an operably linked polynucleotide in cone cells. In one embodiment, a polynucleotide of the invention comprises a nucleotide sequence of an interphotoreceptor retinoid-binding protein (IRBP) gene that is positioned upstream of a promoter nucleotide sequence of a cone transducin alpha-subunit (GNAT2) gene. Another aspect of the subject invention concerns methods for expressing a selected polynucleotide in cone cells. The selected polynucleotide can be provided in a polynucleotide of the invention wherein the selected polynucleotide is operably linked to a polynucleotide promoter sequence of the invention. In one embodiment, the selected polynucleotide sequence is provided in a polynucleotide vector of the invention.Type: ApplicationFiled: October 29, 2012Publication date: September 18, 2014Inventors: Sanford Leon Boye, Frank Markus Dyka, William W. Hauswirth