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: 20230103708Abstract: Recombinant vectors operably encoding a CR2-FH fusion protein comprising a CR2 portion comprising CR2 protein or a fragment thereof and a FH portion comprising a factor H protein or a fragment thereof, and pharmaceutical compositions comprising the recombinant vector, are described. Also provided are methods of using the compositions for treatment eye diseases such as macular degeneration or glaucoma.Type: ApplicationFiled: June 16, 2022Publication date: April 6, 2023Inventors: Monica Vetter, Alejandra Bosco, Baerbel Rohrer, Stephen Tomlinson, William W. Hauswirth
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Patent number: 11512326Abstract: The present invention is directed to a delivery vector for transferring a small peptide coding sequence to a cell for expression of the small peptide coding sequence within the cell. The delivery vector comprises a secretory signal sequence; a sequence encoding a carrier protein operatively associated with the secretory signal sequence; a sequence encoding a cleavage site operatively associated with the sequence encoding a carrier protein; and a sequence encoding a small peptide operatively associated with the sequence encoding a cleavage site.Type: GrantFiled: May 26, 2010Date of Patent: November 29, 2022Assignee: University of Florida Research Foundation, IncorporatedInventors: Qiuhong Li, William W. Hauswirth, Alfred S. Lewin
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Publication number: 20220186260Abstract: 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: December 1, 2021Publication date: June 16, 2022Applicant: University of Florida Research Foundation, IncorporatedInventors: Shannon E. Boye, William W. Hauswirth, Sanford L. Boye
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Publication number: 20220183613Abstract: The present invention provides reagents and methods for modulating cone photoreceptor activity, and devices for assessment of cone photoreceptor activity.Type: ApplicationFiled: October 13, 2021Publication date: June 16, 2022Applicants: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED, UNIVERSITY OF WASHINGTON, UNIVERSITY OF WASHINGTONInventors: Jay NEITZ, Maureen NEITZ, James A. KUCHENBECKER, William W. HAUSWIRTH
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Publication number: 20220175969Abstract: The present disclosure provides short enhanced human opsin promoters for rod-specific expression that satisfy a need in the art for promoters that are able to control transgene expression both efficiently and selectively in rod cells, with little to no off-target expression in other photoreceptor cells. The disclosure provides vector constructs comprising transgenes operably controlled by this enhanced human opsin promoter that may be delivered and targeted with high specificity to rod cells. The disclosure also provides compositions comprising these vector constructs and methods for administration of these compositions to subjects in need thereof.Type: ApplicationFiled: March 4, 2020Publication date: June 9, 2022Applicant: University of Florida Research Foundation, IncorporatedInventors: Frank M. Dyka, William W. Hauswirth
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Patent number: 11325956Abstract: 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: February 1, 2019Date of Patent: May 10, 2022Assignee: University of Florida Research Foundation, IncorporatedInventors: Sanford L. Boye, Shannon E. Boye, Frank Dyka, William W. Hauswirth
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Publication number: 20220033826Abstract: Aspects of the disclosure relate to methods and compositions useful for treating bestrophinopathies, such as Best Disease.Type: ApplicationFiled: August 30, 2019Publication date: February 3, 2022Applicant: University of Florida Research Foundation, IncorporatedInventors: William W. Hauswirth, Alfred S. Lewin, Cristhian J. Ildefonso, Brianna M. Young
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Publication number: 20210395778Abstract: Provided herein are methods and compositions for expressing Otoferlin, e.g., utilizing adeno-associated viral (AAV) particles. Further provided herein are compositions of AAV particles comprising one or more polynucleotides encoding Otoferlin are codon optimized for expression in human cells. Such methods and compositions may be useful for treatment of diseases and disorders such as Deafness, Autosomal Recessive 9 (DFNB9). Also provided are kits comprising such compositions.Type: ApplicationFiled: November 1, 2019Publication date: December 23, 2021Applicant: University of Florida Research Foundation, IncorporatedInventors: Frank M. Dyka, William W. Hauswirth
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Patent number: 11160488Abstract: The present invention provides reagents and methods for modulating cone photoreceptor activity, and devices for assessment of cone photoreceptor activity.Type: GrantFiled: March 29, 2018Date of Patent: November 2, 2021Assignees: Universty of Washington, University of Florida Research Foundation, IncorporatedInventors: Jay Neitz, Maureen Neitz, James A. Kuchenbecker, William W. Hauswirth
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Publication number: 20210324387Abstract: 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: May 21, 2021Publication date: October 21, 2021Applicants: Univeristy 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 Jacobson
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Patent number: 11118185Abstract: 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: GrantFiled: March 1, 2017Date of Patent: September 14, 2021Assignees: 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 Jacobson
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Publication number: 20210207147Abstract: Aspects of the disclosure relate to methods and compositions useful for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering 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 a subject. In some embodiments, a replacement RHO coding sequence that is resistant to the interfering RNA also is delivered to the subject.Type: ApplicationFiled: June 3, 2019Publication date: July 8, 2021Applicants: 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 Jacobson
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Publication number: 20210130421Abstract: 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: November 18, 2020Publication date: May 6, 2021Applicant: University of Florida Research Foundation, IncorporatedInventors: Sanford L. Boye, Shannon E. Boye, Frank M. Dyka, William W. Hauswirth
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Publication number: 20200157573Abstract: Provided herein are methods and compositions for expressing Otoferlin, e.g., utilizing adeno-associated viral (AAV) particles. Such methods and compositions may be useful for treatment of diseases such as Deafness, Autosomal Recessive 9 (DFNB9).Type: ApplicationFiled: May 4, 2018Publication date: May 21, 2020Applicants: University of Florida Research Foundation, Incorporated, The Regents of the University of CaliforniaInventors: Sanford L. Boye, Frank M. Dyka, William W. Hauswirth, Omar Akil
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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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Patent number: 10533187Abstract: 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: GrantFiled: March 18, 2016Date of Patent: January 14, 2020Assignee: University of Florida Research Foundation, IncorporatedInventors: William W. Hauswirth, Ji-Jing Pang
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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: 10426844Abstract: 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: GrantFiled: August 24, 2016Date of Patent: October 1, 2019Assignee: University of Florida Research Foundation, IncorporatedInventors: Mavis Agbandje-McKenna, William W. Hauswirth, Arun Srivastava, Li Zhong
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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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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